[House Hearing, 117 Congress]
[From the U.S. Government Publishing Office]
FDA USER FEE REAUTHORIZATION: ENSURING SAFE AND EFFECTIVE MEDICAL
DEVICES
=======================================================================
HYBRID HEARING
BEFORE THE
SUBCOMMITTEE ON HEALTH
OF THE
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED SEVENTEENTH CONGRESS
SECOND SESSION
__________
MARCH 30, 2022
__________
Serial No. 117-77
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Published for the use of the Committee on Energy and Commerce
govinfo.gov/committee/house-energy
energycommerce.house.gov
_______
U.S. GOVERNMENT PUBLISHING OFFICE
60-330 PDF WASHINGTON : 2026
COMMITTEE ON ENERGY AND COMMERCE
FRANK PALLONE, Jr., New Jersey
Chairman
BOBBY L. RUSH, Illinois CATHY McMORRIS RODGERS, Washington
ANNA G. ESHOO, California Ranking Member
DIANA DeGETTE, Colorado FRED UPTON, Michigan
MIKE DOYLE, Pennsylvania MICHAEL C. BURGESS, Texas
JAN SCHAKOWSKY, Illinois STEVE SCALISE, Louisiana
G. K. BUTTERFIELD, North Carolina ROBERT E. LATTA, Ohio
DORIS O. MATSUI, California BRETT GUTHRIE, Kentucky
KATHY CASTOR, Florida DAVID B. McKINLEY, West Virginia
JOHN P. SARBANES, Maryland ADAM KINZINGER, Illinois
JERRY McNERNEY, California H. MORGAN GRIFFITH, Virginia
PETER WELCH, Vermont GUS M. BILIRAKIS, Florida
PAUL TONKO, New York BILL JOHNSON, Ohio
YVETTE D. CLARKE, New York BILLY LONG, Missouri
KURT SCHRADER, Oregon LARRY BUCSHON, Indiana
TONY CARDENAS, California MARKWAYNE MULLIN, Oklahoma
RAUL RUIZ, California RICHARD HUDSON, North Carolina
SCOTT H. PETERS, California TIM WALBERG, Michigan
DEBBIE DINGELL, Michigan EARL L. ``BUDDY'' CARTER, Georgia
MARC A. VEASEY, Texas JEFF DUNCAN, South Carolina
ANN M. KUSTER, New Hampshire GARY J. PALMER, Alabama
ROBIN L. KELLY, Illinois, Vice NEAL P. DUNN, Florida
Chair JOHN R. CURTIS, Utah
NANETTE DIAZ BARRAGAN, California DEBBIE LESKO, Arizona
A. DONALD McEACHIN, Virginia GREG PENCE, Indiana
LISA BLUNT ROCHESTER, Delaware DAN CRENSHAW, Texas
DARREN SOTO, Florida JOHN JOYCE, Pennsylvania
TOM O'HALLERAN, Arizona KELLY ARMSTRONG, North Dakota
KATHLEEN M. RICE, New York
ANGIE CRAIG, Minnesota
KIM SCHRIER, Washington
LORI TRAHAN, Massachusetts
LIZZIE FLETCHER, Texas
------
Professional Staff
TIFFANY GUARASCIO, Staff Director
WAVERLY GORDON, Deputy Staff Director
NATE HODSON, Minority Staff Director
Subcommittee on Health
ANNA G. ESHOO, California
Chairwoman
G. K. BUTTERFIELD, North Carolina BRETT GUTHRIE, Kentucky
DORIS O. MATSUI, California Ranking Member
KATHY CASTOR, Florida FRED UPTON, Michigan
JOHN P. SARBANES, Maryland, Vice MICHAEL C. BURGESS, Texas
Chair H. MORGAN GRIFFITH, Virginia
PETER WELCH, Vermont GUS M. BILIRAKIS, Florida
KURT SCHRADER, Oregon BILLY LONG, Missouri
TONY CARDENAS, California LARRY BUCSHON, Indiana
RAUL RUIZ, California MARKWAYNE MULLIN, Oklahoma
DEBBIE DINGELL, Michigan RICHARD HUDSON, North Carolina
ANN M. KUSTER, New Hampshire EARL L. ``BUDDY'' CARTER, Georgia
ROBIN L. KELLY, Illinois NEAL P. DUNN, Florida
NANETTE DIAZ BARRAGAN, California JOHN R. CURTIS, Utah
LISA BLUNT ROCHESTER, Delaware DAN CRENSHAW, Texas
ANGIE CRAIG, Minnesota JOHN JOYCE, Pennsylvania
KIM SCHRIER, Washington CATHY McMORRIS RODGERS, Washington
LORI TRAHAN, Massachusetts (ex officio)
LIZZIE FLETCHER, Texas
FRANK PALLONE, Jr., New Jersey (ex
officio)
C O N T E N T S
----------
Page
Hon. Anna G. Eshoo, a Representative in Congress from the State
of California, opening statement............................... 2
Prepared statement........................................... 3
Hon. Brett Guthrie, a Representative in Congress from the
Commonwealth of Kentucky, opening statement.................... 5
Prepared statement........................................... 7
Hon. Frank Pallone, Jr., a Representative in Congress from the
State of New Jersey, opening statement......................... 11
Prepared statement........................................... 13
Hon. Cathy McMorris Rodgers, a Representative in Congress from
the State of Washington, opening statement..................... 15
Prepared statement........................................... 17
Witnesses
Jeff Shuren, M.D., Director, Center for Devices and Radiological
Health, Food and Drug Administration........................... 21
Prepared statement........................................... 23
Answer to submitted questions................................ 118
Richard J. Kovacs, M.D., Q.E. and Sally Russell Professor of
Medicine, Indiana University School of Medicine, Chief Medical
Officer, American College of Cardiology........................ 82
Prepared statement........................................... 84
Mark Leahey, President and CEO, Medical Device Manufacturers
Association.................................................... 88
Prepared statement........................................... 90
Janet Trunzo, Senior Executive Vice President, Technology And
Regulatory Affairs, Advanced Medical Technology Association.... 91
Prepared statement........................................... 93
Diane Wurzburger, Executive of Regulatory Affairs, GE Healthcare. 99
Prepared statement........................................... 101
Submitted Material
H.R. 7084, the Protecting and Transforming Cyber Health Care Act
of 2022, submitted by Ms. Eshoo \1\
H.R. 7192, the Diagnostic Device Advisory Committee Act 2022,
submitted by Ms. Eshoo \1\
H.R. --------, the Medical Device User Fee Amendments of 2022,
submitted by Ms. Eshoo \1\
Letter of March 25, 2022, from U.S. PIRG, IAMERS, to Mr. Eshoo,
et al., submitted by Ms. Eshoo................................. 114
----------
\1\ Legislation has been retained in committee files and also is
available at https://docs.house.gov/Committee/Calendar/
ByEvent.aspx?EventID=114541.
FDA USER FEE REAUTHORIZATION: ENSURING SAFE AND EFFECTIVE MEDICAL
DEVICES
WEDNESDAY, MARCH 30, 2022
House of Representatives,
Subcommittee on Health,
Committee on Energy and Commerce,
Washington, DC.
The subcommittee met, pursuant to notice, at 9:01 a.m. in
the John D. Dingell Room, 2123 of the Rayburn House Office
Building, Hon. Anna Eshoo (chairwoman of the subcommittee),
presiding.
Members present: Representatives Eshoo, Matsui, Castor,
Sarbanes, Welch, Schrader, Cardenas, Ruiz, Dingell, Kuster,
Kelly, Barragan, Craig, Schrier, Trahan, Fletcher, Pallone (ex
officio); Guthrie (subcommmittee ranking member), Upton,
Burgess, Griffith, Bilirakis, Long, Bucshon, Hudson, Carter,
Dunn, Curtis, Crenshaw, Joyce, and Rodgers (ex officio).
Staff present: Vincent Amatrudo, FDA Detailee; Jacquelyn
Bolen, Health Counsel; Waverly Gordon, Deputy Staff Director
and General Counsel; Tiffany Guarascio, Staff Director; Stephen
Holland, Senior Health Counsel; Zach Kahan, Deputy Director
Outreach and Member Service; Mackenzie Kuhl, Press Assistant;
Una Lee, Chief Health Counsel; Aisling McDonough, Policy
Coordinator; Meghan Mullon, Policy Analyst; Kaitlyn Peel,
Digital Director; Caroline Rinker, Press Assistant; Chloe
Rodriguez, Clerk; Kylea Rogers, Staff Assistant; Andrew
Souvall, Director of Communications, Outreach, and Member
Services; Charlton Wilson, Fellow; Caroline Wood, Staff
Assistant; Hilary Carruthers, Minority Fellow; Alec Aramanda,
Minority Professional Staff Member, Health; Grace Graham,
Minority Chief Counsel, Health; Nate Hodson, Minority Staff
Director; Peter Kielty, Minority General Counsel; Emily King,
Minority Member Services Director; Clare Paoletta, Minority
Policy Analyst, Health; Kristin Seum, Minority Counsel, Health;
Kristen Shatynski, Minority Professional Staff Member, Health;
and Olivia Shields, Minority Communications Director.
Ms. Eshoo. The subcommittee on Health will now come to
order.
Due to COVID-19, today's hearing is being held remotely, as
well as in person.
For members and witnesses taking part remotely, microphones
will be set on mute to eliminate background noise. Members and
witnesses, you will need to unmute your microphone when you
wish to speak. Since we will have some witnesses that appear
virtually from our next panel, I ask my colleagues in the
hearing room to mute themselves whenever they are not speaking,
so we can clearly hear the witnesses' response.
Since members are participating from different locations at
today's hearing, recognition of members for questions will be
in the order of subcommittee seniority.
Documents for the record should be sent to Meghan Mullon at
the email address we have provided to your staff, and all--
excuse me, all documents will be entered into the record at the
conclusion of the hearing.
The Chair now recognizes herself for 5 minutes for an
opening statement.
OPENING STATEMENT OF HON. ANNA G. ESHOO, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF CALIFORNIA
Every day, Americans rely on safe and effective medical
devices. From the joy of an ultrasound during pregnancy to the
distress of a cancer diagnosis via an MRI, medical devices
treat, diagnosis (sic), and monitor the health of patients.
When I was working on the original legislation that created
the Medical Device User Fee Agreement process in 2002, we could
not have imagined the innovative devices that are on the market
today. And without the user fees supplementing the FDA for the
past 20 years, many of these innovations would be stuck in a
backlog, instead of helping patients.
A few months ago I visited a hospital in my district, El
Camino Hospital, which is using radiation technology with AI to
individually target tumors. This is just one example of the
hundreds of devices that the FDA has approved or authorized
since MDUFA was last authorized in 2017.
With this impressive innovation comes an increasingly
complex FDA review process. Over the past 20 years, the user
fee agreements have evolved to make sure that the FDA has the
resources necessary so that its reviews are timely,
transparent, and predictable. MDUFA V is the latest evolution.
The recently announced draft agreement will provide FDA $1.78
billion over five years in user fees. This is about ten times
the amount provided in the original 2002 user fee agreement.
But it--when you compare it with pharmaceutical drugs, they are
very different.
With this funding, the FDA's Center for Devices and
Radiological Health will be able to hire 387 new, full-time
employees, and also meet rising payroll costs. The user fees
will also fund successful FDA policies, such as the use of
Real-World Evidence, the harmonization of international medical
device regulatory activities, and patient engagement to inform
the evaluation of products.
While MDUFA V is a significant increase in user fees from
medical device makers, it is important to keep in mind that
user fees cannot and should not relieve Congress from its
responsibility to fund the FDA in a robust way. That is why I
was pleased to see President Biden's budget included a $95
million increase for FDA's medical product safety work.
Today we will hear from representatives from the FDA,
private industry, and public health about the negotiated
Medical Device User Fee Agreement. As the proud mother of
MDUFA, I look forward to shepherding the agreement through
reauthorization before the program expires on September 30th.
[The prepared statement of Ms. Eshoo follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. The Chair is now pleased to recognize the
distinguished ranking member of our subcommittee, Mr. Guthrie,
for his 5 minutes for an opening statement.
OPENING STATEMENT OF HON. BRETT GUTHRIE, A REPRESENTATIVE IN
CONGRESS FROM THE COMMONWEALTH STATE OF KENTUCKY
Mr. Guthrie. Thank you, Madam Chair. Thank you for holding
this important hearing.
And today we are building off the work we have done over
the past several weeks to find additional opportunities to
foster American biopharmaceutical innovation. The focus of
today's hearing is to discuss the recently announced Medical
Device User Fee Agreements, MDUFA. This will be critical to
continuing to enhance our medical device ecosystem here in the
United States.
Like the prescription drug industry, innovators working to
develop new and innovative medical technologies experience
significant delays in getting their products reviewed by the
Food and Drug Administration experts. That is why Congress,
regulators, and industry all came together to develop a
solution in the Medical--or MDUFA, Modernization Act of 2002,
that would streamline the review process and help get these
devices to patients more quickly. This agreement has been
authorized by Congress every five years.
The original MDUFA gave the FDA the necessary tools to hire
more clinical experts to review device applications. It also
offered industry the same assurance of being able to hold the
FDA to higher performance standards. The successes of this
partnership are clear at the FDA's Center for Devices and
Radiological Health. CDRH has granted novel technologies four
times--as many approvals marketing authorization as clearances
over the past decade, largely resulting from policies made
possible by past MDUFA authorizations.
The agreement before us today represents an ambitious
agenda set by industry and CDRH experts. The goal is to ensure
FDA is doing everything it can to protect patient safety, while
also supporting the development of medical device technologies.
Highlights include authorizing the FDA to collect 1.78 billion
from industry, and potentially up to 1.9 billion over the next
five years to bolster CDRH's workforce, and to help get
products reviewed and approved as quickly and as safely as
possible.
Of note is the creation of the new Total Life Cycle
Advisory Program, which CDRH states will help promote the long-
term sustainability of the Breakthrough Devices Program. I was
proud to support the creation of the Breakthrough Devices
Program that was created as part of the bipartisan 21st Century
Cures Act. In 2021 CDRH granted breakthrough designation to 213
devices, and there have been over 600 designations made since
the program's inception. This includes a device that harnesses
machine learning to help healthcare providers diagnose autism
spectrum disorder.
However, I am still frustrated by the Biden
Administration's actions to undermine the bipartisan-supported
Trump-era medical coverage of innovation technologies rule that
would have helped to get breakthrough devices to seniors once
the breakthrough device is approved by the FDA. This directly
conflicts with the earnest efforts made by Congress, CDRH, and
the medical device industry to encourage investments in these
emerging technologies.
I encourage CMS to work to reverse this decision, and work
with the industry as well as their FDA partners to address
outstanding concerns.
To that end, I am also continuing to push for the
codification of the 2018 FDA guidance that permits pre-approval
information exchanges between product sponsors and payers.
These information exchanges help get products covered more
quickly once they are approved by the FDA. My bill, the Pre-
Approval Information Exchange Act, would do just this, and help
public and private payers to make coverage determinations
earlier based off real-time healthcare, economic information
exchanged between entities.
Additionally, offering needed clarity around the FDA's 2016
guidance on emerging signals is another important priority of
mine in the device policy space, and I am working on a solution
to offer needed regulatory certainty on this issue. Outlining a
process that affords companies the chance to work with
regulators on addressing reported adverse health events
associated with their devices will not only protect patients,
but will also create regulatory predictability that will
protect against gaps in care for patients who rely on these
devices.
I look forward to working with my colleagues over the next
several months to re-authorize this important user fee
agreement that will promote even greater innovation for decades
to come.
Thank you, and I appreciate Dr. Shuren for being here, and
I look forward to having questions, and I will yield back.
[The prepared statement of Mr. Guthrie follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. The gentleman yields back.
Colleagues, we are going to break at 9:45 so that members
can attend Dear Don's funeral, and then we will resume at one
with the second panel today.
So we want to hear from Dr. Shuren and get as many
questions in as possible. But before we go to that, we will go
to the chairman of the full committee, Mr. Pallone, for his
opening statement.
OPENING STATEMENT OF HON. FRANK PALLONE, Jr., A REPRESENTATIVE
IN CONGRESS FROM THE STATE OF NEW JERSEY
Mr. Pallone. Thank you, Chairwoman Eshoo. Today we are
continuing our work to re-authorize the FDA user fees, which
provide critical resources for the agency's medical product
review programs. All of the other user fees expire on September
30th of this year. Or--I said all of them do. And Congress must
pass these re-authorizations well ahead of that deadline to
ensure FDA can continue to operate without interruption.
At today's hearing we will review the Medical Device User
Fee Program, also known as MDUFA. And throughout the COVID-19
pandemic, the FDA's Center for Devices and Radiological Health,
or CDRH, has been at the forefront of regulating and adapting
guidance to help develop and authorize diagnostic tests. It has
also managed the supply chain for critical items like gloves,
masks, respirators, swabs, and ventilators.
And the staff at CDRH have been working day and night to
stay ahead of the virus, and they deserve our recognition and
appreciation. Their work over the last two years has
underscored the importance of ensuring that FDA resources are
in place to make sure we have a safe and effective medical
device supply chain.
The draft agreement that we are discussing today between
FDA and industry will substantially increase funds for CDRH,
which will lead to a significant increase in staff capacity at
the agency, as the chairwoman mentioned.
The performance goals included in the draft agreement will
also allow for innovation through the creation of the Total
Product Life Cycle Advisory Program pilot, or the TAP Pilot.
And this pilot program will allow for earlier interaction
between FDA and developers, and will facilitate regular
engagement throughout the medical device review cycle. And this
will hopefully lead to a sustainable program that builds safety
and efficacy discussions into the front end of development to
speed innovation in a responsible way.
Now, the draft also lays out new transparency measures that
will ensure funds are being spent efficiently and going to the
programs authorized by the agreement in the legislation we
passed. And when I mention transparency, I want to also note
the importance of the process we are undertaking here in the
committee today, and the process Congress has laid out for FDA
and industry to reach the agreement we are now reviewing.
By statute, as part of the MDUFA reauthorization, FDA is
mandated to consult with regulated industry, patient, and
consumer representatives and healthcare professionals, receive
public comment, and submit recommendations to Congress no later
than January 15th of this year. This deadline is not a mere
suggestion. It is actually the law. And the process is
important, because it allows for FDA, industry, and members of
the public to examine what has worked well and where review
programs can be improved through the reauthorization process.
It also provides Congress with sufficient time to thoroughly
review these recommendations, and re-authorize the program
ahead of the funding deadline.
Now, you know FDA just released this draft commitment
letter to the committee last Tuesday, which is more than two
months after the January 15th deadline. FDA has not received
public comment on the draft, and this is troubling, considering
there are serious questions about numerous issues, including
how the agency and industry contemplated the extensions of
programs due to the sunset in their agreement. And there is
still a lot to review and more work to be done, and we must act
quickly. So failure to re-authorize the program on time would
be catastrophic for patients relying on safe and effective
medical devices.
I am just trying to say--I am not trying to beat you up,
Dr. Shuren, but, I mean, the bottom line is, you know, we get
this two months later--we are going to meet our deadline
because we don't want to have the pink slips. But I remember a
few years ago, when the pink slips went out, and everybody was
saying, ``Well, Congress, you know, why didn't you do this
quicker?'' Well, in this case, it is your fault. I mean, I
don't know how else to put it.
So we are not going to miss the deadline, though. And I
appreciate FDA and industry being here today to help us
understand their proposal. And I also think it is important for
us to discuss how we can improve the process so this does not
happen again in the future.
And we will also review two other common-sense proposals:
one bill from Representative Schrier would create a new
advisory panel at FDA to bring an independent public health
focus to regulatory decisions, evolving diagnostic tests, the
importance of which are still being seen during the COVID-19
pandemic; and we have another bill from Dr. Burgess that would
incorporate cybersecurity into medical device applications,
which is also critical as medical devices become more
interconnected and technologically advanced.
So look forward to the discussion today. And I yield back,
Madam Chair.
[The prepared statement of Mr. Pallone follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. The gentleman yields back.
The Chair is pleased to recognize the ranking member of our
committee, Representative Cathy McMorris Rodgers, for your 5
minutes for an opening statement.
OPENING STATEMENT OF HON. CATHY McMORRIS RODGERS, A
REPRESENTATIVE IN CONGRESS FROM THE STATE OF WASHINGTON
Mrs. Rodgers. Thank you, Madam Chair. Today this
subcommittee will hold its third hearing to consider the
reauthorization of the FDA user fee programs.
Congress has acted to authorize the Medical Device User Fee
Amendments, or MDUFA, four times before, and we remain
committed to reviewing this authority on time and through
regular order.
I would like to thank our witnesses for testifying today,
and would also like to welcome back Dr. Shuren. Dr. Shuren came
before this subcommittee when we last re-authorized these
programs in 2017.
Before we discuss the proposed amendments and the two bills
for today's hearing, I would like to join in expressing my
disappointment with the failure of FDA and the regulated
industry to deliver their proposed agreement to Congress by the
January 15th statutory deadline. MDUFA negotiations have been
going on for over a year, and we have had just one week to
review the proposed amendment language and commitment letter
before this hearing. This delay hinders Congress's oversight
responsibilities. Re-authorizing these programs on time is a
goal shared by all of us on this committee, and failure to do
so will result in delayed patient access to needed medical
technologies.
Further, I have raised serious concerns about the lack of
transparency throughout this process. In November I wrote to
then-acting Commissioner Woodcock about the delay in posting
minutes, meeting minutes from FDA industry negotiations. To
ensure transparency and progress, documentation of meeting
outcomes and action items are supposed to be made part of the
official record, and made publicly available. While this
posting minutes publicly takes no more than two to three weeks,
during MDUFA V negotiations we saw delays of more than six
months. Even today, there are no meeting minutes posted for any
meetings that took place after June 30th, 2021.
I know that my colleagues and I are looking forward to
getting answers today on what took so long for the proposed
agreement to be delivered to our committee, and how we improve
this process going forward.
Now, regarding the proposed MDUFA V agreement, as well as
two pieces of legislation introduced by Representatives Burgess
and Schrier, Dr. Burgess's bill ensures the cybersecurity of
devices is approved or cleared by FDA. Dr. Schrier's advances
on the real world impact of medical device diagnostics (sic).
We want to make sure FDA has the resources to keep up with
cutting-edge medical technology, such as artificial
intelligence, robotic prosthetics, and facilitate innovation
and production of the more routine devices we rely on:
syringes, gloves, gowns. We need to make sure these resources
are used wisely and improve people's quality of life.
The promise of American innovation will allow medical
technology to help keep patients healthier, enable treatment at
or close to home, and improve timely diagnostic--diagnosis and
treatment. This reauthorization requires FDA to leverage
digital health technologies and Real-World Evidence in the
review and clearance or approval of medical devices where
appropriate.
The proposed enhancements also direct significant
investment in hiring and retaining world-class scientific and
technical staff. There is no question that the COVID-19
pandemic severely disrupted business for the FDA to review
applications and make timely decisions. The Center for Devices
and Radiological Health has especially had a daunting task. FDA
has fallen behind on the accountability part of the deal,
missing three review goals during Fiscal Year 2020 and six
during Fiscal Year 2021. I hope that FDA will improve going
forward, and that the hiring commitments and performance goals
agreed to under MDUFA V will get us back on track.
I am also encouraged that the commitment letter contains
enhancements to improve performance, accountability, and
financial transparency. FDA is committed to publishing an
annual 5-year financial plan which will include hiring targets
and a full accounting of where user fee funds are being spent.
MDUFA V also continues enhancing its Patient Science and
Enhancement Program, which--excuse me, which will prioritize
including the voice of patients in the review process.
The goal of these improvements will improve pre-submission
communications with innovators, make sure patients are heard,
and improve overall efficiency, integrity, and effectiveness of
medical device reviews.
Re-authorizing MDUFA before September's deadline will allow
agency operations to continue, and will also enhance patients
benefit for medical innovation and advancements. This is the
goal that I know is shared by all of our colleagues.
I look forward to today's discussion. I yield back.
[The prepared statement of Mrs. Rodgers follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. The gentlewoman yields back.
Pursuant to committee rules, all Members' glorious written
opening statements will be made part of the record.
I now would like to--well, he really doesn't need to be
introduced, but I am going to introduce him anyway. Our witness
for our first panel, we all know Dr. Jeff Shuren. He is the
able director of the Center for Devices and Radiological Health
at the FDA.
Welcome back to the hearing room, Doctor Shuren. It is
really wonderful to see you again in person back to the
subcommittee. We are very happy to have you with us today, and
we look forward to your testimony.
You are familiar with the lights, so I don't have to walk
you through that. But a warm welcome. You have 5 minutes for
your testimony.
STATEMENT OF JEFF SHUREN, M.D.
Dr. Shuren. It is nice to be back. Chair Eshoo, Ranking
Member Guthrie, and members of the subcommittee, thank you for
the opportunity to testify today about the fifth
reauthorization of MDUFA.
The investments made in previous MDUFA re-authorizations
have paid off dividends, with an increasing number of
innovators bringing their devices to the U.S. first, and a more
robust pipeline of innovative new devices, which ultimately has
led to more timely patient access.
I want you to know that I personally regret that we missed
the statutory deadline to deliver our recommendations to
Congress. I and the entire agency take this obligation very
seriously. I am pleased to report, however, that the long
deliberations have ultimately produced a strong, thoughtful
agreement on recommendations to Congress that, if enacted, will
continue to advance medical device innovation, while
maintaining the FDA's standards to protect patients.
CDRH continued to meet and exceed most performance goals
through the first half of MDUFA IV. However, we missed some
goals later on. During this time we saw a rise in our workload
for which we were not fully funded. For example, so far, during
MDUFA IV, FDA received over 3,000 more pre-submissions than we
were resourced to review, including more than 1,000 in Fiscal
Year 2020 alone. And since Fiscal Year 2018, FDA has granted
more than 600 breakthrough device designations, more than 200
in the last Fiscal Year alone. Medical devices have and
continue to be increasingly more complex, and the review of
their pre-market submissions more resource intensive, while the
number of submissions we receive annually has increased, as
well. And we expect these trends to continue.
Then COVID hit. It pushed us into a continuous all-hands-
on-deck operations in order to facilitate the development and
availability of pandemic-related medical devices. We have
received approximately 8,000 emergency use authorization and
pre-EUA requests, and we are still receiving about 130 of these
submissions a month. We have granted emergency use of full
marketing authorization to over 2,200 medical devices for
COVID-19, including 15 times more EUAs than all other previous
public health emergencies combined. This has truly been a
perfect storm, and my center has been battling against it for
two years.
Moreover, our efforts to grant emergency use authorizations
are not covered within the scope of MDUFA, so they don't count
toward our performance.
On the other hand, the magnitude of the emergency response
inevitably led to a backlog, and delayed review times, and we
fell short on some of our MDUFA goals. I and my center take
these commitments seriously. We know this has had a great
impact on companies across the country. This is why we have
been transparent, communicating about impacts publicly and
regularly, and we have worked hard to address delays for COVID
and non-COVID devices through hiring more staff and
contractors, reallocation of staff, and changes in policy,
procedure, and practice, with many of my staff burning the
midnight oil and burning out in the process.
We greatly appreciate the support from Congress,
particularly in the form of supplemental funding, and we have
now turned the corner. CDRH has reduced the backlog of non-
COVID device submissions by 44 percent, and we are targeting to
have most of the center back to normal operations later this
year.
Despite these challenges, during MDUFA IV we authorized
record numbers of novel devices, over 100 a year during the
pandemic. The MDUFA V proposal takes important steps to address
resource gaps that began to show before COVID-19, and to
support improved performance.
It also features a new accountability mechanism for add-on
payments under which FDA would receive additional user fees if
it meets specified goals. These additional funds come with even
more ambitious goals for the later years of MDUFA V.
The agreement includes a new voluntary pilot to provide
earlier, more frequent, and more strategic engagement with
sponsors of breakthrough devices, and those included in the
Safer Technologies Program, incorporating lessons learned from
the pandemic, where we saw how engaging with sponsors through
the pre-EUA process to problem-solve and answer their questions
in real or near real-time was critical for facilitating
important technologies coming to market quickly and safely.
The MDUFA V proposal would also support advancement of the
patient perspective in regulatory decisions, continuation--
expansion of the use of national and international consensus
standards, leveraging of Real-World Evidence for regulatory
decisionmaking, and enhanced coordination with international
regulators to advance global harmonization, among other
priorities.
We appreciate Congress's patience and support. Thank you
again for the opportunity to testify today. I am happy to
answer your questions.
[The prepared statement of Dr. Shuren follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. Thank you, Dr. Shuren. We will now move to
member questions, and the Chair recognizes herself for 5
minutes to do just that.
Dr. Shuren, in your testimony you said you have received
approximately--did you say 80,000 or 8,000?
Dr. Shuren. Eight thousand.
Ms. Eshoo. Eight thousand EUA requests during the pandemic.
This has, obviously, strained your center's capacity,
especially since EUAs do not generate user fees.
Are you still receiving a heavy volume of EUA requests in
2022, so far?
Dr. Shuren. Yes, for EUAs, pre-EUAs, it is still about 130
a month.
Ms. Eshoo. A hundred and thirty a month. What is your long-
term plan to balance COVID-19, EUA requests, with your
center's--I guess what I would call your regular workload?
And how does MDUFA V help address the center's capacity
gaps? Because they are--it is really jaw dropping, these
numbers.
Dr. Shuren. Yes, the numbers are phenomenal, and really, a
credit to my team for all the hard work. And I appreciate the
support of Congress in doing so.
So some of the steps we have taken is to sort of narrow the
focus on where we put our resources. We are in such a different
place today as a country than we were at the beginning of the
pandemic. And I think, over the coming months, really, the goal
is to start to turn off the spigot on EUAs, that, you know,
there is enough product out there, and it is now turn and use
more of our resources on the non-COVID products that are there.
Ms. Eshoo. In January you issued final guidance to engage
patients in the design and the conduct of medical device
clinical studies. Since publishing the guidance, have you seen
medical device clinical studies include more diverse patients?
It is an area, on a bipartisan basis here, at our
subcommittee, a commitment to really reform clinical trials so
that they are diverse, because they are not today. Tell us how
you are doing with that.
Dr. Shuren. Well, too early to tell with the new guidance.
That said, I want you to know that one of our strategic
priorities for the center for 2022 to 2025 is advancing health
equity. At the top of that is increasing the representation of
diverse populations in clinical trials for devices.
At the same time, we want to do this responsibly. So one of
the actions we will take is putting out a framework about when
that is absolutely critical, and what circumstances, what
devices, and where that will be helpful to have.
Ms. Eshoo. You don't have any legally binding standards,
though, do you?
Dr. Shuren. We don't.
Ms. Eshoo. You don't.
Dr. Shuren. We do need the evidence to support the use in
intended populations. And quite frankly, if we are going to
provide high-quality healthcare, then no patient should be left
behind.
Ms. Eshoo. In 2021, June 2021, FDA issued draft guidance
that included what FDA sees as the distinction between
servicing and re-manufacturing medical devices. Has that draft
guidance helped clarify the apparent confusion between
servicing and re-manufacturing, at least amongst the entities
that perform these activities?
And does the term ``re-manufacturing'' need further
clarification in statute, which is, obviously, where we come
in?
Dr. Shuren. Well, at this point, because it is draft
guidance, it is still--we are getting feedback. It is not
finalized as official policy.
That said, there is value for providing greater clarity,
and maybe even doing so through statute with further expansion
than through guidance.
When we saw reports come in that there are allegations
about problems with servicing, most of those turned out to be
re-manufacturing. And so clarity about what constitutes and
doesn't constitute re-manufacturing is critically important.
Ms. Eshoo. OK. The Chair now recognizes the ranking member
of our subcommittee for his 5 minutes of questions.
Mr. Guthrie?
Mr. Guthrie. Thank you, and thank you, Dr. Shuren, for
being here.
I will tell you, watching over what happened over the last
couple of years, I know it has to be absolutely exhausting for
you, but it also has to be exhilarating. I mean, you are--the
FDA, in the whole Operation Warp Speed effort, I think, rose to
the occasion. We have things we have to look at and questions
we need to ask as we move forward, but absolutely, ensuring
that we had products out--I know you are on the device side,
but just the vaccines, having the products and the testing that
you--out as quickly as it did, I mean, it just--any time you
have a mission that brings you together like that, exhausting
as it is, has to be fulfilling, as well. And helping American
people get through the pandemic that we are still getting over,
hopefully, or getting--figuring out how to live with it, moving
forward.
But--so just a couple of questions on the agreement as you
were looking--I know a big part of it is the hiring goals, and
some more money for hiring goals moving forward. And so my
question is, how does CDRH plan to meet your hiring goals set
forth in the agreement?
Dr. Shuren. Well, MDUFA V also provides us with additional
funding to take advantage of the Cures Authority for hiring
that was in 21st Century Cures, and I really thank Congressman
Upton for his leadership in moving that bill forward.
So that, and I think the greater flexibility that we are
now offering, in terms of work circumstances with telework and
remote work, is going to help us recruit. And we have seen
better recruiting in the past few years than we saw previously.
But I will put on the table something, if Congress is
interested to help us, is the ability to have direct hire
authority, regardless of whether or not someone is on under
21st Century Cures. So if we find the right person, let's bring
him in as quickly as possible. And that will help us be
successful on implementation of, I think, MDUFA, but all of the
UFAs.
Mr. Guthrie. Yes, thank you. I think that is the--
throughout our--and we have to figure out how to have hiring
that is correct and right. I know we put a lot of these in
place, little things that, in the way past, were political.
But I can tell you, from my VA, local VA clinics, this
was--when hospitals are lining up for nurses graduating from
nursing school, and we have to go through the process we have
to go through, then it makes it difficult to get people to--I
am sure you are competing with the same kind of groups. We need
to look at that, or the proper committee needs to look at that,
as well.
So I mentioned earlier about the signaling, merging signal.
Could you explain how CDRH's emerging signals process works?
And will you commit to working with me and other members of
the Committee on ways to address concerns about the
manufacturer input during this process?
Dr. Shuren. So we have not only policy that has been
issued, we have an entire program that is focused on what we
call signal management.
So if we get an indication there may be a problem with a
device--it could be through an adverse event report, a study
that is published out in the literature--we will then go ahead
and do an assessment on that. We have a whole process for how
we do that review, and then make decisions around, if this
requires more data, is this sort of a real signal or not. And
then if so, what is the appropriate action to take?
Part of that includes, in certain circumstances, putting
information out on what we call an emerging signal, because
this is really important to get this information out to the
public. As a part of that process, we generally engage with the
manufacturers in that signal evaluation process. And then, if
we are going out with the communication, we give advance notice
to the manufacturers, and we tell them about the general
content of the communication, unless there is--it is not
feasible. There are so many manufacturers--like we did with
warnings about using masks with metal if you are having an MRI
scan.
But those communications do need to be FDA communications.
They need to be--we need to be independent. If we are back
sharing it, and then we are going to end up in negotiations
with companies, and we need to avoid delaying tactics, where
companies that try to, if you will, preempt us, and put their
own spin on the science, that will undermine public health. It
is absolutely critical we have our independence to get
important information out to doctors and patients so they can
take appropriate steps.
Mr. Guthrie. Thank you. I appreciate that. That is
something--absolutely.
Also, could you explain the differences between pre-
submission program and the Total Life Cycle Advisory Program,
and how you ensure the TAP program doesn't divert resources
from other important programs? You have about 30 seconds for
that.
Dr. Shuren. Yes, so pre-submission is very popular, very
important. And over half of them are requested by small
companies, startups. Here, important questions that really take
more time to answer or provide to us. And then we review if it
is appropriate. Then, you know, within 70 days we are going to
provide--or at least five days before meeting--written
feedback. It is this stage gate approach.
If you really want to engage in problem solving, what TAP
does, it says, rather than the stage--questions takes time,
more questions come back. We work with that developer of
innovative technology in a fluid manner, trying to answer
questions as close to real or near real-time as possible, and
have the capacity to engage in strategizing with the company on
how to get to yes. Obviously, the data has got to support that
it is safe and effective.
But this is to address the challenges with that valley of
death. We really go from concept to market, go beyond what we
have in the MDUFA today, which is just focused on pre-market
review. If we can solve the challenges before you send us a
submission, we are not talking about saving days, we are
talking about saving months and years----
Ms. Eshoo. Years.
Dr. Shuren [continuing]. And getting to yes more
efficiently. TAP can be a game changer, and this is what we
learned from COVID that really works. It is part of the secret
sauce that got those 2,200 devices out onto the marketplace so
quickly.
Mr. Guthrie. Thank you. I appreciate your work.
I yield back----
Ms. Eshoo. The gentleman yields back. The Chair now
recognizes the chairman of the full committee, Mr. Pallone, for
his 5 minutes of questions.
Mr Pallone. Thank you, Chairwoman Eshoo.
Dr. Shuren, you know--you could tell from my opening
statement that I don't want to be--you to send out pink slips
again. And, you know, my concern, obviously, is, you know,
people start looking for other jobs, and the process of
approving medical devices gets delayed. So can you describe
what would happen to your center at FDA and to the medical
device supply chain if we enter August or September and
Congress has not acted? What would this mean for patients, if
you will?
Dr. Shuren. And again, my apologies for our being late. I
know it puts Congress in a very tough bind. But if it is not
authorized in time, then we have to move forward to issue those
pink slips, and we start letting people go, and we wind down
the program.
The program is absolutely essential for assuring that we
get safe and effective technology to patients. If we are under-
resourced, it is going to take more time. There will be delays.
We will start losing the edge we have got now in medical device
innovation here in the U.S. with more important technologies
coming here first. We will lose all of that, and we will not be
well positioned to also protect patients from unsafe products.
Mr. Pallone. Thank you. And, you know, I don't want to keep
dwelling on the delay here, but, you know, maybe what we should
talk about is how we can improve this process going forward.
So you, obviously, were one of the participants in these
negotiations with industry. Can you help us understand what
caused the delay this time, and provide any ideas on how to
improve the process when it is time to re-authorize again, you
know, five years from now?
Dr. Shuren. I have been involved in MDUFA re-authorizations
since 2005. So, you know, we got a late start, too. And this
was us and industry both said, ``We are getting hammered with
COVID. We need more time.''
One thing Congress could do is maybe, rather than just have
the date about when you have to come to Congress, have the date
when we have to sit down and get this started. You know, so we
have got enough lead time, you know, to get it done. And maybe
then, you know, think about--we could be a bit more accountable
publicly if we are going to be late.
And I appreciate, too, our delay on the meeting minutes.
That puts you in a tough bind, as well, to make well-informed
decisions.
Mr. Pallone. All right, thanks. I wanted to note that, as I
think has already been discussed by you and the chairwoman,
that the proposed MDUFA V significantly increases funding above
what was laid out on MDUFA IV. So could you explain why this
increase in resources is necessary, how it will help with
product reviews, and how FDA determined what resources were
needed this time to ensure the agency is funded over the next
five years?
Dr. Shuren. Well, one of the challenges was, you know, as I
mentioned in my opening statement, is under-resourced in MDUFA
IV. Look, we make our best estimates on what the costs are
going to be, but there is no way to really adjust that as we
move along. And some things are just, you know, out of control.
But at the same time--so what MDUFA V is going to do, deal
with those gaps, but give us the ability to further improve our
performance, which is important. It is going to create that
pilot. We are going to test drive TAP, and that, to me, is a
major game changer. But I think we are doing it responsibly. Do
a pilot. Learn from it. See if it is worth keeping, and go from
there. And then greater investments to do more work on bringing
the voice of patients into the picture, we will continue to
have funding for Real-World Evidence, and then better leverage
that moving forward, better use of national and international
consensus standards, and drive toward greater international
harmonization.
This is really--I view it as, like, the next frontier,
where we need to go for a program.
Mr. Pallone. All right. Thank you.
And I know we are running out of time, Madam Chair, so I
will yield back.
Ms. Eshoo. The gentleman yields back. The chair recognizes
the ranking member of the full committee for her 5 minutes of
questions.
Mrs. Rodgers. Thank you, Madam Chair.
In December, FDA published two draft guidance documents to
provide the agency's policy for device manufacturers planning
to transition products granted emergency use authorization
during the pandemic to regular marketing submissions. These
guidance State that products currently marketed under an EUA
would need to submit a pre-market application and change their
product labeling within 180 days of the end of the public
health emergency.
Manufacturers have expressed that this is not sufficient
time to submit applications, particularly for those that are
still gathering clinical data. Others raised concerns about the
burden that updating the label twice is going to raise, once
during the application review, and then again during the
approval decision. This is going to be a burden on
manufacturers.
I wanted to ask, is FDA taking these concerns into account,
so as not to make supply chain challenges worse and hurt
patient access to devices that will continue to be needed, even
once the public health emergency has ended?
And then, can FDA even process an influx of applications
within 180 days, and meet MDUFA goals?
Dr. Shuren. So we are taking all the feedback we are
receiving into account.
And I will mention, you know, during the pandemic we issued
28 guidances, but most of those guidances were immediately in
effect because, as a public health emergency, we wanted to move
quickly. We made the decision that, for transition, it was
absolutely critical we get public input before we finalize. We
made an exception in this case, because we wanted to hear from
manufacturers and others, and we do want to get this right.
I will say I also encourage manufacturers, don't wait for
us to tell you at some point in the future you need to come in
with a pre-market submission. You are out there in the
marketplace. If you want to stay on the marketplace in the long
term, get your data, come in the door, and we will--of course,
if you submit a data for an EUA, we are going to be leveraging
that in our final decisionmaking, too.
Also, if you come in the door, remember the product is on
the market. So it doesn't matter if it takes a little bit
longer to review a pre-market submission. We are more focused
right now getting new product on the market. The transition
devices will be second, but we are not going to disenfranchize
anyone. No product would come off if something is in the door.
Mrs. Rodgers. OK, OK. Thank you.
As I mentioned in my opening statement, I have expressed
concerns about the lack of transparency throughout the cycle of
MDUFA and the negotiations. The requirement for FDA to publish
meeting minutes is a--is in place so that policymakers and the
public can monitor the status of the user fee negotiations in
near real-time, not months later, and stay informed about the
key issues. They aren't optional, and we expect them to be
published quickly.
FDA has not published meeting minutes since June. How many
negotiation meetings have been held since June 30th, 2021?
Dr. Shuren. I will get back to you with the number. But I
have to say a lot. So----
Mrs. Rodgers. Can you estimate how many?
Dr. Shuren. I am going to say over a dozen.
Mrs. Rodgers. OK. Would you speak to why the meeting
minutes were not published on time?
Dr. Shuren. First of all, I will again apologize for that,
because we should.
I have to tell you, negotiations on MDUFA, it is more like
an international treaty: lots of parties, lots of perspectives.
And the same happens with the meeting minutes. There is a lot
of back and forth on them. I don't mean that by way of an
excuse, but everyone wants to be comfortable with what is in
there. Folks were so focused on let's get the deal wrapped up.
And as you know, we went late, and we were all pushing, and we
wanted to get accord. We felt it was important to get
consensus, and that meant more discussions to do it. So we put
the priority with our limited, you know, bandwidth on getting
the deal done in the meeting minutes. But again, my apologies
because that does put you all at a disadvantage.
Mrs. Rodgers. OK. Well, we are missing a lot of information
because of that. And we--and Congress has made multiple
requests. Can you speak to how many times FDA and industry met
in December and January leading up to the January 15th
statutory deadline?
Dr. Shuren. Somewhere in January--there were offline
discussions, not a lot of in-person meetings while other
information was being gathered and other issues were being
dealt with. I don't have the exact number, but I will--I can
get back to you with all of those details.
Mrs. Rodgers. Thank you. I just want to conclude by
expressing concerns about a proposal in the President's budget
that would significantly expand the scope of mandatory device
supply chain reporting requirements that were just put in place
for the first time during the pandemic.
With less than two years since FDA was first given this
authority, I am unaware of any study or review that has been
conducted to understand the benefits and the burdens of this
data collection. I am open to understanding how FDA can better
utilize its current flexibility authorities to efficiently
review changes to components or sourcing. But imposing sweeping
government mandates and more paperwork requirements on
businesses is only going to disincentivize innovation and
reduce competition.
I yield back.
Ms. Eshoo. The gentlewoman yields back.
Dr. Shuren. Could I respond to that? Because I--if it is
possible?
Ms. Eshoo. Sure.
Dr. Shuren. Just to say I appreciate that.
First off, the authority, that broader authority, as you
say, already applies for drugs. We are asking for parity on
that. We have used--and I want to say thank you for the
authorities in the Cares Act, because we used that during the
pandemic. Those notifications helped us prevent or mitigate
shortages with test supplies, and ventilators, surgical masks,
respirators, dialysis systems, defibrillators, even needles and
syringes being used for vaccines.
The problem is shortages occur outside of a public health
emergency. In fact, for a public health emergency--in COVID it
started before the public health emergency was declared. So we
were behind the eight ball because of that. And that hurts our
frontline workers. It hurts patients. And even during the
pandemic, we had a shortage of resin because of a winter storm.
The only reason we got notified is because it happened in a
pandemic, which helped us prevent large-scale shortages of
tests. If this was not in the setting of a public health
emergency, no obligation to tell us, and patients will get
hurt.
We know--we have dealt with shortages for years, but we
have not--we need this authority. We were flying blind without
it. When the pandemic hit, without that authority, and before
public health emergency, we had to reach out to about 1,000
manufacturing facilities over 12 countries, cold calling them.
And we got maybe responses in about a third, and often
incomplete responses. And that put people's lives at risk.
This is something simple to fix. We don't want to be over-
burdensome, but at least parity with the drug program.
Mrs. Rodgers. Well, this merits a longer discussion.
Medical devices are different than drugs, and I think we need
to consider that.
Thank you. I yield back.
Ms. Eshoo. Before the reforms, the approval by--the
approvals by FDA were based on the yardstick by which
pharmaceutical drugs were measured. So, you know, we really
have made progress.
Colleagues, we are now going to recess for Congressman Don
Young's memorial, and we are going to resume at one this
afternoon when members, of course, will continue to question
Dr. Shuren, and to host our second panel. So, Dr. Shuren, you
have time for breakfast and lunch. How's that? And we will see
you back at one.
Dr. Shuren. All right, thank you.
Ms. Eshoo. We will be in recess until then. Thank you,
everyone.
[Recess.]
Ms. Eshoo. The Health Subcommittee will come back to order.
Thank you again for your patience, Dr. Shuren. And I
believe--who is next? The Chair recognizes the former chairman
of the full committee, a great member of this subcommittee, the
gentleman from Michigan, Mr. Upton.
Mr. Upton. Well, thank you again, Madam Chair, for holding
this hearing. And we all regret the loss of our good friend and
colleague, Don Young, which is why we broke for his private
service, with many of us there attending.
Dr. Shuren, I really appreciate your leadership,
particularly over the last couple of years. You were--for those
that don't know, you were a major help as we got 21st Century
Cures done. Not only did you travel around the country, but you
helped us in a number of roundtables to make sure that we did
it right. And the proof is in the pudding. And we are very
pleased with a good number of the results since President Obama
signed that bill into law.
I guess I have got, really, two questions. I hope I can get
through both of them while we are here. I have heard from a
number of the medical device manufacturers and, as you might
know--I am sure you are aware--they are very concerned about
the potential on these new regulations that may be coming out
as it relates to the surveillance once they are done. They are
very afraid that, in return for the faster approvals--and they
did this with the EUA--that it would shorten the time to get
some of those out. But they are concerned that the hammer may
be out there for a long time, perhaps afterwards. And I just
want to get maybe a couple of quick comments from you, and
maybe just have the opportunity down the road.
I don't have language, or--but I just wonder if you could
work with us as we relate it to those potential changes. I know
that there would always be a comment period, et cetera, but I
just wonder if you might be able to look at some constructive
ideas that would alleviate some of the fears that the device
industry might have as it relates to these. I don't even know
if there are proposed regs yet. I don't know if it is--if they
are actually out or not. But if you could just sort of walk us
through that process, that would be helpful to
Dr. Shuren. And just to clarify on surveillance, is this in
terms of the--you had mentioned with EUAs, is this on the
transition to EUAs?
Mr. Upton. Yes, the mandatory reporting--the manufacturers
are experiencing increased demand or having issues with
components that are life-supporting, life-sustaining, or
intended for emergency medical care during surgery. These would
be targeted toward the devices in terms of the reporting of
issues that they might have after they were approved.
Dr. Shuren. Yes. So that pertains to, you know, proposed
legislation that is really in Congress's court that goes back
to supply chain shortages.
Mr. Upton. Right.
Dr. Shuren. And in shortages, we are always talking about
is there permanent discontinuance of the device, or is there a
meaningful disruption in the supply, and we are just
clarifying. One of those circumstances is where the demand
really goes up, and the manufacturer cannot make, you know,
sufficient--and there is going to be a real shortage with
meaningful, meaningful impact.
We saw that in COVID. You remember, with personal
protective equipment, the needs for healthcare workers
skyrocketed, and we had massive shortages of those products.
And it made a big difference. In fact, the question came up,
you know, devices are different than drugs.
I would kind of put to you, ask our healthcare workers how
important it was to them that they have, like, N95 respirators.
Mr. Upton. Great.
Dr. Shuren. Our doctors and nurses. And they didn't have it
in the beginning of this pandemic. And some of those issues, in
fact, started before even a public health emergency. So here is
a case where one of the causes is demand goes up way above
supply. And it is another example of issues that start before a
public health emergency and why, too, we don't want to limit it
to just those circumstances.
Mr. Upton. But are there some regulations, then, that are
pending as it relates to the reporting of issues or not?
Dr. Shuren. I think this is in reference to what we put out
for our--in legislation. But we always are continuing to
provide greater clarity on reporting that is in the CARES Act.
But here we have talked about making sure that, if we are doing
something in supply chain, let's be clear on the circumstances
that are important, that are leading to it.
Mr. Upton. Great. So I may come back with maybe a letter,
and try to----
Dr. Shuren. We are happy to have--talk about this, because
we want to get to the right place. This is a major problem for
the United States----
Mr. Upton. OK, so----
Dr. Shuren [continuing]. And for healthcare.
Mr. Upton. The last question I want to ask quickly is that
a common refrain that we are hearing from patient groups is
that CMS is taking a long time to make payment decisions on new
drugs once they make it through the approval process at FDA.
While I know that FDA is part of the payment process decision,
are there ways that FDA and CMS can better communicate so that,
once a drug or device is approved, it can make it through the
payment process more quickly?
Dr. Shuren. To date, you know, we have a very good working
relationship with CMS, and there are a number of opportunities.
For example, we have our parallel review program, the chance
for a manufacturer to ask to meet with CMS and us in advance to
kind of get our expectations for what it takes for FDA approval
and for CMS, you know, coverage determination.
We are also working through the Medical Device Innovation
Consortium, and CMS is a part of that. And there is already a
workstream regarding to reimbursement and things, too, to
facilitate. And we stand ready to work with our CMS colleagues
on whatever is helpful to them to sort of streamline that
pathway from FDA approval to Medicare coverage.
We know in the U.S. one of the big drivers, either to help
or to harm innovation, is to have, you know, predictable
pathways for reimbursement. Certainly, that is a broad
challenge here in the U.S. and, again, something we are very
happy to----
Mr. Upton. We are looking to try and help with the Cures
2.0 as part of that.
With that, Madam Chair, I yield back my time.
Dr. Shuren. Thank you----
Ms. Eshoo. The gentleman yields back. That is a--it is a
huge issue. And I am glad that you are attempting to align and
have cooperation between the agencies. I don't know what it is
producing, but it is a constant complaint, and it is a
legitimate complaint. So thank you for what you are doing, and
anything that you can--you think that we can get into the
legislation which would advance this case, I know that you will
work with us.
The Chair now has the pleasure of recognizing the gentleman
from Maryland, Mr. Sarbanes, for 5 minutes of questions.
Mr. Sarbanes. Thanks very much, Madam Chair.
Dr. Shuren, thank you for being here today. I appreciate
your testimony. Obviously, it is very important as we are
considering the MDUFA performance goals letter, and Re-
authorizing the Medical Device User Fee Agreement.
While we have you here, I was interested in your
perspective on the importance of increasing clinical trial
diversity, and ensuring that trials for medical devices better
reflect the patient population that might utilize the device in
the future. We sometimes don't think about that in this context
as much as we do in other contexts.
Can you talk about the importance of enrolling trial
participants that reflect the intended patient population of a
device?
Dr. Shuren. Now, we consider this a critically important
area. If you want to know if the device works, it is intended
for a particular population, you have got to go ahead and, you
know, assess it in that population. We have put this as one of
our strategic priorities over the next years as part of our
advancing health equity.
It is also reflected in the MDUFA V agreement, where there
are commitments around increasing, for example, participation
of patients, you know, across broad populations in device
trials. And that includes leveraging technology as a way to get
more patients enrolled in clinical studies. If they don't have
to come out of, for example, their home setting, it will make
it easier for data collection, and that will make it easier
across populations who otherwise have been feeling more
disenfranchized from the ability to participate in clinical
studies.
Mr. Sarbanes. Has FDA had the opportunity to kind of pilot
that in any significant way, and see what the benefits of the
technology are? Could you describe some of that in a little
more detail?
Dr. Shuren. There is already work underway, and we have
tried to facilitate the use of such technology in the setting
of COVID, because we knew that it would be more challenging for
people who otherwise would be enrolled in a clinical study to
get to a clinical trial site, and so have really--have put out
guidance on this.
And there is more that we, as an agency, will be doing in
this space to, again, facilitate these sort of remote clinical
trials. And a linchpin for it is technology.
Mr. Sarbanes. It is another example--we have been seeing
this across, it seems, every arena, that the pandemic push us
to new opportunities that we can then seize upon and deploy in
a more permanent way going forward.
Talk to me a little bit about the relative responsibility
with respect to inclusive and representative trials between the
FDA, on the one hand, and industry on the other.
Dr. Shuren. Well, industry will come to us--for clinical
trials that pose a significant risk. We get a submission to be
able to review in advance, and also companies come to us
through the pre-submission process to seek our advice.
One of the things that we think could be helpful here is to
provide clarity on a framework for those circumstances for
technology where it is important that a diverse population is
included in the clinical trial. That could help facilitate
manufacturers assuring that, if you will, their clinical trial
is fit for purpose, for the intended use for the technology
that they wish to get authorized.
Mr. Sarbanes. Are there things that you think we can be
doing in Congress to incentivize and encourage greater
diversity when it comes to the clinical trial side of things?
Dr. Shuren. I think this is something I really would like
to take back to the agency. This certainly goes beyond medical
devices, and I want to make sure that we are speaking with one
agency voice, since this affects lots of different products.
Mr. Sarbanes. Well, I appreciate it. I want to thank you
for your testimony. Obviously, as you can tell, I am interested
in how we increase the use of digital health technology to spur
greater trial participation. You have alluded to that being one
of the goals in MDUFA V, and I certainly appreciate that.
So we will keep an eye on it. And if you generate some
interesting data in--as you begin to pilot this, and invite
industry to bring a perspective to it as well, [inaudible] with
us, because it may inform our ability to do some things here on
the policy side.
Thanks very much, Madam Chair.
Ms. Eshoo. The gentleman yields back. The Chair is pleased
to recognize the gentleman from Virginia, Mr. Griffith, for
your 5 minutes of questions.
Mr. Griffith. Thank you very much, Madam Chair.
Doctor, at the beginning of the COVID-19 pandemic the CDC
tests for COVID were not accurate. What role did the FDA play
in the approval of these faulty tests?
Dr. Shuren. Well, the test itself, the design of the test,
was fine. And so we authorized that test. But, you know, from
our review there was an issue around the manufacturer. We
believe that there may have been contamination that occurred in
later batches of the test that was produced.
Mr. Griffith. And will you provide this committee with the
FDA's after-action analysis and report on the various causes,
whether it was manufacture or otherwise, of this significant
failure?
Dr. Shuren. I am happy to provide you what information we
can. We did not have an official report of--coming from the
agency. But I did have the director of our in vitro diagnostics
office we had sent over to the CDC to facilitate looking into
this matter. He has many years' history of developing tests,
both in the laboratory and at commercial manufacturers. And----
Mr. Griffith. If you could share that with us, I would
appreciate it.
Dr. Shuren. I would be happy to.
Mr. Griffith. Switching gears a little bit, how many
emergency use authorizations were granted in the last two years
vis a vis the 2-years prior to that?
I don't expect you to have that answer here today, but
could you provide that to the committee, as well?
Dr. Shuren. Yes. I think, if you are talking about all
medical devices, I think we are somewhere over 870 EUAs
granted.
Mr. Griffith. During the last two years?
Dr. Shuren. In the last two years.
Mr. Griffith. OK. If we could just get that----
Dr. Shuren. We will double----
Mr. Griffith [continuing]. Comparison of pre-COVID and
post-COVID, what the use of that was. All right.
[The information appears at the conclusion of the hearing.]
Mr. Griffith. Digital health is an important component of
MDUFA, and in the MDUFA commitment letter I am glad the agency
will put such a strong focus on this important area. But I
wonder whether device, drug, and biologic centers will operate
in silos which could hurt digital health innovation because of
inconsistent regulations.
What specific actions will the agency take to ensure that
this does not occur?
Dr. Shuren. One of the steps we took is to create a digital
health center of excellence out of the Center for Devices,
which serves as also a resource and a convener for the rest of
the agency. And we have already an intra-agency group that
serves to advise the center and to facilitate coordination
between the different parts of the agency on cross-cutting
matters relevant on some of these aspects for digital health.
Mr. Griffith. All right, I appreciate that.
It has also--a little bit different, but in the same area,
it has come to my attention that a significant challenge
associated with incorporating digital technologies in
healthcare is distinguishing between a medical device which
requires FDA approval and a consumer product which falls under
the FTC's jurisdiction.
Do you agree that there should be more cooperation between
these two agencies, as we determine how best to regulate
devices that can be helpful in ourhealthcare?
Dr. Shuren. Well, we do have a good working relationship
with them, and there have been a number of cases with just
medical--with products more generally, where there has been an
issue on, you know, which side of the line it sort of falls.
And we have coordinated with them, such as on cribs, risk of
strangulation on cribs. And we will continue to do so, because
that is an important relationship.
I do think the committee was very helpful in 21st Century
Cures, for example, on clarifying certain circumstances where
the software is not a medical device, and so falls on the other
line. And that clarity then helps, you know, for these
jurisdictional issues.
Mr. Griffith. All right, I appreciate that.
The MDUFA commitment letter also describes several
activities the FDA plans to undertake to support better
harmonization among medical technology regulators across the
globe. What international harmonization efforts are currently
underway?
Dr. Shuren. Right now this is all through the International
Medical Device Regulators Forum. And there is a particular
focus right now on harmonization pertaining to artificial
intelligence--biggest focus on machine learning.
Mr. Griffith. And how do you see this work evolving in the
future, specifically referencing artificial intelligence and
the support of that in decisionmaking and in clinical work?
Dr. Shuren. It has become increasingly more important in
the work that we do. We have already authorized, you know, over
300 devices with AI ML capabilities--just 50, I think, you
know, in the last year.
So we have a whole action plan that goes through a number
of steps we are taking to sort of facilitate the development of
AI technologies, and to ensure they are safe and effective.
Mr. Griffith. And one of the things we have to work on as
we work on AI is to make sure that we are using that, and
helping to bring down healthcare costs, because there are a lot
of things, if it is not something serious, that we could
actually use AI, as opposed to actually using one of our
healthcare providers, who--onsite. So if you combine AI and
telemedicine, we could do an awful lot to bring down costs and
bring service to people who may not otherwise have access to
the medical care that they deserve.
Dr. Shuren. No, we agree. I will say one of the challenges
we face is that, you know, the device frameworks, you know,
that are in place, they are about 45 years old. So they were
really designed for, literally, my grandmother's technology.
You know, it is hardware-based, and we are talking about
software. And it is just not lined up, you know, with the
innovation cycles that you see.
And I would personally say I wish I had the flexibility
that we have in COVID on tailoring the pathway to the
technology in the least burdensome way, and have that ability
to do it in peacetime. Not change the U.S. standard of market,
but have the flexibility to offer it voluntarily, you know, and
then you pick the traditional route, pick the new route. And,
you know, if we don't do that in this software area, like with
artificial intelligence, we are going to kill important
technology that will make a big difference to patients. That I
do worry about.
Mr. Griffith. Let us know what we can do on that.
I yield back, Madam Chair.
Ms. Eshoo. The gentleman yields back. The Chair recognizes
the gentlewoman from Michigan, Mrs. Dingell, for your 5 minutes
of questions.
Mrs. Dingell. Thank you, Chairwoman Eshoo and Ranking
Member Guthrie, for having this really important hearing today.
Since 2002, user fees have supplemented funds appropriated
to FDA to support timely review of medical device pre-market
applications, facility registrations, and other activities.
These funds enable FDA to hire more staff that have the
necessary subject matter expertise to review the complex data
that, as a result, applications may be reviewed in a shorter
period, shorter amount of time, while FDA standards for safety
and effectiveness--and that matters--are still met.
For the first time, MDUFA V, FDA, and the industry have
agreed to an increase in fees for the last three years of the
new cycle if the goals are met in the first three years,
beginning in 2023. If FDA meets the initial goals and fees
increase, the corresponding review goals for FDA in Fiscal Year
2025 through 2027 will also escalate.
Additionally, if FDA doesn't meet its hiring goals,
registration fees would be reduced. This should create
additional incentives for FDA's Center for Devices to review
pre-market submissions by the agreed-upon goal dates. But Dr.
Shuren, I do have some questions.
Since user fees were first considered decades ago, there
have always been questions about whether payments by regulated
industry to the regulating agency create a potential conflict
of interest. How does the CDRH assure that the fees and the
goals agreed upon in the MDUFA only impact review times, and
not review outcomes?
Dr. Shuren. Well, we do assure, you know, that is baked
into the agreement. We are making no commitments regarding
policy decisions. We make no commitments on decisions regarding
individual products. This is basically the fee for service.
The other is--you mentioned the add-on payments. And they
will only kick in for more money. At least we are not talking
about cutting funds if we have a net performance.
But the other thing that is sort of assured is that, with
additional funds that may lead to faster review times, it
doesn't undermine the quality of the decisions that we make
because this is a bit of a queuing issue. And so, if we have
more people, we are able to do things, we have more people to
spread it out, we can reduce the overall time on a review.
The other added advantage is that it allows us to bring on
board more experts, and we have a deeper bench on expertise
like around digital health--assures that we make, you know,
well-informed decisions.
Mrs. Dingell. So do the new performance incentives present
any risk that speed may sometimes have a negative impact on the
quality of the pre-market review?
For example, is it more difficult to identify and explain
submission deficiencies for a greater number of submissions in
a shorter period of time?
Dr. Shuren. Well, I do think, if we are identifying
deficiencies, then--and we do have enough time to identify them
if we have the added people for doing the work. Like I said, it
is a bit of a--it is a queuing issue. And the reason why there
is a certain timeframe isn't because you take a file and you
spend 100 percent of your time reviewing it. Our reviewers have
a stack of files sitting there, and they are looking at one,
they are moving to the other, and that is why it takes a
certain amount of time.
Some of this, if you have more people, they have fewer
files on their desk, they can spend more time on the file, and
it takes less time. That is not taking away from their ability
to identify deficiencies and communicate those.
Mrs. Dingell. Thank you, because I worry about it.
Also, can you discuss how the new performance improvement
adjustment can come about, and is there evidence supporting
this incentive structure?
Dr. Shuren. Well, this was discussed as an accountability
measure for the FDA. In fact, there are a number of things
baked into MDUFA V to increase the level of accountability on
the agency, the add-on payments being one of them. This is the
first time we are doing it in any of the user fee agreements.
And I do think, you know, we will get experience from this.
But we felt that this would be a reasonable thing to try in
MDUFA. And we worked with industry to design it in a way that
we think can support our being successful.
Mrs. Dingell. Thank you. Thanks for your response. I look
forward to discussing these issues further, as well as ways to
improve post-market surveillance, as my colleagues have
mentioned, in the weeks and months ahead.
I yield back, Madam Chair.
Ms. Eshoo. The gentlewoman yields back. It is a pleasure to
recognize the gentleman from Florida, Mr. Bilirakis, for 5
minutes.
Mr. Bilirakis. Thank you, Madam Speaker, I appreciate it
very much.
Dr. Shuren, your testimony mentions the popularity of
Breakthrough Devices Program with significant growth in pre-
submissions for breakthrough-related devices. Can you tell me
about how this MDUFA agreement expands upon the successes of
that program, and how the new product Life Cycle pilot will
help innovators earlier in the development?
Dr. Shuren. So the funding that we are going to get from
industry is going to allow us to hold more pre-submission
meetings within the specified timeframes. And that is an
advantage to anybody who takes advantage of that program. And
it is very popular, and that is why we have seen, you know, the
number of requests continue to go up, because manufacturers
find it very helpful to have those meetings.
TAP moves away from that sort of stage gate approach and
longer time for meetings, trying to make this a much more fluid
interaction with the innovators of very important technologies
like breakthrough devices, to try to--and also give us the
capacity to not just give feedback, but----
Mr. Bilirakis. [Inaudible] asking questions----
[Pause.]
Dr. Shuren. To also problem-solve with the developers. So
the goal here is let's deal with not just the issues around
pre-market review, shorten that timeframe, but focus on what is
even more impactful, what leads up to the pre-market
submission. And if we can work with developers in more real
time there and problem-solve, we shorten that time from,
really, concept to pre-market submission. And if all things
look good in a pre-market submission, we are actually in a
position to maybe even review it more quickly because there
aren't issues, we have dealt with them beforehand.
Mr. Bilirakis. Thank you, Doctor. I appreciate it. Another
question for you. I want to ask you about the use of both
unique device identification, UDI, numbers and the national
drug codes on certain over-the-counter medical devices for
reimbursement purposes.
This impacts, like, again, the items like the test strips,
needles, and syringes, which are critically important to be--
again, to help patients manage chronic conditions, so very
important.
For years, FDA has exercised enforcement discretion to
allow both numbers on the label--both numbers on the label.
Since the UDI number cannot currently be used for reimbursement
purposes, I think it is time to find a permanent solution. I
believe you probably agree with me. For example, FDA could
allow both numbers to remain on the label permanently, or until
such time that the reimbursement systems support using the UDI
number.
Will you work with the committee to find a permanent
solution?
If not, are you planning to at least retain enforcement
discretion to reduce uncertainty in the industry, and keep
patient access to these OTC devices?
So if you could answer that for me, I would appreciate it,
Doctor.
Dr. Shuren. We have had outreach regarding UDI and the NDC
code. Of course, there has been some talk about changes in the
NDC code, and the implications there. And so we are looking at,
you know, opportunities to assure we do not disrupt the
marketplace, as you have raised, you know, one of them being
continued enforcement discretion.
That said, we would be very happy to have conversations on,
you know, what is the--what really is the right solution at the
end of the day.
Mr. Bilirakis. Please, please. Let's followup on that.
Thank you, Doctor.
Thank you, Madam Chair. I will yield.
Ms. Eshoo. Good to see you, Gus, real close to the camera.
Nice glasses.
[Laughter.]
Ms. Eshoo. The Chair now recognizes the gentlewoman from
California, Ms. Matsui, for your 5 minutes of questions.
Ms. Matsui. Thank you very much, Madam Chair, and thank
you, Dr. Shuren, and, ultimately, the industrial witnesses, for
being here today, as well.
When the COVID-19 pandemic began, FDA was able to utilize
and fine-tune the emergency use authorization process to
authorize over 100 different diagnostic tests by the summer of
2020. I commend the agency for their work in this area.
However, I understand these tests with different technologies
or platforms have been validated in a variety of ways and
varying levels of accuracy.
Of course, hindsight is 2020, but it seems that in the
future there may be a more effective and efficient way to
develop and utilize accurate diagnostic tests against a highly
infectious virus. To that end, along with MDUFA, the MDUFA
agreement, today we are discussing the Diagnostic Device
Advisory Committee Act, legislation that will establish a panel
of experts on diagnostic devices at FDA.
Dr. Shuren, what lessons has FDA learned from COVID-19, in
terms of development, validation, and use of diagnostic testing
as part of the coordinated public health response to a
pandemic? Dr. Shuren?
Dr. Shuren. Well, thank you for the question. Let me
mention maybe three things, because, quite frankly, to date we
have issued about--a little over, I think, 450 authorizations
for tests and self-collection kits. And we should never be in
that position again.
If you want to solve it, pre-position manufacturers of
tests in advance of the public health emergency. Have contracts
with them, so that when they are asked, they are set to do it.
And you do it with manufacturers who make these kind of
technologies, and they can make a lot of it very quickly. That
is what South Korea did. They even had two companies who
started to make tests before they even got asked.
Second, de-risk the enterprise. You know, we did this for
vaccines. We pumped all this money in to take the risk off of
production. You knew you weren't going to get reimbursed. That
didn't happen, you know, with diagnostics. And so you had
manufacturers who are, ``I don't know if there is a
marketplace,'' and they were skittish about going into it. We
had to convince some of them to even make tests. So what you do
is you have guaranteed minimum purchasing agreements if you get
authorized, and guaranteed reimbursement. South Korea did that,
as well.
Third, I would say, we found that, rather than having the
companies validate their tests or do all of it, have it done
independent of them. You know, because, in the beginning,
conserve your resources for the material you need to validate.
And you can assure it is done right and it is done quickly. We
wound up doing that for antibody tests, and now for over-the-
counter antigen tests. South Korea had that set up with their
CDC. And so, if there is funding to go do that, the country
could be able to make these decisions also a lot faster.
Ms. Matsui. Could I ask you----
Dr. Shuren. So a few developers, large numbers, quick
decisions.
Ms. Matsui. Well, could I ask you, could the agency utilize
a panel of experts on diagnostic devices to assist in future
public health crises?
Dr. Shuren. Well, expert--outside expert, you know, input
is, you know, always helpful, and we look for those
opportunities to bring them involved. And so this is something
we would be very happy to have conversations about regarding
the proposal, and work with all of you.
Ms. Matsui. OK. The CARES Act of 2020 sought to prevent
shortages by requiring device manufacturers to notify FDA about
any discontinuances and interruptions in the production of
devices critical to public health emergency.
Dr. Shuren, has this notification from device manufacturer
has been useful to the FDA during the COVID-19 emergency?
Dr. Shuren. It has been exceptionally helpful. And again,
thank you to Congress for those authorities.
We have been able to prevent or minimize a variety of
different shortages from--you mentioned test supplies--a number
of personal protective equipment, defibrillators, dialysis
systems, really, across the board. And again, those situations
can arise both just before the public health emergency is
declared, as we found with COVID, and from other causes. And if
we are not well positioned to deal with that, we are going to
have important shortages that aren't resolved.
A quick example, outside of a public health emergency, we
had facilities using ethylene oxide to sterilize medical
devices. In fact, a little over 50 percent of devices that
require sterilization use ETO. When those facilities were
closed, we had no window as to whether shortages were going to
be caused. A few companies told us, many did not. We had to
manually go back in our systems, try to identify which products
were being sterilized there, and see if there was going to be a
shortage. In fact, we got complaints, once a shortage happened,
from the users because we never heard from a company. And this
is all because there was no requirement for a notification. It
puts--it really puts people at risk.
And what does it matter, the cause on the shortage?
Because, at the end of the day, patients don't care the cause
of the shortage. They just care they didn't get the medical
device they needed that may be saving their life. And the
doctors, nurses, other healthcare workers care that they could
not provide the necessary treatment to patients. And as a
doctor, I find that----
Ms. Matsui. Absolutely, Dr. Shuren.
Dr. Shuren [continuing]. You know, difficult to swallow.
Ms. Matsui. I have run out of time. I really can't--so I
yield back. Thank you.
Ms. Eshoo. The gentlewoman yields back. It is a pleasure to
recognize the gentleman from Utah, Mr. Curtis, for your 5
minutes of questions.
Mr. Curtis. Thank you, Madam Chair. It is great to be here
with you, Mr. Ranking Member. It is a great----
Ms. Eshoo. Great to be with you. Thank you.
Mr. Curtis. Dr. Shuren, clearly, many of my colleagues are
familiar with you. This is my first hearing with you, and it is
a delight to be here.
I am really excited to talk about this portion, because
Utah really excels. As a matter of fact, we have the fastest-
growing life sciences community in the Nation, BioHive, and I
love to brag about these companies. The first artificial kidney
came from Utah, and perhaps many in this room remember the
Jarvik heart that came from Utah. Merit Medical is a Utah born
and bred company. It was founded on the design of a
polycarbonate coronary control syringe designed to replace
dangerous glass syringes. Merit Medical was once a small
company, and really the heart blood of my district are these
small and medium-sized companies.
But I also feel like--that sometimes we are the hardest on
these small and medium-sized companies. And I think my first
question to you, Doctor, is it appears that they are
disadvantaged, compared to some of these larger corporations
who can weather longer time approvals, and they tend to have
far less capital and lack the established relationships that
the bigger companies have.
What can be done to level this playing field, and help
these startup companies who are so critical later on, right, as
they grow and become more important? Any ideas on leveling this
playing field?
Dr. Shuren. Well, I have to tell you, and so much
innovation comes from these, you know, small companies. And
they do not have the resources also for the help of what they
need to do to figure out--to actually get to the marketplace.
That is one of the reasons we had proposed this TAP pilot,
is to help. And the big focus is because most of these
innovative technologies coming through with the breakthrough
device designation are these small companies, and let's be
there to help them. If you will address the questions that they
are finding challenges with, and they don't have the outside--
you know, the big companies have so many experts, maybe it is
less helpful to them. But the small companies, in particular,
need that.
And in fact, the person who heads this up, I hired a year
ago, is my deputy center director for science. He was a venture
capitalist for three decades. He started a bunch of small
companies. He gets it. And he came to the FDA specifically to
do just what you are asking for. How do we help, you know,
these companies deal with these issues and get through that
valley of death, if you will, and safe and effective to the
marketplace.
Mr. Curtis. Yes, and I just really need to emphasize how
much more difficult the process is the smaller you are. So
thank you for addressing that.
We have been discussing for months the importance of FDA
keeping pace with industry and the role of these agreements and
the FDA working effectively and efficiently. It has been
brought up a number of times today, these negotiations are
running two months behind PDUFA, GDUFA, and BsUFA, which were
submitted to Congress in January. It is troubling to me that
this agreement was delivered to Congress well past the
statutory deadline, impacting our ability to ensure that they
are authorized on time.
We have also discussed at length the many instances we are
finding that COVID-19 created problems and concerns that we are
seeing and experiencing in our healthcare system. Over many of
these are things that were there before the pandemic. COVID-19
highlighted these existing problems, making them things we
cannot and should not ignore. I don't think that we should be
using COVID-19 as an excuse, a shield the hide behind, instead
of addressing root causes. It was a factor, and we recognized
the initial delay it created. Yet you spoke earlier about
concern over pink slips going out, and I share those same
concerns.
It is my understanding that MDUFA initially--meeting was
delayed by COVID from March 2020 until October 2020, but PDUFA
and GDUFA and the initial public meetings in July 2020. Why did
it take MDUFA--why didn't it move forward as promptly as the
others?
Dr. Shuren. Well, COVID hit the medical device industry,
and it hit us very, very hard. And so we mutually felt we
needed more time to get started.
And then there were a lot of issues, you know, ultimately
to work through. And there is--the medical device industry is
very heterogeneous, and it has very diverse opinions, and that
can take time to work through.
Regardless, we should have had that to you on time. And
that is our fault, ours collectively, and we take
responsibility for that.
Mr. Curtis. You have been very good in taking
responsibility. But I want to point out that BsUFA had an
initial meeting in November after MDUFA, and they still made
their deadline on time.
Dr. Shuren, there are many Utah medical device industry
stakeholders that have vocalized concerns to me over
communication breakdowns between them and FDA. What measures
can the FDA put in place to ensure this communication is
better?
Often when I hear from the complaints, it is communication
more than anything. What can my office do in working with you
in facilitating this? We hesitate, right, to step in to the
middle of this when we hear from them, but we would just love
your advice on, like, how we help these companies in a way that
helps you and is not counterproductive.
Dr. Shuren. Well, if they feel that they are not getting,
you know, the interactions are supposed to, they are not
getting the answers they are supposed to, they are identifying
issues with our program, talk to us. And quite frankly, you can
send them directly to me.
Mr. Curtis. And Doctor--I didn't realize we are out of
time, Madam Chair--I would love to continue that dialog with
you to figure out how to better coordinate with them.
And I yield my time.
Ms. Eshoo. You know, there is something that hasn't been
mentioned in this relative to timing, and meeting deadlines,
and all of that. And it is one aspect. It is understandable,
but I think it should be stated, that there was a--you know,
some real schisms between the very large advocacy or--you know,
for large medical device companies and the small companies. And
they did not see eye to eye. It is not a surprise, because each
one has its own--you know, its own self interest. But that took
time, as well.
So everything is not--doesn't rest with the agency. They
have to negotiate with people. And if they are not coming to an
agreement within the industry itself, that slows things down,
as well. So I think it is fair just to put it out there. We are
all thrilled. I was thrilled when I found out that they, you
know, came to an agreement so that everything could move along,
but that was a part of this.
And as you pointed out, the smaller companies have--they
may be small, but they want their voices heard. So bravo to
them.
OK, it is a pleasure to recognize the gentlewoman from
Illinois, Ms. Kelly, for your 5 minutes of questions.
Ms. Kelly. Thank you, Madam Chair and Ranking Member
Guthrie, for holding this hearing on the FDA user fee
authorizations for medical devices.
According to the newly released MDUFA performance goals and
procedures, the FDA is committed to hiring 200 new employees in
the coming five years. The FDA Diversity and Inclusion
Workforce Strategic Plan of 2018 through 2021 outlines FDA's
commitment to, and I quote, ``cultivate and promote a diverse,
inclusive culture'' in their workplace to reflect the diverse
backgrounds of those served by the agency's work.
Doctor, what metrics will FDA use to ensure that there is
adequate representation of racially and ethnically diverse
employees across all levels of positions in these FDA new
hires?
Dr. Shuren. So we collect that information already as to
what the representation looks like.
But I will tell you, we have already just issued for our
center our diversity, equity, inclusion, and belonging roadmap
on steps we are taking that includes hiring, addressing that,
and it is part of the strategic priorities. I mentioned one:
advancing health equity. The second is on, you know, a modern,
diverse workforce that, if we are going to represent a diverse
country, we need to reflect that diversity in our center. And
that is a commitment from us, and there are already
workstreams.
And so that includes our outreach for hiring in the first
place in different places, so that, again, we can bring that
sort of talent, diverse talent, into the center.
Ms. Kelly. Thank you so much, and great to hear.
You discussed the importance of patient voices in the
development of medical devices. How can patient preference
information, PPI, and patient-reported outcomes, PROs, and
patient-generated health data be leveraged to ensure clinical
care is culturally relevant for racially and ethnically diverse
individuals?
Dr. Shuren. Well, for example, for patient-generated, you
know, health information, here is a great opportunity where
using technology--you know, technology is much easier to push
out into settings where people are living their life. And so
those who may have a hard time getting to a clinical trial site
or, you know what, they have some discomfort of doing that, any
number of reasons, if instead they can provide that information
in the comfort of their home, at work can make it easier for
individuals who don't have that same access. So we think that
is a very important route.
You deal with patient preference information--I will just
mention if you have intended populations, you want to make sure
that is represented too in the patients in whom you conduct
that study. Because we see the preferences of patients are not
uniform at all. They kind of stratify on a variety of factors.
Ms. Kelly. OK. The MDUFA agreement outlines the use of
patient input to inform clinical study design to increase
recruitment and retention of a diverse clinical sample. From a
clinical and device efficacy perspective, why is it important
for clinical trials to have racially and ethnically diverse
participants?
Dr. Shuren. Well, it is important that, if you are going to
use a device in an intended population, that you know it is
going to work in that population. And we have seen, you know,
plenty of instances where there may be a difference in how that
technology works. That may be due to a variety--it may be race,
it may be gender, or any number of things. And so you want to
make sure you have looked at it in those appropriate
circumstances so you know it works in the intended population.
The other is, even if your intended population is small, we
have got to be thinking about, if that technology could add
value in other populations, we should be looking at that so
that we don't have devices simply made for certain segments of
the U.S. population. We ultimately have high-quality healthcare
for all.
Ms. Kelly. Thank you. And that is why I have been working
with my colleagues on the DEPICT Act and the NIH Clinical Trial
Diversity Act that would ensure diversity in clinical trials.
Thank you so much for your patience, and thank you for
being here.
I yield back.
Ms. Eshoo. The gentlewoman yields back. The Chair is
pleased to recognize one of the wonderful doctors we have on
our subcommittee, Dr. Bucshon from Indiana, for your 5 minutes
of questions.
Mr. Bucshon. Thanks, Dr. Shuren. I would like to talk to
you today about a topic that isn't included in today's hearing,
but that I thought maybe ought to be, and that is diagnostic
testing reform, and specifically the VALID Act, which I have
been working on, which--you have also been working on this
issue, I know, for many years.
I was driven to start working on diagnostic testing reform
based on my experience as a doctor before coming to Congress.
Health care providers and patients routinely use and
increasingly rely on diagnostic tests to make difficult
decisions about the best course of care and treatment.
Unfortunately, we continue to see examples of some tests that
don't meet the level of analytical and clinical accuracy that
are needed to make reliable medical decisions, causing some
patients to go through with life-changing procedures that may
not have been necessary.
This is why I believe Congress must provide certainty, and
that is why we are trying to accomplish what we are trying to
accomplish through the bipartisan and bicameral VALID Act,
which I have been working on with my friend, Representative
Diana DeGette, in the House for about five years. We are
working to provide certainty for patients that the results of
their tests are clinically accurate, and provide certainty for
doctors that the tests they are administering and making
healthcare decisions based on are accurate.
And last, we want to provide certainty for test developers
and labs that the regulatory framework won't suddenly change,
and that they will have a clear understanding of what is
expected from them within the risk-based framework.
I would also like to note that the sponsors have been
mindful throughout this process to make sure we are balancing
patient safety while promoting innovation. For example, VALID
provides certain flexibilities to help facilitate development
and support innovation for diagnostic tests for rare patient
populations, all while keeping in place high standards for
patient safety. This is instrumental as we continue to move
toward the future of----
[Audio malfunction.]
Mr. Bucshon [continuing]. Will enable physicians to provide
more individualized patient care to discover a cure and treat
diseases that were previously unknown and untreatable, which is
why I am somewhat concerned that the committee is seemingly
ignoring the issue and the legislation all together. I have
repeatedly called for hearings on VALID so, as a committee, we
can better understand the issue and the legislation needed to
promote innovation and provide clinical and analytical
certainty.
Therefore, I would ask the Chair of the--Eshoo and Chairman
Pallone to work with me in the coming weeks, and with
Congresswoman DeGette, to have a hearing on VALID, the VALID
Act, so that Congress can help better serve patients, as I
truly believe the time for Congress to clarify the rules of the
road for diagnostic testing is now.
So, Dr. Shuren, it is my understanding that the FDA
currently does not have a process tailored specifically to
diagnostic test review. And rather, the FDA uses the existing
medical device process for diagnostic testing review, even
though the two are quite uniquely different. Is that true? Is
that accurate?
Dr. Shuren. Well, the pathway we have for in vitro
diagnostics is different for other devices. I mean, the law is
very clear that IVDs, regardless of who makes them, are called
medical devices, but how we regulate them is different, and we
really tailor that to that kind of technology.
Mr. Bucshon. OK. Does this--so you wouldn't say this
process limits your ability to validate that all diagnostic
tests out there today are analytically and clinically accurate?
Dr. Shuren. Well, the answer is no. We have had this policy
of enforcement discretion for--since the start of the program
for tests made by laboratories. And at the time that made
sense. They are low risk----
Mr. Bucshon. Right.
Dr. Shuren [continuing]. Locally, but they are far more
complex, riskier. And we have seen, over the years, you know,
problematic tests from laboratories to market.
At the same time, though, those LDTs play a critically
important role in healthcare. And, as you note, increasingly,
they and tests made by commercial manufacturers are important
for making clinical decisions. And we have to assure,
ultimately, that they work. Those assurances are in place if it
is made by a commercial manufacturer, then not in place if they
are made by a laboratory.
And so having a legislative framework that clarifies an
overarching approach to assure that all developers, whether
they are commercial manufacturers or laboratories, are working
with FDA and all of us acting consistently under a modern
framework--you know, I mentioned the frameworks are years old.
It is time for an upgrade. This is a time, really, to do it.
And that could have a big impact on public health, but done in
a way that is protecting patients, but driving--you know,
supporting that innovation.
And I, you know, thank you and Representative DeGette on
your leadership on trying to push this forward. We do think the
time is right. We have publicly stated for years we would--we
were holding off on administrative action because we thought a
legislative solution was really the best way to go.
Mr. Bucshon. Thank you for that response. I couldn't agree
more.
I yield back.
Ms. Eshoo. The gentleman yields back. The Chair recognizes
the gentleman from Vermont, Mr. Welch, for your 5 minutes of
questions.
Voice. Oh, sorry, let's go to Cardenas.
Ms. Eshoo. Oh, I am sorry. Who is it?
Voice. Mr. Cardenas.
Ms. Eshoo. Oh, OK. The gentleman from California, Mr.
Cardenas, is recognized for your 5 minutes of questions.
Mr. Cardenas. Thank you very much. I appreciate this
opportunity for us to discuss this important issue, Madam
Chairwoman, and also Ranking Member Guthrie.
I appreciate you, Dr. Shuren, for joining us to discuss
what we should be doing, and continue to do for the American
people. Dr. Shuren, once again, thank you. And obviously, it is
critical that the devices we bring to market are safe,
effective, and work for everyone.
It is one of my top priorities to ensure that our approval
process at the Federal level includes diverse perspectives, and
that medical therapies and devices are tested in trials that
include demographics that mirror the Nation as broad as we are
as a Nation. How will FDA incorporate the perspectives of
patients and stakeholders from diverse backgrounds?
Dr. Shuren. One of the commitments I will highlight under
MDUFA V is to expand exactly that: patient perspectives in the
design, conduct of clinical trials, as well as to facilitate
participation.
So one of those approaches is really using technology as a
way for patients to participate in clinical studies without
having to keep going to clinical trial sites could facilitate
more patients participating, particularly those who have less
access to the healthcare system.
Mr. Cardenas. Well, less access to the healthcare system,
sometimes that comes from a lack of access to transportation, a
lack of access to technology, et cetera. So what you are saying
is, by removing some of those daily barriers, we are hopefully
going to be looking at more diverse input, which means a better
output.
Dr. Shuren. That is correct.
I mean, another step is, in designing studies and looking
for patients, work directly with those centers that are in the
communities where you are trying to recruit. It is very
important to have that kind of partnership.
Also, it can drive a greater participation from diverse
populations. You have got to go to where people are----
Mr. Cardenas. OK.
Dr. Shuren [continuing]. And meet them----
Mr. Cardenas. Thank you, and I think that----
Dr. Shuren [continuing]. As opposed to asking them to meet
ours.
Mr. Cardenas. And I think what you just described not only
is diversity in more ways than one, it is also rural, as well.
So thank you.
I also recognize that it is critical to ensure that we are
expediting the time it takes to approve devices without
sacrificing a review that will determine safety and efficacy.
What steps will FDA take to ensure patients will not be harmed
by devices that have been approved using more expedited
processes?
And how do you, you know, work with and--with these
concerns?
Dr. Shuren. So any of the times we reach an accord with
industry, we are never doing it where we believe it would ever
sacrifice the quality of our decisions, and then put at risk
our authorizing an otherwise unsafe device because of it.
And we believe the extra resources--will it allow us to
meet the commitments that we have laid out in the commitment
letter in a responsible way? We have more people. It means that
we have more folks with fewer files on their desk. They can
move through it more quickly. But it does not compromise the
quality of that review. If anything, by expanding our expertise
in the center, we may have more experts to help out,
particularly things like in digital health, bringing--and that
is one of our commitments. Bringing on more expertise into the
center can be helpful so we have better-informed decisions, but
we can do it in a more timely manner.
Mr. Cardenas. With higher user fee collection, how may that
affect diversity in clinical trials?
And you just mentioned having more experts. I am sure with
more and better funding, we can actually have higher and have
better and more experts to do the job. So how does the funding
and allocation affect that?
Dr. Shuren. The funding also expands our patient engagement
program, the people who are working directly with patient
groups and working with communities. And part of that mandate
under MDUFA V is we will be using some of those additional
resources to facilitate a greater participation by diverse
populations in clinical studies.
Mr. Cardenas. OK, thank you. I appreciate you sharing your
insights and thoughts. And once again, thank you for the work
that you do.
It is imperative that we ensure timely access to innovative
devices, while still confirming that they are going to work as
intended, without undue risk to the users.
And once again, if the information coming in is more
diverse, then we stand a greater chance that efficacy will work
in all communities----
Voice. That is fine.
Mr. Cardenas [continuing]. Not just some.
And with that, my time looks to be expiring. I yield back.
Thank you, Madam Chairwoman.
Ms. Eshoo. I thank the gentleman, and he yields back. It is
a pleasure to recognize the gentleman from Pennsylvania,
another one of our doctors on the committee, distinguished
physicians, Dr. Joyce.
Mr. Joyce. Thank you, Madam Chair Eshoo, for yielding, and
for convening this hearing.
The approval of new, cutting-edge medical devices and
safely getting innovation into the hands of patients and
physicians is critical to improving health outcomes in the
United States.
To that end, Dr. Shuren, how can the FDA and CMS work
together better and earlier to ensure that beneficiaries of
Medicare do not face additional barriers to coverage once
that--the FDA approves or clears an innovative and lifesaving
medical device?
Dr. Shuren. Currently, both FDA and CMS are members of the
Medical Device Innovation Consortium. And about a year ago a
workstream was started that is focused on health economics and
value that really is on the reimbursement side of the house,
and what steps might be able to responsibly streamline that
pathway, like including the voice of patients in
decisionmaking.
We also were engaged in discussions with them on MCIT, and
certainly stand ready to facilitate discussions too on whatever
is helpful to them on establishing predictable pathways for
reimbursement. I mean, we are not insurers. We can't stand in
their place, but we all--have always been there to facilitate
as best we can.
Mr. Joyce. Would it be helpful for CMS to communicate with
the FDA at early stages of development the important issues
that might be addressed in clinical trials to help facilitate
timely Medicare coverage upon market entry?
Dr. Shuren. We do think the voice from CMS early on can be
very helpful. We offer that in the parallel review pathway,
which is voluntary, you know, for companies who may qualify.
I will say a challenge for CMS--so I am going to tin cup
for my sister agency--they don't have enough people. You know,
if we really want to do something there, like for coverage,
national coverage determinations, they need more people. And I,
by the way, used to work over there many moons ago, so I know
exactly what it is like. And they can--they could use some
help.
Mr. Joyce. Thank you, Dr. Shuren. I would like to touch on
an area that Chair Eshoo mentioned before the recess regarding
the distinction between servicing and re-manufacturing of
medical devices.
Just to be clear, is it your opinion that it will be
helpful for Congress to further clarify what constitutes re-
manufacturing in statute?
Dr. Shuren. We do think that that can be helpful. Again,
the devil is in the details as to what it always looks like.
But we know that, even though we have got guidance that is
going through, there is a lot more comfort sometimes--it is
guidance, that there is more comfort if certain things are
baked into the statute.
So again, we think this could be helpful, again, depending
upon what that provision looks like, and we would be--if there
is interest, we would be happy to work with the committee on
it.
Mr. Joyce. Thank you. And I would like to conclude by
thanking my colleague, Representative Peters, for working with
me on the introduction of Clarifying Re-manufacturing to
Protect Patient Safety Act, which I believe would provide the
necessary clarity on what constitutes a significant change to a
medical device, as well as what constitutes re-manufacturing.
Thank you. I see my time has expired. Again, thank you,
Madam Chair Eshoo, for convening such an important hearing.
Ms. Eshoo. The gentleman yields back. Thank you for your
kind comments. The Chair is pleased to recognize the
gentlewoman from California, Ms. Barragan, for 5 minutes.
Ms. Barragan. Thank you, Madam Chair.
Dr. Shuren, several non-profit consumer advocate groups and
public health organizations have raised concerns over the lack
of transparency regarding the FDA's non-public negotiations for
the Medical Device User Fee Amendments program. How involved
were patients and consumer advocate groups during the
negotiations?
Dr. Shuren. We had held, I think it was, monthly
stakeholder meetings to provide updates and to seek comments on
the MDUFA V negotiations.
Ms. Barragan. So my understanding is that there are--no
public stakeholder calls were held this year, and only one
public stakeholder call was held in 2021. Do you know if that
is accurate?
Dr. Shuren. No, I don't believe that that is accurate. We
can get you the details.
Ms. Barragan. Great, I appreciate that. Thank you.
Dr. Shuren, according to a January 2022 report by the GAO,
the FDA lacks an agency-wide strategic workforce plan, and has
no process in place to measure agency performance. The report
emphasized that creating a centralized workforce strategy is
vital for the FDA. Does the FDA plan to adopt an agency-wide
strategic workforce plan?
Dr. Shuren. I would like to take that back, since it is the
agency speaking. But I have to tell you that those
recommendations were taken seriously, account--and there have
been a lot of efforts to facilitate our ability to hire and
bring on board the people that we need in the agency to get our
mission accomplished.
Ms. Barragan. Great. Well, you know, for the first time in
MDUFA's history, the FDA will publish the 5-year financial plan
with hiring targets for the MDUFA program. So I would like to
know how the FDA is going to build and retain a diverse FDA
workforce that accurately reflects, you know, our country when
trying to meet these new hiring performance goals. Is that
something you can comment on today?
Dr. Shuren. Yes. So we are--already issued a roadmap on
diversity, equity, inclusion, and belonging, where this is one
of our actions. It is part of our strategic priorities on a
modern, diverse workforce.
Moving forward, such activities include recruiting from
targeted areas so that we are more reflective of the diversity
in the country. I mean, there is a lot of diversity in CDRH to
begin with, but there is a better job that we can be doing that
that is reflected across all layers in the organization.
Ms. Barragan. Well, thank you. This is of great importance
to me. The--you know, the Hispanic Caucus, and making sure that
we have diversity and inclusion, and the perspectives of those.
So I just wanted to thank you for that, and I look forward to
following up with you, and seeing anything more you have on
this.
With that, Madam Chairman, I yield back.
Ms. Eshoo. The gentlewoman yields back. The Chair is
pleased to recognize the gentleman from Georgia, Mr. Carter,
for your 5 minutes of questions.
Mr. Carter. Thank you, Madam Chair, and thank you, Dr.
Shuren, for being here. I appreciate it.
Dr. Shuren, way back in 2017, I--since that time I have
really appreciated your engagement on legislation that that
myself and others on this committee have authored to establish
over-the-counter hearing aids. I am a pharmacist by profession,
and I see firsthand, and have seen throughout my professional
career, the need for this. And I want to tell you that I
appreciate your engagement in this.
The agency proposed a rule on October 19th, as I understand
it, of last year that got a lot of things right. And I want to
thank you for that, as well. Any idea when--or any indication
that you can give us when the FDA might finalize this proposed
rule?
Dr. Shuren. Well, sir--and first of all, thank you for that
provision. We couldn't agree more. This is--these technologies
are very, very important for public health.
We are supposed to, you know, issue that 180 days from the
end of the comment period. So that turns out to be about July
15th. And our goal is to do that. We know this is important to
the Administration.
I can't--you know, some of it is out of our control, but
that is our goal, to try to meet that statutory deadline.
Mr. Carter. And you said it would be, what, June?
Dr. Shuren. July 15th.
Mr. Carter. July 15th? OK.
Dr. Shuren. Yes.
Mr. Carter. We will look forward to that. I hope it will be
before then. I will tell you there--again, my--and my
experience has led me to believe and to offer to you that this
is needed. I mean, you know, we got reading glasses. I mean, we
ought to have over-the-counter hearing aids. I get it if there
is a need for more severe cases. But for most people--like
myself, who are getting on up there a little bit--you know, you
do need a little bit of help, and there is no reason why we
shouldn't be able to do this. So I look forward to that, and
thank you again for your work on that.
Dr. Shuren. We agree. I will mention we received about
1,000 comments. So there is just--we want to make sure we get
it right.
Mr. Carter. You received 1,000?
Dr. Shuren. Yes, about 1,000 comments.
Mr. Carter. Pro, con, or can you indicate?
Dr. Shuren. Mostly pro. Some had suggestions. You know,
there are some differences of opinion, let's say, around where
you set the output limits.
Mr. Carter. Right.
Dr. Shuren. For example.
Mr. Carter. And I get that. And you are right, we want to
get it right. We want--you know, we don't want to do--I mean,
you know, the Hippocratic Oath, do no harm. So we don't want to
do that. But at the same time, you know, we can help people,
and we need to be doing that.
Let me ask you about the FACTS Act, if you are familiar
with that, the FDA Advancing Collection of Transformative
Science Act. That is legislation that Dr. Burgess on this
committee and I have cosponsored. And it was considered at a
hearing 2 weeks ago that we were in, and it has important
ramifications about the medical device community, and Real-
World Evidence, and a Clinical Laboratory Improvement
Amendments, CLIA, waiver for EUA authorization, and for EUA-
authorized diagnostic tests. And of course, again, this is very
important. And I hope that the committee will continue to move
this bill forward. It is a very important piece of legislation.
Will you commit to working and continuing to work, as you
have, with this committee to improve and advance both the Real-
World Evidence and Clinical Laboratory Improvement Amendments
waiver provisions of this legislation?
Dr. Shuren. We are happy to continue to, you know, talk
with folks through that. We are looking--I should tell you, in
those transitions from an emergency use authorization to full
marketing authorization, we are taking advantage of what has
already, you know, been provided.
We will not--like for a CLIA waiver, we are not planning to
ask folks to go ahead and do usability studies. You know, there
is no need. They have been out there. The biggest focus is
really going to be on having enough data just to make sure that
they work----
Mr. Carter. Right.
Dr. Shuren [continuing]. Because we have relied on so
little data to put them out on the marketplace.
Mr. Carter. Right. Good, good. Well, again, thank you.
These are important issues. And thank you and the agency for
your attention to these.
And, Madam Chair, I will yield back.
Ms. Eshoo. The gentleman yields back. The Chair is pleased
to recognize Dr. Schrier from Washington State for your 5
minutes of questions.
Ms. Schrier. Thank you, Madam Chair. And thank you, Dr.
Shuren, for coming today to discuss the medical device user fee
agreements. Thank you for all you have done to help the
American people get through the worst of the pandemic. And it
is very nice to see you and talk with you again.
I would love to focus on what the FDA and industry can do
together to get the right product to the right market at the
right time and, frankly, even in the right quantity and the
right price for a clearly defined purpose. And I have something
clear in mind.
You and I have been in touch on and off for about a year
and a half regarding the rolling out of rapid home COVID tests.
And as you know, for most of that time I was feeling pretty
frustrated, because the process just seemed so slow, and seemed
unnecessarily difficult to get these tests approved and into
people's hands, even though the technology is pretty simple.
And I was hearing from universities and researchers and
companies that had submitted applications but were still
waiting for FDA emergency use authorization. And it seemed like
at every stage there were barriers, but barriers that, with the
right panel of public health experts and industry advisers all
in a room together, could have really improved communication,
and maybe been resolved quickly.
I am delighted that now we have 17 home antigen tests and a
couple of molecular tests on the market for--with emergency use
authorization, although the price point is still too high for
most people to use that for screening. But that means I was
even happier when the Administration started sending free tests
to every home in the country during the Omicron peak.
But still, reflecting on a year and a half, I felt like we
were still lagging behind. And having an advisory panel to
bring all of these specialists, public health, industry,
consumers, and the FDA all together, could have defined that
goal, set some standards that everybody agreed upon, figured
out how you were going to test them, and even sped up that
approval process.
My bill, the Diagnostic Device Advisory Committee Act to
create such a panel will do just that, and it will convene a
group of experts meeting with FDA to discuss the real-world
impact of diagnostics. And if passed, this will engage the
diagnostics experts, consumers, public health, and you to talk
about the risks, uses, needs, and the applications of these
devices. And I think it will bring a lot of transparency and,
hopefully, expediency.
So, Dr. Shuren, with the lens of lessons learned, I was
just wondering if you could talk a bit about how such a panel
might help future discussions, and how it might expedite
getting things to market more quickly.
Dr. Shuren. Well, certainly, input from the outside experts
can be very informative. Certainly, things in advance, when we
deal in a public health emergency, things moving quickly, just
a--sometimes a little bit more challenging. But this is
something we would certainly welcome the opportunity to talk
with you about, and work with you on.
I will say in the case--I don't want to throw the baby out
with the bathwater, you know--for over-the counter antigen
tests, which we put as a priority, actually, in the spring of
2020, and were one of the first countries to authorize, where
we saw that you had lots of tests is where you invest in the
marketplace.
It is not about having a lot of different tests. It is
about having a lot of tests made through high manufacturing
capacity. And you have a country like the UK. When they put
that money in through large government contracts that were
going to then support large manufacturing volume, and they only
did it with a handful of, you know, companies, and subsidizing
so that your tests are low cost or free, massive increase in
what was available. And many more developers came to their
marketplace.
And I agree with what the Administration did. When they got
money, you know, they invested in the marketplace. And we saw
the same thing happen, a rapid increase, you know, of--well,
increase in production now over an order of magnitude. And that
really makes a difference. And if that is not there, the
numbers drop, you know, because the companies--if there isn't
that demand or guaranteed with contracts, they are going to cut
production. We saw it happen in the U.S. There were more tests
available in late spring than in late summer, because demand
dropped, nothing propped up the marketplace. You know, a
company closed a manufacturing facility.
Ms. Schrier. And Dr. Shuren----
Dr. Shuren. So that is a key piece that we need to have
there.
Ms. Schrier. I could not agree more.
Dr. Shuren. And a last thing is independent review. When we
were able--funding to support NIH with us to do the ITAP
program, now we are able to make sure, without changing any
standards, they were able to do the evaluations very quickly,
just a few weeks, evaluate, and we authorize where the data was
there. Our performance standards are really the same as we have
seen, you know, that 80 percent sensitivity, as with other
countries.
But the technology isn't as simple, though, actually
getting those antibodies right on the strip. We have seen
problems. In fact, the UK had the same experience. Most of the
tests that came to them they never authorized, because of
problems either with the test on validation--we have had the
exact same experience with the U.S. In fact, many of the folks
we have seen are the same folks that made all those bad
antibody tests, with the same technology that came onto the
U.S. market.
So I 100 percent want to work with you. I also want to make
sure, also, that we deal with some of these other issues, to
assure that we, in the future, have the tests we need.
Ms. Schrier. Thank you very much----
Ms. Eshoo. The gentlewoman's time has expired.
Ms. Schrier. I yield back.
Ms. Eshoo. The Chair now recognizes the gentleman from
Texas, Mr. Crenshaw, for your 5 minutes of questions.
Mr. Crenshaw. Thank you, Madam Chair. And thank you, Dr.
Shuren, for being here with us today.
I certainly share my colleagues' concerns about how MDUFA V
came together, and I hope you will work with us for a better
process in the future.
We are excited to have you here to talk about the next
frontier of medical devices. and how our phones and devices can
deliver digital health. You know, we carry these things around,
and they have tremendous possibility to improve patient health
and well-being.
One that stands out to me in particular is the app
connected to the continuous glucose monitor, which allows
parents to track on their phones the glucose levels of their
children with diabetes. A constituent of mine talks about this
innovation as an absolute game changer. She used to wake up
multiple times a night to check her son's glucose levels. Now,
just as an app that notifies her when he drops to dangerous
levels.
The FDA uses the framework of safe and effective to
evaluate medical devices. I am always going to be a little
skeptical of that mandate to regulate effectiveness, and
whether the FDA is best suited for that. That is a conversation
for another time. But giving us the framework we currently
have, do you think FDA is suited to properly regulate things
like artificial intelligence?
Dr. Shuren. So, first off, we are the place for doing it.
And, you know, we have authorized now over 300 devices with
artificial intelligence, particularly machine learning--I think
just 50 in the past year.
But I do think that we need a regulatory flexibility that
we don't currently have to better tailor the pathways to that
kind of technology.
Mr. Crenshaw. OK.
Dr. Shuren. The pathways in the law now are many years old.
But I do think--and I am happy to continue the conversation--
effectiveness matters. You know, as a physician, too, we want
to know that benefits outweigh the risks. And that is really,
at the end of the day, what we are saying: benefits outweigh
the risk. We have got to know it helps patients and that,
again, you know, the risks----
Mr. Crenshaw. And the reason I ask about the artificial
intelligence question is because three or 4 years ago FDA had a
paper that said they might need different authority to regulate
digital health. You are saying right now there might need to be
some changes. And maybe--and we don't have--we have 2-minutes
and 47 seconds, I don't think we are going to get through it
right now. But if you would please followup with us on what
those changes need to be, that would be exceptionally helpful
for this committee.
The other question I want to ask you in our time left is,
you know, one of the problems we often see with medical devices
and other treatments is FDA approves something, then CMS has to
evaluate it again, seemingly with the exact same set of tests
and standards, just to determine if they will pay for it.
How do you think--and maybe you weren't expecting a
question like this--but how do you think these agencies can
work together to get patients what they need, once it has been
approved by FDA?
Dr. Shuren. Well, we do have different standards. You know,
they are reasonable and necessary, you know, safety and
effectiveness. And what they have, not different than other
insurers.
But we believe it is very important that that pathway from
an FDA marketing authorization to CMS--or, by the way, any
insurer's decision to coverage-reimburse, we have got to
streamline that. Because, quite frankly, patients don't have
real access to technology, particularly if it is expensive, if
it is not covered and paid for, right, because people just
can't afford some of these things out of their pocket, and they
just won't get it.
And the U.S. is complicated. The reimbursement structure is
much more complicated than some other countries. And providing
predictability, however we do it--I am not a payer, so, you
know, I can't tell you, and it is not for me to say what is the
best thing from a CMS perspective. But I do think, as a Nation,
solving that problem to have more predictable reimbursement is
absolutely essential for us to drive better technology for
patients. And if we don't do it, we are at risk of losing our
edge on innovation to other countries.
And I will tell you who is knocking at our door, is China.
Mr. Crenshaw. Yes, and I couldn't agree with you more. I
agree with the sentiment. I suppose we could delve into this a
little deeper at a later time on whether CMS is duplicating the
processes that occur at FDA already. I think that is what we
are concerned about. You know, looking at cost effectiveness
seems to be like something a payer would do. But do they really
need to do extra safety tests when FDA has already done it?
That would be our issue. And they are not here right now. So,
you know, it is--I am just curious what your thoughts were.
Ms. Eshoo. The gentleman yields back. The issue was raised
earlier in our hearing today, and I think that, working with
FDA, with Dr. Shuren--and Cures 2.0, I think, presents an
opportunity for changes that we can work together on. But this
is a concern on both sides of the aisle, very legitimate.
All right, all committee member--staffers, members of the
subcommittee, I will stay to finish out the questions if we
only have three more. Congress members Kuster, Dunn, and
Trahan. Do we have anyone else? Can you ping us? Otherwise, I
am going to stop, and go and vote, and come back, and Dr.
Shuren is going to have to wait again.
So why don't we go to the gentlewoman from New Hampshire?
And I hope the offices respond.
Ms. Kuster. Thank you very much, Madam Chair----
Ms. Eshoo. The gentlewoman from New Hampshire is
recognized.
Ms. Kuster. Thank you, Madam Chair.
Thank you, Dr. Shuren, for being with us today. I want to
jump right in, and ask you about the proposed pilot program at
FDA known as the Total Product Life Cycle Advisory Program,
also known as TAP.
This program is intended to foster earlier interaction
between the FDA and industry to identify risks earlier in the
development with input from outside stakeholders. While the
pilot will begin in 2023 with only 15 products, importantly it
will increase to 325 products by the end of the MDUFA in 2027.
I am interested in learning about how this program will achieve
its intended goals of improving patient outcomes, streamlining
regulatory engagement, and increasing efficiency in the pre-
market review process.
Dr. Shuren, can you elaborate on how TAP will achieve these
goals?
Dr. Shuren. One of the lessons learned from COVID is that,
to facilitate technology coming to the marketplace, and safe
and effective technology, developers sometimes are hitting, you
know, roadblocks. Particularly if you are dealing with
innovative technology, you may be dealing with new science.
And when we had the ability, as we did in COVID, to engage
with those developers in near or real time to answer their
questions, to work with them hand-in-glove to problem-solve, we
could expedite product coming to market, because we solve
problems more quickly. They were more efficient. You can be
more efficient in how you spend your money--you, as the
developer--and you can reduce that time, ultimately, on the
development evaluation cycle and then, ultimately, for FDA
authorization.
So TAP is just taking from those lessons learned, and now
piloting that, if you will, in peacetime. And by doing this,
the reason you will see the growth also in products is that we
are sort of rolling this out. We will start with one of our
offices, you know, learn from that, be iterative, be like an
innovator, do this like a skunkworks, and then we will start
rolling it out to other offices, and then include the
opportunity for more products to come in.
And this, in particular, can be a major game changer for,
you know, your small, innovative companies who don't have the
same bandwidth for trying to get that product to market. Again,
it has got to be safe and effective, the science has got to
support it. But we know, from experience, these are the things
that can really make a big difference.
Ms. Kuster. And how do you expect FDA will balance the
resource needs of the program with the demands of a growing
stack of pre-market submissions?
Dr. Shuren. Well, this is one of the reasons too we got
added resources in--or would get, you know, if enacted--that
would then give us the capability for handling those additional
submissions that are coming in the door outside of TAP.
And the add-on payments that are put in as an
accountability factor is another mechanism for kind of assuring
that we keep, if you will, our eyes focused on a variety of
actions that we have committed to meet and to take over the
course of MDUFA V.
We also sort of factored in for the pilot--is that it is up
to a certain number. So if it turns out, you know, we are not
completely right on the resource needs, and we would have
needed more, we can kind of scale back, if you will, the number
of products that come into it. So we really can test drive, and
that is the nice thing about doing a pilot in this case. We
really can learn, factor that in. And if things look good, we
will have a conversation with industry about where we go from
here. And if it doesn't look good, you know, we can pull the
plug.
But most of the people we are hiring are your review folks
who are doing the other bread-and-butter work. So we see this
as a win, and we think MDUFA V has already built in a number of
aspects to assure that we are well positioned to make good on
our other commitments, as well.
Ms. Kuster. Great. And one last one: How will the FDA
ensure that patient advocates and outside stakeholders are
involved in the process?
And how will you ensure that the focus remains on safety
and efficacy?
Dr. Shuren. Well, it is very important that the agreement
for MDUFA V, whatever gets enacted, does not change our
independence on decisionmaking, it is not to get money in
return for making a particular policy decision or any decisions
on product. Absolutely essential that this is really about
improving performance, not to influence our decisionmaking. And
we think what we have gotten to with industry, in trying to
reach consensus around there, achieves that objective.
And of course, you know, moving forward, we do view the
perspective of patients as being very important in the work
that we do. And investments from MDUFA V are going to expand
our abilities to advance that work on the science of patient
input, and broaden patient engagement into medical device
development evaluation.
Ms. Kuster. Great. Thank you so much. My time is up----
Ms. Eshoo. The gentlewoman's time is----
Ms. Kuster [continuing]. I yield back.
Ms. Eshoo. Yes, the gentlewoman's time has expired. The
Chair is pleased to recognize another one of the doctors on our
subcommittee, Dr. Dunn of Florida, for your 5 minutes.
Mr. Dunn. Thank you very much, Madam Chair, for hosting
this hearing today to discuss the agreement between the FDA and
the industry regarding medical user fees.
And let me say thank you, Dr. Shuren, for your insights
today, and for your stamina putting up with these marathon
questions, and for your enthusiasm for this. It is--it shows
through. We appreciate a good witness who comes and really
informs us. Thank you.
Let me say innovation in the device space is exciting for
patients and doctors. Like, more and more advanced medical
devices come to market, and Congress just has to make sure that
FDA is adequately equipped to properly evaluate these new and
emerging technologies.
We also have to guarantee that the patients have access to
the latest and greatest technologies without significant
delays, and that those delays that collect patient information
keep that information secure.
We have learned a lot over the course of the pandemic. We
witnessed the FDA efficiently grant emergency use
authorizations to numerous diagnostic tests and devices. And I
would like to see us continue to tune that process to continue
these rapid and safe approvals to make it more available to the
patients. And I certainly appreciate you being here to inform
us on that today, Dr. Shuren.
I understand that, during the MDUFA IV reauthorization
process, the committee sought additional information regarding
the servicing of medical devices. As a result, the FDA put out
in 2018 a report on the matter, concluding that OEMs and third-
party servicers both provide high-quality, safe, and effective
servicing of medical devices. I know this to be true from my
own personal experiences. I ran a large practice with a number
of complex machines, used linear accelerators for radiotherapy,
PET scanners, CT scanners, et cetera.
Utilizing third-party services was critical to maintain
high-volume, high-quality care in a cost-effective manner. So I
commend the FDA for following through on this committee's
concerns about enhanced post-market surveillance and
transparency by requiring disclosure of servicing on the MDR
3500 Form, and especially for providing clarity on the
definition of re-manufacturing in your draft guidance.
So, Dr. Shuren, I understand CDRH has worked hard to
provide a clear, transparent process for entities to understand
if they are engaged in servicing or in re-manufacturing of
devices. And is it correct that the FDA concluded in 2018 there
was not--no significant safety concerns related to the third-
party servicing of medical devices?
Dr. Shuren. We did conclude that there weren't widespread
concerns.
Mr. Dunn. Excellent, excellent. How much feedback has the
agency received on your 2021 draft guidance regarding re-
manufacturing?
Dr. Shuren. Actually, I don't know the numbers, offhand.
Mr. Dunn. Do you have a feel--some of it, not much of it?
Dr. Shuren. I would rather get you the right information.
Mr. Dunn. OK. Didn't--OK. When do you expect that guidance
to be finalized?
Dr. Shuren. It is, you know, on our list to try to move
forward in the coming year.
Mr. Dunn. In this year?
Dr. Shuren. In this year.
Mr. Dunn. Excellent, excellent.
As you know, in the CARES Act, Congress granted FDA
temporary authorities limited to the duration of the emergency
to require additional reporting related to medical device
shortages. You addressed that earlier in one of your comments.
The FDA subsequently listed only 30 devices on a shortage list
such as PPEs, diagnostics, ventilators, et cetera.
In January of this year the FDA issued draft guidance
outlining their vision for reporting requirements beyond the
public health emergency. It is my understanding that they are
pursuing authorities to issue blanket requirements for the
entire industry, including hundreds of thousands of devices. It
seems to me, surely, there are only a few hundred critical
devices we should be tracking in that detail.
And can we look at some kind of threshold for reporting on
all those requirements? Does the CDRH even have the expertise
in the supply chain management to look at those things?
Dr. Shuren. Yes, so what we are putting forward is still a
narrow list of devices.
Mr. Dunn. Oh, excellent.
Dr. Shuren. Yes, it is, and----
Mr. Dunn. We would like to know what that is, but that--
thank you.
Dr. Shuren. Yes. No, it is, and it is really the things
that are critically important.
Mr. Dunn. And I actually got your point earlier that it is
critically important. I just didn't want to see, you know, the
bureaucracy bogged down with chasing hundreds of thousands of
different supply chains for reporting, especially on a biweekly
basis.
Dr. Shuren. We don't want to, either.
Mr. Dunn. It seems to me--yes. No, I mean, I think--I don't
know that I could do biweekly reporting like that, so--thank
you very much again, Dr. Shuren, you have been a really
excellent witness, and a great guy. Thank you.
I yield back.
Ms. Eshoo. Isn't that lovely? Isn't that nice for you to
hear, Dr. Shuren?
Thank you, Dr. Dunn.
All right. We have one more member to question before we
take a break. There are eight votes, and I will return ASAP
upon the last vote being cast for the second panel.
The gentlewoman from Massachusetts, Congresswoman Trahan,
is recognized for your 5 minutes.
Mrs. Trahan. Well, thank you, Chairwoman Eshoo, Ranking
Member Guthrie, for convening us here today to discuss the
importance of medical devices.
You know, I just want to start with an issue that has
dominated the conversation around supply chain shortages, and
that is semiconductor chips. Over the last few months my office
has heard from several companies facing dire chip shortages,
critical devices, from mammography screening to defibrillators
to diagnostic scanners. The chip shortage is hindering
companies' ability to upgrade equipment, meet market demands,
and sustain a thriving job market.
So, Dr. Shuren, in your position have you heard much about
this challenge?
Is there a role for the FDA, either through cooperation
with the Department of Commerce or working with companies, to
facilitate upgrades from legacy chips to advanced chips?
Dr. Shuren. The answer is yes. We have heard a lot. We have
talked to a lot of manufacturers. We have had conversations
with the Department of Commerce and other folks in government
to sort of convey the importance of these chips to a variety of
medical devices.
And, of course, the shortage on chips is, of course,
leading to and contributing to shortages on medical devices.
And appreciate, too, those chips are used in a variety of
technologies, and there is lots of needs out there. But we have
really been trying to advocate for the needs here in public
health.
And certainly, when it comes to, like, the Defense
Production Act too, we have got HHS, who plays, you know, the
lead role here, or one of the lead roles in trying to advance
that, in partnership with other parts of the government.
Mrs. Trahan. Great. I appreciate that.
You know, in recent years we have seen an influx of
software using artificial intelligence in the medical device
space, ranging from imaging tools used in radiology to insulin
pumps that automatically adjust. And, unlike a scalpel or X-ray
machine, AI-powered medical software needs to be continually
updated by design. These tools are built so that they continue
to learn and improve from data they collect while they are
deployed.
And the FDA requires that software that undergoes
significant changes go through that approval process a second
time. This is a necessary safeguard, but can slow the approval
process for updated versions of medical software, especially
those using data collected post-market.
So, as a part of its proposed artificial intelligence
action plan, the FDA has described accepting a pre-determined
change control plan as a part of the pre-market submission
process for software as a medical device. And in the control
plan a manufacturer could--would detail ahead of time how it
plans to change its software and establish how those changes
will not alter the safety and effectiveness of the software.
So how would pre-determined change control plans in pre-
market applications affect the approval process for software as
a medical device?
Dr. Shuren. So where it may be applicable to have a plan
like that where the manufacturer is laying out here are the
changes we will--we want to make, and here is how we will
assure that those changes are not, for example, adversely
impacting the safety or effectiveness of the technology, and if
we are looking at that, and that plan makes sense, and that is
going to work, and we go ahead and authorize that either as
part of authorizing the device--or a company could come in
later and just come in with that plan, then you are expediting
updated, modified devices within the context of that plan,
expediting patient access, because the FDA isn't going to look
at it. We have already looked at what the manufacturer will be
doing.
What is important, though, of course, is assuring that you
have got the safeguards in place, that when those changes are
made, in fact, it remains safe and effective technology, and we
have the ability to take action if problems arise in the
future.
Mrs. Trahan. That is great. And there are other regulatory
tools that the FDA can use to promote continued evaluation of
software as a medical device throughout its product Life Cycle?
Dr. Shuren. Well, we think this is a perfect opportunity
for sort of marrying up, you know, more of a continued
evaluation from Real-World Evidence.
That is why, you know, part of our investment in those
real-world data sources is can we be using that to, you know--
if you got technology out there, and we are just--you know, we
are learning from it, we are kind of keeping tabs. And software
as a medical device, including with AI capabilities, is really
ripe candidates for doing that. Even, you know, where we can,
leveraging information that the device itself is collecting on
itself--almost like a black box in an airplane. So we think
those kinds of methods are really things we would incorporate.
Where we are limited, though, is some of the constraints
that we have under the current law, and another reason why we
think having more flexibility to tailor pathways that better
fit these and other kinds of technologies, but do it
voluntarily, you know--so, Company, pick the old way or pick
the new way, and if you like--and want to change your mind,
flip to the other later.
But if we can do that and build it right, then we can take
advantage and get the best of both worlds. We would actually
have better assurances of safety and effectiveness, and more
expedited time to the market.
Mrs. Trahan. Thank you. I know my time----
Ms. Eshoo. The gentlewoman's time has expired.
Mrs. Trahan. Thank you.
Ms. Eshoo. Dr. Shuren, thank you. Thank you for your
patience today with our schedule. Thank you for the work that
you have done, what your entire team, the center--this has been
a test like no other, the last two years. And, you know, the
work that you and everyone at--you know, at the center at the
FDA have done--extraordinary work under extraordinary
circumstances. So bravo to you, to all of the people there.
Thank you for the work that has been put into this negotiation.
We will, of course, move it along. And we look forward to
continuing to work with you to really produce for the American
people. So thank you.
The committee is going to recess now, and I just want to
close--because she won't be here when we come back for the
second panel--to recognize Kim--what is the matter with me? Too
much talking today. Kim, we miss you, but it is great to see
you out there.
And Dr. Shuren, you are fortunate. You are fortunate to
have Kim right there with you. Bravo.
OK, so we are going to go and vote. We will be back as soon
as we can after the eighth vote is cast, and hear from our
second panel. Thank you again. Bravo.
[Recess.]
Ms. Eshoo. The Health Subcommittee will reconvene, and I
want to thank our--the witnesses of our second panel, and I now
would like to introduce them.
Ms. Janet Trunzo is the senior executive vice president of
technology and regulatory affairs for the Advanced Medical
Technology Association. We know them as AdvaMed.
Welcome to you, and thank you.
Ms. Diane Wurzburger is the executive of regulatory affairs
for GE Healthcare, and is testifying on behalf of the Medical
Imaging and Technology Alliance, MITA, M-I-T-A, where she
serves on the board of directors and chair of the technical and
regulatory committee.
Mr. Mark Leahey is the president and chief executive
officer of the Medical Device Manufacturers Association, MDMA.
And with us here in person--patiently waited, I am sure,
just about all day--Dr. Richard Kovacs. He is the chief medical
officer and past president of the American College of
Cardiology. He is also a practicing cardiologist and a
professor at the Indiana University School of Medicine.
Thank you very much, Dr. Kovacs, and welcome to our
subcommittee.
We thank all of the witnesses for joining us today. We are
looking forward to your testimony.
And we will go straight to you, Dr. Kovacs. You are
recognized for 5 minutes. I think you probably know what the
lights are.
Turn your microphone on, and a warm welcome to you, and the
gratitude of the entire committee for being with us.
STATEMENT OF RICHARD J. KOVACS, M.D., Q.E. AND SALLY RUSSELL
PROFESSOR OF MEDICINE, INDIANA UNIVERSITY SCHOOL OF MEDICINE,
CHIEF MEDICAL OFFICER, AMERICAN COLLEGE OF CARDIOLOGY; MARK
LEAHEY, PRESIDENT & CEO, MEDICAL DEVICE MANUFACTURERS
ASSOCIATION; JANET TRUNZO, SENIOR EXECUTIVE VICE PRESIDENT,
TECHNOLOGY AND REGULATORY AFFAIRS, ADVANCED MEDICAL TECHNOLOGY
ASSOCIATION (ADVAMED); AND DIANE WURZBURGER, EXECUTIVE OF
REGULATORY AFFAIRS, GE HEALTHCARE
STATEMENT OF RICHARD J. KOVACS, M.D.
Dr. Kovacs. Chairwoman Eshoo and Ranking Member Guthrie and
the distinguished members of the subcommittee, I am Dr. Richard
Kovacs. I have been introduced. I am proud to represent the
ACC, a 54,000-member professional society whose care team
members work to transform cardiovascular care and improve heart
health.
The college's activities include leading in education,
bestowing credentials on highly qualified individuals,
accrediting high-quality institutions, publishing leading
medical journals, and maintaining national cardiovascular data
registries to improve care.
Today I am here to discuss re-authorization of MDUFA from a
clinician's perspective, with special emphasis on four topics:
listening to the patient; attention to the Total Product Life
Cycle; use of Real-World Evidence for safety and efficacy; and
advancing regulatory science.
We and my colleagues use medical devices on a daily basis
to serve and heal our patients. We ask our patients what is
important to them, and we listen carefully.
Let me give you an example. Before I flew to Washington
last night, I saw Richard, an 86-year-old man from northwestern
Indiana. He likes to work in his yard. He, unfortunately,
suffered from aortic stenosis, a severe narrowing of the main
outlet valve of his heart. Three weeks ago he couldn't walk
fifty feet from his car into the hospital door because he was
so short of breath. He received a transcatheter aortic valve
replacement, left the hospital within 48 hours, has no scar on
his chest, and feels great. When I saw him yesterday, he is
back in his garden.
But in the longitudinal care, our care doesn't stop with
the implantation of a device like this.
A few weeks ago I had to say goodbye to another patient,
Carlos, a 70-year-old man from northeastern Indiana who I met
in 1989, when he needed an aortic valve replacement. We
selected a mechanical valve. It was implanted surgically. Yes,
he did have a scar, but that valve functioned flawlessly for
the next 33 years, until he passed away from another disease.
So we urge you to listen to patients, and engage the
patient voice from the earliest phases of development and
throughout the life cycle of the medical device. The patients
will tell you what really matters to them.
We have specific recommendations in our written submission.
They correspond a lot to what Dr. Shuren said earlier today.
Clinicians like me use these devices every day. And like
the example of Carlos, I may manage a patient for decades.
Cardiology is a specialty where the pace of change is rapid,
and innovations in our DNA. Clinicians have a great deal to
offer in this process. We support the TPLC Advisory Program and
its efforts to facilitate the early involvement of clinicians
in the product life cycle.
It is impossible to know everything about a device from
early clinical trials. So Real-World Evidence of safety and
efficacy, evidence that can be gleaned from clinical data
registries like our National Cardiovascular Data Registry is
what I rely on through the product life cycle. We should
leverage these data for the public good.
Registries can be cost savings. Sponsors that have used
registries to house post-approval studies have achieved savings
of 40 to 60 percent over the usual clinical trials.
Safety and efficacy are also best supported by sound
regulatory science. Science is a team sport these days, and
teams of industry employees, academics, and regulators can
solve these problems. The Cardiac Safety Research Consortium is
such a collaboration, and it has made important advancements in
drug safety. The ACC supports the FDA Network of Experts
program, and can expand access to experts in cardiovascular
disease.
Finally, I want to say we also support the efforts to
provide additional cybersecurity. I have had the experience of
working in two hospitals that have been hacked, and the care
deteriorates dramatically.
So thank you for your interest. And on behalf of our
patients and our profession, thank you for allowing us to be
part of this process, and I look forward to any questions.
[The prepared statement of Dr. Kovacs follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. Thank you, Doctor.
Next, Mr. Leahey, you are recognized for your 5 minutes of
testimony, and thank you.
[Pause.]
Ms. Eshoo. We have a problem, or----
Voice. He needs to go off mute.
Ms. Eshoo. Pardon me?
Voice. He needs to go off mute.
Ms. Eshoo. Oh, I see.
Mr. Leahey. Can you hear me now?
Ms. Eshoo. You have to unmute yourself, Mr. Leahey.
Mr. Leahey. Yes, can you hear me?
Ms. Eshoo. We can hear you, yes.
Mr. Leahey. OK, great.
STATEMENT OF MARK LEAHEY
Mr. Leahey. Thank you, Chairwoman Eshoo, Ranking Member
Guthrie, and members of the subcommittee, for the opportunity
to testify today. My name is Mark Leahey, and I am the
president and CEO of the Medical Device Manufacturers
Association, a national trade association representing hundreds
of medical technology companies.
MDMA was founded in 1992 to be the voice of the innovative
and entrepreneurial sector of the industry. While the industry
is broadly represented throughout the United States, one of the
unique components of this vibrant part of America's innovation
ecosystem is that the majority of companies are small
businesses. According to data from the Department of Commerce,
over 98 percent of med tech companies have fewer than 500
employees, and more than 80 percent have less than 50
employees, yet they are the major source of innovation in
America's competitive advantage in medical technology.
Our industry is dedicated to one mission: to alleviate
human suffering and to improve patient care. Our industry has a
proud tradition of answering the needs of patients and
providers, and perhaps no example is more profound than what
innovators have done since the outset of the COVID-19 pandemic.
Whether it was respiratory devices, diagnostics, advanced
patient monitoring, or personal protective equipment, the
medical technology industry worked tirelessly to help the
United States and the entire world to confront this challenge,
and they continue to do so today.
In addition to the extraordinary efforts of this industry
and to healthcare professionals, I would also like to take a
moment to acknowledge the dedicated professionals at the FDA
who worked 24/7--COVID and non-COVID medical technologies to
improve patient care during the pandemic. Their efforts ensured
that patients had timely access to safe and effective medical
technologies.
The MDUFA V draft agreement that we are discussing today
and the historic increase in user fee funding that it contains
demonstrates our commitment to provide additional capacity and
expertise to further advance the FDA's mission. MDUFA V five
provides over $2 billion in investable funding to FDA.
As a point of reference, MDUFA totaled approximately $150
million over the five years of the program. While each MDUFA
typically provides funding for an additional 200 new hires,
under MDUFA V FDA will be able to hire a minimum of 273 people,
and up to 387 new people to support the MDUFA program. This
represents a historic increase in both overall funds and
people, and it is our hope and expectation that this will be
the last major investment needed for the MDUFA program, and
that moving forward, any necessary increases will be much more
modest and targeted.
With these significant investments, MDUFA V also
establishes more transparency around the use of funds,
including ensuring that annual hiring targets are met. FDA will
also conduct an H.R. assessment during the MDUFA V to identify
how many MDUFA-funded vacancies exist. Currently, CDRH is only
able to track MDUFA IV and later FTEs. Public reports in 2016
indicated MDUFA-funded vacancies exceeded 25 percent, and
innovators want to ensure that the additional capacity that we
are funding through user fees is realized in new additional
hires and backfilling any vacancies that arise.
Beyond the financial accountability and transparency
provisions that MDUFA V contains, performance goals associated
with the de novo and PMA total time to decision also improved
over the course of the agreement.
One goal that was elusive under MDUFA IV was the 510K total
time to decision goal in Fiscal Year 2022 of 108 days. As was
mentioned earlier, COVID did impact FTE capacity, including the
ability to meet certain MDUFA IV goals. Under MDUFA V, the 510K
total time to decision goal will ramp down each year, hopefully
achieving 108 days by Fiscal Year 2026.
MDUFA V also expands investments in patient science and
engagement to enhance the patient perspective into the medical
device evaluation process. As we all know, America's medical
technology ecosystem was not built overnight. It took decades
of work between countless stakeholders including Congress, the
FDA, innovators, physicians, patient groups, and more to design
the regulatory pathways that has resulted in the gold standard
of safety and efficacy. At the same time, we all recognize that
this is a delicate balance to ensure that the right policies
are in place to support innovation and to spur the next
generation of cures, therapies, and diagnostics that patients
are relying on.
As I noted, this is a historic investment in the FDA, and
it will be critical over the coming years to meet the goals and
milestones within the user fee agreement help ensure that the
United States remains the global leader in medical technology
development.
It is also critical that Congress continues its vital
oversight role in providing the necessary resources and
investments to FDA for it to achieve its mission.
MDMA and our member companies remain committed to working
closely with you to reach our shared goal of providing safe and
effective medical technologies to patients and providers in a
timely manner. Thank you once again for this committee's
passionate leadership on this important work, and I look
forward to answering any questions that the committee members
may have. Thank you very much.
[The prepared statement of Mr. Leahey follows:]
Prepared Testimony of Mark Leahey
President and CEO, Medical Device Manufacturers Association (MDMA)
``FDA User Fee Reauthorization: Ensuring Safe and Effective Medical
Devices'' March 30, 2022
House Energy and Commerce Committee, Subcommittee on Health
Thank you Chairman Pallone, Ranking Member McMorris
Rodgers, Subcommittee Chairwoman Eshoo, RankingMember Guthrie
and Members of the committee for this opportunity to testify
today. My name is Mark and I am the President and CEO of the
Medical Device Manufacturers Association (``MDMA''), a national
trade association representing hundreds of medical technology
companies. MDMA was founded in 1992 to be the voice of the
innovative and entrepreneurial sector of our industry.
While the industry is broadly represented throughout the
United States, one of the unique components of this vibrant
part of America's innovation ecosystem is that the majority of
companies are small businesses. According to data from the
Department of Commerce, over 98% of med tech companies have
fewer than 500 employees, and more than 80% have less than 50
employees, yet they are the major source of innovation and
America's competitive advantage in medical technology. Our
industry is dedicated to one mission: to alleviate human
suffering and improve patient care.
Our industry has a proud tradition of answering the needs
of patients and providers, and perhaps no example is more
profound than what innovators have done since the outset of the
COVID-19 pandemic. Whether it was respiratory technologies,
diagnostics, advanced patient monitoring, or personal
protective equipment, the medical technology industry worked
tirelessly to help the United States and the entire world to
confront this challenge, and they continue to do so today. In
addition to the extraordinary efforts of this industry and
healthcare professionals, I would also like to take a moment to
acknowledge the dedicated professionals at the FDA who worked
24/7 on COVID and non-COVID medical technologies to improve
patient care during the pandemic. Their efforts ensured that
patients had timely access to safe and effective medical
technologies.
The MDUFA V draft agreement that we are discussing today,
and the historic increase in user fee funding that it contains,
demonstrates our commitment to provide additional capacity and
expertise to further advance their mission.
MDUFA V provides over $2B in investable funding to FDA. As
a point of reference, MDUFA I totaled approximately $150M over
the five years of the program. While each MDUFA typically
provides funding for an additional 200 new hires, under MDUFA
V, FDA will be able to hire a minimum of 273 FTEs and up to 387
new FTEs to support the MDUFA program. This represents a
historic increase in both overall funds and people, and it is
our hope and expectation that this will be the last major
investment needed for the MDUFA program and that moving
forward, any necessary increases will be much more modest and
targeted.
With these significant investments, MDUFA V also
establishes more transparency around the use of the funds,
including ensuring that annual hiring targets are met. FDA will
also conduct a HR assessment during MDUFA V to identify how
many MDUFA funded vacancies exist. Currently, CDRH is only able
to track MDUFA IV and later FTEs. Public reports in 2016
indicated MDUFA funded vacancies exceeded 25%, and innovators
wants to ensure that the additional capacity we are funding
through user fees is realized in the new additional hires and
backfilling any vacancies that arise.
Beyond the financial accountability and transparency
provisions that MDUFA V contains, performance goals associated
with De Novos and PMA Total Time to Decision (TTD) also improve
over the course of the agreement. One goal that was elusive
under MDUFA IV was the 510(k) Total Time to Decision Goal in
FY22 of 108 days. As was mentioned earlier, COVID did impact
FDA capacity, including the ability to meet certain MDUFA IV
goals. Under MDUFA V, the 510(k) TTD goal will ramp down each
year, hopefully achieving 108 days by FY26. MDUFA V also
expands investments in Patient Science and Engagement to
enhance the patient perspective into the medical device
evaluation process.
As we all know, America's medical technology ecosystem was
not built overnight. It took decades of work between countless
stakeholders, including Congress, the FDA, innovators,
physicians, patient groups and more to design the regulatory
pathways that has resulted in the gold standard of safety and
efficacy. At the same time, we all recognize that this is a
delicate balance to ensure that the right policies are in place
to support innovation, and to spur the next generation of
cures, therapies and diagnostics that so many patients are
relying on. As I noted, this is a historic investment in the
FDA, and it will be critical over the coming years to meet the
goals and milestones within this user fee agreement to help
ensure that the United States remains the global leader in
medical technology development. It is also critical that
Congress continues its vital oversight role, and providing the
necessary resources and investments to FDA for it to achieve
its mission. MDMA and our members remain committed to working
closely with you to reach our shared goal of providing safe and
effective medical technologies to patients and providors in a
timely manner. Thank you once again Chairman Pallone, Ranking
Member McMorris Rodgers, Subcommittee Chairwoman Eshoo and
Ranking Member Guthrie for your passionate leadership on this
important work, and I look forward to answering any questions
that the committee members might have.
Ms. Eshoo. Thank you, Mr. Leahey. And for all the work that
you and your colleagues put into the negotiations, bravo.
Ms. Trunzo, you are now recognized for your 5 minutes of
testimony.
[Pause.]
Ms. Eshoo. You need to unmute.
[Pause.]
Ms. Eshoo. Can you hear me?
Ms. Trunzo. Yes, I can.
STATEMENT OF JANET TRUNZO
Ms. Trunzo. Thank you----
Ms. Eshoo. OK.
Ms. Trunzo [continuing]. Very much, Chairwoman Eshoo,
Ranking Member Guthrie, and members of the committee. Thank you
so----
Ms. Eshoo. There you are.
Ms. Trunzo [continuing]. Much for inviting----
Ms. Eshoo. Thank you.
Ms. Trunzo [continuing]. The Advanced Medical Technology
Association, or AdvaMed, to testify on the reauthorization of
the Medical Device User Fee Program.
This legislation is critical to patients continuing to have
access to innovative, safe, and effective medical technologies,
and we are grateful for the opportunity to offer our insights
today.
AdvaMed is the world's largest trade organization
representing medical technology companies. AdvaMed represents
more than 400 medical device manufacturers, of which there are
300 small companies. I had the pleasure of representing AdvaMed
during the discussions of the very first user fee program, the
Medical Device User Fee and Modernization Act of 2002, and each
of the re-authorizations since then.
The ongoing public health emergency created uncertainties
that presented a significant challenge to our MDUFA
discussions. Yet AdvaMed believes that the collective efforts
of the industry and FDA have produced an agreement that will
further strengthen the medical device pre-market review
program. This will advance the ultimate shared goal of patients
having timely access to safe and effective medical devices.
From the very first user fee program in 2002, the
underlying principle is that user fees supplement existing
appropriations so that FDA has the resources necessary to
support timely review of submissions. While user fees support
overall timeliness and predictability, they neither guarantee a
particular result nor guarantee the timing of any particular
application review. Those remain completely under FDA's
authority.
Industry and FDA have taken the opportunity during each re-
authorization to refine and improve the goals. Each MDUFA cycle
included significant increases in investments by increasing the
number of new FTEs to support the anticipated workload. For
MDUFA V, AdvaMed and the industry representatives approached
the re-authorization with the same two overarching principles
we have had in the past: patients must continue to benefit from
access to safe and effective medical devices; and the
associated goals of the user fee program should be refined and
improved.
However, we also recognize that the COVID-19 public health
emergency had required a significant effort on the part of FDA.
As a result, we believe the device center needed to focus on
the fundamentals of the device review program, which we refer
to as Back to Basics. AdvaMed believes the package is well
crafted to provide significant resources and capacity for FDA,
greater predictability for the industry, and is in the best
interests of patients.
It has the following key components.
First, the general goal structure for submissions is
unchanged. Over the course of MDUFA V we expect to see
improvements in review times compared to MDUFA IV goals, or
compared to current performance, depending upon the submission
type.
Second, the package provides significant additional
resources to ensure that FDA can provide timely feedback to
companies seeking pre-submission guidance from the agency. This
process enhances the likelihood of an efficient review of the
product submission.
Third, this package will fund targeted initiatives to
support the pre-market review program. For example, there is
increased funding for patient science and engagement to enhance
the incorporation of the patient experience into the medical
device evaluation process.
Fourth, this package contains specific accountability
measures for the evaluation of the program by funding a quality
management program and two independent assessments of the
review process.
Finally, this agreement provides enhanced public
transparency of MDUFA financing, including clarity on the use
of the carryover balances.
On behalf of AdvaMed, we look forward to working with
Congress, FDA, and stakeholders on the re-authorization of the
Medical Device User Fee Program so that our common goal of
timely patient access to safe and effective medical devices is
realized. Thank you.
[The prepared statement of Ms. Trunzo follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. Thank you, Ms. Trunzo. You have been there from
the beginning, so bravo to you. And it is wonderful to have you
as a witness.
Next, Ms. Wurzburger, you are recognized for 5 minutes for
your testimony, and welcome, and thank you.
STATEMENT OF DIANE WURZBURGER
Ms. Wurzburger. Thank you and good afternoon, Chairwoman
Eshoo, Ranking Member Guthrie, and distinguished members of the
subcommittee. Thank you for the opportunity to appear before
you today to discuss the FDA's Medical Device User Fee Program
on behalf of the Medical Imaging and Technology Alliance, also
known as MITA.
MITA is the primary trade association and standards
development organization representing the manufacturers of
medical imaging technologies, including magnetic resonance
imaging, medical X-ray equipment, computed tomography scanners,
ultrasound, nuclear imaging, radiopharmaceuticals, AI-enabled
imaging software, and other products. MITA member companies'
technologies play an essential role in our Nation's healthcare
infrastructure and are integral in the care pathways of
evaluating, staging, managing, and effectively treating
patients with cancer, heart disease, neurological degeneration,
COVID-19, and numerous other medical conditions.
By catching disease early, reducing the need for invasive
inpatient procedures, and facilitating shorter recovery times,
medical imaging saves money and improves efficiency in the
healthcare system. Medical imaging technologies have
revolutionized healthcare delivery in America and around the
world, extending human vision into the very nature of disease.
A consistent and timely FDA review process is essential to
timely patient access to these technologies. MITA continues our
strong support for an effective, well-resourced FDA capable of
fulfilling its mission to protect and promote the public
health. The medical imaging industry supported enactment of
FDA's user fee programs in 2002 and its subsequent re-
authorizations in 2007, 2012, and 2017. We participated
alongside our industry colleagues in the MDUFA V negotiations,
and support enactment of the proposed agreement, which will
provide the FDA device program with ample resources, establish
new accountability measures, and allow for exploration of new
review paradigms such as the Total Product Life Cycle Advisory
Program, also known as TAP.
User fees provide for an efficient pre-market review
process, allowing the safe and effective medical device
innovations to get patients--to get to patients and healthcare
providers in an expedient, consistent, and transparent manner.
Supplementing FDA funding with user fee brings stability and
predictability to the device review process and timelines.
The goals of the medical device industry and FDA commit to,
and FDA's subsequent performance are critical to timely
patients' access to safe and effective medical advancements.
Without a consistent and timely FDA review process conducted by
well-trained FDA staff, access to diagnostic imaging
technologies will be delayed, and industry's ability to deliver
technological advancements will be compromised.
We, therefore, will continue to partner with FDA and other
stakeholders in asking Congress to re-authorize this important
program that supports patient access to safe and effective
medical imaging innovations.
MDUFA V was negotiated during turbulent times for all parts
of our healthcare system, including innovators, regulators,
healthcare providers, and patients. The COVID-19 pandemic
strained every part of our society. FDA and industry strived to
meet the challenges presented by this public health emergency
by ensuring safe and effective medical devices could be
delivered to patients in an expeditious manner.
The last several years created significant resource
challenges for FDA, and as it seeks to recover its operations
and get back to pre-pandemic performance, it will need to be
sufficiently resourced to meet its obligations and continue to
review products for safety and effectiveness.
The MDUFA V agreement will raise the Center for Devices and
Radiological Health's funding significantly, allowing the
center to meet its pre-market review commitments. It will also
be able to hire new FTEs and meet rising payroll costs. And the
agency will also continue to invest in successful programs that
support the use of standards and Real-World Evidence in
regulatory pre-market decisions; the advancement of digital
health technologies; the expansion of patient engagement
opportunities to inform the development and evaluation of
innovative technologies; FDA's engagement with international
regulators and the promotion of regulatory convergence; as well
as continued FDA collaboration with accredited third-party
reviewers to support a voluntary alternate review pathway.
MDUFA V will bring new accountability measures and ensure
FDA--excuse me, user fee dollars are being appropriately
invested in shared goals, and also support multiple independent
assessments of performance and generate recommendations on how
the center can continue to improve its operations.
In closing, MITA urges Congress to move quickly to
enactment of MDUFA V. This agreement, negotiated between FDA
and the medical device industry over the last year-and-a-half,
will ensure ongoing patient access to safe and effective
devices.
Thank you for the opportunity to present our views today. I
am happy to answer any questions you may have.
[The prepared statement of Ms. Wurzburger follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. Thank you, Ms. Wurzburger. And that concludes
the testimony of our five witnesses. And thank you once again
for being with us today, and your patience in terms of the
House schedule. We will now move to member questions, and the
Chair will recognize herself for 5 minutes to do so.
Dr. Kovacs, in your written testimony you said that more
needs to be done to use patient input to inform clinical study
design in order to recruit and retain a diverse patient sample.
In your view, what else should the FDA and the medical device
manufacturers be doing to recruit more diverse patients in
device clinical investigations?
Dr. Kovacs. I think that Dr. Shuren had many good points to
make this morning: going to where the patients are, to approach
them in their environment; to use telemedicine and
telecommunications to reach patients that otherwise are
unreachable. And I would add one additional point to what Dr.
Shuren mentioned this morning, and that is also to diversify
our investigators.
The actual clinical investigators need to look like the
patients that are enrolled in these trials. Outside the scope
of MDUFA, but within the scope of what could be--help with
legislation to improve the training, to increase our pipeline.
The college is working on this, but we realize we are a small
organization trying to get way upstream of this to diversify
our investigative team.
Ms. Eshoo. Excellent. Well, Dr. Shuren stayed because he
wanted to hear your testimony and that of the others that are
with you today. So he is listening very intently.
To Ms. Trunzo, you said that the device review program
needs to, quote, ``go back to the basics'' in order to balance
COVID-19 demands with the center's regular workload. How does
MDUFA V help address the center's capacity gap, in your view?
[Pause.]
Ms. Eshoo. You need to unmute.
[Pause.]
Ms. Eshoo. Are we putting each other to sleep?
[Laughter.]
Ms. Trunzo. So sorry.
Ms. Eshoo. That is all right.
Ms. Trunzo. I believe that the investment from MDUFA V that
we have discussed in our testimony of about $1.78 billion in
guaranteed funding allows for FDA to hire the additional FTEs
needed so that there is sufficient resources and capacity for
FDA to get back to basics.
It is--if you look at the number of additional FTEs that
FDA will get as a result of this investment, it will support
the medical device review program and get us back to basics.
Thank you.
Ms. Eshoo. Thank you.
To Ms. Wurzburger, do you think the term--and we discussed
this earlier today with Dr. Shuren--do you think the term ``re-
manufacturing'' needs further clarification in statute, despite
the FDA's draft guidance? And if so, why?
Ms. Wurzburger. Thank you. Yes. MITA agrees with Dr. Shuren
that clarification is needed, and the legislation
recently[inaudible] Representatives Peters, Schrier, and Joyce
provides that clarity.
While the FDA guidance remains in draft form, there is
vaule for providing greater clarity via statute for the
activities that could significantly be for performance or
safety specifications, or intended use of the device are clear.
Additionally, the legislation--legislative proposal also
includes provisions to provide public education and
transparency or awareness of manufacturers' regulatory
responsibilities, and to promote compliance. Thank you.
Ms. Eshoo. Thank you.
I am going to yield back my time and recognize our
wonderful ranking member of our subcommittee, Mr. Guthrie, for
your 5 minutes of questions.
Mr. Guthrie. Thank you. Before I get to my questions,
earlier today--I am glad Dr. Shuren is still here--we were
talking about device shortage, the proposal, and we are all for
patient safety. That is premier, first and foremost, moving
forward. And I think someone said--I don't remember who,
exactly--the difference in pharmaceutical and device. And it is
all about patient safety.
So those--there is no difference between the two, except I
know that, when we did the pharmaceutical, I was hearing a lot
of calls from people who were--I had, like, people driving
ambulances say they didn't have basic pharmaceuticals. They
were canceling surgeries because they didn't have the basic
pharmaceuticals. And when you looked at it, it was small. It
was high volume, small margin, usually one supplier, and so
forth.
And we were really concerned that you just couldn't plan.
It would be like you are getting 100,000--I am making the
number up, but say you are supposed to get 100,000 a week from
this supplier, and you get 50 one week, 120 one week, 30 next
week, it depends on the disruptions. And that is what we were
looking for, and that is what hearings are for. Maybe there is
the same problem in device.
But there is a difference. I am a manufacturing--not--
aluminum parts, not pharmaceutical. But there is a big
difference in just not hitting your targets week in and week
out, and all of a sudden committing to 100,000, and all of a
sudden you need a million. I mean, that is kind of what
happened with our pandemic. And that is a different problem.
And it is just a different problem.
I just want to--if you are going to expect somebody to go
from 100,000 to 10 times, or 5 times, or however much they need
there, the government has to buy the capacity or they have to
store it. I mean, the storage. And that is something that we
need to work through, and to make sure we have the--it
correctly.
But if the same problem is we are just not getting the
devices on a regular basis, like the same thing, we need to
address that, too. We need to address that, too. So that is
what we need to sort out in the hearing.
But the other thing I asked about this morning with Dr.
Shuren is emerging signals. And again, we are all--patient
safety is premier. But Ms. Trunzo, is there a way that you
can--we can balance, or make sure that we have--we promote
innovation and we get this right with the signaling without
compromising patient safety?
[Pause.]
Mr. Guthrie. Ms. Trunzo? Did----
Ms. Trunzo. Sorry. I believe there is----
Mr. Guthrie. OK.
Ms. Trunzo. I am sorry. I believe there is a way to balance
the emerging signals program. I think it is really important
that the program allows for, if FDA does detect an emerging
signal, that there is an opportunity for the company to
interact with FDA, because sometimes the company may have
supplemental information that may be crucial to the evaluation
process that FDA is undertaking.
And I think the other important factor in an emerging
signal program is the ability for FDA to--because it is
emerging signals and it may not be confirmed, and if it later
is confirmed, or later confirmed ought to be an emerging
signal, then it would be important for the FDA to somehow
communicate that to healthcare providers during that process.
But there are----
Mr. Guthrie. OK, on that--I should have muted my phone, so
we don't get that--I mean my talk button, so I don't get the
feedback, but I--but on that, Dr. Shuren, I thought, brought a
valid point about how the timing that could take to get that
done. If it is--is there a proposal that you are moving forward
that would say, if it is an emergency situations--I understand
that there is--FDA detect emerging signal, and you want--need
the time to respond, because you want to make sure that you
have the opportunity to, and I understand that and fully
support that, except is there a criticalness to the time, the
timing of some are and some aren't, I guess? And so how do we
decide which ones are and which aren't?
Ms. Trunzo. I believe that there--that is a delicate
balance to achieve of the actual timing of that information.
And I think that goes back to why it is so important.
If FDA, through the data sources that FDA has access to, if
FDA does determine that there might be an emerging signal, that
initial interaction with the company is really--it should be
part of the process, because the company may be able to provide
additional information, which would then make that process more
efficient and timely in the final determination that FDA will
make.
Mr. Guthrie. OK. I have one quick--if I can get it in
really quickly. So the--Ms. Trunzo, the Breakthrough Device
Program, we believe it has been innovative. And what
improvements can we make to the path--this pathway to
incentivize further investments in emerging technologies?
Ms. Trunzo. Well, I think the breakthrough process has seen
a lot of emphasis, most recently--especially after the 21st
Century Cures Act, where the whole breakthrough process was
well defined, there was a timeline built into the breakthrough
designation process so that FDA had a specific time of 60 days
to respond to requests for getting that breakthrough
designation.
And I think the investment in the Total Product Life Cycle
Program that is part of MDUFA V, which will--once that
designation is made, and that sponsor participates in this
program, there are--will be significant resources to support
the pre-submission process, such that when that final
submission is made to FDA as a result of that investment of the
additional resources to help the company through that process,
that product will have a more efficient review and get into the
hands of patients and healthcare providers.
Mr. Guthrie. Thanks. My time is expired. I appreciate the
answers, and I yield back.
Ms. Eshoo. The gentleman yields back. You know, on this
issue of shortages, it is not just in the--on the
pharmaceutical side. Lucile Packard Children's Hospital, right
in the heart of my congressional district, reported to us that
they have a heparin syringe shortage right now. So, you know,
we have to look after all of this.
And Dr. Shuren, you are here, and I know you are going to
followup on that. So thank you.
All right, the Chair now recognizes the gentleman from
California, Mr. Cardenas, for your 5 minutes of questions.
Mr. Cardenas. Thank you. Thank you very much, Madam
Chairwoman and Ranking Member, I really appreciate this
opportunity to talk to this second esteemed panel.
In your--Dr. Kovacs, in your testimony you talk about the
importance of the patient perspective, and share some stories
from your own experience. Many times we talk about improving
devices and therapies, and somewhere along the way the impact
on real people can get lost.
My first question for you is, how big of a difference can
these devices make in a person's life?
And when we talk about the expeditious approval of safe and
effective devices, what does that actually look like for
patients in their day-to-day lives?
Dr. Kovacs. Thank you for the question. These devices range
from lifesaving devices in what I do in cardiology, to a life-
altering devices: the difference between being able to work or
not work, the difference to being able to be mobile or not
mobile, the difference between being able to enjoy one's family
or not. So these make huge differences.
But the differences that they make to the patients, I would
reemphasize, we should be asking the patients. What is the most
important thing to the patient? What may be important to one
patient in one situation may be different to another patient.
This revolves around the whole science of patient-reported
outcomes to the statistical analysis of these patient-reported
outcomes, and to bringing these into part of the equation for
designing the trials in the first place.
So we need to--and as we said, we need to diversify the
number of the types of patients that are in these trials to
understand the differences in patient desires for the outcomes
that they are hoping for these novel therapies.
Mr. Cardenas. Thank you. You also note in your testimony
the importance of emphasizing patient engagement in the medical
device approval process. Among your recommendations to improve
these processes, you advocate for ``patient input to inform
clinical study design,'' which would reduce barriers for
diverse patient samples.
How would you recommend the FDA receive and operationalize
this kind of input?
Can you explain what this would look like on the ground,
from the patient perspective?
Dr. Kovacs. This would look like, first of all, engaging
the patients, engaging that diverse patient population into the
design of the studies, and the endpoints of the studies which
determine the scientific rigor of the study.
Is this--this goes all the way back to the definitions.
What is a patient-reported outcome? Is it meaningful to be able
to walk from--for 50 feet? Is it more meaningful to be able to
walk for a mile? And those are nuanced, those required crisp
data definitions, and they require careful analysis by the FDA,
hopefully in conjunction with the patients and other experts.
Mr. Cardenas. OK. So you are describing a collaboration of
sorts, an understanding of what is going on with these studies,
and getting feedback from the patient, and also FDA to be
involved in that, as well?
Dr. Kovacs. Correct. I hang around with a lot of movement
disorders neurologists who tell me that when they--and they use
telemedicine, they want to observe these patients with
Parkinson's disease, for example, in their environment. And
what that therapy does to their ability to function in their
daily life is what is important to that patient. Not
necessarily a biomarker or a test result, but what the how the
patient actually functions.
Mr. Cardenas. Well, thank you. In their own environment.
Thank you very much.
Why is it important to ensure patient input is elevated,
and that diversity is a priority in the trial process?
How much of an impact will this ultimately have on patient
experience?
Dr. Kovacs. The patients that we want to apply these
therapies to--the patients in the trials that approved these
devices should look like the patients that we intend them--they
intend them to.
We have numerous examples of unintended consequences of not
including the right types of patients in clinical trials to not
understand whether a device is effective in a significant
proportion of our population. Women, for example, respond
differently to device therapy than men, and we need to
understand that going forward.
Mr. Cardenas. Thank you very much. My time is expired.
Thank you so much, Madam Chairwoman. I yield back.
Ms. Eshoo. The gentleman yields back. Thank you for
participating in this part of our hearing today, Mr. Cardenas.
The Chair is--oh, the chair is pleased to recognize the--go
to Dr. Joyce?
OK, back to you, Dr. Joyce. You are recognized for 5
minutes for your questions, the gentleman from Pennsylvania.
Mr. Joyce. Thank you, Madam Chair. Thank you, everyone, for
being here at this hearing, which we recognize was originally
convened at nine this morning.
During the first panel we heard from Dr. Shuren--and Dr.
Shuren, thank you for being here this afternoon, as well--on
this committee and proposed expansion of shortage reporting on
medical devices beyond the context of the public health
emergency. My question is first for Ms. Trunzo.
Can you please comment on the burden that this proposal
would place on device manufacturers, particularly the impact it
may have on small manufacturers, as well as what manufacturers
do already to ensure supply chain continuity?
[Pause.]
Ms. Trunzo. I can start----
Mr. Joyce. Ms. Trunzo----
Ms. Trunzo. Yes, I can start with the latter. The--our--the
companies take great efforts in managing their supply chains.
It is an art and a science to manage those supply chains to
ensure that there is not a shortage.
As far as the burden goes of what is [inaudible], it
depends on what is asked to be reported on, what kinds of
information is part of the reporting, and does it apply to all
medical devices or just a subset of medical devices, and does
it go beyond reporting, beyond the public health emergency, or
in advance of the public health emergency. So the burden is
variable, depending upon the extent to which the reporting is
required.
Mr. Joyce. Well, specifically, beyond the context of the
current public health emergency, would that add additional
burdens?
Ms. Trunzo. We believe that it would. We support--I will be
very clear to you, first of all, that we are very much
supportive of working with the committee on any kind of
additional mandatory shortage reporting.
But the way medical device manufacturers often--there are
multiple manufacturers for a specific device type. And so what
might not be a disruption in the supply chain for one
manufacturer doesn't necessarily mean that there are--there is
a shortage for that particular device type on it entirety.
Having shortage reporting be in place at all times for all
medical devices could very much be burdensome to our industry.
Mr. Joyce. Thank you.
Mr. Leahey, I am going to ask you to weigh in on this,
particularly the impact on small manufacturers to ensure the
supply chain is ready, the impact and the burden of this
reporting.
Mr. Leahey. Thank you very much. Well, as Janet just said,
you know, there are instances through the public health
emergency where issues have arose.
But I think it is important to recognize the difference
between drugs and devices. For the overwhelming majority of
medical devices, there are multiple companies selling competing
devices, and that competition creates resiliency. And we have
seen in global demand for devices needed to respond to the
pandemic created supply challenges, no doubt, early in the
pandemic. But industry has responded.
We think the CARES Act, which allows HHS and the FDA to
collect shortage information in advance and during the public
health emergency, is appropriate. But our members would have
concerns about broad new authorities to collect supply chain
information for hundreds of thousands of devices on the market
at--not at risk of supply chain disruption.
Mr. Joyce. And I would like to turn to Dr. Kovacs.
Clinically, I practiced medicine for 25 years, and my decision
to go into medicine was because, at the age of six, I lost my
5-year-old brother after an atrial septal defect repair,
something which is now done as an outpatient, which is done by
interventional cardiologists, and these young people who have
atrial septal defects have this and are, literally, sent home
within hours from the procedure.
What is the impact of the ability to advance the
development of medical devices, and how do those medical
devices impact you in your clinical practice?
Dr. Kovacs. The practice of cardiology--and I am sorry to
hear about you're your sibling, but the--and Happy Doctors Day.
Mr. Joyce. Thank you, sir. Happy Doctors Day.
Dr. Kovacs. My specialty is one that is crucially dependent
on this, and crucially dependent on innovation to advance this.
As I mentioned two patients in my testimony, one who
probably spent ten days in the hospital recovering from cardiac
surgery, one who went home without a scar within 48 hours. That
has ripple effects down the line entirely in hospital care,
fewer hospital days, lower costs, less time in the hospital,
less recovery time, less burden on the family to take care of a
family member who has been incapacitated. The benefits go on
and on.
We need to continue to spur innovation. Cardiology is a
particularly innovative sub-specialty, and we need to remove
barriers to that innovation.
Mr. Joyce. I thank you for your answer.
And Madam Speaker, my time has expired, and I yield.
Ms. Eshoo. The gentleman yields back. I may be the
chairwoman, but I know I am not Speaker.
[Laughter.]
Ms. Eshoo. But thank you for the elevation for three
seconds.
The Chair is very pleased to recognize the ranking member
of the full committee, Congresswoman McMorris Rodgers, 5
minutes for your questions.
Mrs. Rodgers. Thank you, Madam Chair.
Ms. Trunzo, as medical device technology increasingly
relies more on software updates and algorithm changes to
improve performance, how can FDA ensure that patient safety is
preserved, while also enabling these updates to be made in a
timely manner?
Ms. Trunzo. Look, I think that--I believe that one way in
which that can be accomplished is with a pre-determined change
protocol approach, where those medical device software medical
devices are constantly being updated because of the nature of
the device being a software base, that if there is a pre-
determined change protocol in place, it ensures that the
manufacturer's company will be able to do those updates under a
pre-approved protocol that FDA has pre-approved, and ensures
the safety, and at the same time allows those--to be made in a
safe manner. And I think that is the way to solve that problem.
Mrs. Rodgers. Thank you. As a--I would also like to ask--
the FDA currently has limited authority to collect information
on potential device shortages during or in advance of a public
health emergency.
Is the experience of your member companies--or in the
experience of your member companies, how has FDA used this data
to prevent or mitigate shortages thus far?
And that was for Ms. Trunzo and Mr. Leahey.
Ms. Trunzo. Well, so the way--the information has been
submitted to FDA as a result of the shortage reporting
requirement during the public health emergency, and in advance
of one.
The way in which FDA uses that information is--we are not
exactly sure how FDA uses the information and what actions FDA
takes with that information. Presumably, there is an analysis
of it. And then, once the information is published, there is a
list published on the FDA website that identifies where the
shortages are, the information that continues to be presented
to FDA, how they analyze that. And then, when the--when that
shortage no longer exists, and how they--how FDA changes that
shortage reporting list, I think is not well--it is not well
understood, from our perspective.
Mrs. Rodgers. OK, OK, OK. Thank you.
Mr. Leahey?
Mr. Leahey. I would echo what Janet said. Obviously, there
is a devine scope right now publicly available, but how that
information is being analyzed, used to provide flexibility
maybe for substitution in parts of which a shortage, I think
that is an area where we don't have a lot of visibility, but
FDA has been reaching out, I think, to industry with the group
that is handling supply chain resiliency.
So again, we are supportive of FDA having these
conversations with industry, trying to work through these
problems. But broad-based new authorities here are certainly of
concern to our members.
Mrs. Rodgers. OK. Are there--as a followup, are there
certain types of medical devices for which you think it would
be helpful for FDA to collect this information?
Mr. Leahey. Again, I think the current list right now that
exists related to products during a public health emergency--
you know, we know PPE, there were ventilator issues, other
areas that likely could continue, and the Secretary, under the
CARES authority, has the ability to continue this during a
public health emergency or in advance of one.
So I think the scope of the universe of products that FDA
is looking at right now seems right size. If there are other,
you know, targeted areas that we can have conversations around,
I think we are open to that. But again, having something that
is cascading that would, you know, apply to orthopedic implants
and cardiovascular devices and everything across the sun just
seems well beyond the scope of an efficient regulatory process.
Mrs. Rodgers. Ms. Trunzo, would you care to add anything?
[Pause.]
Mrs. Rodgers. Maybe--oh, is she muted?
Ms. Trunzo. I believe--yes, I believe that the current list
that FDA has published for purposes of reporting shortages
during the public health emergency is a sufficient and good
list.
Mrs. Rodgers. OK, OK. Thank you.
Thank you, Madam Chair. I yield back.
Ms. Eshoo. The gentlewoman yields back. I am not aware of
any other members that are----
Mr. Guthrie. No, none----
Ms. Eshoo. Not on the Republican side and not on the
Democratic side. So let me thank the witnesses of our second
panel.
Dr. Kovacs, thank you so much. You gave wonderful
testimony, all through the lens of your patients, and telling
their stories. So, you know, the way you presented, the way you
addressed this overall issue was made very real by--you
personalized the testimony. We appreciate it very much, and
also the patience of each one of you.
So to Mr. Leahey, it is great to see you. Thank you for the
work that you have done on this.
To Ms. Trunzo, thank you for your testimony. Thank you for
unmuting.
And thank you to Ms. Wurzburger.
You are all real pros. You know all of this, certainly in
your lane, for whomever you are representing.
But I think that, you know, the best thing that we learned
today is that it was a combination of all, you know, the
stakeholders, patients, the organizations that negotiated with
FDA so that we can move this legislation forward.
And also, we heard many things that were raised of what we
have learned during the pandemic, and what we need to be really
cognizant of as we move forward. That is very important, that
we are wise enough to examine our shortcomings so that we--
another day, another time it won't be experienced again.
So I have a request, unanimous consent, to enter the
following document--we only have one--into the record. It is a
letter from public interest and healthcare organizations.
Mr. Guthrie. No objection.
Ms. Eshoo. OK, so without objection, so ordered.
Ms. Eshoo. And I think--is there anything else that we need
to include at the end of the hearing?
Members do have ten business days to submit additional
questions for the record.
So to the witnesses, please respond promptly if you receive
questions from members.
And at this time, the subcommittee is adjourned.
[Material submitted for inclusion in the record follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
[all]