[House Hearing, 115 Congress]
[From the U.S. Government Publishing Office]
EXAMINING FDA'S PRESCRIPTION DRUG USER FEE PROGRAM
=======================================================================
HEARING
BEFORE THE
SUBCOMMITTEE ON HEALTH
OF THE
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED FIFTEENTH CONGRESS
FIRST SESSION
__________
MARCH 22, 2017
__________
Serial No. 115-18
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Printed for the use of the Committee on Energy and Commerce
energycommerce.house.gov
______
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COMMITTEE ON ENERGY AND COMMERCE
GREG WALDEN, Oregon
Chairman
JOE BARTON, Texas FRANK PALLONE, Jr., New Jersey
Vice Chairman Ranking Member
FRED UPTON, Michigan BOBBY L. RUSH, Illinois
JOHN SHIMKUS, Illinois ANNA G. ESHOO, California
TIM MURPHY, Pennsylvania ELIOT L. ENGEL, New York
MICHAEL C. BURGESS, Texas GENE GREEN, Texas
MARSHA BLACKBURN, Tennessee DIANA DeGETTE, Colorado
STEVE SCALISE, Louisiana MICHAEL F. DOYLE, Pennsylvania
ROBERT E. LATTA, Ohio JANICE D. SCHAKOWSKY, Illinois
CATHY McMORRIS RODGERS, Washington G.K. BUTTERFIELD, North Carolina
GREGG HARPER, Mississippi DORIS O. MATSUI, California
LEONARD LANCE, New Jersey KATHY CASTOR, Florida
BRETT GUTHRIE, Kentucky JOHN P. SARBANES, Maryland
PETE OLSON, Texas JERRY McNERNEY, California
DAVID B. McKINLEY, West Virginia PETER WELCH, Vermont
ADAM KINZINGER, Illinois BEN RAY LUJAN, New Mexico
H. MORGAN GRIFFITH, Virginia PAUL TONKO, New York
GUS M. BILIRAKIS, Florida YVETTE D. CLARKE, New York
BILL JOHNSON, Ohio DAVID LOEBSACK, Iowa
BILLY LONG, Missouri KURT SCHRADER, Oregon
LARRY BUCSHON, Indiana JOSEPH P. KENNEDY, III,
BILL FLORES, Texas Massachusetts
SUSAN W. BROOKS, Indiana TONY CARDENAS, California
MARKWAYNE MULLIN, Oklahoma RAUL RUIZ, California
RICHARD HUDSON, North Carolina SCOTT H. PETERS, California
CHRIS COLLINS, New York DEBBIE DINGELL, Michigan
KEVIN CRAMER, North Dakota
TIM WALBERG, Michigan
MIMI WALTERS, California
RYAN A. COSTELLO, Pennsylvania
EARL L. ``BUDDY'' CARTER, Georgia
Subcommittee on Health
MICHAEL C. BURGESS, Texas
Chairman
BRETT GUTHRIE, Kentucky GENE GREEN, Texas
Vice Chairman Ranking Member
JOE BARTON, Texas ELIOT L. ENGEL, New York
FRED UPTON, Michigan JANICE D. SCHAKOWSKY, Illinois
JOHN SHIMKUS, Illinois G.K. BUTTERFIELD, North Carolina
TIM MURPHY, Pennsylvania DORIS O. MATSUI, California
MARSHA BLACKBURN, Tennessee KATHY CASTOR, Florida
CATHY McMORRIS RODGERS, Washington JOHN P. SARBANES, Maryland
LEONARD LANCE, New Jersey BEN RAY LUJAN, New Mexico
H. MORGAN GRIFFITH, Virginia KURT SCHRADER, Oregon
GUS M. BILIRAKIS, Florida JOSEPH P. KENNEDY, III,
BILLY LONG, Missouri Massachusetts
LARRY BUCSHON, Indiana TONY CARDENAS, California
SUSAN W. BROOKS, Indiana ANNA G. ESHOO, California
MARKWAYNE MULLIN, Oklahoma DIANA DeGETTE, Colorado
RICHARD HUDSON, North Carolina FRANK PALLONE, Jr., New Jersey (ex
CHRIS COLLINS, New York officio)
EARL L. ``BUDDY'' CARTER, Georgia
GREG WALDEN, Oregon (ex officio)
C O N T E N T S
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Page
Hon. Michael C. Burgess, a Representative in Congress from the
State of Texas, opening statement.............................. 1
Prepared statement........................................... 3
Hon. Gene Green, a Representative in Congress from the State of
Texas, opening statement....................................... 4
Hon. Frank Pallone, Jr., a Representative in Congress from the
State of New Jersey, prepared statement........................ 100
Witnesses
Janet Woodcock, M.D., Center for Drug Evaluation and Research,
Food and Drug Administration................................... 5
Prepared statement........................................... 8
Jeff Allen, PH.D., President and CEO, Friends of Cancer Research. 54
Prepared statement........................................... 56
Kay Holcombe, Senior Vice President of Science Policy,
Biotechnology Industry Organization............................ 71
Prepared statement........................................... 73
Anne Pritchett, PH.D., Vice President of Policy and Research,
Pharmaceutical Research and Manufacturers of America........... 84
Prepared statement........................................... 86
Submitted Material
Statement of Rare Disease Legislative Advocates, submitted by Mr.
Guthrie........................................................ 101
Statement of the Epilepsy Foundation, submitted by Mr. Guthrie... 102
Statement of NVCA, submitted by Mr. Guthrie...................... 104
Joint statement of the National Organization for Rare Disorders
and Friends of Cancer Research, submitted by Mr. Guthrie....... 106
EXAMINING FDA'S PRESCRIPTION DRUG USER FEE PROGRAM
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WEDNESDAY, MARCH 22, 2017
House of Representatives,
Subcommittee on Health,
Committee on Energy and Commerce,
Washington, DC.
The subcommittee met, pursuant to call, at 10:15 a.m., in
room 2322 Rayburn House Office Building, Hon. Michael Burgess
(chairman of the subcommittee) presiding.
Present: Representatives Burgess, Guthrie, Upton,
Blackburn, Griffith, Bilirakis, Long, Bucshon, Brooks, Hudson,
Carter, Green, Engel, Butterfield, Matsui, Sarbanes, Schrader,
Kennedy, Cardenas, and Eshoo.
Staff present: Adam Fromm, Director of Outreach and
Coalitions; Jay Gulshen, Legislative Clerk, Health; Carly
McWilliams, Professional Staff Member, Health; Alex Miller,
Video Production Aide and Press Assistant; Jennifer Sherman,
Press Secretary; Danielle Steele, Policy Coordinator, Health;
and John Stone, Senior Counsel, Health; Jeff Carroll, Minority
Staff Director; Samantha Satchell, Minority Policy Analyst;
Kimberlee Trzeciak, Minority Health Policy Advisor; and C. J.
Young, Minority Press Secretary.
OPENING STATEMENT OF HON. MICHAEL C. BURGESS, A REPRESENTATIVE
IN CONGRESS FROM THE STATE OF TEXAS
Mr. Burgess. I ask everyone to take their seats. The
subcommittee will come to order, and I will recognize myself
for an opening statement for 5 minutes.
Today's hearing marks the Health Subcommittee's second
opportunity to consider the reauthorization of several key FDA
user fee programs. The Prescription Drug User Fee Act
authorized the Food and Drug Administration to collect user
fees from industry to support the approval of new drugs and
biologics, and is a top priority for this committee.
This was first authorized in 1992, and while there is
always room for improvement, the Prescription Drug User Fee
Agreement has been a success bringing safe and effective new
products to patients in a timelier manner. Every 5 years since,
pursuant to a process set forth in statute, Congress has
reauthorized the program after reviewing the recommendations
from the Food and Drug Administration, industry, patient
groups, and other stakeholders.
The committee has been reviewing the Prescription Drug User
Fee Agreement since December when it was transmitted to
Congress and publicly posted. As I stated in our hearing on the
generic and biosimilar programs earlier this month, Chairman
Walden and I are committed to shepherding the user fee
legislation through committee following regular order and
getting it to the House floor with ample time to spare.
Reauthorization of the user fee agreements every 5 years
provides an opportunity, an opportunity to examine, an
opportunity to improve upon the state of discovery,
development, and delivery of medical therapies in America. For
instance, in 2012, the reauthorization of the user fees in the
Food and Drug Administration's Safety and Innovation Act
established the Breakthrough Therapy Designation. This program
expedites the review and approval of promising new drugs that
show early evidence of efficacy in serious, life-threatening
diseases with an unmet clinical need.
Under this program, over 165 products have been granted
breakthrough designation which means more treatments, which
means more cures, are being prioritized for patients suffering
from some of the most debilitating conditions. I am pleased
that the user fee agreements considering now will continue to
build upon the success of the Breakthrough Therapy program.
A unique factor in the negotiations of these user fee
agreements was its overlap with the development of the 21st
Century Cures Act, a bill enacted in December of last year
after a multi-Congress effort led by Representative Fred Upton
and Representative Diane DeGette. Over the course of the 113th
and 114th Congresses, members of this subcommittee worked to
uncover opportunities to strengthen and opportunities to
streamline the process by which cures are discovered and then
made available to patients. The resulting law touches each step
of the process through which new treatments come to the
bedside.
I am encouraged to see in our witness's testimony that the
Prescription Drug User Fee Agreement VI will dedicate resources
to complement the implementation of the many priorities, the
many priorities of the 21st Century Cures bill. In particular,
I like the fact that the FDA will formalize a structure to
incorporate patient input and patient experience into the
benefit-risk assessment of products that are actually under
development. This is a good thing. Patients have the most at
stake and they deserve to be heard.
I am also encouraged that the Food and Drug Administration
will dedicate resources to modernize clinical trials and
evidence development including the utilization of real-world
evidence in investment in biomarkers. Real-world evidence has
the potential to increase sufficiency and foster robust data
collection and analysis. Advancing development of biomarkers
has significant promise to accelerate regulatory decision
making and expedite the pace of clinical trials without
sacrificing standards for efficacy and safety.
Other provisions incorporated into the proposal for PDUFA
VI--OK, you made me say it--PDUFA VI. I was trying to just call
it the user fee agreements--reflects the top priorities of this
committee in the 21st Century Cures Act. Again I want to
reiterate my commitment to ensuring that this reauthorization
stays on track. We all know there are a lot of competing
influences this year, but this year will mark the fifth renewal
by Congress, and it is widely agreed that the prescription drug
user fee agreements will provide for the timely review of new
drug and new biologic license applications. Again I want to
underscore that is a good thing.
I thank all of our witnesses for being here, particularly
Dr. Woodcock. Thank you, and welcome again back to our humble
little subcommittee for one more hearing. I look forward to
hearing from each of you and more about the agreement that is
before us today, and I will yield back the balance of my time.
The chair now recognizes the gentleman from Texas, the
ranking member of the subcommittee, Gene Green, 5 minutes for
an opening statement, please.
[The statement of Mr. Burgess follows:]
Prepared statement of Hon. Michael C. Burgess
The Subcommittee will come to order.
The Chair will recognize himself for an opening statement.
Today's hearing marks the Health Subcommittee's second
opportunity to consider the reauthorization of several key FDA
user fee programs. The Prescription Drug User Fee Act (PDUFA)
authorized FDA to collect user fees from industry to support
the approval of new drugs and biologics, and is a top priority
for this Committee. It was first authorized in 1992 and, while
there is always room for improvement, PDUFA has been a
remarkable success, bringing safe and effective new drug
products to patients in a more timely manner. Every 5 years
since, pursuant to a process set forth in statute, Congress has
reauthorized the program after reviewing recommendations from
FDA, industry, patient groups, and other stakeholders.
The Committee has been reviewing the PDUFA VI agreement
since December, when it was transmitted to Congress and
publicly posted. As I stated at our hearing on the generic and
biosimilar user fee programs earlier this month, Chairman
Walden and I are committed to shepherding the user fee
legislation through Committee, following regular order, and
getting it to the House floor with ample time to spare.
Reauthorization of the user fee agreements every 5 years
provides an opportunity to examine and improve upon the state
of discovery, development, and delivery of medical therapies in
America. For instance, the 2012 reauthorization of PDUFA in the
Food and Drug Administration Safety and Innovation Act,
commonly known as FDASIA, established the Breakthrough Therapy
Designation. This program expedites the review and approval of
promising new drugs that show early evidence of efficacy in
serious, life-threatening diseases with unmet clinical need.
Under this program over 165 products have been granted
breakthrough designation, which means more treatments and cures
are being prioritized for patients suffering from some of the
most despairing conditions. I am pleased that PDUFA VI will
continue to build upon the success of the breakthrough therapy
program.
A unique factor in the negotiation of PDUFA VI, was its
overlap with development of the 21st Century Cures Act, a bill
enacted last year after a multi-year initiative led by
Representative Upton and Representative DeGette. Over the
course of the 113th and 114th Congresses, members of this
subcommittee worked to uncover opportunities to strengthen and
streamline the process by which cures are discovered and made
available to patients. The resulting law touches each step of
the process through which new treatments and cures come to
market. I am encouraged to see in our witnesses' testimonies
that PDUFA VI will dedicate resources to complement the
implementation of many of the priorities in 21st Century Cures.
In particular, I am pleased to see that FDA will formalize
a structure to incorporate patient input and experience into
the benefit-risk assessment of products in development.
Patients have the most at stake, and they deserve to be heard.
I am also encouraged to see that FDA will dedicate resources to
modernize clinical trials and evidence development, including
the utilization of real-world evidence and investment in
biomarkers. Real-world evidence has the potential to increase
efficiency and foster robust data collection and analysis.
Advancing development of biomarkers has incredible promise to
accelerate regulatory decision-making and to expedite the pace
of clinical trials without sacrificing standards for efficacy
and safety.
Numerous other provisions incorporated in the proposal for
PDUFA VI reflects the top priorities of this Committee in the
21st Century Cures Act, and I want to reiterate my commitment
to ensuring this reauthorization stays on track. This year will
mark the fifth renewal by Congress, and it is widely agreed
that PDUFA VI will provide for the timely review of new drug
and biologic license applications. I thank our witnesses for
being here today and I look forward to hearing more from each
of you about the agreement before us today.
OPENING STATEMENT OF HON. GENE GREEN, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF TEXAS
Mr. Green. Thank you, Mr. Chairman. I thank all our
witnesses, both Dr. Woodcock, welcome back again, and our
second panel for being here this morning. Today we are
examining the sixth Prescription Drug User Fee Agreement, PDUFA
VI. I think it is fair to say that we all support a strong FDA
that is responsive to the needs of the patient community and
the innovations of scientific research and healthcare delivery.
I am pleased that Congress is moving judicially through the
process of reauthorizing the user fee programs and honoring
their negotiations that have led to the agreements, and PDUFA
is the most mature of the user fee programs having first been
enacted in 1992. Sometimes our committee seems like we are a
little mature.
The law lays out a detailed process for reauthorization
that requires FDA to negotiate with industry to develop
recommendations and that the agency solicit public input and
hold public hearings and consult with Congress and patients and
consumer advocates and other relevant parties. The
recommendations that are a result of this process must also be
available publicly for a period for public comment, and
ultimately are required by statute to be transmitted to
Congress.
I was disappointed to see the line in the administration's
testimony that they do not stand behind these agreements and
hinted towards reopening the painstakingly negotiated products.
As we know, we are here today as the result of months of work
between FDA and stakeholders to examine the program, figure out
what is working and what can work better, and come to an
agreement on how the program should be for the next 5 years
through a public, drawn-out process.
This process is a long one and the statutory deadline for
reauthorization is coming up quickly. Congress has never
flirted with neglecting its obligation to reauthorize in a
timely and responsible manner. I sincerely hope that this holds
true for the sixth reauthorization of PDUFA. Along with the
other user fee programs, it must be reauthorized so FDA can do
its work and patients maintain access to new therapies without
a major disruption in the medical product ecosystem.
PDUFA was first enacted as a way to reduce the time it took
FDA to review new drugs and biologics and improve access to
medical treatments more quickly. Over the years, the user fees
provided under PDUFA have allowed the FDA to hire additional
staff and improve the efficiency and predictability of the
review process.
Prior to the first PDUFA, the median time for FDA for
approval of standard applications was 28 months. Today, the
median time for approval for standard applications has been
reduced to 12 months, and first-cycle approval rates are at 95
percent. The U.S. remains the gold standard for drug approval
and evaluation of safety and efficiency.
The commitment letter for PDUFA VI includes a number of
performance goals meant to help the agency with recruiting and
retaining the scientific and professional staff needed to keep
pace with the science. For the first time, PDUFA VI also
includes specified agency hiring goals. This builds on the
hiring provisions in the 21st Century Cures that will help the
agency to compete with the private sector in terms of
competitive salary, and gives the agency the authority to hire
scientific and technical staff needed to support medical
project review.
There have been some that have criticized FDA for being a
barrier to the access to innovative new drugs. This is
inaccurate. Contrary to the description by the President and
others who want to roll back patient safety measures, the FDA's
approval process is not slow and burdensome. Today, more than
two-thirds of novel drugs are approved first by the FDA rather
than anywhere else in the world.
It is clear that PDUFA has been successful in meeting the
goal of improving efficiency of the drug review process at FDA
and ensuring patients have access to novel therapies. The
policies and goals included in the agreements reflect what
these stakeholders value and will help ensure advancements and
improvements within the FDA and ultimately health care more
broadly.
I want to thank the agency and the stakeholders for their
leadership on this agreement that will continue the trajectory
of patient-centered innovation at the FDA. 21st Century Cures
did a great job to advance such reforms and help get new cures
from the lab table to the bedside. I look forward to hearing
from the FDA and other witnesses on how this agreement will
build on these successes and continue to advance the modern,
efficient FDA and a healthy pipeline of medical breakthroughs.
And I yield back my time.
Mr. Guthrie [presiding]. The gentleman yields back his
time.
Mr. Green. Do we have any other opening statements? No, OK.
Mr. Guthrie. We have none on our side. OK, we will turn to
the witnesses. We want to thank all of our witnesses for being
here today and taking the time to testify before the
subcommittee. And each witness will have an opportunity to give
an opening statement followed by a round of questions from
members.
And we have two panels of witnesses today, and we will
begin with our first witness, Dr. Janet Woodcock, Center for
Drug Evaluation and Research, Food and Drug Administration. We
appreciate you being here. And, Dr. Woodcock, you are now
recognized for 5 minutes to give an opening statement.
STATEMENT OF JANET WOODCOCK, M.D., CENTER FOR DRUG EVALUATION
AND RESEARCH, FOOD AND DRUG ADMINISTRATION
Dr. Woodcock. Thank you, and thanks to the members of the
subcommittee for inviting me to testify at this important
hearing. We are talking here about a program that has been
going on for 25 years, the prescription drug user fee program.
And as result, as we have already heard, over that time U.S.
patients have gone from being one of the last in the world to
obtain access to new drugs to in most cases being the first
patients in the world who can get access to innovative new
therapies, all at the same time maintaining the standards that
FDA has for safety and effectiveness of these therapies.
At the same time, we have moved from multiple cycle
scenario to predominantly first-cycle approval for these drugs,
meaning that the industry and FDA have enough communication,
the standards are clear enough, they are able to submit a
complete application that can be reviewed and approved without
further delay. And this is a great time efficiency and resource
efficiency for industry for the FDA and for the medical
community alike.
Also, this program has allowed us to accommodate the
advances in medical science that have occurred recently over
the last several decades. Congress and the U.S. investment in
NIH and in biomedical research has caused tremendous growth in
scientific understanding. Now we are really contemplating, we
have approved drugs for example that are antisense
oligonucleotides that act directly on people's DNA. We are
looking at multiple applications for gene therapies although
none have been approved yet. We are looking at multiple
cellular therapies that are under development.
And so this promise that you have been hearing about
science is really coming about and we have approved cures for
various conditions such as hepatitis C, which has long been a
scourge of people.
So the next programmatic proposals, the enhancements for
the sixth iteration of this, try to build on the
accomplishments that we already have. And as has already been
said, the first one is really aligned with the Cures
legislation that was passed and that is enhancing the ability
to capture patient voice in drug development. Not just on
benefit-risks, but patients want to tell us what we should
study, what matters to them. What do they want ameliorated
about their disease? What is most important? How should we
study it?
They want to know, they want to tell us how trials should
be designed that work for patients. People always wonder why
there is so many dropouts in the trials, missing data. Well,
because we designed the trials in a way that patients couldn't
participate. So the patient voice is critical, and then at the
end of the day how much risk are people willing to trade off in
uncertainty for the benefits, the potential benefits of any
given therapy. And this will require a rigorous process to
generate and develop all these data and bring the patients in,
in a rigorous way. It is envisioned in Cures and laid out in
Cures, and the programmatic enhancements of PDUFA VI would
bolster our ability to do that in a timely manner.
Also, there is support for the Breakthrough Therapy
Program. Now what I will say about that is that is probably the
first program that has really shortened drug development. As we
have all said, drug review isn't the problem. It occurs now in
a timely manner, predictable manner, based on PDUFA. But drug
development is still a very gnarly problem. It takes too long
and it costs too much, right, and there are many failures.
Breakthrough has been the first program as actually drug
development time has been shortened, and you have heard that
before this committee from a number of witnesses, taking
several years off of drug development in the overall time it
takes to get those drugs. Part of it is the quality of the
compounds, the molecules that are developed under and given
breakthrough, but also part of it is the support that FDA is
willing to give. And so the new program would give additional
resources.
There is also, as was envisioned in Cures, support for
biomarker qualification also for the better use of surrogate
endpoints, an advancement of clinical trial design, something
dear to my heart and I would be happy to talk to you about;
advances in the use of real world evidence, which is also a
Cures theme; better communication with industry to make sure
that we are always on the same page and we move things along;
and then administrative improvements including oversight of
some of the administrative processes, reports, and financial
oversight, to make sure the management and planning of the
program is as good as it can be.
So I believe this captures in the programmatic proposals
many of the modern themes that need to now address improvements
in drug development and drug approval, and I would be happy to
answer any questions.
[The prepared statement of Dr. Woodcock follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Mr. Guthrie. Thank you. I want to thank you for your
testimony. We will now move to the first Q&A portion of the
hearing, and I will begin the questioning and recognize myself
for 5 minutes.
So Dr. Woodcock, as part of 21st Century Cures, this
committee included provisions that set up FDA Intercenter
Institutes of Excellence in major disease areas to improve
coordination across the agency. FDA has since established the
Oncology Center of Excellence. Can you provide us with an
update on how things are going so far and if there is anything
we can do to help ensure smooth and timely implementation?
Dr. Woodcock. Yes. The Oncology Center of Excellence is
considered a joint venture by the three medical products
centers, Center for Biologics, Center for Devices and
Radiological Health, and Center for Drugs. And so we put this
together jointly, it resides up in the office right above us.
Dr. Richard Pazdur is the director of that office.
And the procedures that we are running, they will review
the clinical oncology, the medical oncology portion of any
product that comes in with a medical oncology indication to any
of the three centers. So they will do the medical part of that
review, and Rick will direct it, but it will include
oncologists from Drugs and Biologics as appropriate to that
particular cancer area.
So we have worked out the procedures and so forth and we
expect those applications then will go before the Oncologic
Drugs Advisory Committee and be heard. And then the Center,
whichever Center has the product, will complete the rest of the
product review which is about the quality of the product and
the control of that quality, and then we will actually approve
the application using the clinical recommendations from the
Oncology Center of Excellence.
And the Center also will be the outfacing, outward facing
group that will interact with the medical oncology and patient
community.
Mr. Guthrie. OK, thank you. I want to yield time to my good
friend from Virginia, Mr. Griffith.
Mr. Griffith. Thank you very much. I appreciate that. Dr.
Woodcock, prior to the FDA's encouraging the development of
abuse deterrent opioids, manufacturers should be incorporating
these technologies into their products and testing whether they
deter various routes of potential abuse, intranasal,
intravenous, et cetera. If they do, manufacturers need to be
able to include this data in their product labeling and
communicate this useful information to healthcare providers.
I understand a recent exclusivity determination by FDA
calls into question whether multiple manufacturers in the same
product class could make such claims even if their data
justifies it and even if they are using different technologies.
Is that accurate?
Dr. Woodcock. It is likely accurate. I think like many of
the laws governing exclusivity that Congress passed long ago,
they certainly didn't foresee some of the situations. And we
struggle all the time with trying to figure out how to apply
exclusivity fairly and justly to everyone and yet not
disadvantage public health goals that we may have.
Mr. Griffith. And so you would agree it probably would
discourage a manufacturer if they can't go forward and discuss
that; a company or a manufacturer might not invest as much if
they think that somebody has beaten them to the punch by a few
months. And so what you are recommending, if I understood your
previous answer is, is that we probably should take a look at
it and change the law?
Dr. Woodcock. Well, I can't go that far because of course
that is your purview.
Mr. Griffith. Yes, ma'am. Thank you.
Dr. Woodcock. But I do believe that times have changed.
Mr. Griffith. Yes, ma'am. I appreciate that. Speaking of
product manufacturers being able to share useful scientific
data and information about their products with doctors, I
understand that some previous leaders at the Department of
Health and Human Services would not allow FDA to work with
Congress to clarify in a responsible and constitutionally sound
manner how manufacturers can communicate truthful and non-
misleading off-label information.
Now as you and I were just discussing, I prefer that
Congress work with you all to make the rules as opposed to
leaving it to the courts to decipher. Will you commit to
working with us to set up some clear rules of the road so folks
know what they can communicate and what they can't?
Dr. Woodcock. Certainly. We will work with the
administration, through the administration with you on this
issue.
Mr. Griffith. All right, I appreciate that very much.
With that Mr. Chairman, I appreciate the time and I yield
back.
Mr. Guthrie. I yield back my time and I recognize Mr. Green
for 5 minutes to ask questions.
Mr. Green. Thank you, Mr. Chairman. And thank you, Dr.
Woodcock, again for being here this morning. As I mentioned in
my opening statement, I was disappointed to see the line in the
administration's testimony that they do not stand behind these
agreements and hinted toward a reopening of painstakingly
negotiated products. Can you explain to the committee what
would happen should Congress fail to reauthorize PDUFA and the
other user fees before the statutory deadline in September?
Dr. Woodcock. If there is not a reauthorization, we must
initiate our reduction in force process where we would prepare
to let go of for the Center for Drugs is maybe 70 percent of
the staff working on the process of review in new human drugs.
And we do have carryover balance within the user fee agreements
that we are supposed to hold some money back. In case the
program terminates we can, over several months, have an orderly
process to let go of the staff. And that we would have to start
thinking about that in July because there are complicated
personnel rules that have to do with who has to be notified
first and so forth.
Mr. Green. OK, thank you. There seems to be a
misunderstanding about the drug development process. We hear
often that new therapies take about 10 years to develop and
some seem to think that means the application languishes at the
agency for a decade. In fact, the FDA review is the final step
in the development process and more efficient than ever.
Can you explain to this committee how PDUFA VI builds on
the past successes of the program and helps the agency work
with stakeholders to not be a bottleneck but a strong partner
in getting these new treatments to patients in need?
Dr. Woodcock. Certainly. Obviously we have prioritization
programs for breakthrough drugs and for priority drugs where
they are reviewed in shorter times. We have gotten some reviews
out in 3 months, and a drug approved and on the market after
the application is submitted where it is a breakthrough.
So, but that doesn't mean that the development time is
short. The development time still is quite long and the failure
rate of drugs in development is still very high. Perhaps in
some areas nine out of ten drugs that get into human testing
fail during human testing at huge cost. So a lot of the efforts
we are working on, I believe patient focused drug development,
the innovative clinical trials, the surrogate endpoints, and
biomarkers, all of which are encompassed in the proposed
programmatic changes, will help with this drug development
phase and making it as short as possible.
As I said, the Breakthrough Therapy Program has actually
worked and some of those development programs in the clinic
have only been a couple years, so the time to patients has been
shortened dramatically.
Mr. Green. Can you explain to this committee how PDUFA VI
builds on the successes of the program in the past and helps
the agency work with stakeholders to not be a bottleneck but a
strong partner in getting new treatments to the patients in
need? Or that is my question. OK, let me get to the next one.
Many provisions of the user fee agreements resemble ideas
we advanced in Cures, things like biomarker qualification
programs, incorporation of the patient perspective in decision
making, and the advancement of innovative clinical trials are
goals we have shared. I am concerned about the impact that the
administration's proposed hiring freeze would have on FDA, and
could it be made hard to advance these shared goals?
User fees under PDUFA also assist the FDA in hiring and
retaining staff necessary to support the activities associated
with review of drug applications. The commitment letter for
PDUFA VI includes a number of performance goals meant to help
the agency with recruiting and retaining the scientific
professional staff needed to keep pace with science. In fact,
for the first time, PDUFA VI also includes specific agency
hiring goals. This builds off the hiring provisions in the 21st
Century Cures.
Can you discuss further how the PDUFA VI will help the FDA
to hire and train the scientific technical workforce needed to
fulfill the goals agreed to in the commitment letter?
Dr. Woodcock. Well, for really the first time, this
programmatic proposal in PDUFA VI really focuses on some of the
administrative processes and tries to set in place some
oversight over hiring and so forth, and some new scientific
recruitment staff and so forth that would enable us to hire
scientists. As I said, the science has really come along, and
so we are talking about really high-tech kind of treatments in
humans, such as gene therapy in humans and so forth, and we
need the scientific staff that are qualified to evaluate those
and make sure they are safe as well as that they work.
Mr. Green. Well, my time is almost up. And I know our goal
is to make sure you have the resources to do it quicker and not
lay in another level of bureaucracy to make it even longer.
Thank you, Mr. Chairman.
Mr. Guthrie. Thank you. The gentleman yields back. Mr.
Upton is recognized for 5 minutes.
Mr. Upton. Well, thank you, Dr. Woodcock. It is great to
see you again, and I know all of us on both sides of the aisle
here really appreciated your work and your input from the very
beginning on getting 21st Century Cures ultimately to the
President. And we knew that by expediting the approval of drugs
and devices we were going to need require you all at the FDA to
help us and to help chart that course for us and provide the
right resources so that you would be able to do your job.
And obviously PDUFA VI is very important, very important.
And alarming of course to us, a good message to us is if we
don't get it done by summer or show that we have made progress
by July and August, certainly by September, that you would
actually have to RIF 70 percent of the staff, is quite alarming
and ought to serve as turning up the burner for us to get our
job done, as we have in the past in a very strong bipartisan
way, ultimately getting this bill to the President.
A question for you, Diana DeGette and I sent a letter a
couple weeks ago to OMB asking about the federal hiring freeze
that the President announced as it relates to the
implementation of 21st Century Cures. And of course as you know
we came up with offsets, dollar for dollar matching to help
with the half billion dollar increase that we gave for the FDA.
Yesterday, it is my understanding that you told the Senate
Health Committee--and I have to again compliment Lamar
Alexander and the great work that they did over there. But
yesterday you told the Health Committee that the White House
did give the FDA permission to move forward with hiring on the
select user fee positions needed to implement Cures. I don't
know if that is a quote or not, but that is my understanding.
Can you provide some more detail? We have not heard back.
It is my understanding we have not heard back from our letter
that we sent to the White House, but can you tell us more
details about the type of hiring that is going to be needed to
implement 21st Century Cures and what guidance you have been
able to get from both OMB and the White House and which select
user fee positions is the FDA hiring?
Dr. Woodcock. Well, I am not in a position to discuss that
particularly, I can talk about what programmatic needs there
will be. Clearly, the patient focused drug development is going
to need different kind of scientists than we have traditionally
had. We have had laboratory scientists who are looking in test
tubes and doctors who are--we are going to need social
scientists and other folks who can actually talk to people and
get rigorous evidence about what their needs and preferences
are and who can work on instruments, say, patient reported
outcome measures and so forth. So that is one category that we
don't necessarily have enough of.
In addition, on real-world evidence that is some different
types of science that we will need to have people who can
analyze that, big data, and so forth. And interesting, we have
already had multiple outside parties approach us who are using
real-world evidence in different ways and they want to
collaborate with us and we are working with them, some of them
in the Oncology Center of Excellence. So we will need data
scientists of that sort.
And Breakthrough Therapy, which I know isn't Cures, but
will need basically people who in specific disease areas
particularly rare diseases. We also commit to integrate rare
disease expertise within the review teams where there are rare
diseases. We are seeing more and more rare diseases being
treated.
So we have very focused needs in specific places for a
specific kind of scientist, and I am sure that the Biologic
Center with the rise in gene and cell therapy and that more or
less explosion and also regenerative medicine, they are going
to need specific types of scientific expertise.
Mr. Upton. Great. Well, thanks again for your work and we
look forward to continuing the process as we get this thing
done too. I yield back. Thank you, Mr. Chairman.
Mr. Guthrie. Thank you. The gentleman yields back and Mr.
Schrader is recognized for 5 minutes.
Mr. Schrader. Thank you very much, Mr. Chairman, and
thanks, Ms. Woodcock, for being here again. Very impressive
results; I don't know if it is appropriate, but the graphs that
are in your written testimony, I think, are pretty dramatic and
you should share those with us when you come in and give your
testimony. It would be pretty interesting, I think, for
everyone to see that with the work of the committee and follow-
through by FDA that the first drug approvals have dramatically
increased and it is really pretty impressive. So make sure you
show yourself to advantage when you become before us here.
I appreciated these comments on the Breakthrough Therapy
Program with regard particularly to new and innovative drugs
with unmet needs. We are finding that in the generic area that
once again there are unmet needs despite the fact there may
have been a product and there is either a sole source or no
source alternative. Any thought of how the breakthrough process
you are currently using on the brand name side might translate
into the generic sphere of development?
Dr. Woodcock. Our analysis of the issues with generics is
more that, you know, about ten percent of what we call
reference drugs, the brand drugs, never get a generic filed to
them. And so this seems to be a market phenomenon, competition
is only attracted where people think they can make money by
competing, and the small source products and so forth.
Now in the generic drug user fee program, the second one
that we are proposing, the programmatic enhancements include a
program to help with complex generics. And those are ones that
actually people might not try to enter the market because it is
hard, where, say, you are using an autoinjector or you are
using a very complex molecule and so it might be hard.
So there, and we have agreed that we would set up a pre-
program similar to kind of like the prescription drug user fee
where before they send in the application we have meetings with
them and we give them advice and we help them develop their
products, so by the time they get the application in the door
it actually could be approvable.
So that would help with those types of products, but the
small----
Mr. Schrader. I guess where I was going--and that is very
good and I think that is outstanding and hopefully a benefit to
a lot of the companies out there. But I was going, say we are
able to encourage a manufacturer to come to market through a
variety of different means. We have a bill, Gus Bilirakis and I
are trying to find what is the appropriate way to get and
incentivize folks to come to market; make it worth their while
as you put it.
But once they are there it would be nice if you used that
breakthrough approach that has been so successful to also, you
know, hasten things through. And I think to, and help us
encourage them to come to market and be successful, if they
knew that breakthrough approach was going to be applied that
might incentivize things also.
Dr. Woodcock. Well, we would be happy to work with you. In
medicine we have a saying: First do no harm. And sometimes
there are unintended consequences and I think it would be worth
discussing, because there is such a commercial hit that the
innovators take when they get a generic competition on the
market that any provisions that they can sue us about or send
us citizen petitions or obstruct a process cause delays, and I
believe that needs to be taken into account as you think about
incentivizing.
Mr. Schrader. We are trying to do that. We have a REMS
portion of our bill to try and make sure that it is being used
appropriately for safety purposes and not block competition in
the market. So we are trying to listen to you and your advice.
Dr. Woodcock. We would be happy to work with you in this.
Mr. Schrader. Second question on the biomarkers. I think
that is a great idea because it takes as you said, many times
it takes awhile to get these drugs to market and many of them
do fail. And so a lot of the manufacturers want to have some
idea if they are on the right track and you want to have some
idea if they are on the right track, so early intervention and
changing things would be appropriate.
Taking a blood pressure measurement or whatever the
appropriate biomarker is, how do you follow through on that as
the medication goes through the market, or a product goes
through the market, to make sure that number one that the
biomarker does turn out to be an accurate reflection of what
the drug's ultimate outcome is, and then, once the drug is on
the market, how do you go back and reassess the biomarkers to
make sure they are actually meaningful indicators for you and
for the companies?
Dr. Woodcock. We have a program known as Accelerated
Approval. For some biomarkers such as blood pressure, their
benefit is unequivocal and we don't need to keep proving over
and over again that lowering blood pressure keeps you from
getting strokes and so forth. We know that. But many other
biomarkers are, quote, surrogate endpoints that we aren't a
hundred percent sure that they are going to translate into
benefit, and therefore we would give an accelerated approval it
is called. That is kind of a misnomer, kind of misleading, but
what that means is we are approving us based on the biomarker,
but they have to do further studies. They are required to do
further studies after approval to show that their drug actually
causes clinical benefit.
So you get on the market earlier, that is the accelerated
part, but you still have to deliver that proof.
Mr. Guthrie. Thanks. The gentleman's time has expired. I
will recognize Mr. Lance for 5 minutes for questions.
Mr. Lance. Thank you very much, Mr. Chairman. It is always
good to see you, Doctor. I am encouraged to see that the Rare
Diseases Program staff will be integrated into review teams for
rare disease development programs to provide unique expertise.
Could you please speak to the relationship between PDUFA and
21st Century Cures as it relates to drug development tools such
as real-world evidence, complex trial designs, and biomarkers,
and the importance of getting the agreement to the President's
desk by the end of July?
Dr. Woodcock. Certainly. Well, what was negotiated in PDUFA
VI bolsters certain aspects of Cures with additional resources,
and also would have specific timelines put in place and
agreements. Some of those are slightly different, but we can
reconcile them all kind of defaulting to whatever the earliest
thing we agreed to is, we would do it then, all right.
Mr. Lance. Yes, Dr. Woodcock.
Dr. Woodcock. So, for example, real-world evidence, their
guidance and so forth we would put out. 21st Century Cures has
a broader qualification process, so it includes patient
reported outcomes, clinical outcome assessment, as well as
biomarkers, whereas the PDUFA agreement is about biomarkers.
However, we are going to put up the same process for
everything, the Cures process, which puts in place timelines
and obligations on both the submitters and the agency. So we
will put that across the board.
We expect, as you all know from our discussions, the
biomarkers to be the most difficult part of this, and so the
PDUFA gives, envisions more support for the biomarker
qualification process.
Mr. Lance. Thank you. Dr. Woodcock, 21st Century Cures
included a provision on combination products and that provision
directs the agency to improve coordination between the Device
and Drug Centers. Considering both Centers are involved in this
process, should there be some coordination between the
agreements?
Dr. Woodcock. Yes, and actually I believe there is. PDUFA
VI provides some resources actually are envisioned for the
Device Center, all right, to conduct these reviews. But I am
pleased to say under the leadership of Dr. Rachel Sherman, who
is deputy commissioner, we have made considerable progress
already in combination products. We have put together a
council, we have mapped the processes, we have improved the
processes, we have developed standardized templates and so
forth. So I think we have made a lot of progress already and
that these efforts in Cures and in the user fee agreements will
enhance that.
Mr. Lance. Do both Centers receive part of the user fee for
combination products?
Dr. Woodcock. Yes.
Mr. Lance. Is that the way it works?
Dr. Woodcock. Yes.
Mr. Lance. And as I understand it, the goal in fiscal year
2019 is 50 percent, when the goal in fiscal year 2021 is 90
percent; is that accurate?
Dr. Woodcock. I believe so. That is how we typically
structure these goals. If we haven't been keeping track the
first year or so we try to find out what our baseline is. It
may be 80 percent--we might hope so, OK--and then we ratchet it
up after that.
Mr. Lance. Thank you, Dr. Woodcock.
And Mr. Chairman, I yield back 1 minute, 25 seconds.
Mr. Guthrie. The gentleman yields back and Mr. Cardenas is
recognized for 5 minutes for questions.
Mr. Cardenas. OK. I will try to yield back a minute and 25
seconds or more to keep up with the program here. Thank you
very much, Mr. Chairman, for holding this hearing.
Dr. Woodcock, what is the significance of September 2017 as
far as your professional world goes?
Dr. Woodcock. If these various user fee programs are not
reauthorized at that time, we must initiate processes to let go
of the staff and wind down the program. There is money in all
these agreements to do that. There is some money held back.
Mr. Cardenas. Yes, money held back to wind it down----
Dr. Woodcock. That is all.
Mr. Cardenas [continuing]. Which only expends over a few
more months.
Dr. Woodcock. That is correct.
Mr. Cardenas. Are many of the people that would be let go,
per se, if we legislatively failed to do our job here, would
that--you are talking about jobs, people who are specialists,
or what kind of jobs are they?
Dr. Woodcock. Most of these are doctors and scientists.
They are almost all at the Ph.D. or M.D. level. The physicians
are generally some specialists, so we would have nephrologists
or people who are specializing in medical imaging, and so hard
to find people.
Mr. Cardenas. Is it fair to say that getting so close to
September 2017 creates kind of a little bit of nervousness
amongst people who are trying to get their work done in such an
environment?
Dr. Woodcock. Well, what we would expect is the
productivity would slow down as we approach the brink,
tremendously. This has happened once before where we approached
it and we lose staff. Our people are heavily recruited into
other jobs and they aren't paid as we have all discussed, they
aren't paid as much as private sector. And so I would expect we
would start to lose people very early who would leave before
they got their notice.
Mr. Cardenas. So to that point, if and when this, it seems
to have happened before, the ramping up, once there is a
restoration after the fact, isn't the ramping up many times
harder than it was in the ramping down?
Dr. Woodcock. It is indeed. At least in the New Drugs
Program where we need to hire physicians, the last time, and we
didn't come to a reduction in force, we just came sort of close
to that, it took more than a year for the New Drugs Program to
recover its losses, and its recruitment rate was slowed down
which it already is slow, because people have kind of lost
faith in the viability of the program.
Mr. Cardenas. And something such as a year of that
revamping to just restore back to where it was, doesn't that
cause a compounding effect potentially when it comes to the
actual work being done going forward not only within the
department but in the industry that happens to interact with
you?
Dr. Woodcock. Well, we would have to prioritize very
carefully what work would be done. Public safety would come
first, obviously, and we would probably not be able to give all
the advice that we would like to give or that people would like
to have from us.
Mr. Cardenas. So the stress is--I am interpreting this
conversation as there would be stress involved in many ways
actually expands beyond the department if in fact we weren't
able to timely, in a timely fashion get this restored.
Dr. Woodcock. I believe that is very accurate.
Mr. Cardenas. Do our job legislatively by the September '17
deadline.
Dr. Woodcock. Yes.
Mr. Cardenas. OK. So briefly, Dr. Woodcock, when it comes
to what we have done on 21st Century Cures, and your department
is complicit in making sure that we do well with that. But at
the same time, when it comes to biomarkers can you please
discuss further how PDUFA VI will help with these efforts and
what further biomarker development activities PDUFA VI will
provide resources for?
Dr. Woodcock. Certainly. Both 21st Century Cures and the
program envisioned in PDUFA VI both envision more effort going
to biomarkers. 21st Century Cures sets up a structured program
for what we call regulatory qualification, and what that means
is new biomarkers, a different sort, we would tell people, the
public, you can use this biomarker to make this decision about
patients.
Now that can be a very heavy decision say if it is a safety
biomarker. We are saying we are trusting human lives to the
results of this biomarker. So there is a lot of scientific work
that has to go in to make sure that biomarker is providing
reliable information to make that decision.
And so what we are going to do, or are instructed to do
under Cures and also under this PDUFA VI, is develop the
evidence standards, OK. How much evidence, so everybody
understands what kind of evidence you need in order to rely
upon a new biomarker, and also then evaluate new nominated
biomarkers through the Cures process that was set up against
those evidence standards. So we have to do both of these, so we
need the kind of scientists who are able to do that sort of
work.
Mr. Guthrie. OK, thank you. The gentleman's time has
expired.
Mr. Cardenas. Thank you, Chairman.
Mr. Guthrie. Thank you. I now recognize Mr. Long for 5
minutes for questions.
Mr. Long. Thank you, Mr. Chairman, and thank you, Dr.
Woodcock, for being here today. I would like to spend my time
with you discussing a very vulnerable population, one that I
personally focused on helping. Every year, nearly 200,000
newborns in the United States are admitted to neonatal
intensive care units for treatment. Due to the numerous
challenges and despite current pediatric incentives, the last
new drug for this population was approved in 1999.
Last year my colleague on this committee Ben Ray Lujan and
I introduced the Promoting Life-Saving New Therapies for
Neonates Act and are working to introduce the bill this year.
Our bill would create a new incentive model by providing a
narrowly targeted, transferable exclusivity voucher to drug
sponsors who successfully develop products for neonates.
Do you believe the current pediatric incentives have been
successful in stimulating therapy development for newborns?
Dr. Woodcock. No, not particularly, I do not.
Mr. Long. Given the lack of development, can you identify
the challenges that you see from a regulatory perspective at
FDA as well as research and development challenges for the
industry?
Dr. Woodcock. Well, I believe that we have taken steps
recently along with the American Academy of Pediatrics and
others, and our new head of pediatrics at FDA is a
neonatologist. And together with her and others we have put
together a network of NICUs, because part of the issue is the
NICUs did not standardize their treatment protocols and so
everyone had a different treatment protocol. So if we were
going to ask a developer, a drug developer, to develop a drug
in NICUs, every NICU director would want a different protocol.
So the first thing that had to be done was say what is the
standard of care in the NICU, in the neonatal intensive care
unit, and then you can say what are the biggest unmet medical
needs for neonates, and then you can start talking about, OK,
do we have a trial network or do we have some type of
infrastructure that could actually evaluate a new therapy were
it developed? And they are working on doing that
internationally which is really good news. So I would be happy
to update you on the progress on that.
Mr. Long. This is a tough population to test drugs on.
Dr. Woodcock. That is right.
Mr. Long. Are there steps you believe we could take in the
upcoming user fee process to help spur much needed development
for this vulnerable patient population?
Dr. Woodcock. I don't know in user fee process. My belief
is, and I have talked to the American Academy of Pediatrics
about this, that the heads of neonatal intensive care units
need to get their program together, decide what the standard of
care is, decide what the unmet medical needs are, develop trial
structures so they could test new drugs, and if they make--if
you build it they will come, in my opinion. If you make a
pathway clear that developers could use, then I believe they
will develop products for neonates, sick neonates. And I
believe it is needed.
Mr. Long. OK, thank you. And once again thank you for being
here today taking your time to be with us.
And Mr. Chairman, I yield back.
Mr. Guthrie. The gentleman yields back. Ms. Matsui is
recognized for 5 minutes for questions.
Ms. Matsui. Thank you, Mr. Chairman.
Thank you, Dr. Woodcock, for being here. It is wonderful to
have you here. First of all, I want to mention that I am
concerned with the President's budget proposal and how it might
impact the work that this committee is doing to reauthorize
these vital agreements that they have. And I don't think it
would be wise to renegotiate the user fee agreements that FDA
and the industry have worked so hard to reach, nor do I think
it would be wise to impose drastic cuts to the agency's budget
authority that would endanger the FDA's ability to collect
these user fees. FDA performs many critical functions to keep
our food and drugs safe and we cannot afford to compromise
that.
Now I am particularly concerned about both the development
and the final price of drugs for patients with rare diseases.
These populations are often neglected and left with little or
no treatments or cures. I want to ensure that we take advantage
of our robust research efforts in this country for these rare
disease patients.
I am pleased that there are many provisions in the
negotiated PDUFA agreement that would make important advances
for this rare disease community, particularly building on the
effort to include the patient experience in drug development
ensuring that staff at FDA who have expertise in rare disease
are integrated across different centers.
Dr. Woodcock, can you elaborate on the provisions in PDUFA
that would help patients with rare diseases?
Dr. Woodcock. Well, I believe the biggest help is actually
going to be in the patient focused drug development. And why is
that, because rare diseases often are so rare there are not any
doctors who really know what happens to the people. And so what
we are encouraging and we are seeing now is the patients are
getting together and they are having their own patient focused
drug development meetings.
They are collecting, and we have given some grants out to
help with this, they are collecting natural history on their
disease so people actually know what happens to someone with
the rare disease. Often it is very disparate. Not everyone with
that rare disease has the exact same course, so then it is even
harder to study them.
So we are encouraging them to develop natural history so we
can help with trial designs and then maybe even outcome
measures, like what is the most burdensome part of the disease?
What would they like ameliorated? So that then if a company
comes along and wants to develop they have a pathway to
develop. So that is all baked into these agreements in rigorous
ways of collecting that information and helping patient groups
so they can develop these things.
But also of course there is an agreement to integrate rare
disease staff into review teams so that there is more, it is
not all about blood pressure meds and gigantic trials and heart
disease, OK, it is about people who have the very rare diseases
pose different problems in development.
Ms. Matsui. Right. OK, can you talk more about the
Breakthrough Therapy Program and what successes had it had and
what additional resources help FDA with approval of innovative
orphan therapies?
Dr. Woodcock. Well, we were completely surprised after the
Breakthrough provisions were passed that we got so many
applications, all right, and so it has been extremely
successful in getting designations. We are only supposed to
designate drugs that preliminary data, their early data they
develop in the clinic shows it may be a game changer in the
disease. It may change the disease; it isn't proven yet.
I can get back to you with the actual numbers, but we have
designated hundreds of these to our surprise--we thought it
would only be a handful--and we have approved many. And so this
is great news for patients, because many times when we approve
these they actually are a game changer for that disease.
Ms. Matsui. OK. That is wonderful. You testified that
surrogate endpoints have been the basis for 60 percent of rare
disease approvals. Can you explain surrogate endpoints in
laymen's terms and why they are important for rare disease
approvals?
Dr. Woodcock. Surrogate endpoints are something other than
how a patient feels or functions or how long they live. So that
is our gold standard for approval, it makes you feel better or
makes you function better or it makes you live longer. But
often diseases take a really long time, OK, to have their
manifestations. And say for diseases where you are missing an
enzyme--that is many rare diseases. So you are missing an
enzyme and you start accumulating that substance inside your
body instead of eliminating it.
Ms. Matsui. Right.
Dr. Woodcock. And we can give back these enzymes now, so
sometimes we have accepted the fact that in vital organs that
material goes away, all right. Well, that has to be really good
news. It is not a hundred percent sure that doing that will
reverse the symptoms of the disease, but it is pretty
plausible, right? So often we give an accelerated approval like
we were talking about saying, OK, we will get it on the market.
All the patients can start taking this because is it removing
this stuff from the body, but we want you to show with a
registry or other that actually they are feeling better
eventually, to make sure that is the truth.
Ms. Matsui. OK. Thank you very much, and I have gone past
my time and I yield back.
Mr. Guthrie. Thank you. The time has expired, and we
recognize Mr. Bilirakis for 5 minutes for questions.
Mr. Bilirakis. Thank you so much. I appreciate it, Mr.
Chairman. And thank you, Dr. Woodcock, for coming today again.
Recently the FDA issued a request for comment on a proposed
Office of Patient Affairs. Can you tell us what the goal of
this office is, how it fits into the agency with its current
patient related programs, and how this office would benefit
patients?
Dr. Woodcock. The thought is that many patients don't
understand the structure of FDA. FDA has long been divided into
Centers, and if you are kind of inside Washington, you know you
call the Biologic Center and you call the Drug Center. But what
do you do if you don't know even who to call, right. So the
thought is for medical products, not for foods or whatever, but
for medical products, if people have questions about medical
products there ought to be a little bit of a front-facing,
patient-facing unit that can help people figure out who to ask
the question. And so that is, I think, a lot of the rationale
behind it.
Mr. Bilirakis. All right, thank you. Dr. Woodcock, in the
21st Century Cures Act we were able to pass reform language to
modernize the Office of Combination Products. As you know,
combination products are products on the market that have
elements of a medical device and a drug, like inhalers and
insulin injectors. Many patients need and rely on combination
products, as you know.
While we worked on the 21st Century Cures, I asked FDA
about innovation in the drug and device space as more and more
innovative products may be combination products. At the time
there were complaints from innovators about the slow and
burdensome FDA process for approving combination products. One
of your colleagues at FDA stated in an hearing, ``That is a
place that does require probably further discussion and whether
or not there are changes to be thought about to make that
intersection work better than it currently does.''
Can you update us on what the FDA is doing on the drug side
to implement the Cures language for combination products and
what was agreed to in the user fee agreement?
Dr. Woodcock. Certainly. Well, what we are doing, the Cures
product calls for work on this and the user fee program, the
drug user fee program, actually provides resources for review
of the device portion, OK, so that has been agreed to. But in
advance of that we have set up a combination product council at
the agency. We have mapped the different processes. We have
revised them to make them more efficient. We are tracking them.
We have standard forms and so forth, and I think everyone
agrees that that is all going much better now.
So even in advance of implementing these we have gotten
sort of the basics down about how to do these reviews more
effectively given that we agree with you, this is the future of
products. But the PDUFA program proposes that more resources be
given to the Device Center to conduct these reviews of drug
related, drug-led combination products of which many of these
are.
Mr. Bilirakis. OK, very good. I yield back, Mr. Chairman.
Thank you.
Mr. Guthrie. The gentleman yields back, and Ms. Eshoo is
recognized for 5 minutes for questions.
Ms. Eshoo. Thank you, Mr. Chairman. Welcome, Dr. Woodcock.
Dr. Woodcock. Thank you.
Ms. Eshoo. It is always good to see you. Mr. Long is not
here, but I wanted to say for the record that in FDASIA when we
built that and passed it, I had language in that that required
neonatologists being hired, and that was back in 2012. So I
will talk to him later. I will be happy to work with him, but I
think that that is important to set down for the record.
The PDUFA was enacted in 1992. I was running for Congress
when that was put into place. And at that time drug review
times were lagging and the FDA simply really couldn't keep up
with the flood of new drug applications. So through these user
fees paid by applicants it has given the FDA the resources it
needed to hire and support more staff very specifically to move
the applications forward.
I think the program overall has been successful at reducing
review time backlogs, and even though the President criticized
the FDA during his joint address to the Congress for, ``having
a slow and burdensome approval process,'' I think the facts
really dispute that claim. And we are always looking to improve
it, but it has been instrumental in promoting the improvements
we have seen over the past 25 years.
Now I want to talk about two bills that I authored. I am
very proud of them. One the BPCA, the Best Pharmaceuticals for
Children Act; and the other, PREA, the Pediatric Research
Equity Act. Both of these programs were permanently
reauthorized in 2012, but I think today we need some
improvements. We know that children are not just small adults;
that drugs work differently in them than in adults and they
have to be studied specifically for their use. That is why I
authored both of these pieces of legislation. I think they have
a track record of success, because more than 664 drug labels
have been revised with important pediatric information as a
result of the two bills.
So my question to you, Dr. Woodcock, is what is the
implication of the orphan drug exemption in PREA on children's
health? Are there examples of orphan drugs that would have
benefited from a pediatric study but were not studied as a
result of the orphan drug exemption in PREA?
Dr. Woodcock. I have to get back to you on specifics, are
there any. In general, the orphan diseases, the rare diseases
are sort of throughout life. Many of them start in childhood
and so children are usually studied.
So much of the pediatric drug development problems were the
fact that drugs were studied for adult diseases and there would
be a few children who had them, relatively speaking, and they
weren't ever studied, right, and it was used off-label in them,
but in many of the rare diseases that rare disease starts in
childhood and continues through.
But there may be some instances, and we can get back to you
about where the rare disease predominates in adults. There are
only a few children, and perhaps then the exemption means that
those children may not be studied, but in talking to the rare
disease and the orphan staff and the pediatric staff they don't
believe this is a large problem.
Ms. Eshoo. Well, orphan drugs are, as you know they are
currently exempt from PREA's----
Dr. Woodcock. Yes.
Ms. Eshoo [continuing]. Pediatric study requirements and
that is why I am asking about it. Before the BPCA and PREA, the
vast majority of drugs, more than 80 percent used in children
were used off-label and without data on their safety or
efficacy, and today that number has been reduced to
approximately 50 percent, but that is still a lot. That is
still a lot.
Why, if FDA has the authority to issue civil monetary
penalties for other violations of the Food, Drug, and Cosmetic
Act, including violations of post-marketing requirements, do
you think that the FDA should be prohibited from using that
authority to ensure compliance with PREA post-marketing
requirements?
Dr. Woodcock. That is a legal question and we would be
happy to work with you and get back to you on that.
Ms. Eshoo. But do you have any thoughts on it? You deal
with legal all the time.
Dr. Woodcock. I do.
Ms. Eshoo. You live within a legal framework.
Dr. Woodcock. That is correct. The civil money penalties
provisions and those provisions are apparently rather difficult
to operate and implement, but so I would prefer getting back to
you with the agencies.
Ms. Eshoo. Sure. That is fine. Thank you very much.
Thank you, Mr. Chairman.
Mr. Guthrie. Thank you. The lady yields back, and I now
recognize Mrs. Blackburn for 5 minutes for asking questions.
Mrs. Blackburn. Thank you, Mr. Chairman.
Dr. Woodcock, you were so patient to come to us regularly,
and we do appreciate it because we are so interested in making
certain that things that are supposed to be done are tended to.
As Chairman Upton mentioned, 21st Century Cures, the
implementation there, of course the Children Count Act which I
had had that component, that is something that we are going to
watch very closely and so we do appreciate the updates. We know
it takes a lot of time to come up here, but we are very
appreciative. I just want to quickly look at the abuse
deterrent opioids and the components that are there. March
2016, you did the draft guidance. When is there going to be the
final guidance on that? What is the expectation?
And then I want to know, I know we have touched around the
edges on this, but talk a little bit about the actions that can
and should be taken from you all to advance the abuse deterrent
opioids and to get these into the marketplace, just a little
bit there. And that is really my only two questions.
Dr. Woodcock. Certainly. Well, as far as the guidance, it
is very difficult ever to give a firm date certain when a final
is going to come out.
Mrs. Blackburn. Just an expectation or timeline.
Dr. Woodcock. Well, let me just assure you that we are
putting great effort into this, because really what we need to
do, we think, is incentivize innovator development of various
abuse deterrent formulations. The current ones, as we have
already discussed, are kind of version 1.0 and surely we can do
better, right. And so there has to be probably some incentives
there.
And then generics, we need a pathway so that the generics
understand what they would have to do to show that they source
exactly the same as the innovator, because uptake of these
abuse deterrent formulations is lower because there are a lot
of old opioids on the market that are very inexpensive that are
not abuse deterrent.
And that is often for health systems the preferred opioid
to use to save money, so we need a progression of incentives
and also a clear pathway. But the innovation needs to go from
the innovators, the people who are out there trying to figure
out better ways to deter abuse.
Mrs. Blackburn. Right, but are we talking 6 months, a year?
I mean, when do you think there will be a final decision----
Dr. Woodcock. A final guidance for the generic?
Mrs. Blackburn. Yes.
Dr. Woodcock. I would hope within 6 months, I certainly
would.
Mrs. Blackburn. That is great. And then if you will speak
just a little bit toward what further the FDA can do to spur
the abuse deterrent opioids.
Dr. Woodcock. Sure. There are many things we are trying to
do, one of them though is trying to incentivize development of
drugs, new drugs that don't have these abuse liabilities to
treat pain, and we have approved a number of them. They are
often for specific conditions.
For example, we approved one for neuropathic pain and that
is now being used by the neurologist, those drugs, instead of
opioids because opioids aren't very good for neuropathic pain.
So that cuts out one category of people who are getting these
opioids.
So we want to stimulate and we have been working on this
for years with the outside world, scientific world, trying to
stimulate the development of drugs that aren't opioids that
don't have these abuse liabilities, because people are going to
continue prescribing opioids for people in pain unless they
have something else to offer. So also we have workshops and we
work on abuse deterrent formulations to try and stimulate and
work with innovators on new ways to deter abuse.
Mr. Griffith. The gentlelady yields back. Mr. Butterfield
is recognized for 5 minutes for questions.
Mr. Butterfield. Thank you very much, Mr. Chairman.
Let me just begin by thanking you and Dr. Woodcock, for
coming back. You have been at that table many times and thank
you so very much. Chairman Burgess, who is not here today, but
I want to thank him for holding this hearing on the
Prescription Drug User Fee Agreement reauthorization.
Since I came to Congress some 12 years ago back in '04,
Congress has come together under the leadership of both
Democrats and Republicans to pass this important bipartisan
legislation. Just last year we passed the 21st Century Cures
Act that this committee drafted and passed unanimously to help
boost the resources of the Food and Drug Administration and
encourage the development of new treatments.
For my constituents in North Carolina, developing new
treatments can literally make the difference between life and
death. Health outcomes for many in the communities that I
represent are deeply concerning. Many of my constituents are
African American citizens. By most measurable health
statistics, outcomes for African Americans lag far behind.
Supporting the reauthorization of PDUFA is important to finding
new treatments to help reduce health disparities for my
constituents and indeed Americans all across the country.
Through additional resources made possible by PDUFA V, the
FDA has been able to work with industry to make available new
treatments for rare diseases through the Breakthrough Therapy
Program. Through November of last year, FDA has granted, I am
told, 165 breakthrough therapy requests. This includes
treatment in many areas that disproportionately impact my
constituents and African Americans throughout the country.
Breakthrough designations have been granted for diseases
like HIV and hepatitis C, and colorectal cancer, all of which
impact minorities at high rates. PDUFA VI has the potential to
make advancements in areas from breakthrough therapies and
real-world evidence to clinical trials and biomarkers. The
additional resources made possible through the proposed new fee
structure can help FDA build the workforce needed to complete
these new tasks.
However, this administration's executive actions and
proposed budget do not seem to understand the importance of
FDA's mission to help patients and improve public health. The
impact of a hiring freeze on the FDA implemented by the Trump
administration is still unclear. Also the administration's
budget proposal fails to understand the good-faith effort that
has been put forth by the FDA and by industry and patient
advocacy groups all working together. Now is the time to come
together to support the FDA. Our constituents are counting on
us, my colleagues, to work together in this space.
Dr. Woodcock, I am excited by the innovations occurring in
cancer drug development as cancer drugs are now being developed
by molecular target. By identifying the drivers of the cancers,
these new molecularly targeted drugs are achieving great new
strides in treatment and providing greater health to cancer
patients. These targeted drugs are often effective for many
types of cancers.
Question, are innovative new cancer treatments for adults
also tested for children with the same targets as adult
cancers?
Dr. Woodcock. Not generally. The paradigm is changing and
typically over time treatments for cancer as well as other
disorders have been according to disease. So in cancer it is
what we call histology, which basically means the organ that
the cancer originated in. That is why we call it colon cancer
or we call it whatever cancer, lung cancer, right.
But these molecular alterations may go across diseases and
it may be only a small subset of each of these cancers are
driven by the same molecular alteration. That isn't something
that has really been looked at very closely in children. It may
be that there are rare mutations in children that are the
similar as the mutation in adult for these molecular targeted
therapies.
Mr. Butterfield. OK. Also my colleague Representative
McCaul of Texas and I introduced the RACE for Children Act,
H.R. 1231, to promote the discovery of new cures for children
with cancer. First, the RACE for Children Act would provide
that a drug company will provide a pediatric study plan of a
drug pursuant to the Pediatric Research Equity Act if the drug
is, ``intended for the treatment of an adult cancer and is
directed at a molecular target considered to be germane to the
growth and progression of such pediatric cancer.''
Do you believe this provision would create greater access
to novel cancer drugs for pediatric cancer research?
Dr. Woodcock. I am not able to comment on that at this
time, but we would be happy to work with you on this.
Mr. Butterfield. All right. We are deep into this and we
would like all the help we can get. Secondly, the RACE for
Children Act would end the--I am over, yes. I yield back.
Mr. Guthrie. I yield back. Thank you for yielding back. I
see no questions on the majority side. Mr. Sarbanes, you are
recognized for 5 minutes for questions.
Mr. Sarbanes. Thank you, Mr. Chairman.
Thank you, Dr. Woodcock, for joining us. I have been here
10 years, I think on four or five different committees. You are
my all-time favorite witness. I just want you to know that.
Because you are so professional in your presentations, so
knowledgeable, and you play things straight, so I appreciate
your being here.
I am fascinated by this idea of including, incorporating
real-world evidence in regulatory decision making. I mean the
implications of it are kind of humorous because it suggests
that up until now there hasn't been real-world evidence in the
process, but I certainly understand what it is meant to convey.
And I was wondering if you would just talk a little bit
about that topic. And obviously the agency is going to have to
come up with, and I know you are in the process of doing this,
a kind of formal process for identifying what qualifies as
real-world evidence and then how it gets gathered and then how
it gets translated back to the agency, how much weight is given
to it as part of the overall analysis that is done by the staff
there at the FDA. So if you could maybe just talk about that a
little bit more that would be helpful.
Dr. Woodcock. Certainly. Well, FDA runs actually one of the
largest real-world evidence gathering operations that is around
in the health area, which is our Sentinel System, which is for
drug safety and was mandated by Congress. And there we have 193
million different people's claims data that we can access, all
right, anonymously, and we use that for drug safety analysis.
And the Congress told us that that should be used first
rather than requiring companies perhaps to do specific
observational studies. And recently, as we have
institutionalized this system there are four programs where we
are able to do something in Sentinel and not require additional
outside studies, but that is safety.
Now on the effectiveness side, obviously you would only
collect real-world data if the drug were on the market, OK,
because before a drug gets on the market data is collected into
clinical trials, it is not just collected into doctor's
offices. So that would be after a drug is marketed can we
collect patient experience data to perhaps broaden the
indication or add new indications or whatever.
Mr. Sarbanes. Can you comment on how sort of off-label use
relates to that?
Dr. Woodcock. Well, it does relate to it, because often,
for example, let's take these oncology drugs and these targeted
drugs. So they target a specific target. We may approve it for
a number of tumors, but then there may be somebody who comes in
and they have a rare tumor or a tumor that this is an unusual
type and they have that mutation. So the physician may treat
them with the targeted agent and that would be considered off-
label use although it is completely rational, right.
So what we are working with a large number of outside
parties who are gathering this information up in different ways
and then they want to collect that experience of the patients,
those rare patients, and then perhaps if they responded and we
can document that then maybe we can add that to the label and
say if you have a rare patient like this come in they should be
treated with this targeted therapy too. So that is an example.
There also are registries, and some people consider those
real-world evidence and some don't. But we are trying to put
registries together, get that registry information, make sure
it is----
Mr. Sarbanes. Just on that point again intrigued me. What
would make certain people consider a registry real-world
evidence and other people not consider it real-world evidence?
Dr. Woodcock. Yes. Well, some people are sort of purists
and they consider real-world evidence only collected like in
the course of ordinary medical care and of stock, OK. Other
people consider it evidence that is collected during the course
of treatment even if you add a few bells and whistles. So we
really don't care. This is evidence outside of standard
clinical trials, so let's figure out for all of it what can we
do to gather more information about performance of drugs
outside of your traditional clinical trial.
Mr. Sarbanes. Would you imagine that at the end of this
process adding this to your portfolio, if you want to call it
that, that there would be maybe some kind of advisory council
or group that the FDA would bring into the process of
identifying real-world evidence? I mean, what kind of
structures do you think we might see, or is it premature to----
Dr. Woodcock. I think what you would see is a series of
policy sort of guidances or pronouncements by the FDA as we are
able to broaden our uses of and examples of how we have used
it. And in some of those cases we may take it to a specific
advisory committee, say we think we should add these, say,
tumors to the label because here is the real-world experience
and it looks like these people respond and they would never get
in a clinical trial because they are rare or whatever.
So that I think is the kind of accumulating information. We
can't just have people sort of think great thoughts absent
examples of what can be done.
Mr. Sarbanes. Right, thanks very much. I yield back.
Mr. Guthrie. Yes, thanks, time has expired. And seeing no
other members here to ask questions--well, thank you, Dr.
Woodcock, for being here. I concur it is always great to have
you here and you always do a good job. I appreciate it.
Dr. Woodcock. Thank you.
Mr. Guthrie. We will now transition to our second panel.
Thank you. We want to thank all of the witnesses for being
here today and taking the time to testify before the
subcommittee. As a reminder, each witness will have the
opportunity to give an opening statement followed by a round of
questions for members.
Our second panel of witnesses includes Mr. Jeff Allen,
President and CEO, Friends of Cancer Research; Ms. Kay
Holcombe, Senior Vice President of Science Policy,
Biotechnology Industry Organization; and Dr. Anne Pritchett,
Vice President of Policy and Research, Pharmaceutical Research
and Manufacturers of America.
We appreciate you all being here, and we will begin the
panel with Mr. Allen, and you are now recognized for 5 minutes
for an opening statement.
STATEMENTS OF JEFF ALLEN, PHD, PRESIDENT AND CEO, FRIENDS OF
CANCER RESEARCH; KAY HOLCOMBE, SENIOR VICE PRESIDENT OF SCIENCE
POLICY, BIOTECHNOLOGY INDUSTRY ORGANIZATION; AND, ANNE
PRITCHETT, PHD, VICE PRESIDENT OF POLICY AND RESEARCH,
PHARMACEUTICAL RESEARCH AND MANUFACTURERS OF AMERICA
STATEMENT OF JEFF ALLEN
Mr. Allen. Good morning, Vice Chairman Guthrie, Ranking
Member Green, and members of the subcommittee. It is an honor
to be here today to provide the perspective of Friends of
Cancer Research. The current pace of scientific discovery
represents an unparalleled opportunity to improve human health.
The critical component to this is an FDA that is highly
responsive to public health needs and able to evolve with
cutting edge science.
Prior to the initial user fee authorization in 1992,
patients in other parts of the world were gaining access to new
medicines more readily than Americans with only about ten
percent of new treatments reaching U.S. patients first. Today
that paradigm has been reversed. Funds through the PDUFA
mechanism have allowed the FDA to make the review process more
predictable, efficient, and accessible. In fact, our data
indicates that for new cancer drugs approved by both the FDA
and the European counterpart, 97 percent were available in the
United States first. Furthermore, the FDA approved them on
average nearly 6 months faster.
This sixth authorization of the user fee agreement comes at
a critical time for the agency and for patients. It will
support numerous initiatives, a couple of which I would like to
mention today. PDUFA VI advances the role of patients and their
experiences. PDUFA V, in the 21st Century Cures Act, provided
important steps to incorporate the patient perspective in drug
development.
The PDUFA VI agreement will further assist organizations,
researchers, in collecting patient experience data, create
channels for providing such data to the FDA, and it will help
develop methods for analyzing it. PDUFA VI supports the
Breakthrough Therapy Designation. This designation to expedite
the development of highly promising new drugs has been rapidly
implemented. To date, 170 designations have been granted
leading to 79 approvals.
Upon examining the pre-market development time of new
cancer drugs, we found that it was over 2 years shorter for
breakthrough designated drugs than for those without the
designation. PDUFA VI will provide resources necessary for
continued success. PDUFA VI promotes qualification and use of
drug development tools that can help identify patients for
which a drug is likely to work, offer early indicators of
toxicity to help improve patient safety, and in some cases
indicate that a drug will have long term benefit. The agreement
will help create a process in which new tools can be accurately
assessed and ensure their appropriate use.
PDUFA VI enhances the use of real-world evidence. Once a
drug reaches real-world populations there may be unanswered
questions about its effects, particularly in patients not
represented in clinical trials. The collection of real-world
evidence allows for a greater understanding of drugs currently
in use. By allocating user fee funding toward these programs,
the FDA and other stakeholders will be able to identify
limitations and explore different opportunities for the use of
data collected from post-market experience.
To that end, FDA approved labels should be a vitally
important source of information to guide the safe and effective
use of prescription drugs. However, in some instances, such as
drugs that have gone off patent, labels may have become
outdated and no longer reflect optimal use. This is illustrated
by extensive discrepancies between FDA approved labels and
widely accepted practice guidelines.
The FDA could play a greater role in evaluating the
relevant data to update the product label as appropriate and
adjudicate between the uses backed by strong evidence and those
backed by less persuasive information. This would establish a
high standard for post-market evidence and make the product
labels more useful. For the programs of this proposed user fee
agreement to succeed, the full budget of the FDA must be robust
and the capacity of which the agency can maintain and hire the
best scientific minds must be unencumbered.
Despite opportunities afforded by PDUFA VI, the passage of
the 21st Century Cures Act, and the enormous contributions of
this committee, I would be remiss to state that the FDA and the
people who rely on it are optimally positioned at present. The
proposed cuts to biomedical research will put the brakes on the
engines of discovery and jeopardize the development of new
medicines for patients. Holding the FDA budget authority at
stagnant levels prevents progress on agency functions that are
not covered by user fees.
Among the challenges that have been exacerbated in the
current environment is the implementation of the FDA Oncology
Center for Excellence, an innovative approach and a new model
for collaboration. The potential of a detrimental budget and
the presence of the current hiring freeze put the OCE and so
many other transformational opportunities at significant risk.
For the people who currently depend on safe and effective
medicines, for those who are holding strong for the
breakthroughs to come, and for every future patient, there
isn't time to waste. We urge Congress to swiftly pass the sixth
reauthorization of PDUFA. Thank you.
[The prepared statement of Jeff Allen follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Mr. Guthrie. Thank you. Thank you for your testimony, and I
now recognize Ms. Holcombe for 5 minutes for your opening
statement.
STATEMENT OF KAY HOLCOMBE
Ms. Holcombe. Mr. Vice Chairman, Ranking Member Green, and
members of the subcommittee, Bio appreciates the opportunity to
speak with you today about the sixth reauthorization of the
Prescription Drug User Fee act. Let me begin by stating
unequivocally that BIO strongly supports this PDUFA VI user fee
agreement and its timely authorization.
Nearly 25 years ago, completing action begun by this
committee, Congress passed the first PDUFA after agreeing with
FDA and the biopharmaceutical industry that providing
additional staff funded by user fees would help FDA review
applications more quickly. You were shown to be spectacularly
right. Today in this final year of PDUFA V, FDA is the most
efficient drug regulatory agency in the world. American
patients are the beneficiaries.
The success of PDUFA in bringing down the time of new drug
review has led over the years to substantial expansion of the
program in terms of the numbers and kinds of commitments FDA
has made annually from increasing its efficiency in
communicating with drug developers to enhancing its post-market
surveillance and monitoring of drugs throughout their life
cycles to applying best review practices across all review
divisions to enhancing processes to review and approve
therapies for rare diseases to executing systematic approaches
to measuring the benefit-risk ratio of potential drugs and to
seeking and incorporating patient perspectives in that
assessment.
What has worked relative to review of applications has also
made a difference in drug development. In PDUFA VI that is
taken to a new level. FDA formal review time is the mere tip of
the iceberg of time patients wait for new drugs. Review
timelines are significant not only because they are short, but
also because they are predictable and predictability is
critical for companies making investment decisions. It would be
highly desirable if the same sort of efficiency and
predictability were achieved throughout drug development.
PDUFA VI builds on the proven premise that greater and more
productive interaction between drug developers and FDA works.
It leads to better outcomes and to more efficient development
programs. A greater focus on drug development improvements in
PDUFA VI is not at the sacrifice of what has been achieved for
review times, 8 months for priority applications and 12 months
for standard.
The PDUFA VI goals, including expanding expertise in
diverse statistical methods, piloting innovative clinical trial
designs and computer modeling and simulation, use of biomarkers
as surrogate endpoints, and more frequent and appropriate use
of real-world evidence or big data will transform drug
development. All of these goals which attempt to bring 21st
century science to the fore in this agreement will augment, not
completely replace, tried and true methods of data collection.
In the end, these new approaches will add to the old to make
drug development more efficient while not compromising the
statutory gold standard of substantial evidence of safety and
effectiveness.
PDUFA VI also will take patient focused drug development to
new levels. The message of these commitments is that the
patient voice truly matters, in the beginning when early
studies show a promising treatment and at the end when FDA is
making its decision about a product's benefit and risk. PDUFA
VI will bring the patient voice to the forefront, changing it
from a voice with a compelling story to a voice that provides
evidence, verifiable, valid evidence that is appropriate for
the drug label.
Finally, Mr. Chairman, I want to emphasize how crucial it
is that FDA has the ability to hire and retain the people it
needs to carry out its PDUFA goals and to do that without
jeopardizing the other significant parts of its public health
mission. PDUFA is a carefully negotiated agreement that takes
account of input from all stakeholders including FDA, industry,
patient and consumer groups, and others. The key questions on
the table are what needs to be changed or enhanced and what is
the actual verifiable cost of achieving those goals?
The majority of costs paid by user fees are for personnel.
This agreement is carefully crafted to ensure that FDA can
bring those people on board who are needed to meet the goals,
employees who costs are paid by user fees. In PDUFA VI, the
annual hiring goals are included in the agreement. This allows
the public a line of sight into whether goals may fall by the
wayside as a result of an inability to hire.
Mr. Guthrie. Thank you. You need to summarize or is that
your conclusion?
Ms. Holcombe. In conclusion, I want to reiterate BIO's
strong support for this agreement. It satisfies our basic goals
of financial transparency, long-term program viability, hiring
and retention improvements to ensure stability and achieve the
agreed-upon goals. The vision of PDUFA VI is the vision of 21st
Century Cures. Put patient needs----
Mr. Guthrie. Thank you.
Ms. Holcombe [continuing]. For access to new medicines
first.
[The prepared statement of Kay Holcombe follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Mr. Guthrie. Thank you very much. Now Dr. Pritchett, you
are recognized for 5 minutes for opening statement.
STATEMENT OF ANNE PRITCHETT
Ms. Pritchett. Good morning, Vice Chairman Guthrie, Ranking
Member Green, and members of the subcommittee. I am please to
appear before you to provide PhRMA's perspective on the timely
reauthorization of PDUFA. PhRMA, as you know, represents the
country's leading innovative biopharmaceutical research
companies which are devoted to developing new medicines that
enable patients to live longer, healthier, and more productive
lives. We appreciate the opportunity to testify and share our
views on PDUFA VI.
For over 2 decades, PDUFA has helped to bring innovative
medicines to patients by providing greater clarity and
predictability in the science-based drug review process. Today,
I just want to briefly share PhRMA's perspective on the PDUFA
program and key elements of the PDUFA VI agreement.
First, I just want to note that we view user fees as an
important mechanism to support the critical work of the FDA and
human drug review process, and note as a result of this program
over 1,500 new drugs and biologics have been approved since
1992. The number of new medicines being approved on their first
review cycle is at a historic high, and as we heard earlier,
the review times have dramatically dropped by more than half
since the 1990s as a result of this agreement.
As a result of PDUFA, the U.S. leads the world in the
introduction of new medicines and is a global leader in
biopharmaceutical R&D. I would note at a time when other
countries are seeking to attract and grow their own
biopharmaceutical presence due to its far-reaching economic
impacts, it is more critical than ever that we ensure that the
U.S. retains its competitive advantages which includes a
science-based gold standard regulatory system in the FDA, one
that facilitates the ability of our industry to harness the
latest scientific and technological advances and to translate
those into new treatments and cures for patients.
I would note that PDUFA VI is a result of extensive
negotiations between the FDA and the innovative
biopharmaceutical industry and it really includes unprecedented
input across all stakeholders. Patients and patient advocates
in particular played an important role by providing input on
potential PDUFA VI goals through formal stakeholder meetings
with the agency as well as frequent interactions with industry,
and that feedback is reflected in several of the provisions. I
would note failure to reauthorize PDUFA in a timely manner
would obviously negatively impact the FDA's ability to carry
out its important role in fostering the introduction of new
medicines to patients.
And I want to briefly touch on some key elements of the
agreement. First, obviously PDUFA VI facilitates science-based
integration of the patient perspective into the development and
regulatory review of innovative medicines. Over the course of
PDUFA VI, FDA will be holding a number of workshops to gather
stakeholder input to inform a range of guidances that are
focused on how do we better incorporate the patient element
into all stages of drug development and review.
Second, PDUFA VI enhances the FDA's access to the tools,
processes, and expertise necessary to ensure that the FDA is
ready for the 21st century, the latest scientific advances in
drug development and regulation. Specifically, as mentioned by
other witnesses, there will be an increase in the FDA's
capacity to qualify biomarkers. The agreement advances the use
of real-world evidence building on 21st Century Cures
facilitates the appropriate use of innovative clinical trial
approaches.
Third, PDUFA VI will accelerate the development and
availability of new medicines to patients while providing the
scientific and regulatory predictability that will foster a
continued biopharmaceutical innovation. PDUFA VI not only
builds upon the highly successful new molecular entity review
program, which has led to shorter review times and an increase
in first cycle reviews, but it builds upon it by incorporating
additional metrics.
Fourth, PDUFA ensures that the FDA will be able to hire and
maintain a strong scientific medical and regulatory workforce
to advance its public health mission. For the first time, PDUFA
VI includes detailed hiring goals and includes dedicated
resources for the recruitment and retention of a world-class
scientific workforce. And I would note it also includes
independent outside consultants to help facilitate the agency
in developing a comprehensive hiring strategy.
And finally, the agreement builds on key provisions of the
21st Century Cures Act by further advancing real-world
evidence, incorporating that into a structured benefit-risk
framework, patient-focused drug development, biomarker
qualification, as well as includes a number of improvements to
combination product review.
I want to conclude by saying PhRMA and its member companies
we are committed to working closely with the FDA, your
committee, Congress, and all stakeholders to ensure the
continued success of PDUFA in bringing safe and effective
innovative medicines forward to address unmet medical needs for
all patients. We believe that moving all of the UFA's forward
in a timely manner is important to supporting the FDA's mission
of protecting public health and promoting innovation, as well
as critical to supporting our shared goals of fostering
continued competition and innovation. Thank you for the
opportunity to testify today.
[The prepared statement of Anne Pritchett follows:]
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Mr. Guthrie. Thank you all for your testimony, and we will
now move into the question and answer portion for our second
panel. And I will begin the questioning and recognize myself
for 5 minutes.
First, I would like to request unanimous consent for
entering the following statements in the record: the Rare
Disease Legislative Advocates, the RDLA blog, PDUFA RDLA,
Congress Begins Process of Reauthorizing Prescription Drug User
Fee Act; a letter from the Epilepsy Foundation, letter; number
three, National Venture Capital Association blog post on PDUFA;
and four pieces I am asking to enter into the record, National
Organization for Rare Diseases and Friends of Cancer Research
joint statement on PDUFA.
Mr. Green. No objection.
Mr. Guthrie. No objection, so ordered.
[The information appears at the conclusion of the hearing.]
Mr. Guthrie. So Mr. Allen, actually Mr. Sarbanes was
talking about this earlier with Dr. Woodcock, and where I kind
of wanted to look at about labeling. And after a drug is
approved, more and more information is often learned about it.
This can include new uses, more tolerable doses, et cetera. And
for cancer drugs, can you talk about the disparity between the
information in products labeling and how the drug is actually
being prescribed and administered by oncologists?
Mr. Allen. Sure. So typically a manufacturer would, if they
are pursuing an additional indication for a drug, would take it
and submit it through the supplemental new drug application at
the FDA for that information to be evaluated. But in some
instances like I mentioned, when a drug is off patent or
perhaps even for a particularly rare population where a
clinical trial is difficult or infeasible, or in some cases
because the drug has been on the market for so long may be
unethical, the label oftentimes doesn't reflect the practice
and the use of that drug over time.
And what we found by comparing the FDA labels to practice
guidelines that are constructed by medical oncologists is that
the uses that are recommended by expert oncologists are usually
far beyond that of what is contained in the FDA label. So the
question is, should the product label have a role in the agency
which we trust to evaluate medicines for them to get on the
market, should they have a more active role in looking at
potential label modifications further down the life cycle of
the drug to ensure it is supported by the highest quality
evidence.
Mr. Guthrie. Do you think FDA needs to clarify when and how
companies can provide such information?
Mr. Allen. In terms of when it can be supplied to them?
Mr. Guthrie. Yes.
Mr. Allen. I think it could----
Mr. Guthrie. When it needs to be.
Mr. Allen. The agency certainly does in terms of safety,
but the same mechanisms aren't in place with regards to
alternative uses. So one could imagine that perhaps it is worth
having a longer discussion about the ability for over a certain
period of time perhaps after the patent expires that there be
some process for review of post-market evidence in order to
make sure that the way the drug is being used is supported by
the highest quality of evidence, so the people who are
prescribing and using it are able to tell the difference
between what is high quality and what is just an anecdotal use.
Mr. Guthrie. OK. And I had another question, but I think
you did answer it that do you believe FDA should play a more
active role in updating product labeling, and you answered that
actually when you answered the first question.
Mr. Allen. Yes. I think it is worth a longer conversation
because obviously there are resource implications. But given
the oncology anyway there are highly qualified professional
guidelines that might help the FDA conduct post-market analyses
when it is appropriate to review a growing body of evidence.
Mr. Guthrie. Right. Yes, that is something that we just
need to work to make sure we can clarify that because I think
Dr. Woodcock was sharing similar to that when she was talking
to Mr. Sarbanes about, some oncologists has a different tumor
that this actually has effect for and works for and would be
logical to use, but we need to make sure that it can be done
through the process.
So thanks for your testimony, and actually I will yield
back my extra minute and I will yield to Mr. Green, 5 minutes
for questions.
Mr. Green. Thank you, Mr. Chairman.
Ms. Holcombe, current statute outlines a detailed process
for reauthorization of the PDUFA. The FDA is charged not only
with negotiating with the industry to develop recommendations,
but also to solicit public input and hold public hearings and
consult periodically with Congress and patients and consumer
groups, among others. The recommendations that are a result of
this process must also be available publicly for the public
comment and ultimately required by statute to be transmitted to
Congress by January 15th of this year. The process that led to
the ultimate transmission of FDA PDUFA VI recommendations
kicked off nearly 2 years ago in July of '15.
Ms. Holcombe, can you further discuss industry's role in
the reauthorization of PDUFA and particularly the timeline for
these activities?
Ms. Holcombe. So as you point out, Mr. Green, this process
began in July of 2015 with a public meeting at which all
stakeholders were provided an opportunity to testify, and
industry took advantage of that opportunity and presented our
views about the importance of PDUFA in general and about some
specific enhancements to the program that we would be seeking
in our negotiations with FDA. Those negotiations kicked off
approximately 2 months later and lasted then for over 12
additional months and were intensively focused on the
calculation of what could be done and how much it would cost to
do each one of those things.
And both FDA and industry put ideas on the table, and those
ideas were frequently, if not every single time, informed by
the input of other meetings that were going on simultaneously
with patients in the other groups that you mentioned, so it was
a very long process and I would say mathematically and
statistically a very precise process.
Mr. Green. Well, since this is our sixth time on it,
hopefully we learn something every time.
Ms. Holcombe. Well, we are getting faster.
Mr. Green. OK. The statute requires that a recommendation
be transmitted to Congress no later than July 15th of '17, a
deadline they met. Does the statute allow the FDA to transmit
recommendations for reauthorization at an alternative date?
Ms. Holcombe. Not that I am aware of.
Mr. Green. OK. PDUFA expires on September 30th of '17. What
would be the impact for your member companies if Congress did
not pass the reauthorization of PDUFA before the September 30th
deadline?
Ms. Holcombe. I think we would describe the implications as
titanic in nature. FDA would be required, if this were not
reauthorized, to reduce its force by probably in the Drug
Center alone about 5,000-plus individuals, and those are the
people who review our applications. But even more importantly
than the review of applications, which as we have all said
today is merely the tip of the iceberg of our process of drug
development, it would absolutely disable any process that FDA
has for talking to us during drug development about how to be
more successful in our program.
Mr. Green. Do you support and your industry support PDUFA
VI recommendations as transmitted to Congress?
Ms. Holcombe. Yes, we do.
Mr. Green. OK. You note in your testimony that the drug
development process, the most time-intensive part of bringing a
drug to market, it is my understanding that on average it takes
between 10 to 12 years to develop a drug. Recognizing this, we
included in the 21st Century Cures a revision that would have
FDA host a public workshop and issue guidance on innovative
trials and designs and approaches.
Would you please explain further how innovative trial
designs may help your member companies in bringing treatments
to market quicker and, in addition, would you discuss how PDUFA
VI builds off of the Cures' effort to support the use of
innovative drug trial designs?
Ms. Holcombe. Yes. So as my testimony pointed out great
minds think alike, and we in the industry as well as FDA itself
agreed a hundred percent with you in your identification in
21st Century Cures of the importance of thinking of different
ways to do clinical trials than the traditional way of
randomized controlled trials.
Often clinical trials have to enroll many, many people and
they take a long time. And as Dr. Woodcock pointed out, they
often have high dropout rates. Patients can't stay in them, and
these cause drugs to fail and at great expense to companies
that are developing them. So innovative ways of thinking,
creative ways of thinking could we do trials differently and
therefore make them shorter and smaller but still come out with
the substantial evidence of safety and effectiveness that we
all need to have and the answer to that is yes, we can.
Mr. Green. OK. Mr. Chairman, I know my time has expired.
And Mr. Allen, I would like to submit some more questions to
you, but obviously FDA is working with you on the off-label
usage that practitioners learn, and we need to be able to learn
what they are learning so we can actually make those
pharmaceuticals available and go back through the FDA process
as brief as we could do to make sure those cures are there. So
thank you, Mr. Chairman.
Mr. Guthrie. Thank you. Thank you. The gentleman's time has
expired. Dr. Bucshon, you are recognized for 5 minutes for
questions.
Mr. Bucshon. Thank you, Mr. Chairman. First of all, I mean,
I also want to go on the record saying I have concerns about
the budget proposal as it relates to research, the NIH and
above. I don't think that is a partisan issue.
The question I have is as we transition to new models to
approve medicines we have been talking about that whether that
is changes in clinical trials or other things, how do you see
the legal environment evolving to allow this to happen? I will
start with Dr. Pritchett. Because, I mean all of us are
realists. I was a medical doctor before, as you transition to a
new way to approve a product there is going to be people out
there that are looking to throw a wrench into the gears by
saying it didn't prove what it was supposed to prove and that
is why my particular client has been hurt. And so I mean there
are legal ramifications of trying to do this also, any
thoughts?
Ms. Pritchett. So I think as I understand your question, as
we think about looking at innovative, new approaches to
clinical trials, collection of real-world evidence and how do
we apply this to drug development and then how do we ensure
that we are using them in a robust way so that we are reducing
any potential concerns related to liability, ensuring that we
aren't approving medications without appropriate evidence----
Mr. Bucshon. Essentially that is the question, because that
is one of the things that drives up costs of drug approvals.
Everybody has to look at those issues, but a percentage of the
drug costs are because of these type of issues. My question
would be basically is, I mean, yes, what are our thoughts about
that? Do we need, I mean obviously we are working on tort
reform in other areas. Any thoughts on that process as it
relates to the drug product development?
Ms. Pritchett. So I am not prepared to discuss that today.
We would be happy to come back and have further discussions
with you. I would note that as we look at the PDUFA process,
part of the engagement by FDA in providing very clear guidances
to industry is to help avert any potential concerns from a
liability perspective, et cetera, but I think this is an
important topic that we would welcome to have further
discussion.
Mr. Bucshon. OK, because I could see that the FDA and
others very quickly retract their thoughts on these things as
soon as we have some big class action lawsuit against the FDA
and everybody else.
Ms. Pritchett. I think that is a very important issue that
you are raising and we would greatly appreciate coming back and
having further discussion on that topic. I appreciate you
raising it.
Mr. Bucshon. OK, great, any other comments from the other
panelists?
Ms. Holcombe. I think one of the important things to
recognize about PDUFA VI is that in some respects takes account
of this type of concern by initiating under PDUFA VI pilot
programs, where the agency and the industry are going to work
together to pilot these various trial designs or model-informed
drug development approaches and determine whether in fact with
input from outside stakeholders, whether these kinds of
designs, this kind of way of developing drugs is going to
produce the sort of good evidence, solid evidence that we need
to make sure this product is safe and effective when it goes on
the market.
Mr. Bucshon. OK, anything else? Well, anyone, for all of
you, can anyone give us a sense of how they envision the FDA
utilizing the authorities in 21st Century Cures Act in PDUFA
VI, that the provisions involving the use of real-world, so-
called real-world evidence to support their decision making how
we would envision that being incorporated?
Ms. Holcombe. Well, I think as Dr. Woodcock said, FDA
already uses real-world evidence in the determination, making
determinations about potential safety signals of marketed
drugs. So the question is in PDUFA VI, is it possible to use
real-world evidence, i.e., patient experience with drugs in the
marketplace from medical records or from your Apple watch? Is
it possible to harness those data, to validate those data, and
to use those data to understand more about how the drug is
working, and would those data be helpful then in making a
decision about perhaps broadening an indication for a drug that
is very narrowly indicated or adding an indication, which goes
to the point that Dr. Allen was making about how the drug label
can become out of date when medical practice is ahead of what
was known at the time the drug was produced.
Mr. Bucshon. OK. I will just make a quick comment. That is
why other issues we are working on like interoperability of
electronic medical records is going to be really key to this
type of thing. I think everyone would agree to that and I yield
back.
Mr. Guthrie. The gentleman yields back. Seeing no questions
from this side, Ms. Brooks from Indiana is recognized for 5
minutes for purpose of asking questions.
Mrs. Brooks. Thank you, Mr. Chairman.
Dr. Pritchett, PDUFA VI creates a significantly revised fee
structure which replaces current levies on manufacturing
facilities and on products. Can you explain this revised fee
structure and how is it beneficial for all parties?
Ms. Pritchett. I would actually yield to Kay who was
directly involved in the negotiations and I wasn't, who I think
would be better.
Mrs. Brooks. I wondered about that after one of her
previous answers, but I was suggested to you. So Ms. Holcombe,
would you like to share with us?
Ms. Holcombe. The reason for looking at a different way of
collecting fees was to try to make sure that we had a system
that was administratively as simple as it could be so that it
was not so costly or so burdensome on either the FDA and
companies. So the way fees used to be collected was they were
divided one third among manufacturing facility fees, product
fees, and application fees. There were two things wrong with
that, at least two.
One thing was that it placed equal emphasis in terms of
percentage of dollars collected on all three components,
including the application fee which is the least predictable
source of revenue. There is very little perfect ability to
predict the number of applications that are going to come into
FDA on an annual basis. They have a lot of experience so they
can give you a range, but if you collect 50 instead of 60 that
makes a huge difference in the total revenue that you are
collecting. So could we reduce the dependence on the
application fee and increase the dependence on other fees?
The second thing that was wrong was the manufacturing
facility fee, which is a nightmare to calculate mostly because
drugs aren't just manufactured like I make mine in my facility
and you make yours in your facility. Lots of people make drugs
in the same facility, so figuring out whose was where when and
how often and so forth. So could we offload that fee, could we
calculate the rest of the fee based on number of products?
And so we calculated what would that mean if we did certain
percentages of collection from that new fee and certain
percentage from the application fee? And we developed, or FDA
actually developed, not I personally, a tool that companies
could use and they could plug into this tool how much money
they had paid in fees in previous years and how much they would
pay under some new sort of split of the fees.
Mrs. Brooks. What is the tool referred to by the FDA?
Ms. Holcombe. We called it a widget, OK.
Mrs. Brooks. OK.
Ms. Holcombe. Yes, my terminology, sorry. It was a tool
that allowed you to manipulate the percentage based on, so the
total fee collection, let's say, is $800 million. If you
collected ten percent from applications and 90 percent from
products, what would that mean for each company? How much would
they pay?
So they would plug in their own numbers, like how many
applications did they think they would be submitting next year
and how many products do they have on the market? And they
figured out, bingo, up came this number; this is how much you
pay. Well, then they could change that from 10/90 to 20/80. How
much would you pay based on your number of products on the
market and the number of applications you anticipate
submitting, how much would you pay?
And using that tool, we figured out collectively with all
of our companies that the ratio that had the least negative
impact on the most companies was a 20 percent application fee,
80 percent program fee collection. And although a small number
of companies on a percentage basis, out of all the long list of
companies that pay fees, did see that their fees would go up
slightly, the vast majority of companies saw that their fees
actually would go down.
How could that even be, right, but it was. I mean, math, it
is just a wonderful science.
Mrs. Brooks. And if those fees go down what happens?
Ms. Holcombe. The total amount of money collected is still
the same because it is spread out across all fee payers.
Mrs. Brooks. OK, thank you. I am sorry my time is up, but
thank you for explaining it.
Ms. Holcombe. I am sorry I don't know the math involved in
it.
Mrs. Brooks. I yield back. OK, thank you.
Mr. Guthrie. Thank you, and the gentlelady yields back. Mr.
Bilirakis is recognized for 5 minutes for questions.
Mr. Bilirakis. Thank you. Thank you, Mr. Chairman. I
appreciate it very much.
Ms. Holcombe, back in the 2000s it was recognized that
there was a lack of good information on the safety and efficacy
of drugs for the pediatric population. Can you talk about some
of the incentives that came about to encourage more pediatric
clinical studies?
Ms. Holcombe. So the Best Pharmaceuticals for Children Act
combined with the Pediatric Research Equity Act have been a
very successful way to get more drugs studied in pediatric
populations so that information can go on the drug label and
pediatricians know how to dose the drugs for children. The
incentive that has caused that to be so successful was the
addition to whatever regulatory exclusivity the company might
have on its product for adults of 6 months for doing these
pediatric studies.
And we believe at BIO that incentives such as this one can
be very effective in increasing the number of products that are
developed in areas for which there is high unmet medical need
but very difficult populations or areas of interest, such as,
for example, intractable antibacterial resistant conditions.
These are tough and there is just no good way of doing it. So
incentives can be very effective and they have been for
pediatric studies.
Mr. Bilirakis. What about the rare disease space? Again
there are about 500 approved rare disease drugs, but 7,000 rare
diseases affecting approximately 30 million Americans. They are
taking medication off-label, and I know the stories because I
hear from my constituents on a regular basis. They take the
drugs off-label not knowing if their drugs are safe and
effective for their conditions or if it is proper dosage--that
is so important--and fighting with their insurance companies on
coverage of these medications.
Does it make sense to incentivize development for a
targeted population when there are clearly defined needs?
Ms. Holcombe. Yes.
Mr. Bilirakis. Thank you. Last month my colleague and I,
G.K. Butterfield, introduced the OPEN Act for the second time.
Much like the BPCA, it creates an incentive to run more
clinical trials in the rare disease space where 95 percent of
diseases have no FDA approval treatments. This would bring more
approved drugs to these patients.
The OPEN Act has the potential to result in hundreds of new
drugs and treatments for individuals with rare diseases. Only
150 rare disease patient groups, over 150 at last count, I
think it is more than that support this bill. The OPEN Act was
part of the House 21st Century Cures Act, and while it fell out
at the 11th hour, unfortunately, I am going to continue to push
for this legislation. It is a priority for me. Do you have any
comments on that? And of course I welcome cosponsors for this
legislation as well.
Ms. Holcombe. So it is clear that without the Orphan Drug
Act we would not have 500 treatments for rare diseases. It is
also a tragedy that we don't have 7,500. And again we believe
incentives can work. We don't have an official position on your
proposal, but definitely it merits more evaluation.
Mr. Bilirakis. All right. Well, thank you very much. I
yield back, Mr. Chairman.
Mr. Guthrie. Thank you. The gentleman yields back, and
seeing no other members wishing to ask questions I would like
to thank all of our witnesses for being here today.
And pursuant to committee rules I remind members that they
have 10 business days to submit additional questions for the
record, and I ask the witnesses submit their response within 10
business days upon receipt of those questions.
Without objection, the subcommittee is adjourned.
[Whereupon, at 12:18 p.m., the subcommittee was adjourned.]
[Material submitted for inclusion in the record follows:]
Prepared statement of Hon. Frank Pallone, Jr.
Thank you, Mr. Chairman. I appreciate the opportunity today
to discuss the reauthorization of the Prescription Drug User
Fee Act. PDUFA has been incredibly successful at bringing
reviews of new drug applications down by more than half, and
providing patient access to treatments more quickly, and often
before any other country.
PDUFA has also encouraged innovation by bringing stability
and predictability to the review of new drug applications. FDA
has been able to hire the review staff and scientific and
technical experts needed to keep pace with science and increase
the efficiency of the review process. However, more work needs
to be done and I am encouraged by how PDUFA VI builds off of
the successes of this user fee program.
I am therefore disappointed to see what can only be
referred to as a disclaimer in the testimony today from FDA.
While it is true that the reauthorization proposals were
negotiated under a previous Administration, the goals of the
PDUFA program and the drug approval process remain the same--a
fully resourced and staffed FDA, and an efficient and timely
drug review process that is keeping pace with the scientific
and regulatory advancements in this field. It is my hope that
the Administration would understand how carefully crafted the
current agreement is, and recognize that the reauthorization
process started nearly two years ago.
The agreement before us today is the result of many
negotiations with industry and stakeholders, consultations with
patients and consumers, and solicitation of public input. The
resulting recommendations were transmitted to Congress in
meeting the January 15, 2017 statutory deadline. Transmitting
new recommendations at this point would go against this
requirement, and run the very real risk of PDUFA not being
reauthorized before the program expires on September 30,
endangering the review of innovative new drug treatments and
threatening the jobs of thousands of FDA employees.
I intend to continue to work with my colleagues on the
Committee and across the Capitol, as well as industry, to
ensure that we do not let this happen. This is a strong
agreement, and one that deserves our support.
Thank you.
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