[Senate Hearing 115-261]
[From the U.S. Government Publishing Office]
AGRICULTURE, RURAL DEVELOPMENT, FOOD AND DRUG ADMINISTRATION, AND
RELATED AGENCIES APPROPRIATIONS FOR FISCAL YEAR 2018
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TUESDAY, JUNE 20, 2017
U.S. Senate,
Subcommittee of the Committee on Appropriations,
Washington, DC.
The subcommittee met, pursuant to notice, at 10:35 a.m., in
room SD-192, Dirksen Senate Office Building, Hon. John Hoeven
(chairman) presiding.
Present: Senators Hoeven, Collins, Rubio, Merkley, Leahy,
and Tester.
U.S. FOOD AND DRUG ADMINISTRATION
STATEMENT OF SCOTT GOTTLIEB, M.D., COMMISSIONER, FOOD
AND DRUG ADMINISTRATION
opening statement of senator john hoeven
Senator Hoeven. The hearing will come to order. I would
like to thank the members for being here, Ranking Member
Merkley.
And I certainly want to welcome FDA Commissioner Gottlieb.
Thank you for being here this morning. We appreciate it.
Today's hearing will focus on the Food and Drug
Administration's fiscal year 2018 budget request. Thank you
again for being here, Dr. Gottlieb. Obviously, this is a good
opportunity to talk about FDA's priorities for the upcoming
year.
Congratulations on your confirmation. We certainly want to
welcome you in your first appearance before the subcommittee,
and we look forward to working with you.
The agency you head has authority over approximately $0.20
of every dollar spent in America. Americans expect that the
food they eat and the drugs they take will be safe and
effective.
FDA's reach is vast. The agency has authority over more
than 300,000 foreign establishments and 185,000 domestic
establishments, ranging from food processing plants to
facilities that manufacture lifesaving medications. In addition
to facilities themselves, FDA is tasked with the regulatory
responsibility of individual products.
In delivering these regulatory responsibilities, your
private sector partners expect transparency and certainty from
the FDA.
When I speak to small businesses and AG producers in North
Dakota, their overwhelming concerns are that often overly
burdensome regulations coming out of Washington, D.C., can
stifle innovation and hinder their ability to create jobs.
While we all support the FDA's mission, we must also be mindful
of these concerns.
I believe that FDA must avoid the trappings of one-size-
fits-all solutions, and urge you and your staff to take a
common-sense approach.
In regard to the budget request itself, I am concerned that
this request relies on a significant increase in user fees that
is not feasible and unlikely to gain congressional approval. I
am concerned that the proposed cuts to budget authority may
negatively impact food safety programs and slow the agency's
important work on drugs and medical devices.
That being said, Dr. Gottlieb, I also recognize that these
decisions were made before you were confirmed, so I hope you
will pledge to work with Congress to ensure that FDA has the
resources necessary to meet its critical mission.
We have many other issues to cover this morning, so at this
point, I will turn to Senator Merkley for his opening comments.
Senator Merkley.
opening statement of senator jeff merkley
Senator Merkley. Thank you. I am going to keep this very
brief, because we are hoping to hear your testimony and have a
round of questions before we go to the vote at 11.
The department you head covers products that constitute 20
percent of what consumers spend both in food and drugs. It
makes that work very important, and this budget before us very
important on everything from scientific research, support for
State and local health organizations, blood safety work,
medical devices, post-market surveillance, medical product
exams, and so much more.
And so I look forward to hearing your thoughts on the
budget and getting to our inquiries.
Thank you and congratulations.
Senator Hoeven. I would suggest, at this point, we go right
to Dr. Gottlieb's statement, so that we can come right back for
questions and then any opening statements as well that other
members may have.
All right, Dr. Gottlieb.
summary statement dr. scott gottlieb
Dr. Gottlieb. Thank you. Mr. Chairman, Mr. Ranking Member,
and Members of the Subcommittee, I appreciate the opportunity
to testify today regarding the President's budget.
I have talked to you in the past about the steps that FDA
is taking on the generic drug side to try to bring more low-
cost opportunities to patients when it comes to new drugs and
improved access. I want to just briefly touch on some of the
things we are going to be doing on the new drug side. The most
tangible way we are going to reduce health care costs is by
finding better treatments for a lot of costly diseases.
breakthrough drugs
Toward these ends, we will be announcing soon a medical
innovation development plan that will include a broad range of
steps we will take to make sure that our own regulatory tools
and policies are modern and risk-based, and designed to
facilitate the development of potentially breakthrough new
treatments.
One area of focus of this new plan is going to be on
targeted drugs, especially those that affect rare diseases or
diseases for which there is no effective therapy. Among other
things, FDA will be updating various guidance documents on the
kinds of drug development techniques that help facilitate the
discovery and development of targeted therapies. This includes
guidance on clinical trial enrichment strategies to improve
efficiency and adaptive trial designs so we can modernize the
statistical tools we use to evaluate safety and effectiveness.
We will also be taking a fresh look at policies that support
innovation to allow drugs to be targeted only to those patients
who are most likely to benefit from medicine. We will also be
taking a broad range of new steps.
Among these new actions, we will be issuing a new guidance
document within the next 6 months on the clinical evaluation of
targeted therapies for rare disease subsets. This new policy
will address targeted drugs and how we can simplify the
development of drugs targeted to rare disorders that are driven
by genetic variations and where diseases all have a similar
genetic fingerprint, even if they have a slightly different
clinical expression.
One example is a cancer, where a drug targets a particular
molecular subset of cancer, regardless of where the tumor
arises. We will clarify when we can give a broad approval to a
drug in multiple different kinds of molecularly similar
cancers, which are not particular to the tumor being in one
specific tissue or organ. In other cases, rare subsets may be
grouped by lab testing, so they can be studied in a single
clinical trial.
This sort of genetically driven medicine is more common as
we understand the genetic basis of disease. Now, our new policy
will describe when we will approach drug review less by how a
disease is expressed and more by how it is driven by a common
set of genetically driven factors.
Many of these targeted drugs are aimed at rare and orphan
diseases. But right now, we have a backlog of about 200 orphan
drug designation requests where we have not responded to
sponsors on whether the drugs will receive an orphan drug
designation from FDA. As part of our new plan, we are
committing today that, in 90 days, we will completely eliminate
this backlog of requests and provide an answer back to the
sponsors.
To help eliminate the backlog, we have created a special
orphan designation SWAT team. Moreover, we will never again
develop a backlog. Going forward, we are committing today that
every orphan drug application will receive a response from FDA
within 90 days of the request. To enable more efficient reviews
and timely responses to sponsors, we are also implementing a
new streamlined orphan designation review template.
These are just some of the things we are working on. I look
forward to discussing with you how FDA's budget and this
committee can support all of the agency's key priorities,
including food safety, and enable consumers to improve their
lives.
Thanks a lot.
[The statement follows:]
Prepared Statement of Dr. Scott Gottlieb, M.D.
Good morning Chairman Hoeven, Ranking Member Merkley, and Members
of the Subcommittee, I am Dr. Scott Gottlieb, Commissioner of the Food
and Drug Administration (FDA). Thank you for the opportunity to appear
before you today to discuss the President's fiscal year 2018 Budget
request for FDA.
First of all, I would like to thank you all for your continued
support of FDA. FDA has received strong bipartisan support throughout
the appropriations process in recent years. This funding is critical to
the agency fulfilling its mission. Without your support, we could not
meet the critical public health challenges confronting the nation.
I am honored to have been chosen by the President and confirmed by
Congress to lead FDA. As a physician, an entrepreneur, a cancer
survivor, and a father, I know personally the importance of FDA's role
in improving and protecting the lives of all Americans. Every person in
this country is affected in one way or another by the decisions made by
FDA. For this reason, I am honored and humbled to serve as FDA's
Commissioner.
FDA's fiscal year 2018 Budget requests $5.1 billion--a nearly 10
percent ($456 million) increase over the fiscal year 2017 Continuing
Resolution (CR) funding level. Mindful of the larger pressures on the
Federal budget, FDA has focused our request on the most urgent needs.
The fiscal year 2018 Budget aims to protect the public health by wisely
investing taxpayer dollars, requiring industries that benefit from the
FDA's review process to pay their share, and advancing regulatory and
administrative efficiencies.
fda plays a critical role in america's public health system
As a science-based regulatory agency, FDA's broad mission is to
promote and protect the nation's public health and touches the lives of
all Americans. Over $2.4 trillion annually, roughly 20 cents of every
dollar, is spent by consumers on a product that FDA regulates. These
products include human and animal drugs, medical devices, biologics,
such as vaccines and blood, dietary supplements, and cosmetics. Tobacco
is another product within FDA's purview--the agency protects the public
health of future generations by reducing tobacco use by America's
children.
FDA's regulation of food is another critical part of FDA's mission.
FDA works to assure that the nation's food supply is safe, sanitary,
wholesome, and appropriately labeled. FDA has made great strides in
promoting the safety of the foods we eat as envisioned by Congress in
the FDA Food Safety Modernization Act (FSMA). Thanks to the support of
this Committee and your colleagues in the House, we have been working
closely with our state partners, to educate and assist industry during
the implementation of FSMA's provisions. These include preventive
controls for manufactured human and animal foods, sanitary
transportation of our food, and as of May 30, verification that our
high food safety standards have been met by foreign suppliers. FDA
remains committed to working with industry to facilitate innovation to
make safe and healthy food choices available to consumers.
fda has a proven track record of success, but there's more work to do
In the last year, FDA has helped bring new treatments, including
several life- saving cures, onto the market. FDA's Center for Drug
Evaluation and Research approved 22 novel drugs in 2016; approvals
included the first treatment for patients with spinal muscular atrophy,
a new drug to treat patients with a rare chronic liver disease known as
primary biliary cirrhosis, and two new treatments for patients with
hepatitis C. Additionally, 2016 marked the highest number of generic
drug approvals and tentative approvals in the history of the FDA's
generic drug program--more than 800 in total. In September 2016, FDA
approved the first ``artificial pancreas,'' a medical device that
automatically monitors blood sugar and provides insulin doses when
needed. This device has the potential to improve the lives of roughly
1.5 million Americans living with Type-1 diabetes.
As highlighted by the above examples, FDA's collaboration with
innovators brings products to the market that make a difference in the
lives of all Americans. Since the creation of the first user fees in
1992, user fees have been instrumental in allowing FDA to build
capacity and improve the timeliness of the medical product review
process without compromising the agency's high standards. The user fee
programs provide FDA with the critical and stable funding we need to
hire and train the highly-qualified reviewers needed to keep pace with
innovation.
However, the medical products field is ever-changing and advancing,
and to ensure the agency has the critical resources needed to keep pace
with this field, the Fiscal year 2018 Budget recalibrates how the
agency finances our medical product review work. Calling for an
increase of $1.2 billion in user fees, the fiscal year 2018 Budget
includes a total program level of $3.2 billion for medical product
safety investments, which is $505 million above the fiscal year 2017 CR
level. The Budget finances the full cost of FDA pre- market review
through user fees. These resources will dramatically increase the
agency's capacity for pre-market review, and bring more new products to
market faster than ever before.
cures implementation
The fiscal year 2018 Budget's focus on medical products complements
Congress' direction last December in passing the 21st Century Cures Act
(Cures). Cures provided a dual directive to FDA-- to support innovation
while maintaining the evidentiary standards that provide assurance to
the American public about the safety and efficacy of medical products.
This includes advancing patient-focused drug development and using
real-world evidence in modern clinical trial design. As a result, Cures
will help FDA facilitate more patient-centered, efficient, and less
costly medical product development, ultimately leading to more timely
patient access to important medical products. The fiscal year 2018
Budget requests a total of $60 million to support this critical work,
and we look forward to working with Congress, and this Committee, as
FDA continues its work on implementing Cures.
promoting innovation by prioritizing regulatory efficiency
As FDA's Commissioner, part of my job is to ensure the Agency has
the policies and processes in place needed to address the important
public health issues of our day, as well as emerging threats of
tomorrow. We must hold true to our consumer protection mission, while
not hampering innovation.
The Administration is committed to the goal of reducing barriers to
innovation and spurring innovation on behalf of patients. At FDA, we
understand the impact our regulations have on industry and the public--
which is why we have, and will continue to engage in robust dialogue
with outside stakeholders to ensure our actions strike the right
regulatory balance while maintaining our gold standard.
The fiscal year 2018 Budget includes proposals designed to make
sure we are taking a risk-based approach to our work and make the
process for developing safe and effective medical products more
efficient. By leveraging FDA's statutory mandates, including recent
enhancements made by Cures, the agency is working to reduce review
times by improving processes and gaining efficiencies to the greatest
extent possible. These proposals will help reduce uncertainty in
medical product development by increasing engagement and early
interactions with manufacturers. Improved regulatory science and
policies will not only lead to more efficient approvals and increased
competition that can help reduce costs to consumers, but more
importantly, they will improve patient- outcomes. By streamlining
clinical trials, integrating patient voice throughout the regulatory
process, and promoting greater preparedness for novel and emerging
public health threats, Americans will get better products, faster.
prioritizing administrative efficiencies
In addition to regulatory efficiencies, FDA is taking a close look
at all of our programs, policies, and procedures to ensure that every
dollar dedicated to administrative costs is spent wisely. The fiscal
year 2018 Budget proposes the establishment of a Working Capital Fund
(WCF) to support agency-wide business services. A WCF will allow FDA to
operate in a more efficient and transparent business environment. Over
time, this WCF will also allow FDA to recapitalize resources to support
IT infrastructure, reduce cost redundancy and improve service delivery
for mission critical needs.
Dollar for dollar, FDA remains one of the smartest investments made
by the American taxpayer. The fiscal year 2018 Budget also identifies
targeted reductions and program changes totaling $127 million in budget
authority while preserving core mission activities. These reductions in
budget authority are targeted to certain areas where better tools and
policies will allow us to do more with less, and will be coupled with
policy efforts to improve the efficiency of the programs that see
reductions, to make sure that we are improving our effectiveness and
taking a risk-based approach to our consumer protection mission.
conclusion
Today, we are at an inflection point in public health. Cures for
diseases we once believed were incurable are now within our reach. The
fiscal year 2018 Budget will protect and advance the health and well-
being of every American, while providing American taxpayers the
assurance that we are requiring industries that benefit from the FDA's
review process to pay their share. I look forward to answering your
questions today and to working with all of you going forward.
Senator Hoeven. Thank you, Doctor. We will start with
questions.
FISCAL YEAR 2018 RESOURCES
As I noted in my opening comments, I have concerns with the
administration's proposal to essentially double user fees. It
does not appear that the HELP Committee will move forward with
that proposal. Instead, Congress will likely pass the
previously negotiated user fees.
So my question is, based on the fiscal year 2017
appropriation, are you confident that you are going to be able
to meet the program needs, based on your current appropriated
level for fiscal year 2017?
Dr. Gottlieb. Senator, thanks for your question.
The bottom line is, we can always do more with more when it
comes to the resources that the agency has. I am confident that
we have been able to be efficient in everything we do.
I think there are still places that we can look within the
agency to try to improve our operational efficiency. We
recently made an announcement with respect to a realignment
when it comes to the field activities. We are going to continue
to look for places to improve our operational efficiency.
We appreciate very much, though, the resources that we have
gotten from this committee, in particular, the resources on the
food safety side and the resources that we have gotten under
FSMA. That that has dramatically improved the stature and the
base of resources for food safety. It is a very different
agency today than the one that I left 10 years ago, in that
regard.
HIRING FREEZE
Senator Hoeven. You have about 1,000 vacancies. The hiring
freeze has been lifted for your agency. Are you moving forward?
And where are you in that process of filling positions?
Dr. Gottlieb. Thanks for the question. That is right. We
negotiated the lifting of the hiring freeze about 2 weeks ago,
I believe, and we are starting to move forward with filling
those vacancies. I put out a notification recently to the
Office of the Commissioner, as well as the different centers,
in terms of the process of moving forward. But there is already
activity with filling some of those existing open slots.
OPIOID CRISIS
Senator Hoeven. What is the FDA's role in addressing the
opioid crisis? And what are you doing?
Dr. Gottlieb. Well, it is multifaceted. This is, in my
view, the biggest challenge facing the agency, and I think one
of the biggest public health crises facing this country.
There are a lot of different components where the FDA is
going to play a role. You look at medically assisted therapy
where the agency plays an important role trying to address the
current addiction problem, and trying to get better opioids on
the market that are more tamper-resistant.
I think where we can play a particularly important role,
however, is on the new addiction aspects of this crisis. We
know that most people who are going to become addicted to
opioids are first exposed to opioid drugs in the clinical
setting through legitimate prescription. A certain percentage
of patients who are exposed to opioids in the clinical setting
will go on to develop an addiction.
So I think it is incumbent upon all of us to make sure that
only properly indicated patients are being prescribed opioids.
And when they are prescribed opioids, they are prescribed
opioids for a duration that comports with the clinical
circumstance for which the prescription was written in the
first place.
There are things that FDA can do to try to address these
aspects of the problem. So that is going to be a particular
area of focus of ours. We recently developed a steering
committee made up of all the center leadership and senior
clinicians within the agency to look at trying to see how we
can think differently about this problem.
The other place where I have tried to focus some
policymaking attention is looking at the risk in the illicit
setting. We traditionally have looked at the risk of illicit
use as a component of how we evaluate the risk and benefit of
opioids overall. But I want to make sure we have a proper
framework in place for doing this, and we are looking not just
at the risks associated with these drugs in their labeled
indication but also the risks associated with how they might be
abused and misused and diverted and used illicitly. And we have
recently taken an action that factored into it consideration of
how the drug was being used in the illicit setting.
Senator Hoeven. What about approval of drugs that actually
help wean people off some of the opioids? I know there is
development in this area.
Dr. Gottlieb. Right. This is the medically assisted
therapy. We need to continue to develop good drugs in this
area.
One of the challenges, though, has been reimbursement.
While it is outside my mandate, getting these drugs to patients
is an important step as well. So we are looking at things we
can do to help facilitate the development of clinical trials
that move these drugs into different clinical settings to see
how we might better study them in real-world settings.
Hopefully, we will have more to say on that soon.
NUTRITION FACTS PANEL
Senator Hoeven. Last week, the FDA announced a delay, for
compliance to the Nutrition Facts Panel regulations. I know you
are limited on what you can say until it is published in the
Federal Register. But given my comment regarding one-size-fits-
all solutions, will FDA use a common-sense approach toward
those nutrition regulations?
Dr. Gottlieb. Thanks a lot.
I am confident we will, Senator. We announced the delay, in
part, to provide additional guidance to sponsors on how to
interpret aspects of the new Nutrition Facts label.
This is a time limited delay. This is not a suspension of
the regulation. We are not reopening the regulation. We are
just using this time to develop additional guidance documents
that we will be issuing to help inform how people can comply
with the new labeling.
Senator Hoeven. Senator Merkley.
Senator Merkley. Thank you, Mr. Chairman.
And welcome, Dr. Gottlieb.
RESPONSE TO MINORITY REQUESTS
The first question I have for you is, a number of news
reports have discussed the administration's directive to
Federal agencies not to respond to requests from minority
Members of the Senate.
That is completely contrary to the very long and positive
bipartisan history both on this subcommittee and our dealings
with the FDA under both Republicans and Democrats. I think a
dialogue with the executive branch for all Members results in
stronger bills more likely to have support, and it clearly
benefits the FDA and the American public to have that dialogue.
Can we count on you to be engaged in that dialogue and
respond to inquiries and requests from the majority and the
minority?
Dr. Gottlieb. Absolutely, Senator. I responded to all the
requests for new information that I got during my confirmation
process from the minority. I am prioritizing timely responses
equally from both the majority and the minority. We have
reached out to many offices, including your own. I had the
pleasure to meet with you twice.
So we will not pick sides in how we provide information to
Congress. I respect Congress, and we are going to make sure we
are providing you the information you need.
Senator Merkley. Thank you very much. Your outreach has
been appreciated, and I appreciate your commitment to
continuing on that course.
OPIOID ADDICTION
During your confirmation process, you described the
staggering human consequences of opioid addiction and
characterized that epidemic as the biggest crisis facing the
agency.
Now we have a prediction that the CDC, engaged with various
other experts, that the Trumpcare bill coming out of the House,
we do not know what version we will see in the Senate yet,
would cut billions of dollars from treatment for mental health
and substance abuse disorders, and also that millions of people
will lose their insurance, which makes it very unlikely that
they will be seeking medical care in the first place.
We are concerned that this budget would damage the ability
to take on opioid addiction, with its enormous daily toll on
lives. As someone who is leading the charge on this epidemic,
who has presented it as a top priority, do you have concerns
about the loss of access to health care by millions of
Americans, if this bill is enacted?
Dr. Gottlieb. Senator, I am very focused on what we are
doing at FDA right now to address this crisis, as I know you
can appreciate. And you are going to continue to see a series
of activity out of the agency to address this crisis in
different ways that hopefully start moving us to a posture
where we are getting ahead of the problem, instead of always
being one step behind.
I have not focused a lot of attention on the various
legislation moving through, with respect to the Affordable Care
Act. I am very focused on what I am doing at FDA right now,
which is more than a full-time job, as I know you can
appreciate, and I will continue to talk to you about those
priorities.
Senator Merkley. There are 13 Senators who are holding a
series of closed meetings to work to prepare a version of the
bill that will come before the Senate, probably next week. Have
those individuals, or as a group, brought you in to get your
consultation and insights on the opioid epidemic?
Dr. Gottlieb. I do not know what group you are talking
about, but I have had conversations with individual Members
about the opioid epidemic. I met with probably 60 Members in
the run-up to my confirmation process. I would say that this
issue came up in most of those meetings. Since I have been in
this position, we have taken a lot of additional meetings with
both Democratic and Republican Members in the House and the
Senate, and this issue comes up a lot.
So I have consulted widely on this issue, including with
you, Senator, and I appreciate all the dialogue I have had with
Congress.
Senator Merkley. The group that I am referring to are the
13 Republican Senators who have been delegated the authority to
prepare a bill to be brought to the floor, one that will have
no public input. That is the group. Has that group asked you to
come and share your expertise on this?
Dr. Gottlieb. I have had no dialogue with any group working
on legislation as a group, no.
Senator Merkley. Well, I imagine, just knowing your
background, that you probably share my concerns that such
issues get the insight from experts and also that the public
has a chance to weigh in, so that we get, in this ``We the
People'' Republic, a full opportunity to make sure we get kind
of policy right, if you will, when it has an impact on so many
people.
Dr. Gottlieb. Thank you, Senator.
Senator Merkley. Thank you.
Senator Hoeven. Senator Collins.
Senator Collins. Thank you, Mr. Chairman.
Mr. Chairman, I have an opening statement that I would
request be submitted for the record.
Senator Hoeven. Without objection.
[The statement follows:]
Opening Statement of Senator Susan M. Collins
Thank you Chairman Hoeven and Ranking Member Merkley for holding
this important hearing to talk about the fiscal year 2018 Budget
request for the Food and Drug Administration. And thank you,
Commissioner Gottlieb, for testifying before the Subcommittee today.
The volume and complexity of the FDA's work have grown considerably
in recent years. Charged with assuring the safety and efficacy of human
and animal drugs, biologics, and medical devices--FDA must work to
strike the appropriate balance between encouraging innovation and
timely access while protecting the public's health and safety. FDA also
regulates cosmetics, tobacco, and products that emit radiation. And, it
is responsible for ensuring the safety and security of our nation's
food supply.
FDA's core mission is critical to the lives of American families
and seniors, and I will plan to touch on two issues during questions
that are particularly top of mind--the opioids crisis and eliminating
barriers that unduly prevent generic drug competition.
With such an extremely broad mandate, I look forward to hearing
from the Commissioner about the agency's priorities.
Senator Collins. Thank you.
HEALTHCARE PROVIDER EDUCATION--OPIOID PRESCRIPTION
Doctor, we have previously discussed the need for improved
health care provider education with regard to the prescribing
of opioids. Medicaid beneficiaries are prescribed pain
relievers at a higher rate than those with other sources of
insurance. And they also, not surprisingly, given that higher
rate, have a higher risk of overdose from prescription opioids,
heroin, and fentanyl.
What opportunities do you see for greater collaboration
among the FDA, CMS, State Medicaid directors, medical
societies, and other parties, in order to address this problem
of appropriate prescribing of opioids?
Dr. Gottlieb. I appreciate the question, Senator. I would
also add the DEA (Drug Enforcement Agency) to that, because
there might be things we can do in conjunction with our
partners at the Justice Department.
As part of the steering committee that we have set up, we
are currently having discussions about what steps we can take
to improve provider education, and maybe take a look at
packaging as well, as a way to help make sure prescriptions are
more appropriately matched to the clinical circumstances for
which they are being written.
I do not want to get too far ahead of that process, other
than to say that this is something that is at the top of the
list of things that we are looking at right now. This includes
what additional steps we can do under our current authorities,
both through the risk management plans that we currently
promulgate in conjunction with opioids, the approval of opioids
and other narcotics on the schedule of drugs, as well as in
partnership potentially with the DEA. DEA obviously has
authority to potentially look at certain requirements as part
of the process for giving a DEA license to individual
practitioners.
Senator Collins. Thank you.
DRUG PRICES
As you know from our numerous discussions, the Senate Aging
Committee last year undertook a major investigation examining
the explosion in prices of off-patent prescription drugs for
which there is no generic equivalent. In one case, a drug was
purchased by a company that played absolutely no role in
developing the medicine, and then raised its price by 5,000
percent overnight.
One of the problems that we found is that these companies
warded off competition from generic companies by putting their
drugs in closed distribution systems or in specialty
pharmacies. The intent in doing so was to delay access or even
block access to a sufficient quantity of the brand name drug to
do the bioequivalence studies that the FDA requires.
These abuses are serious and contribute to the cost
increases that we are seeing. By one estimate, in 2014, such
abuses resulted in increased costs to consumers of $5.4 billion
per year.
I have had extensive conversations in hearings and
privately with Dr. Janet Woodcock about this problem. She has
testified that FDA has done 150 referrals to the Federal Trade
Commission (FTC) to take a look at this anticompetitive process
without any success. She suggested that there needs to be a law
change in order for the Risk Evaluation and Mitigation
Strategies (REMS) system not to be abused.
I know that you have testified before the House
Appropriations Subcommittee and noted your concern about this
type of anticompetitive behavior. Should Congress revise the
REMS law, as suggested by Dr. Woodcock, since there is only so
much that FDA can do now about the problem?
Dr. Gottlieb. Well, I appreciate the question, Senator.
I know there is some legislation that Congress is currently
contemplating in this regard. We would be happy to provide
technical assistance on that. I think we already have. I
believe there are things we can do within the scope of our
current authorities through administrative action to address
this challenge.
There are two different challenges here. One is the REMS,
which is sometimes misused as a way to block the ability of
generic companies to get access to the samples they need in
order to develop a generic drug. It takes between 1,500 to
3,000 actual doses in order to develop a generic equivalent.
The other issue is things are sometimes embedded in the
contracts with the distributors or the specialty pharma
companies that make it hard for the distributors or the
specialty pharma companies to sell the drugs to the generic
companies when they try to purchase them at fair market value
in the marketplace.
So there are two different issues. Some we can solve, or
address within the scope of FDA, and some might require us, if
we want to try to address it administratively, to partner with
Medicare, where there might be opportunities to do that.
We can identify, to your point, the situations, the
circumstances, where we believe the generic companies are not
able to get the access to the doses and make referrals. We
cannot fully address some of the commercial restrictions that
prevent them from getting access to those doses. But we could,
in partnership with other agencies.
Senator Collins. Thank you.
Senator Hoeven. Senator Tester, I understand that you are
deferring to Senator Leahy. Is that correct?
Senator Tester. I was not going to, but go ahead.
[Laughter.]
Senator Leahy. There has to be some advantage of being vice
chairman of this committee.
Senator Hoeven. Senator Leahy.
Senator Leahy. Thank you. I will be brief. I will put my
full statement in the record.
[The statement follows:]
Prepared Statement of Senator Patrick Leahy
Thank you, Chairman Hoeven and Ranking Member Merkley, for holding
this hearing today to examine the President's fiscal year 2018 budget
proposal for the Food and Drug Administration. And thank you,
Commissioner Gottlieb [got-LEEB], for joining us here today. I thank
the Chair and Ranking Member for giving me the opportunity to offer a
few opening remarks.
The Food and Drug Administration has an enormous responsibility to
ensure the safety of all Americans. From our food supply, to
pharmaceuticals and cosmetic products, the FDA must have the resources
it needs to effectively review these products. That is why I was so
disappointed and alarmed to see the woefully inadequate budget
submission from the FDA, which, when excluding the user fee proposal
and the mandatory funding through the 21st Century Cures Act, includes
a 34 percent cut. The cuts include $119 million from monitoring food
safety and a $55 million reduction in medical product safety. In my
many years in the Senate, I have heard repeatedly that the FDA needs
more, not less, resources to adequately fulfill the agency's mission.
Commissioner Gottlieb, I am curious to hear from you about your
justification for how these proposed reductions will not adversely
impact Americans' health and safety, and help bolster consumer
confidence in our Nation's food supply.
The FDA also has a responsibility to approve new drugs, including
generic alternatives. With the price of prescription drugs on the rise,
crippling households and seniors on a fixed income, I will also want to
discuss with you ways to help ensure more competition in the
pharmaceutical market. Commissioner Gottlieb, as a physician and
someone who has worked in the industry for many years, I am interested
in hearing your thoughts.
Finally, Dr. Gottlieb, I would be remiss if did not make one thing
very clear: President Trump's abysmal budget assumes savings from the
repeal of the Affordable Care Act, and deep cuts to the Medicaid
program. As we talk today about how to increase access to safe,
affordable--life- saving--prescription drugs, we cannot forget the
millions of Americans that would be left without health insurance if
the Affordable Care Act is repealed. Whatever Senate Republicans are
crafting behind closed doors, we cannot--and should not--accept any
proposal that strips essential and affordable healthcare from millions
of Americans.
As I have said in other Appropriations hearings, as Vice Chairman
of this Committee, I will work to craft a budget that truly puts
Americans first. I look forward to working with the other members of
this Committee, on both sides of the aisle, who I believe also want to
fulfill that goal.
Senator Leahy. Commissioner, I am glad you are here today.
I appreciate you being here.
FDA'S FISCAL YEAR 2018 BUDGET SUBMISSION
I was disappointed to see I think a woefully inadequate
budget submission from the FDA. When you exclude the user fee
proposal and mandatory funding to the 21st Century Cures Act
that is about a 34 percent cut, $119 million is cut from
monitoring food safety, $55 million reduction in medical
product safety.
I have been here for over 40 years. I have heard repeatedly
the FDA needs more money, not less. This is the first time I
have seen such a huge cut, especially with the price of
prescription drugs on the rise, crippling opioids, and so
forth.
So I think the budget that the President has submitted is
abysmal. It assumes deep cuts of the Medicaid program. It
assumes repeal from the Affordable Care Act. It is kind of
saying the check is in the mail. But I would really like to
have a budget that puts Americans first, not political slogans
first.
DRUG PRICES AND CREATES ACT
But I want to follow up on something Senator Collins was
saying about drug prices and the Creating and Restoring Equal
Access to Equivalent Samples (CREATES) Act. We care, all of us,
I do not care whether you are Republicans or Democrats, about
the high cost of prescription drugs. I know that some of the
pharmaceutical companies have used inappropriate delay tactics
to limit the ability of generic competitors to enter the
market. Some will not give the generics the samples needed for
testing.
I proposed a bill joined by Republicans and Democrats both
here in the Senate and in the House, the CREATES Act, which
would deter pharmaceutical companies from blocking cheaper
generic alternatives.
So can you explain again the role access to these samples
plays in market competition?
Dr. Gottlieb. Thanks for the question.
The bottom line is that there is no question there are
places where companies do take advantage of rules meant for one
purpose as a way to gain commercial advantage. What I want to
do is make sure we have a framework in place that makes it hard
to do that. I think that when we put in place rules for, in
this case, a risk management plan trying to address drug safety
questions, we do not want to see those rules misused as a way
to try to forestall access to generic competition that Congress
intended under the laws that exist right now.
So I do not want to be playing whack-a-mole with companies
either. I want to have in place a consistent framework and a
consistent set of rules that prevent these kinds of abuses.
I think we can achieve that. I would be happy to work with
Congress on the legislation that you are contemplating. But I
also think that there are things that we can do
administratively through our current authorities. That is where
I am going to be focusing my attention.
Now the REMS is not the only place where this kind of
potentially anticompetitive behavior goes on. We are going to
be looking across all those places. We are going to be
announcing a public meeting very soon to solicit input from the
public on where other people believe there are practices that
could be forestalling access to generic competition, where
branded companies might be taking advantage of certain rules
that we could potentially address through our existing
authorities.
But this is an important focus of mine.
Senator Leahy. You commented recently the FDA is evaluating
whether to waive the law's existing preference for brands and
generics for REMS. And Janet Woodcock, as mentioned already,
testified that a statutory change might be needed.
Which is better for you, statutory, or can you do it from
regulatory?
Dr. Gottlieb. Well, I have been in the job for about 6
weeks now, and I have spent a lot of my time looking at what I
can do, and starting to put those actions in motion. So I have
spent far less time looking at potential statutory solutions.
I think you just referred to the single, shared REMS, where
branded companies and generic companies are obligated to try to
negotiate a single REMS to try to reduce burdens on providers.
And the question there becomes, at what point do we step in and
say, ``You know what? The negotiations have gone on for long
enough, and we are going to allow the generics to move forward
with their own REMS program.''
That is a decision we could make. We have to develop the
administrative record to do that. That is something where we,
through our current policy, can help address a potential stall
tactic.
I think if we put in place a policy signifying that we were
willing to step in and say, ``You know what? These negotiations
have gone on long enough. We are going to allow the generic
company to move on their own,'' I think maybe companies might
reach agreement quicker than they are today.
Senator Leahy. Thank you.
Thank you, Mr. Chairman. I think you are going to find both
Republicans and Democrats are going to be interested in working
with you to do that, because these prices are getting out of
control.
Senator Hoeven. Senator Rubio.
Senator Rubio. Thank you.
Thank you, Dr. Gottlieb, for being here.
PREMIUM CIGARS
As you know, as we discussed for your confirmation, there
are a number of small businesses in Florida that have been
making premium hand-rolled cigars for generations. The industry
is at stake.
Last year, under the previous administration, they
finalized a rule that would require premium cigars, not the
stuff you get from behind the counter, but the premium ones, to
regulate the manufacture, import, packaging, labeling,
advertising, promotion, sale, and distribution, at the premium
cigar level.
So it is already illegal to sell tobacco products to anyone
under the age of 18, which I think addresses the underage
smoking issue. Plus, the premium cigar market really is not
marketed toward that. It is a different market altogether.
Are there any plans to reevaluate the inclusion of premium
hand-rolled cigars from this rule, as was proposed under the
preliminary rule?
Dr. Gottlieb. Thanks for the question.
We are currently looking at aspects of the rule. As you
know, there was a 3-month delay in implementation of certain
compliance dates announced before I arrived at FDA. We are
coming up on the end of that delay.
Whatever we do in this regard is going to need to be
science-based, of course. But we are cognizant of the
challenges faced by small businesses. I also understand that
there are a number of legislative measures to exempt premium
cigars. If Congress were to act, we would be happy to work with
legislators to mitigate any unintended consequences of these
measures.
I do not want to comment too specifically, given that there
is pending litigation right now around this issue, other than
to say that I understand the concerns. You and I have had the
opportunity to talk about them on a few occasions now. I do
understand the concerns of the small businesses that make
premium cigars, Senator.
Senator Rubio. Yes. And just for those who might be
watching, we are talking about the premium cigars, or what the
name implies, a premium cigar.
Dr. Gottlieb. That is right.
Senator Rubio. I mean, it is an expensive product marketed
toward a very specific audience. If it is science-based, I
think it will show, as we have seen repeatedly, that it is
really not a product that it is marketed to people under age,
or the like.
PEDIATRIC CANCER TREATMENTS
Real quick, I really appreciate the assistance FDA has
provided to me and to Senator Bennet on our legislation, RACE
for Children, to close the gap on cancer treatments that exist
between adults and children. I was just wondering if you could
provide some background as to why the FDA initially requested
legislation to close this loophole after years of trying to
encourage development of pediatric cancer treatments only
through the Best Pharmaceuticals for Children Act?
Dr. Gottlieb. Well, I know the legislation, Senator. I
assure you, we want to do everything we can to try to make
products available to pediatric patients, particularly
pediatric patients who have rare diseases where current
available therapy might not fully address their clinical needs.
I know that we have provided technical assistance with respect
to this legislation and have worked with your office and will
continue to do that.
Senator Rubio. Just again, as an aside, what Senator Bennet
and I are aiming at is, right now it is basically on an
incentive system, where we are trying to incentivize companies,
in every one of their trials, to have a pediatric component.
Unfortunately, it is not working.
They are not doing that enough because sometimes the target
audience is not big enough for them, or the target potential
patient mix is not large enough for them to be encouraged to do
that.
So our hope is to be able to drive more of that. I think
everyone has been impacted in their own families by pediatric
cancer, and it is critically important that we develop new
treatment options at that level, because when it strikes a
family, it is devastating.
So again, we thank you for your cooperation and look
forward to continuing to interact with you.
Thank you, Mr. Chairman.
Senator Hoeven. Senator Tester.
TOBACCO PRODUCTS
Senator Tester. Thank you, Mr. Chairman. I am glad I stuck
around for Marco's questions, because the fact is, I think he
is absolutely right on the premium cigars. I would tell you, on
the other side, there are companies that are marketing their
tobacco products to kids, and, hopefully, you can do something
about that, too, because it is ridiculous. It smells bad.
You do not need to comment on that. I just hope you would
pursue that.
GENERIC DRUGS
Would you agree that expediting the review of high-need
generic drugs should be a priority for the FDA?
Dr. Gottlieb. We currently do expedite various categories
of what I think you and I might agree are high-need generic
drugs. In fact, we just announced we are going to be
prioritizing the review of drugs that do not face any
competition. There are 180 generic drugs right now, or drugs
that are off-patent.
FISCAL YEAR 2018 BUDGET
Senator Tester. And the chairman talked about 1,000
vacancies. CBO has estimated that, if we are going to do this,
it could be as many as 500 additional employees over the next 5
years. At $60,000 a year a pop, that is a fair amount of money.
I think it is the right thing to do. I think it is an
important thing to do. But how, under this budget, are you
going to be able to accomplish that?
Dr. Gottlieb. Well, look, as the chairman noted, I was not
involved in the formulation of the budget.
Senator Tester. I got it.
Dr. Gottlieb. I would have to, obviously, make it work, if
the budget were to pass as it was proposed.
These are challenging budgetary times. We are going to have
to figure out ways to do more with less. We have tried to
target the cuts that this budget does distribute. This budget
is an overall increase, but there will be certain cuts
distributed under the budget, because of the way the money is
allocated with the emphasis towards the user fees.
We will have to try to, and we have tried to allocate the
reductions to places of lower priority. But in an agency where
there is an important mission, and a lot of what we do is
important, sometimes it is challenging to find those areas. We
have tried to do the best we can to identify them.
Senator Tester. I think this is really important. I think,
as many of the people on this committee have talked about,
prescription drugs is a huge driver in health care costs. They
are huge. We need to make sure they are safe. But the backlog,
whether it is with generics or orphan drugs, as you talked
about in your opening statement, is critically important.
I love to do more with less, but we have to do a lot more
with a third less, and I just do not see how that works. You do
not have to justify this, because I have heard the ``more with
less'' from a lot of different folks.
But the truth is, in the end, if we are going to be able to
hold you accountable and you come back in and say, ``You know
what? I just did not have the manpower to do it,'' that is our
job here, to make sure you have the manpower to do it. And your
recommendations are really important when it comes to manpower.
And I get it. There is fat in every agency. And there are
priorities in every agency. So I would hope that you would be
honest with us and say, ``You know what? This is a big issue.''
Because I think the generic drug thing is a huge issue. And to
get these high-need generics out I think is something we should
all try to achieve. But you also need to be realistic on the
manpower and the budget that it takes, because it is important.
Dr. Gottlieb. Well, Senator, as you are trying to think
through what you think are the appropriate allocations to help
support the agency's mission, you can rest assured I would be
happy to work with you to provide you any advice you need.
Senator Tester. Not only me but this entire subcommittee on
your budget. I would love to get your recommendations. Because,
like I said, I think this is an incredible driver in health
care, and we need to figure out ways to do reduce costs.
GENERIC DRUGS
I think generics have been one of the bright lights over
the last 15, 20 years, however long they have been around. And
so when we see hedge fund folks buying up prescription drug
companies, doing the kinds of things Senator Collins talked
about, we have to figure out ways to block that, whether it is
through your agency or some other agency.
Dr. Gottlieb. Right. We are also going to contemplate this
very issue in the GDUFA reauthorization that is before Congress
right now.
Senator Tester. All right. Thank you.
IMPORTATION OF PRESCRIPTION DRUGS
Now I want to talk about importation of prescription drugs.
There have been all sorts of efforts over the last 20 years
that I know of, from Montana hauling busloads of folks up into
Canada to buy prescription drugs. There have been bills put
forth here for reimportation, some of them good, some of them
not so good.
Do you think Americans should be allowed to import drugs
from other countries?
Dr. Gottlieb. Well, this question has been put to FDA
Commissioners across both Republican and Democratic
administrations, and there is a certification. It is currently
legal to have drug reimportation, as long as the Secretary of
Health and Human Services can certify the safety of the drugs
that are coming in. That legislation has existed through both
Republican and Democratic administrations.
I have not taken a fresh look at this question. I have not
been asked to. I am happy to look at it. But I would remind the
committee that FDA Commissioners dating back to when I was last
at the agency about 15 years ago have not been able to make
that certification.
Senator Tester. Have not been able to make the
certification of safety, of it being a safe product reimported?
Dr. Gottlieb. Of the ability to put in place the proper
regulatory architecture that, if you have reimportation of
drugs, to make sure that the chain of custody can be
guaranteed, that you are actually getting a drug that was
manufactured by legitimate----
Senator Tester. Right. I think that is a legitimate
concern. I think there is also a legitimate concern of
Americans being gouged for their prescription drugs. I am
almost to the point where I think we may be subsidizing other
countries for their cheaper prescription drugs.
I do not know that to be a fact. But the truth is that when
I go into Montana, and I think the same can be said for North
Dakota or Oregon, we hear about this issue a lot.
Dr. Gottlieb. And I am trying to take steps to address it,
Senator, as you know, through what we are doing to try to bring
more competition onto the market.
I think you are absolutely right. I do not think it is a
debatable proposition. We are subsidizing drugs for other
countries through the high prices we pay here to support the
research and development, and we need to address that, too. I
am not the trade representative, obviously, but I am trying to
do all I can through within the context of my agency.
Senator Tester. Thank you for your answers.
Thank you, Mr. Chairman, for allowing me to go over time.
Senator Hoeven. Absolutely, Senator.
ADDITIONAL COMMITEE QUESTIONS
We have votes that have been called, so at this point, we
are going to adjourn the hearing.
I do want to thank you, Dr. Gottlieb, for being here today.
I appreciate you not only being here but your good work.
For members of the committee, any questions that you want
to submit for the hearing record should be turned into
subcommittee staff within 1 week, which is Tuesday, June 27th.
We would appreciate if we could have a response back from you,
Doctor, within 4 weeks from that point.
Questions Submitted by Senator John Hoeven
alzheimer's
Question. Alzheimer's disease is a major public health threat to
our country. Patients and providers currently have few impactful
therapeutic options when fighting Alzheimer's disease. Costs associate
with Alzheimer's disease have been estimated to be $236 billion, per
year. And unless we get new medicines to slow the progression of the
disease, cure it, and even one day prevent its onset, those annual
costs are projected to top $1 trillion by 2050. In no uncertain terms,
we urgently need new FDA-approved drugs for Alzheimer's disease. To
achieve this important goal, FDA must ensure that the right regulatory
policies and processes are in place to encourage and facilitate
innovation. The status quo with regard to Alzheimer's disease must
change.
What is the FDA's policy of requiring clinical trials for new
Alzheimer's medicines to meet two endpoints, or co-primary endpoints--
one related to cognition and one related to function?
Answer. As stated in FDA's 2013 draft guidance on Alzheimer's
disease (AD), clinical trials in the dementia stage of AD should use a
co-primary outcome measure approach in which a drug demonstrates
efficacy on both a cognitive and a functional or global assessment
scale. The measures of neuropsychological performance are sensitive,
and on their own, these measures may not be clinically meaningful.
Thus, co-primary endpoints are intended to ensure that the drug's
observed effect on cognition is clinically meaningful. Before the onset
of overt dementia, however, milder functional and/or global impairments
are more challenging to assess accurately, especially for patients
early in the spectrum of the illness. Accordingly, as expressed, in
FDA's 2013 draft guidance on AD, openness to the use of a single
primary endpoint, such as the Clinical Dementia Rating--Sum of Boxes
score, that integrates both cognition and function in a single
assessment. Additional assessments may also be appropriate for use in
clinical trials, and FDA is open to considering other assessments that
integrate both cognition and function.
Question. From your new position as FDA Commissioner, do you
believe the co-primary endpoint standard is appropriate? Have you
asked, or will you ask your staff to look into this issue?
Answer. As noted in response to Question #1, and as stated in FDA's
2013 draft guidance on Alzheimer's disease, clinical trials in the
dementia stage of AD should use a co-primary outcome measure approach
in which a drug demonstrates efficacy on both a cognitive and a
functional or global assessment scale. The measures of
neuropsychological performance are sensitive, and on their own, these
measures may not be clinically meaningful. Thus, co-primary endpoints
are intended to ensure that the drug's observed effect on cognition is
clinically meaningful. Before the onset of overt dementia, however,
milder functional and/or global impairments are more challenging to
assess accurately, especially for patients early in the spectrum of the
illness.
Accordingly, as expressed, in FDA's 2013 draft guidance on
Alzheimer's disease, openness to the use of a single primary endpoint,
such as the Clinical Dementia Rating--Sum of Boxes score, that
integrates both cognition and function in a single assessment.
Additional assessments may also be appropriate for use in clinical
trials, and FDA is open to considering other assessments that integrate
both cognition and function. The Agency is prepared to approve new
treatments for Alzheimer's disease that are supported by substantial
evidence of effectiveness on clinically meaningful outcomes. FDA works
closely with drug sponsors to help them develop this evidence.
opioids
Question. The opioid epidemic continues to plague this country.
Both the CDC and World Health Organization recommend treatment for pain
starting with non-opioids prescription medication before using opioids.
What is FDA doing to ensure non-opioid prescriptions receive a
prioritized and timely review?
Answer. FDA's prescription drug efforts are part of a larger,
coordinated Departmental strategy around addressing the opioid crisis.
HHS strategy includes five priority areas:
--Improving access to prevention, treatment, and recovery services,
including the full range of medication-assisted treatments;
--Targeting availability and distribution of overdose-reversing
drugs;
--Strengthening our understanding of the crisis through better public
health data and reporting;
--Providing support for cutting edge research on pain and addiction;
and
--Advancing better practices for pain management.
The FDA continues to support efforts to better understand the
treatment of pain, especially as it relates to balancing the need to
effectively treat pain with the public health crisis related to opioid
use disorder and overdose. Currently, there are FDA-approved drug
treatment options, opioids and non-opioids, available for the
management of pain.
FDA has a number of programs, such as Fast Track and Breakthrough
Designation, which are intended to facilitate the development and
expedite the review of products that, for example, meet unmet medical
needs. Novel non-opioid medications with the potential to provide pain
relief in situations in which opioids often are used could be
appropriate candidates for such programs. FDA is also working with the
National Institute on Drug Abuse to encourage the development of non-
addictive pain medication.
Question. How many non-opioid treatments are currently under review
by FDA and are scheduled for review in the next 12-24 months?
Answer. Although FDA is unable to discuss the substance of any
matters that may be pending before the Agency, FDA can assure you that
the Agency understands the value in and supports the development of new
treatment options for pain. In fact, there are a number of novel non-
opioid products under development.
Question. What if anything can be done by FDA to ensure more
prescription non-opioid medications are available to treat pain?
Answer. Currently, both opioid and non-opioid FDA-approved drugs
are available for the management of pain. There are also medical
devices indicated to treat specific types of pain. FDA understands the
value in new treatment options for pain, and continues to work with the
medical device and drug industries to explore new options for patients
in pain, especially options that have improved safety profiles and are
less likely to result in addiction or abuse. FDA has a number of
programs, such as Fast Track and Breakthrough Designation, which are
intended to facilitate the development and expedite the review of
products that, for example, meet unmet medical needs. Novel non-opioid
medications with the potential to provide effective pain relief in
situations in which opioids often are used could be appropriate
candidates for such programs.
FDA is also working with the National Institutes of Health's (NIH)
National Institute on Drug Abuse to encourage the development of non-
opioid pain medications, and has been involved in discussions with NIH
in a series of meetings to facilitate development of non-addictive pain
treatment. In addition, FDA looks forward to a future in which
substantially all opioid medications are less susceptible to abuse than
the conventional formulations that dominate the market today.
antibiotic resistance
Question. I'm concerned about superbugs--the reports coming out of
Asia and other countries is that much of their food-producing livestock
is ridden with multi-drug resistant bacteria. FDA and our farmers have
eliminated high-value antibiotics from our farm systems, but others
haven't done much at all. There's a lot we can do to deter the
development of antibacterial resistance, but it seems the cat is out of
the bag and we really need a strong and ongoing pipeline of new drugs.
What can we do to develop the next generation of antibiotics?
Answer. Antimicrobial resistance (AMR) is one of the most serious
threats to global health in this century. FDA has taken a leading role
in addressing this critical threat by working with key stakeholders to
identify new approaches to stimulate antibacterial research and
development, including by streamlining clinical trial designs;
enhancing surveillance through systems such as the National
Antimicrobial Resistance Monitoring System; and actively participating
in global efforts to reduce resistance.
Recent FDA efforts include administering the incentives available
to sponsors under the Generating Antibiotic Incentives Now (GAIN) Act.
FDA has granted 136 Qualified Infectious Disease Product (QIDP)
designations, including approximately 71 designations for novel drugs.
FDA has approved ten drug products with QIDP designation. Moreover, FDA
has written, reviewed and revised a number of guidance documents to
provide clarity and predictability on recommended trial designs and
development pathways. The Agency has also engaged the scientific
community through public meetings, partnerships, and regulatory science
research.
Efforts to strengthen the antibacterial drug pipeline must also
address significant economic and scientific challenges. The President's
Advisory Council on Combatting Antibiotic-Resistant Bacteria (PACCARB)
recently finalized their report with recommendations addressed to
Secretary Price for incentivizing the development of vaccines,
diagnostics, and therapeutics/anti-infectives for both human and animal
health to address the issues surrounding antibiotic resistance. HHS'
Biomedical Advanced Research and Development Authority (BARDA) and the
National Institute of Allergy and Infectious Diseases (NIAID) have
helped to launch important, new efforts in this space, such as the
Combating Antibiotic Resistant Bacteria Biopharmaceutical Accelerator
(CARB-X), a public-private partnership that provides funding to
accelerate the preclinical discovery and development of antibacterial
drugs.
As you noted, FDA has been actively working with various
stakeholders, in the animal drug context, to implement judicious use
policies to promote antimicrobial stewardship. Such efforts include
working with animal drug sponsors to transition medically important
antimicrobials used in the feed or water of food-producing animals to a
marketing status requiring veterinary oversight, and eliminating the
use of these products for production (such as growth promotion)
purposes. Implementation of this transition process was completed in
January 2017 marking an important step forward in promoting
antimicrobial stewardship in animal agriculture.
In addition, strengthening the pipeline for antimicrobials or
alternatives to antimicrobials intended for use in food-producing
animals by developing appropriate incentives (including public-private
partnerships) to spur drug and vaccine development is also important to
ensure antimicrobial therapy is available to meet the current and
future needs of these animals and to provide a safe, wholesome food
supply.
foreign high risk inspections
Question. Over the past several years the Committee has provided
FDA with additional resources to develop a targeted, risk-based, and
efficient inspection model for high-risk establishments for onsite
verifications.
Given the challenges of expanded geography, constantly expanding
inventory of foreign exporters, and finite budgetary resources, how
will FDA leverage third party site verification services to complement
FDA foreign site planning and inspection activities?
Answer. In fiscal year 2016, FDA spent the bulk of the money
provided to Office of Global Regulatory Operations and Policy (GO) on
commercial foreign onsite verification reviews in support of expanding
global coverage. FDA contracted with Dun & Bradstreet (D&B) to provide
site verification information on specific facilities. D&B performed
site verifications across all of the regulated commodities. With
respect to medical products, the remaining funds were used in support
of the pharmaceutical GMP mutual recognition initiative and enhancing
the Center for Drug Evaluation and Research (CDER) site selection
model. Regarding foods, the remaining funds were used to enhance the
process and consolidate the responsibilities of foreign food inspection
planning in the Office of Regulatory Affairs.
In fiscal year 2017, Office of Global Regulatory Operations and
Policy, (GO) expanded the number of foreign onsite verifications and
facility data delivered through D&B for foreign medical device and food
establishments. These specific commodities were chosen due to the value
and impact that commercial onsite verifications provide these programs.
For the pharmaceutical program, GO has worked with CDER to issue grants
for analysis that will support the development of quality scorecards
for pharmaceutical facilities and products. A quantitative
characterization of the state of quality can enhance oversight by
improving the site selection model's identification of high-risk
foreign facilities.
As part of the reauthorization of the Generic Drug User Fee
Amendments (GDUFA) in the FDA Reauthorization Act (FDARA), FDA
committed to working on a guidance explaining the risk-based site
selection model as well as outreach activities to better inform foreign
regulatory counterparts about our model.
______
Questions Submitted by Senator Mitch McConnell
opioids
Question. Given your public statements, it seems that the FDA and
Congress agree that combating the opioid epidemic remains a top
priority. How do you plan to coordinate FDA's prescription drug abuse
efforts with other agencies? Specifically, how will the FDA work with
the NIH to accelerate innovative cures and non-opioid pain therapies to
the market faster?
Answer. FDA understands the value in and supports the development
of new treatment options for pain, and is committed to continue working
with our colleagues at the National Institutes of Health, including the
National Institute on Drug Abuse, and other government agencies to
explore new options for patients in pain, especially options that are
not addictive. Since June 2017, FDA has collaborated with NIH via a
series of three meetings to facilitate development of non-addictive
pain treatment and treatment for opioid addiction and overdose
prevention/reversal. Also, in May 2017, FDA hosted a public meeting on
pain management with participation from many Federal agencies to
discuss, among other things, non-addictive pain treatment options. FDA
notes that the Agency's prescription drug efforts are part of a larger,
coordinated Departmental strategy around addressing the opioid crisis.
Question. The FDA Opioid Action plan issued in February 2016
included a commitment to collaborate with advisory committees. FDA
stated that after considering advisory committee recommendations and
reviewing existing requirements, the agency was inviting affected
opioid manufacturers to a meeting to inform them of the agency's
intention to require a Risk Evaluation and Mitigation Strategy (REMS)
for immediate-release (IR) opioids. This meeting was held on January
25, 2017. Where is the agency in the process of expanding REMS to
include IR opioids? What steps has the agency been taking to consult
and work with stakeholders on the expansion of the REMS?
Answer. On July 10, 2017, FDA announced that it intends to update
and modify the existing Risk Evaluation and Mitigation Strategy (REMS)
for extended-release/long-acting (ER/LA) opioid analgesics, and for the
first time, to include immediate-release (IR) opioid analgesic products
in the modified REMS program. FDA expects this action to take place in
the coming weeks. The modified REMS is expected to include revisions to
the existing FDA Blueprint for prescriber education which describes the
content that must be covered in an educational program for it to be
considered REMS-compliant.
FDA sought input from relevant stakeholders in a variety of
settings on a number of issues related to the ER/LA Opioid Analgesic
REMS, including at an Advisory Committee meeting in May 2016, during a
public workshop in May 2017, and through establishment of a docket
announced in a Federal Register notice in May 2017.
On May 3 and 4, 2016, FDA convened a joint meeting of the Drug
Safety and Risk Management (DSaRM) Advisory Committee and the
Anesthetic and Analgesic Drug Products Advisory Committee (AADPAC) to
discuss whether the ER/LA Opioid Analgesics REMS assures safe use,
whether it is unduly burdensome to patient access to the drugs, and
whether it (to the extent practicable) minimizes the burden to the
healthcare delivery system. FDA also sought input on possible
modifications to the ER/LA Opioid Analgesic REMS, including expansion
of the scope and content of prescriber training and expansion of the
REMS program to include immediate-release (IR) opioid analgesics.
On May 9 and 10, 2017, FDA held a public workshop to seek input on
how to best support prescriber and other healthcare provider education
on appropriate pain management and opioid analgesic prescribing. This
meeting convened government experts and representatives from state
licensing boards, professional associations, healthcare systems,
patient groups, and other stakeholder groups involved in the challenges
of improving pain management while addressing the opioid epidemic.
In addition to the panel discussions at the meeting, FDA is also
considering comments submitted to two public dockets which closed on
July 10, 2017. The first docket asked for public comment on how to best
support prescriber and other Health Care Provider education on
appropriate pain management and opioid analgesic prescribing. FDA has
received more than 250 comments to the docket. The second docket under
review considers modifications to the existing FDA Blueprint for
Prescriber Education for Extended-Release and Long-Acting Opioid
Analgesics in light of recommendations from the May 2016 Advisory
Committee meeting. The draft revisions to the Blueprint would broaden
the Blueprint to incorporate information on pain management, including
the principles of acute and chronic pain management, non-pharmacologic
treatments for pain, and pharmacologic treatments for pain (both non-
opioid analgesic and opioid analgesic). More than 680 comments were
submitted to this docket.
Question. Also included in the action plan was the commitment to
expand access to abuse deterrent formulations (ADF) to discourage
abuse. Beyond the FDA's guidance issued in April 2015 related to the
types of studies to assess abuse deterrence efficacy and suggested
labeling, what other actions has the FDA taken to expand access to ADF
or assist manufactures in the development and subsequent approval
process for bringing these formulations to market?
Answer. FDA's support of opioid products with abuse-deterrent
formulations (ADF) is one of a number of steps to which FDA has
committed which is focused on policies aimed at reversing the opioid
epidemic while still providing patients in pain access to effective
relief. To date, the Agency has approved ten opioids with labeling
describing properties expected to deter (though not completely prevent)
one or more routes of abuse. The 2015 final guidance for industry,
Abuse-Deterrent Opioids--Evaluation and Labeling, provides information
on abuse-deterrent formulations for applicants seeking such approval.
Given the lower cost, on average, of generic products, the
availability of generic ADFs is an important step toward balancing the
need to reduce opioid abuse with helping to ensure access to
appropriate treatment for patients in pain. To that end, in March 2016,
FDA issued draft guidance entitled General Principles for Evaluating
the Abuse Deterrence of Generic Solid Oral Opioid Drug Products. The
draft guidance, when finalized, will provide recommendations regarding
studies that should be conducted to show that a generic opioid is no
less abuse-deterrent than a brand name opioid with abuse-deterrent
labeling with respect to all potential routes of abuse.
FDA continues to engage advisory committees, as appropriate, to
provide advice on novel or challenging issues that arise in connection
with the development and evaluation of abuse-deterrent opioids. In July
2017, FDA held a public workshop with expert panel members and
interested stakeholders about the challenges in using currently
available data and methods for assessing the impact of opioid
formulations with properties designed to deter abuse on opioid misuse,
abuse, addiction, overdose, and death in the post-market setting.
FDA remains committed to working with sponsors and other
stakeholders to support the development of and access to opioid
formulations with properties that could lead to a meaningful reduction
in prescription opioids misuse and abuse compared to the conventional
formulations.
Question. FDA requires manufacturers of extended release (ER/LA)
opioids to make available REMS-compliant training for prescribers of
ER/LA opioid analgesics, yet physician participation in these training
programs is voluntary. At the same time, FDA is holding manufacturers
of ER/LA opioids accountable to industry-negotiated performance goals
that manufacturers are struggling to meet. As part of the REMS, the ER/
LA opioid manufacturers agreed to specific performance goals amounting
to 160,000 active prescriber participants in the voluntary REMS-
compliant training programs by March 2016. However, the most recent
report showed that only 66,000 prescribers completed training, meaning
the participation rate amounted to 41 percent of their goal. Beyond
updating the ER/LA opioid analgesics REMS, how will the FDA encourage
prescribers of these drugs to participate in the training?
Answer. Under the current ER/LA REMS, companies are required to
make training available to healthcare providers that prescribe
extended-release/long-acting (ER/LA) opioid analgesics. The companies
may meet this requirement by working with accredited continuing
education providers who offer training to prescribers; however,
prescribers are not currently required to complete this training in
order to prescribe ER/LA opioid analgesics.
The Agency has been carefully evaluating existing requirements and
assessments of the ER/LA Opioid Analgesic REMS, specifically how
prescribers are trained on the use of opioids and management of pain.
To make sure providers are properly informed about suitable prescribing
and the risks and benefits associated with opioid drugs, FDA intends to
update the existing REMS on ER/LA opioid analgesics, and for the first
time, extend these same regulatory requirements to the manufacturers of
immediate release (IR) opioid analgesic products. The new training will
be aimed at making sure providers who write prescriptions for the IR
opioids are doing so for properly indicated patients, and under
appropriate clinical circumstances. In addition to expanding the
training to include more healthcare providers, the training will also
include expanded information on pain, broad principles of acute and
chronic pain management, pharmacologic treatments other than opioids,
and the use of other therapies for pain that do not involve medication.
FDA agrees that all healthcare providers involved in the management
of pain should be educated about the safe use of opioids. Based on the
feedback the Agency has received from two public meetings over the past
year, FDA is actively exploring the question of whether, in the future,
there should be mandatory provider education and how to operationalize
such a requirement. As part of the Opioid Policy Steering Committee's
responsibilities, FDA will be reviewing the data necessary to
understand the most effective way to move forward.
Question. In 2011, FDA issued an Advisory to Drug Manufacturers
regarding glass contamination of injectable drugs and recommended
specific actions the industry could voluntarily take to address the
problem. Does the current administration have plans to update the
Advisory?
Answer. FDA has met with manufacturers who are working to develop
glass medical products to address quality issues with respect to glass
product design and manufacturing for glass products intended for
injectable drugs. The Agency has also initiated a study comparing
various types of glass products intended for use in injectable drug
products and expects to begin analyzing the results of that study by
the end of the year. Additionally, industry experts have initiated
studies of new types of glass products to determine superiority to
current products and suitability with actual drug product formulations.
FDA will use the analysis of its own study, as well as discussions with
industry and any other appropriate available data, when considering
whether to update the 2011 advisory.
electronic distribution of prescribing information
Question. Congress has prevented the implementation of the 2014
Obama administration proposed rule titled the ``Electronic Distribution
of Prescribing Information for Human Prescription Drugs Including
Biological Products.'' Given Congress's explicit concerns, does the
current administration have plans to revise or withdraw this rule?
Answer. FDA is continuing to review its options with regard to the
above rulemaking in light of Sec. 734 of Division A, Title VII, of the
Consolidated Appropriations Act, 2017, which states:
SEC. 734. None of the funds made available by this Act may be
used to propose, promulgate, or implement any rule, or take any
other action with respect to, allowing or requiring information
intended for a prescribing healthcare professional, in the case
of a drug or biological product subject to section 503(b)(1) of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 353(b)(1)),
to be distributed to such professional electronically (in lieu
of in paper form) unless and until a Federal law is enacted to
allow or require such distribution.
______
Questions Submitted by Senator Roy Blunt
drug review time
Question. It has come to my attention that the FDA median new drug
application (NDA) review times vary widely--from 175 days for oncology
and anti-viral drugs to 375 and 400 days for neurology and psychiatry
drugs. In fact, most NDAs have a median review time of more than 300
days. Patient needs are not being met with such long review times.
Could you please provide me the following:
Compare each division's interpretation and implementation of
authority under the Food and Drug Administration Safety and Innovation
Act to exercise flexibility in meeting the approval standard regarding
unmet medical need, including under the accelerated approval pathway.
Answer. CDER has a longstanding commitment to regulatory
flexibility regarding the evidence required to demonstrate that the
approval standard has been met for products that are intended to treat
serious or life-threatening diseases, especially where no satisfactory
alternative therapy exists. This regulatory flexibility extends
throughout the Center and is stated in FDA's regulations at 21 CFR
312.80--Subpart E.
FDA instituted its Accelerated Approval Program--one of the
Agency's expedited programs--to allow for earlier approval of drugs
that treat serious conditions and that would provide a meaningful
advantage over available therapies. Provisions of the Food and Drug
Administration Safety and Innovation Act of 2012 facilitate somewhat
broader use of accelerated approval to expedite patients' access to
important treatments for serious conditions.
Divisions across CDER exercise flexibility in determining whether
drugs meet approval standards (full or accelerated) based on whether
available data indicate that a drug's benefits outweigh the risks for
its intended use(s). Medical need, available alternatives, nature of
the disease, and patient input are among the factors considered in
assessing benefit and risk. Average review times depend on multiple
factors such as the amount and quality of data provided by the
applicant to CDER, available data (including from natural history
studies, if applicable) about the disease or condition to be treated,
and the number of applications under review at the time. Depending on
the drug under consideration, review teams may work across divisions,
leveraging CDER resources to promote collaboration and innovation in
the regulatory process.
Question. Identify policies, practices and procedures, staffing and
resource issues, division culture and precedents, and other factors
which cause or contribute to the differences in review times among
divisions for both priority review and standard review New Drug
Applications.
Answer. Differences in review and approval times between CDER
divisions can be the result of many factors such as the quality of
available data (which depends in part on the rigor of study design)
provided to support an application, complexity of the disease, whether
other effective therapies are available, and the amount and quality of
epidemiologic and natural history data about the disease. All divisions
within CDER are fully focused on our mission of protecting and
promoting public health by helping to ensure that human drugs are safe
and effective, meet established quality standards, and are available to
patients. As such, division culture places these goals as paramount
when reviewing applications, including those being considered under the
Agency's expedited review programs.
Question. Make recommendations for actions that could be taken to
materially reduce differences in New Drug Application review times
among Food and Drug Administration divisions for both priority reviews
and standard reviews and shall include recommendations concerning New
Drug Applications reviewed under the accelerated approval pathway.
Answer. By leveraging FDA's statutory mandates, the Agency is
working to reduce review times by improving processes and gaining
efficiencies to the greatest extent possible. This includes helping to
reduce uncertainty in drug development by encouraging sponsors to
contact the Agency early and often, especially for those products being
developed under expedited review programs. Streamlining clinical
trials, integrating patient voice throughout the regulatory process
based on successful models of participation, and promoting greater
preparedness by establishing nimble approaches to address novel and
emerging public health threats will help Americans get better products,
faster.
FDA is embarking on PDUFA VI, which provides resources for the
highly successful, and resource-intensive, breakthrough therapy program
and streamlines the review of drug/device or biologic/device
combination products. As a result, the Agency suggests that applicants
with products designated as ``breakthrough therapies'' engage in early,
frequent, and meaningful communications with the Agency to better
enable promising safe and effective products to reach patients faster.
Further, PDUFA VI emphasizes patient-focused drug development efforts
through a more systematic, science-based approach to collecting
meaningful patient input. Review times across CDER divisions are likely
to be enhanced by advancing drug development tools, including biomarker
qualification, and increaseing understanding of how ``real-world
evidence'' can be generated and used in regulatory decisionmaking.
Pilot programs to explore novel approaches to the design of complex
clinical trials and the application of advanced modeling techniques to
preclinical and clinical data are also important options to consider
for reducing review times.
fda review time
Question. I am concerned about the cost to the healthcare system
and confusion to consumers and physicians due to Polyethylene Glycol
(Rx PEG) 3350 Abbreviated New Drug Applications remaining on the market
when there is a proven over-the-counter drug available. My
understanding is that the FDA has been looking into this issue for more
than 10 years to confirm that this is a contravention of the Durham
Humphrey Amendments to the Food, Drug, and Cosmetic Act.
Can you explain to me why this review has taken so long and appears
to be ongoing without resolution?
Answer. The distinction between prescription and OTC drugs was
codified by the Durham-Humphrey Amendments, which were enacted in order
to address the marketplace confusion that arose from the simultaneous
marketing of identical or nearly identical drugs on a prescription and
OTC basis for identical or equivalent uses (Public Law 82-215, 65 Stat.
648 (1951). See, e.g., H.R. Rep. No. 82-700, at 5 (1951). The Agency's
detailed rationale for this action is set forth in the Notice of
Opportunity for Hearing published at 73 F.R. 63491 (Oct 24, 2008).
The sponsors of the abbreviated new drug applications (ANDAs) for
prescription PEG 3350 products refused the FDA's request that they
withdraw these products voluntarily. As a result, the Notice of
Opportunity for Hearing was issued, which was followed in May of 2014
by a Draft Proposed Order. Following issuance of the Draft Proposed
Order, several ANDA holders for prescription PEG 3350 drugs submitted
responses asserting that there is a genuine and substantial issue of
material fact that necessitates a hearing.
Consistent with 21 CFR Part 12 and 314.200, the Office of the
Commissioner must review the requests for hearing, the Draft Proposed
Order, and the ANDA holder's objections to the proposed order, and must
determine whether to grant a hearing or enter a final order withdrawing
approval of the ANDAs. The timeframe will depend in part on whether a
hearing is granted or denied. The Office of the Commissioner is
actively considering the matter at this time. The docket for this
matter is available at regulations.gov, Docket No. FDA-2008-N-0549.
Question. How much time is needed until FDA can come to a
conclusion on this issue?
Answer. The sponsors of the abbreviated new drug applications
(ANDAs) for prescription PEG 3350 products refused the FDA's request
that they withdraw these products voluntarily. As a result, a Notice of
Opportunity for Hearing was issued, which was followed in May of 2014
by a Draft Proposed Order. Following issuance of the Draft Proposed
Order, several ANDA holders for prescription PEG 3350 drugs submitted
responses asserting that there is a genuine and substantial issue of
material fact that necessitates a hearing.
Consistent with 21 CFR Part 12 and 314.200, the Office of the
Commissioner must review the requests for hearing, the Draft Proposed
Order, and the ANDA holder's objections to the proposed order, and must
determine whether to grant a hearing or enter a final order withdrawing
approval of the ANDAs. The timeframe will depend in part on whether a
hearing is granted or denied. The Office of the Commissioner is
actively considering the matter at this time. The docket for this
matter is available at regulations.gov, Docket No. FDA-2008-N-0549.
______
Questions Submitted by Senator Dianne Feinstein
antibiotics
Question. I was encouraged that the FDA recently announced the full
implementation of Guidance for Industry #213. This is an important step
in maintaining the effectiveness of antibiotics by ensuring the
judicious use of antibiotics in food-producing animals and limiting the
use of antibiotics to situations that are necessary to protect animal
health.
However, I remain concerned by the significant number of antibiotic
labels that lack defined durations of use, defined dosages, or that
could allow usage under the previous label of ``growth promotion''.
I understand the FDA has collected comments through the Federal
Register on this issue, but what is the timeline as well as the
specific policy actions the FDA plans on taking regarding these
problematic labels?
Answer. FDA agrees that the full implementation of Guidance for
Industry #213 in January 2017 is a critical and important step forward
to foster the judicious use of antimicrobial drugs and curb
antimicrobial resistance. FDA has and continues to express its
appreciation for the cooperation of the animal pharmaceutical industry
for meeting its commitment to fully align all affected products with
the GFI #213 recommendations. FDA recognizes that additional work is
needed to ensure that the labeled use conditions of all medically
important antimicrobial drug products are aligned with the principles
of judicious use. As you noted, one issue of concern is the fact that
some medically important antimicrobial drug products approved for use
in animal feed or water have at least one therapeutic indication
without a defined duration of use.
While this remains a concern, it is important to note that as of
January 2017 it is no longer legal to use such feed or water
antimicrobial products for production (such as growth promotion)
purposes and their use must be under the authorization of a licensed
veterinarian. FDA believes that veterinarian oversight plays a critical
role in ensuring that medically important antimicrobials are used
judiciously.
In September 2016, FDA published a Federal Register notice
requesting comment from the public on this duration of use issue. A key
objective in seeking public comment is to optimize the use of medically
important antimicrobials by using a dosage strategy that maximizes drug
effectiveness, minimizes target animal toxicity, and has an
appropriately targeted duration of use. FDA received numerous
substantive comments on this issue and is still in the process of
analyzing these comments. Once FDA has finished analyzing the comments,
the Agency will develop a timeline with proposed actions for addressing
this issue. It remains an Agency priority to foster stewardship of
medically important antimicrobial drugs in food-producing animals and
help preserve the effectiveness of these antimicrobials in animal and
human medicine.
Question. How will the FDA continue to improve its collection of
data related to antibiotic use on farms and large production
facilities?
Answer. Having access to better data on antimicrobial use and
resistance is critical for assessing the impact of actions already
implemented and for guiding additional steps for fostering judicious
antimicrobial use in veterinary settings. FDA is currently working
closely with the U.S. Department of Agriculture (USDA) to coordinate
data-collection activities across agencies. FDA experts are working
with experts at the USDA/Animal and Plant Health Inspection Service
(APHIS) Center for Epidemiology and Animal Health on strategies for
collecting, analyzing, and reporting data on antimicrobial use. FDA has
taken a number of additional actions to enhance data on antimicrobial
use and resistance. For example, in September 2015, FDA, in
collaboration with the USDA and the Centers for Disease Control and
Prevention (CDC), held a public meeting to obtain input on strategies
for enhancing the collection of antimicrobial drug use and resistance
data. Moreover, in May 2016, FDA issued a final rule revising the
annual reporting requirements for antimicrobials sold or distributed
for use in food-producing animals to require species-specific estimates
of antimicrobial sales or distribution data for major food animal
species.
In August 2016, FDA funded two cooperative agreements with
university researchers to develop and pilot methodologies to collect
detailed information on antibiotic use practices in cattle, swine,
chickens, and turkeys. These ongoing projects are expected to provide
important information on data collection methodologies and will help
support ongoing efforts at USDA to optimize long-term strategies for
collecting and reporting such data. In August 2017, FDA published a
proposed approach for using a biomass denominator to adjust annual data
on the amount of antimicrobials sold or distributed for use in food-
producing animals in the U.S. to take into account the size of animal
populations. This adjusted estimate will provide insight into broad
shifts in the amount of antimicrobials sold for use in food-producing
animals. FDA is inviting comment on the methodology and utility of this
type of data analysis.
Question. Will the FDA continue to enhance coordination with the
Department of Agriculture for on-farm data collection?
Answer. Given the many challenges associated with collecting
nationally representative on-farm data on antimicrobial use across the
various food animal production sectors, FDA believes it is essential to
continue to work in close collaboration with USDA on this issue. FDA is
currently working closely with USDA to coordinate data collection
activities across agencies. FDA experts are working with experts at the
USDA/APHIS Center for Epidemiology and Animal Health on strategies for
collecting, analyzing, and reporting data on antimicrobial use.
Specifically, USDA/APHIS's National Animal Health Monitoring System
provides essential data collection infrastructure for antibiotic-use
practices in livestock and poultry operations and zoonotic pathogen
infection in U.S. food-producing animals via its established survey
cycle and in-plant biological survey capability. Having access to
better data on antimicrobial use and resistance is critical for
assessing the impact of actions already implemented and for guiding
additional steps for fostering judicious antimicrobial use in
veterinary settings. In support of FDA's ongoing work with USDA on this
issue, FDA has taken a number of additional actions to enhance data on
antimicrobial use and resistance. For example, in September 2015, FDA,
in collaboration with the USDA and CDC, held a public meeting to obtain
input on strategies for enhancing the collection of antimicrobial drug
use and resistance data.
In August 2016, FDA funded two cooperative agreements with
university researchers to develop and pilot methodologies to collect
detailed information on antibiotic drug use in cattle, swine, chickens,
and turkeys. These ongoing projects are expected to provide important
information on data collection methodologies and will help support
ongoing efforts at USDA to optimize long-term strategies for collecting
and reporting such data. Moreover, in August 2017, FDA published a
proposed approach for using a biomass denominator to adjust annual data
on the amount of antimicrobials sold or distributed for use in food-
producing animals in the U.S. to take into account the size of animal
populations. FDA's methodology relies on USDA data to estimate the
number of animals in a particular U.S. livestock population, including
annual totals of animals slaughtered and annual totals of livestock
imported into or exported from the U.S. This adjusted estimate will
provide insight into broad shifts in the amount of antimicrobials sold
for use in food-producing animals. FDA is inviting comment on the
methodology and utility of this type of data analysis.
Question. Please provide an update on the progress of collecting
species-specific data for the end-of-year use summary.
Answer. In May 2016, FDA issued a final rule revising the annual
reporting requirements for drug sponsors of antimicrobials sold or
distributed for use in food-producing animals to obtain estimates of
sales by major food-producing species (cattle, swine, chickens, and
turkeys). The additional data will improve our understanding of how
antimicrobials are sold or distributed for use in major food-producing
species and help further target efforts to ensure judicious use of
medically important antimicrobials.
Section 105 of the Animal Drug User Fee Amendments of 2008 (ADUFA
105) requires antimicrobial drug sponsors to annually report to FDA the
amount of all antimicrobial drugs they sell and distribute for use in
food-producing animals. ADUFA 105 also requires that FDA issue annual
summary reports of these sales and distribution data collected from
sponsors. The law further requires that these data be reported out by
antimicrobial drug class. To report summary data in a manner protective
of both national security and confidential business information, only
those antimicrobial drug classes and other categories with three or
more distinct sponsors of approved and actively marketed animal drug
products are independently reported.
FDA is currently reviewing the sales and distribution data
submitted for the 2016 reporting year. The first annual summary report
containing estimated species information is expected to publish by
December 31, 2017.
Question. Please provide an update on the National Action Plan for
Combating Antibiotic-Resistant Bacteria.
Answer. Along with their Federal partners, FDA continues to
implement the National Action Plan for Combating Antibiotic-Resistant
Bacteria and to support the ongoing work of the Presidential Advisory
Council on Combating Antibiotic-Resistant Bacteria (PACCARB). An
assessment of progress being made under the National Action Plan was
published by the PACCARB in March 2016. The full report is available at
https://www.hhs.gov/sites/default/files/paccarb-final-report-
03312016.pdf. More recently, FDA, along with partner agencies, provided
an update to their Year 2 milestones and accomplishments in the
National Action Plan to the council members at the PACCARB's September
13-14 public meeting.
FDA is committed to advancing efforts to foster the judicious use
of antimicrobial drugs in animals. The Agency has issued a number of
important guidance documents and regulations to support its judicious
use strategy and has actively engaged in outreach efforts to support
effective implementation.
diversity in clinical trials
Question. Section 907 of the 2012 Food and Drug Administration
Safety and Innovation Act directed FDA to take a closer look at the
inclusion and analysis of demographic subgroups in applications for
drugs, biologics, and devices--including by sex, race and ethnicity,
and age--and issue a report on findings.
The report that was issued was followed by an ``Action Plan to
Enhance the Collection and Availability of Subgroup Data'', which
included 27 responsive and pragmatic actions, which fall under three
overarching priorities: improving the completeness and quality of
demographic subgroup data collection, reporting and analysis;
identifying barriers to subgroup enrollment in clinical trials and
employing strategies to encourage greater participation; and making
demographic subgroup data more available and transparent.
Please provide an update on the implementation of this Action Plan.
Answer. In February 2016, FDA hosted the public meeting ``Enhancing
the Collection, Analysis, and Availability on Demographic Subgroup
Data,'' to provide a public update on implementation of the Action Plan
(AP). FDA has completed implementation on 26 of 27 action items in the
AP. Highlights are as follows:
To address priority I, data quality, FDA authored two guidance
documents: ``Collection of Race and Ethnicity Data in Clinical Trials''
and ``Evaluation and Reporting of Age, Race, and Ethnicity Data in
Medical Device Clinical Studies.'' Both provide clear and detailed
guidance for regulated industry on clinical trial inclusion data
matters. FDA has updated its MedWatch forms for adverse events
reporting to include fields for race and ethnicity. FDA released its
``Women's Health Research Roadmap'' to better coordinate women's health
research across the Agency.
To address priority II, participation, FDA declared 2016 the ``Year
of Diversity in Clinical Trials'' to make a strong push to promote
representation of diverse groups in clinical trials. Activities like
the Minorities in Clinical Trials Campaign and the Diverse Women in
Clinical Trials Initiative included public service announcements,
educational materials, webinars, and print/digital outreach. Two public
stakeholder meetings were held:
In April 2015, the Institute of Medicine and FDA hosted a joint
meeting titled ``Strategies for Ensuring Diversity, Inclusion, and
Meaningful Participation in Clinical Trials: A Workshop.'' 100+
participants attended and a publication detailing the proceedings was
released in April 2016. In December 2015, FDA and Johns Hopkins held a
joint workshop titled ``Assessing Safety and Efficacy for a Diverse
Population'' to advance understanding of the importance of clinical
trial diversity.
To address priority III, data transparency, FDA launched Drug
Trials Snapshots (DTS). DTS publishes in clear, consumer-friendly plain
language demographic data from clinical trials that supported FDA
approval of new molecular entities and original biologics. 100+ DTSs
have been published.
safety of imported food
Question. The United States imports food products from more than
200 countries and territories through over 300 U.S. ports. Food imports
have more than doubled in the past decade. As the volume of imports
grows, so does the potential for foodborne illness resulting from
imported food.
FDA has bilateral and multilateral food safety-related arrangements
and agreements with numerous countries to ensure the safety of imported
food, and I understand that FDA is also exploring other ways to
leverage the work of the food safety authorities in other countries, in
accordance with section 305 of the 2011 FDA Food Safety Modernization
Act (FSMA). One approach that FDA has been testing as part of this
effort is referred to as ``systems recognition,'' under which FDA would
review certain other countries' food safety systems to ensure that
those systems are comparable to that of the United States.
How do FDA's agreements with other countries ensure that those
countries will effectively address U.S. food safety requirements and
respond in a timely manner to problems that are identified?
Answer. FDA recognizes that food safety issues and outbreaks can
arise in all countries and Systems Recognition accounts for this
reality. Systems Recognition focuses not only on the ability of food
safety systems to help ensure safe food, but also on the ability of
food safety authorities in foreign countries to identify, address, and
contain food safety issues and outbreaks that may arise, learn from
past events, and strengthen their system over time.
Systems Recognition describes whether: (1) a country's food safety
system provides a similar, though not necessarily identical, system of
protections as the U.S. food safety system, and (2) the country's food
safety authority or authorities provide similar oversight and
monitoring activities for food produced under its jurisdiction. Systems
Recognition is based on the premise that food safety systems with
similar elements and similar levels of oversight lead to similar food
safety outcomes.
FDA conducts assessments of foreign food safety systems through the
application of ten standards, outlined in the International
Comparability Assessment Tool (ICAT). These core elements of a food
safety system are important to ensure effective food safety outcomes.
For example, FDA evaluates a system's capacity for responding to
identified problems through surveillance, investigation, response, and
subsequent review of alleged food-related incidents and emergencies
that can reduce the incidence of illness, injury, and outbreaks. FDA
also examines the food safety authority's strategies, procedures, and
actions to enforce and achieve compliance with food safety laws and
regulations and to evaluate the effectiveness of its compliance and
enforcement program. If FDA determines that a country's food safety
system meets the ICAT criteria, FDA implements a Systems Recognition
Arrangement with monitoring and periodic reevaluation by FDA through
the duration of the arrangement.
Question. To what extent has FDA evaluated the effectiveness of
these agreements in keeping the U.S. food supply safe?
Answer. FDA is continuing with efforts to assess potential Systems
Recognition partners to determine whether additional partnerships can
be achieved. At the same time, the Agency is engaged in a program of
monitoring and evaluation for currently recognized partners. To date,
Systems Recognition arrangements have been put into place with New
Zealand, Canada, and Australia. The latter two arrangements were
completed within the past 15 months.
Food safety outcomes typically are judged by microbiological or
chemical levels, incidence of foodborne disease, or some other
quantitative food safety performance measure. Beyond these case-level
measures, FDA is currently exploring additional correlations between
food safety performance measures and the elements making up food safety
systems as a way to measure a food safety system's overall regulatory
performance, in keeping with FSMA's mandate to approach food safety
from a systems perspective.
Before determining whether to enter into a Systems Recognition
Arrangement with another country, FDA uses a tool called the
International Comparability Assessment Tool (ICAT) to assess the
country's food safety system, including the laws, regulations,
programs, and policies upon which a foreign food safety authority
relies to help ensure the safety of food. Based on FDA's experience
with the oversight of domestic state programs and the Manufactured Food
Regulatory Program Standards (MFRPS), from which the ICAT was derived,
FDA believes that the standards reviewed through the ICAT assessments
correspond to comparable food safety outcomes associated with the food
safety system.
To ensure that Systems Recognition Arrangements remain relevant and
effective, each arrangement and corresponding foreign competent
authority undergoes a periodic review and will be reassessed every 5
years. This schedule allows the parties to monitor and modify terms of
the arrangement as needed to account for developments in either party's
food safety regulatory system.
Question. Please provide an update on the results of the systems
recognition pilots with New Zealand and Canada.
Answer. After establishing Systems Recognition Arrangements with
New Zealand in 2012 and Canada in 2016, the countries moved to the
implementation phases for both countries.
Under the arrangements, FDA and each systems-recognized country are
engaged in regular discussions to promote consumer protection
throughout implementation. FDA and the foreign competent authority
continue, through ongoing bilateral communication and periodic reviews,
to examine the performance of each country's regulatory system to help
ensure that the arrangements continue to provide the appropriate level
of public health protection. In addition, the bilateral arrangements
are reviewed periodically, including when the level of food safety
assurance required under domestic law or achieved by one of the parties
significantly changes. For example, FDA has updated its assessment tool
to incorporate new FDA Food Safety Modernization Act standards, and
Canada's Safe Foods for Canadians Act will prompt a re-review of the
bilateral terms next year. In addition, all Systems Recognition
Arrangements include full reassessment at five-year intervals.
Ongoing monitoring of the New Zealand and Canada system-recognized
countries has proved useful in assuring FDA that appropriate public
health outcomes have been achieved under these Systems Recognition
Arrangements.
Question. How, if at all, does FDA plan to use the ``systems
recognition'' approach and integrate it with its broader efforts to
ensure the safety of imported?
Answer. The FDA Food Safety Modernization Act (FSMA) provides FDA
with a variety of new authorities to help ensure the safety of imported
foods and takes into account the capability of the regulatory system of
the exporting country to assure compliance with U.S. food safety
standards for a given food. FSMA also directs FDA to consider bilateral
and multilateral arrangements and agreements to leverage the work done
by foreign competent authorities to help ensure the safety of imported
foods. Both Systems Recognition and commodity-specific arrangements
achieve this directive.
FDA uses Systems Recognition when the Agency determines it can rely
on another food safety authority's implementation of a science-based
food safety system. This level of regulatory partnership and leveraging
requires a rigorous assessment process to support a determination that
FDA is justified in recognizing a foreign food safety system and using
the work of a foreign government to inform FDA's regulatory
decisionmaking process.
Because Systems Recognition takes into account the role of the food
safety system of an exporting country, it informs FDA's risk-based
decisionmaking and resource allocation regarding inspections,
monitoring, admissibility, and follow-up when food safety incidents
occur. Leveraging the regulatory oversight of comparable food safety
systems allows FDA to enhance its risk-based targeting of resources
with respect to import controls, foreign facility inspections, and
foodborne outbreaks. These arrangements with foreign competent
authorities complement FDA's upcoming implementation of the FSMA import
tools, such as the Foreign Supplier Verification Program (FSVP)
regulation, Third-Party Certification, and the Voluntary Qualified
Importer Program. The FSVP regulation incorporates Systems Recognition
by establishing modified requirements for importers of certain types of
food imported from foreign suppliers in good compliance standing with
the food safety authority for a country whose food safety system FDA
has determined to be comparable to the U.S. system under the Systems
Recognition initiative.
food safety modernization act
Question. In September 2016, FDA extended the compliance dates for
many regulated facilities under the Food Safety Modernization Act
(FSMA), especially small and very small businesses that fell under the
main core regulations including Preventive Controls for Human and
Animal Food, Produce Safety Standards, and the Foreign Supplier
Verification Program (FSVP) for food imports. Please provide an update
on the implementation of these regulations.
Answer. In 2016, FDA finalized the last of seven foundational rules
to implement FSMA and is now focusing on implementation of the
regulations. FDA extended certain compliance dates in August 2016 for
very specific activities required in four of the FSMA rules, but these
extensions did not affect the majority of the provisions contained in
the seven rules. FDA is working to address the issues for which the
Agency extended the compliance dates as we proceed with implementation
of the rules. FDA's implementation activities include industry
education, training, outreach, and technical assistance; developing
inspection protocols and strategies to achieve compliance; training for
FDA and state regulators; establishing necessary information technology
systems to support industry, FDA, and states; and coordinating with our
partner organizations.
key highlights of fda's implementation efforts:
Question. FDA initiated inspections to evaluate compliance with two
of the regulations--Preventive Controls for Human Food, and Foreign
Supplier Verification Programs. Rollout of additional inspection
programs are planned for this year.
FDA established a Technical Assistance Network to provide technical
assistance to stakeholders for interpretation of the regulations.
FDA is in the third year of a five-year cooperative agreement with
the National Association of State Departments of Agriculture, NASDA, to
bring together a range of state partners to collaboratively plan
implementation of the Produce Safety regulation and Preventive Controls
for Animal Food regulation, including facilitating outreach and
education and delivery of training to state regulators.
Recently, FDA announced the second round of funding to 43 states
for nearly $31 million to develop produce safety regulatory programs.
In 2016, FDA awarded nearly $22 million to 42 states. The cooperative
agreement is renewable for 5 years, provided availability of funds and
successful performance by states. Given the states' local presence,
knowledge, and relationships with the farm community, FDA believes the
states are very important in helping to provide oversight and direct
technical assistance.
In June 2017, FDA began accepting applications from accreditation
bodies in support of the accredited third-party certification program.
FDA posted the Strategy for FSMA training, which outlines the work FDA
is doing to prepare its staff, state partners, and industry to
implement the regulations. As part of the training strategy, FDA
engaged with the Food Safety Preventive Controls Alliance, the Sprout
Safety Alliance, and the Produce Safety Alliance to develop industry
training on the regulations.
Since October 2016, FDA has released 13 FSMA-related draft or final
guidance documents for industry. In 2017, FDA announced its intention
to consider how it might simplify the agricultural water standards and
to extend compliance dates for those standards (for produce other than
sprouts). FDA also recently announced its intention to continue to
focus on Current Good Manufacturing Practice requirements during
routine animal food facility inspections, but not to begin routine
inspections for Preventive Controls requirements until the fall of
2018.
What steps is FDA taking to ensure that small businesses and
producers understand and are able to comply with the FSMA requirements?
Answer. FDA is engaging in and sponsoring a great deal of outreach
and technical assistance to help small and very small farms and
processors in understanding and complying with the provisions of the
FSMA regulations.
Beginning with the enactment of FSMA in 2011, FDA has been
committed to providing tools to the food industry, particularly small
businesses and producers, in meeting the requirements of the law. FDA
recognizes that small and very small businesses and farmers face unique
circumstances and challenges in implementing FSMA. FDA established
phased-in compliance dates to provide small and very small businesses
as well as producers additional time to comply with the preventive
controls regulations (human and animal food), produce safety
regulation, and foreign supplier verification program regulation. FDA
also began operating a Technical Assistance Network (TAN), which is a
web portal to which questions concerning application of and compliance
with the FSMA regulations can be submitted for an individual response.
FDA continues to engage in a number of activities intended to
assist small and very small businesses as well as producers in advance
of their FSMA compliance dates. To help ensure that our assistance is
targeted to meet their needs, FDA partners with groups involved with
specific communities to facilitate delivery of training and
informational resources to achieve compliance with the FSMA
regulations. For example, in 2016 FDA awarded a cooperative agreement
to the National Farmers Union Foundation to develop and provide
science-based, culturally specific food safety training, education, and
outreach for local food producers and processors.
FDA established a Produce Safety Alliance (PSA) to develop and
deliver training on the produce safety regulation requirements that
would be of particular assistance to small and very small farms, with
training courses available since fall 2016. FDA also established the
Food Safety Preventive Controls Alliance to develop and deliver
training that will help small and very small facilities understand the
preventive controls and foreign supplier verification program
regulations requirements.
In 2016, FDA awarded almost $22 million in cooperative agreements
to 42 States to develop produce safety infrastructure which included
outreach and technical assistance to farms in those states. In July
2017, FDA announced an additional nearly $31 million in cooperative
agreements to 43 States to begin and continue development of these
State activities. FDA has also created a Produce Safety Network (PSN),
which is a regionally-located staff of produce specialists whose
primary focus is to develop relationships with the local farming
community and assist in the implementation of the produce safety
regulation.
In addition, FDA has issued small entity compliance guides for the
current good manufacturing practice (CGMP) and hazard analysis and risk
based preventive controls regulations for both human and animal feed,
intended to inform small and very small domestic and foreign human and
animal food facilities about the CGMP and preventive controls
regulations and enable them to better understand the requirements of
the regulations.
These guidances should be helpful to small and very small farms
that also engage in on-farm manufacturing and processing. FDA also has
issued a small entity compliance guide for the Mitigation Strategies to
Protect Food Against Intentional Adulteration rule. FDA also intends to
issue small entity compliance guides for the produce safety regulation
and the other FSMA regulations.
cosmetics
Question. Virtually every American uses personal care products
daily, yet the safety laws for cosmetics and personal care products
haven't been modernized since created in 1938. There is support among
the industry- both large and small companies- as well as consumer and
health groups to take significant steps to update oversight in this
area. I have worked with Senator Collins to introduce a bipartisan bill
to do just that.
Will you commit to working with us to make meaningful updates,
including evaluating the safety of ingredients in these prod?
Answer. FDA shares your interest in the safety of cosmetics
marketed to American consumers and commits to continuing dialogue on
cosmetics issues.
Questions Submitted by Senator Tom Udall
Question. Last year, FDA issued a long overdue rule, known as the
deeming rule, to enable the agency to begin to oversee e-cigarettes,
cigars, and other previously unregulated tobacco products. FDA was
responding, in part, to the thousands of flavored e-cigarettes that
have flooded the marketplace in recent years--flavors such as cotton
candy, gummy bear, root beer float, and banana split. While youth use
of e-cigarettes dipped last year, youth use of e-cigarettes exceeds use
of regular cigarettes. As the Surgeon General reported last year, use
of e-cigarettes by youth is a public health concern and use of any
product containing nicotine by youth, including e-cigarettes, is
unsafe.
deeming
I am concerned that FDA announced in May a three-month delay in
enforcement of future deeming rule compliance dates and that the
Administration has delayed filing legal briefs defending the deeming
rule from industry challenges. While there is debate about whether e-
cigarettes could help some adult smokers to quit regular cigarettes, we
should all be able to agree that FDA oversight is needed to protect
kids and public health. FDA should be aggressively addressing the
flavors and marketing practices that are increasing e-cigarettes'
appeal to youth.
During the Subcommittee's hearing, you indicated that FDA was
looking at aspects of the deeming rule. Which aspects of the deeming
rule you are examining?
Answer. On July 28, 2017, FDA announced a comprehensive approach to
the regulation of nicotine which includes the Agency's plan to begin a
public dialogue about lowering nicotine levels in combustible
cigarettes to non-addictive levels through achievable product
standards. The Agency intends to issue an Advance Notice of Proposed
Rulemaking (ANPRM) to seek input on the potential public health
benefits and any possible adverse effects of lowering nicotine in
cigarettes. The comprehensive approach also includes, among other
things, a reconsideration of aspects of the implementation of the final
deeming rule with an eye towards fostering innovation where innovation
could truly make a public health difference, and making sure FDA has
the foundational regulations it needs in place to make the entire
program transparent, predictable, and sustainable for the long run.
FDA shares the concerns about youth use of e-cigarettes. On August
8, 2017, FDA announced it would pursue a strategic, new public health
education campaign aimed at discouraging the use of e-cigarettes and
other electronic nicotine delivery systems (ENDS) by kids. The Agency
plans to expand its ``The Real Cost'' public education campaign this
fall to include messaging to teens about the dangers of using these
products while developing a full-scale campaign that will launch in
2018. The campaign is just one component of the Agency's efforts to
restrict youth access, limit youth appeal, and reduce youth exposure to
toxic chemicals from all tobacco products. FDA continues to enforce
existing regulations specifically aimed at addressing youth access to
ENDS and other newly-regulated products, including banning the sale of
tobacco products to those under age 18, requiring age verification by
photo ID, and prohibiting free samples. Since August 2016, FDA has
issued over 6,400 warning letters to brick and mortar and online
retailers for selling newly-regulated tobacco products such as e-
cigarettes to minors.
On August 4, 2017, FDA issued a guidance that extended the
deadlines for the submission of marketing applications for those
products that became newly-regulated by last year's deeming rule and
were on the market as of August 8, 2016. Applications for newly-
regulated combusted products--such as most cigars, pipe tobacco, and
hookah tobacco--would be submitted by August 8, 2021. Applications for
newly-regulated non-combusted products--such as most e-cigarettes--
would be submitted by August 8, 2022. For newly regulated products on
the market as of August 8, 2016, FDA anticipates that manufacturers
will be able to continue marketing products while FDA reviews product
applications submitted by the revised filing dates.
With this additional time, FDA intends to issue other foundational
rules and guidances--addressing topics such as the type of information
FDA expects to be included in marketing applications--that will help
make the product review process more efficient, predicable, and
transparent, while still upholding the FDA's public health mission.
This additional time will not only help manufacturers develop
higher quality and more complete applications, but also allows FDA
additional time to explore clear and meaningful measures to make
tobacco products less toxic, appealing, and addictive, with an intense
focus on youth. In particular, the Agency is pursuing product standards
for ENDS that would address known risks. This could include measures on
battery safety and child-resistant packaging, and product labeling to
prevent accidental child exposure to liquid nicotine. The FDA also
intends to issue an Advance Notice of Proposed Rulemaking (ANPRM) to
seek public comment on the role that flavors--including menthol--in
tobacco products play in attracting youth. The FDA also intends to
issue an ANPRM to request information on how ``premium cigars'' should
be defined, the health effects of these products, and their patterns of
use. Additionally, the Agency plans to explore additional restrictions
on the sale and promotion of ENDS, including restrictions on how
products may be sold and advertised, to further reduce youth exposure
and access to these products.
Question. Will you commit to not delay enforcement of the deeming
rule beyond the current three-month delay?
Answer. On July 28, 2017, FDA announced a comprehensive approach to
the regulation of nicotine which includes the Agency's plan to begin a
public dialogue about lowering nicotine levels in combustible
cigarettes to non-addictive levels through achievable product
standards. The Agency intends to issue an Advance Notice of Proposed
Rulemaking (ANPRM) to seek input on the potential public health
benefits and any possible adverse effects of lowering nicotine in
cigarettes. The comprehensive approach also includes, among other
things, a reconsideration of aspects of the implementation of the final
deeming rule with an eye towards fostering innovation where innovation
could truly make a public health difference, and making sure FDA has
the foundational regulations it needs in place to make the entire
program transparent, predictable, and sustainable for the long run.
FDA shares the concerns about youth use of e-cigarettes. On August
8, 2017, FDA announced it would pursue a strategic, new public health
education campaign aimed at discouraging the use of e-cigarettes and
other electronic nicotine delivery systems (ENDS) by kids. The Agency
plans to expand its ``The Real Cost'' public education campaign this
fall to include messaging to teens about the dangers of using these
products while developing a full-scale campaign that will launch in
2018. The campaign is just one component of the Agency's efforts to
restrict youth access, limit youth appeal, and reduce youth exposure to
toxic chemicals from all tobacco products. FDA continues to enforce
important existing regulations specifically aimed at addressing youth
access to ENDS and other newly-regulated products, including banning
the sale of tobacco products to those under age 18, requiring age
verification by photo ID, and prohibiting free samples. Since August
2016, FDA has issued over 6,400 warning letters to brick and mortar and
online retailers for selling newly-regulated tobacco products such as
e-cigarettes to minors.
On August 4, 2017, FDA issued a guidance extending the deadlines
for the submission of marketing applications for those products that
became newly-regulated by last year's deeming rule and were on the
market as of August 8, 2016. Applications for newly-regulated
combustible products--such as most cigars, pipe tobacco and hookah
tobacco--would be submitted by August 8, 2021. Applications for newly-
regulated non-combustible products--such as most e-cigarettes and other
Electronic Nicotine Delivery Systems (ENDS)--would be submitted by
August 8, 2022. For newly regulated products on the market as of August
8, 2016, the Agency anticipates that manufacturers will be able to
continue marketing products while FDA reviews product applications
submitted by the revised filing dates.
Importantly, the compliance policy described above does not affect
any current requirements from the deeming rule that have already gone
into effect. For example, the deeming rule provisions regarding
mandatory age and photo-ID checks to prevent illegal sales to minors
remain in effect and are subject to enforcement by FDA. It also will
not affect future deadlines for other provisions of the rule,
including, but not limited to, required warning statements, ingredient
listing, health document submissions, harmful and potentially harmful
constituent reports, and the removal of modified risk claims, such as
``light,'' ``low,'' or ``mild,'' or similar descriptors.
______
Questions Submitted by Senator Patrick Leahy
helping farmers comply with food safety regulations
Question. The FDA is still in the long drawn-out process of
implementing new regulations under the Food Safety Modernization Act
(FSMA). This is something that our farmers and food companies in
Vermont have been watching very closely, especially for produce farmers
who will be covered under the new regulations and have compliance dates
that begin in 2018. However, there has already been significant market
pressure for farms of all sizes to demonstrate food safety compliance
now, regardless of whether they are fully subject to the new
requirements. It is critical that the FDA and USDA both provide support
to farmers in order to build the needed capacity to adapt to this
changing food safety regulatory landscape. In addition, food safety
training and technical assistance should not be one-size-fits-all, but
rather tailored to meet the needs of agricultural operations across the
country that vary in types and farm size.
I have heard reports from farmers in Vermont that they feel there
is a shortage of information flowing from the FDA, and a growing need
for trainings that are tailored to the unique needs of small- and mid-
scale diversified farms and local food processors.
The FDA has stated that it will provide additional guidance for
trainers to use in assessing the equivalency of their training
programs, and it has provided some funding to support the development
of training programs tailored toward local food producers. However, at
this point very little information has been provided regarding the
timelines and processes for these much needed equivalent and
alternative trainings, yet compliance starts in 2018. It is imperative
that the FDA provide sufficient resources in order to offer a diverse
array of farmer food safety capacity-building efforts to help American
produce farms of all sizes and stages of development in order to face
these new Federal food safety requirements, access markets, and promote
public health.
What are the specific processes and timelines that the agency will
follow in issuing alternate training and equivalency criteria for
farmers as quickly as possible?
Answer. FDA recognizes that this is the first FDA regulation
specific to produce farms, which can make implementation challenging
for farmers. FDA is actively engaged in activities to ensure that
farmers have the training, guidance, and other technical assistance
targeted to meet their needs and delivered in a timely manner based on
the phased in compliance dates in the final rule. FDA has also
announced its intention to extend the compliance dates for agricultural
water provisions in the rule (other than for sprouts).
Specifically, FDA is actively engaged in activities that will
assist with ensuring that training is and continues to be available to
farmers. FDA established a Produce Safety Alliance (PSA) to develop and
deliver training on the produce safety requirements that would be of
particular assistance to farmers. PSA training courses have been
available since fall 2016. In August 2016, FDA announced a partnership
with the National Farmers Union, through a Cooperative Agreement, to
develop and provide science-based, culturally specific food safety
training, education and outreach, for local food producers and
processors. The emphasis is on those involved in diversified,
sustainable, organic, and identity-preserved agricultural operations;
beginning and socially disadvantaged farmers; value-added farm
businesses and small-size processors; and direct and intermediate
supply chain participants.
In August 2016, FDA announced a partnership with the University of
Arkansas at Fayetteville, through a Cooperative Agreement. The
partnership is to develop and implement food safety training,
education, outreach, and identification of technical assistance
resources for key tribal stakeholders, including farmers, packers, and
manufacturers/processors that grow, harvest, pack, manufacture/process,
or hold food covered by FSMA. Moreover, FDA partnered with the U.S.
Department of Agriculture's National Institute of Food and Agriculture
to provide a National Coordination Center and four Regional Centers to
provide training opportunities for owners and operators of farms, small
food processors, and small fruit and vegetable wholesalers. These
Regional Centers are also responsible for providing technical
assistance to farms and offer yet another mechanism for delivering
training in a form and manner that meets farms' needs. One of the four
Regional Centers is based at the University of Vermont.
In 2016 and 2017, FDA awarded a combined nearly $53 million in
cooperative agreements to 43 States to develop produce safety
infrastructure, which included outreach and technical assistance to
farms in those states. FDA has also created a Produce Safety Network,
regionally-located staff consisting of produce specialists whose
primary focus is to develop relationships with the local farming
community and assist in the implementation of the produce safety
regulation. The FSMA Collaborative Training Forum is co-led by FDA and
USDA and involves all public and private entities engaged in FSMA
training, especially those serving small and very small businesses. The
members of the Forum are actively working to identify and address any
information gaps in the various training materials and help ensure that
training programs meet farms' needs.
added-sugar nutritional labeling for maple and honey:
Question. I understand that the FDA is setting a new compliance
date for the updated nutrition facts label. I am a strong supporter of
transparency and giving consumers information about what is in the
foods they buy. However, I believe consumers should not be misled into
thinking that sugar has been added to products like maple syrup and
honey, which by their nature are pure single ingredient products.
Have you looked into this issue related to the labeling of sugars
in honey and maple syrup? Will you commit to work on this issue to find
solutions in order to avoid consumer confusion for these single
ingredient pure foods like maple syrup and honey so that we do not
adversely impact farmers and producers?
Answer. FDA announced on June 13, 2017, its intention to extend the
compliance dates for the Nutrition Facts label final rule. After
careful consideration, FDA determined that additional time would
provide manufacturers covered by the rule with guidance from FDA, and
would help them be able to complete and print updated Nutrition Facts
labels for their products before they are expected to be in compliance.
FDA will provide details of the extension through a Federal Register
notice.
The Nutrition Facts label final rule defines ``added sugars,'' in
part, to include sugars that are either added during the processing of
foods, or are packaged as such, which includes packages of sugar or
containers of honey. The Agency has heard concerns from the honey
industry about declaring added sugars on a jar of honey since no sugar
is added to the product. The Agency has also heard similar concerns
from the maple syrup industry. Providing industry more information
about the labeling of ``added sugars'' on pure honey, maple syrup, and
other single ingredient sugar products--is an Agency priority, and FDA
is working to address the complex issues related to this labeling
concern. FDA plans to invite further comment in the near future and
intends to follow up with the maple syrup and honey industries and
other stakeholders at a later date.
maple nutritional labels and deceptive marketing
Question. We also must crack down on food manufacturers that use
the word ``maple'' on their labels or utilize maple related graphics on
their packaging, when there is not any maple syrup in their products. I
am concerned that consumers are being misled into thinking that these
manufactured food products contain maple syrup, because to most
consumers ``maple'' is not viewed merely as a characterizing flavor as
is the case with a grape or orange soda. Instead, ``maple'' is
synonymous with the ingredient maple syrup. This must be addressed by
the FDA because these foods that are masquerading as maple syrup-
flavored only serve to deceive and mislead consumers, and can cause
economic harm to American maple syrup producers and food producers
using real pure maple syrup.
Maple syrup as a premium ingredient has a material bearing on the
price and consumer acceptance of food products that contain it, which
is why it is frequently an ingredient named in the foods or displayed
on its packaging. What steps will the FDA take to exercise its legal
authority to investigate and take action against misbranded ``maple''
products in interstate commerce that give consumers the erroneous
impression that such maple syrup is present in the foods they are
purchasing?
Answer. FDA's regulations address labeling requirements for
characterizing ingredients and flavors. Those regulations provide a
basis to allow firms to include statements that are truthful and not
misleading on their labels. In addition, they provide a framework for
identifying maple ingredients or maple flavors in products, whether
involving the use of real maple syrup, or maple flavors derived from
maple sources, or the use of maple flavor that is derived from other
natural or artificial components. If maple flavor is derived from maple
sources other than maple syrup, such as maple sugar or maple extract,
FDA would not object to that product being represented as containing
natural maple flavor. However, if the characterizing flavor is derived
from a non-maple source, such as fenugreek or an artificial flavor, the
food must be labeled as ``artificially flavored'' in accordance with 21
CFR 101.22(i)(1)(ii) and 101.22(i)(2).
Under FDA's regulations, the term ``maple'' is not synonymous with
``maple syrup.'' Such a reading would require, among other things,
evidence that consumers perceive the terms to be synonymous and a
change in FDA's regulations. The process for requesting FDA to issue,
amend, or revoke a regulation is described in 21 CFR 10.30 (Citizen
petition).
FDA shares your concern for the truthful labeling of food products,
and FDA intends to continue to monitor the marketplace for potentially
false and misleading labeling. If a food does not contain the
ingredient maple syrup, the label cannot include the term ``maple
syrup'' in the ingredient statement or as a part of the statement of
identity. FDA will consider taking action, as appropriate, consistent
with our food safety priorities and resources, against products that
are misbranded.
Further, FDA shares your desire to avoid consumer confusion. In
September 2016, FDA developed an FDA Consumer Update to help educate
consumers about the differences in the ways that ingredients and
flavors are declared on product labels, including ``maple'' and ``maple
syrup.'' The Update is available at: https://www.fda.gov/ForConsumers/
ConsumerUpdates/ucm521518.htm. FDA may develop additional educational
materials if further needs are identified. Furthermore, FDA has worked
with the maple syrup industry and would be willing to meet with them to
discuss available data and other industry information on consumer
perceptions regarding maple and maple syrup.
fighting the opioid epidemic
Question. Addiction to opioids has ravaged the nation in recent
years. According to the National Institutes of Health (NIH), more than
two million Americans suffer from opioid addiction today, with the
number of prescriptions written for opioids having tripled in the past
20 years. Overdose deaths from heroin and opioids has also more than
tripled in the past two decades.
This epidemic has had devastating consequences. I watched with
interest the recent announcement by the FDA on its intention to take
steps to address the crisis by exploring new opioid formulations
designed to deter abuse, and by engaging stakeholders to evaluate the
impact of opioid addiction on addicts, their families, and communities
nationwide.
In addition to the FDA's recent announcement, what other ways can
the agency continue to take steps to address the nationwide opioid
epidemic?
Answer. The FDA Commissioner has made reducing the scope of the
epidemic of opioid addiction his highest initial priority. As a first
major step in combatting this crisis, FDA recently created an Opioid
Policy Steering Committee which is considering what more the Agency can
do to confront the challenges of opioid addiction and opioid-related
overdoses and deaths.
Bringing together some of the Agency's senior leaders and members,
the Committee has begun by exploring three questions: whether mandatory
education for healthcare professionals who have the capability to
prescribe opioids is needed; whether extra risk management steps for
opioid prescription need to be taken; and whether a change to the
current FDA framework used to assess the risk of abuse and misuse
during the drug review process is needed.
While the Committee has begun with these core questions, it has a
broad mandate to consider whatever additional questions FDA should be
seeking to answer. The Committee will solicit input, and engage the
public. FDA is committed to looking at all facets of this complex issue
and collaborating on various approaches. To pursue these policies, FDA
plans to continue to have public dialogue through various forums and to
share additional steps and information FDA is considering in addressing
these challenges. FDA continues to collaborate with fellow HHS agencies
as part of a coordinated Departmental strategy around addressing the
opioid crisis.
Question. Regarding the FDA's recent announcement to explore new
opioid formulations designed to deter abuse, has the agency also
considered exploring opioid-alternatives to treat and mange pain?
Answer. FDA continues to support efforts to better understand the
treatment of pain, especially as it relates to balancing the need to
effectively treat pain with the public health crisis related to opioid
use disorder and overdose. Currently, there are both opioid and non-
opioid drugs approved by FDA for the management of pain.
FDA has a number of programs, such as Fast Track and Breakthrough
Designation, which are intended to facilitate the development and
expedite the review of products that are intended to treat a serious
condition for which there is an unmet medical need. Novel non-opioid
medications with the potential to provide effective pain relief may be
appropriate designations, but it will be a product-specific
determination.
SUBCOMMITTEE RECESS
Senator Hoeven. Again, I want to thank everyone for coming
today, most of all the Commissioner. Again, we look forward to
working with you.
Dr. Gottlieb. Thanks a lot.
Senator Hoeven. We are adjourned.
[Whereupon, at 11:15 a.m., Tuesday, June 20, the
subcommittee was recessed, to reconvene subject to the call of
the Chair.]