Health Policy Roundup: Background on FDA User Fee Programs
February 4, 2022
This event was Part IV of the Alliance’s Health Policy Roundup and 2022 Forecast series.
Download a summary of this event »
Since the passage of the first User Fee Act in 1992, user fee programs have helped finance the Food and Drug Administration (FDA)’s review of human medical products such as drugs and medical devices. By authorizing the collection of user fees from biopharmaceutical companies, the FDA can support increased staffing and resource capacity building, as well as generate regulatory predictability. This predictability, in turn, is meant to help incentivize industry investment in clinical research and development. During the pandemic, user fees have also helped enable the FDA to provide prompt recommendations and regulatory guidance for COVID-19 vaccines and therapeutic development. The User Fee Act is subject to renewal every five years, with the next renewal slated for 2022, and involves careful negotiation among manufacturers, the FDA, and Congress. The latest reauthorization is a must-pass piece of legislation for Congress in 2022.
This event will explain user fee programs and their role within the drug development and FDA regulatory review process. Panelists will provide insights on the stakeholders, timeline, the process of reauthorization, the history of these bills being used for broader health legislative initiatives, and explore lessons learned from almost thirty years of user fee programs as we approach our next iteration.
For additional information on the U.S. Food and Drug Administration review process, see Chapter 4 of the Alliance’s Health Policy Handbook.
Speakers:
- Anna Abram, Senior Advisor, Akin Gump Strauss Hauer & Feld LLP
- Jeremy Sharp, Managing Director, Waxman Strategies
- John Stone, J.D., Principal, BGR Group
- Emilia DiSanto, J.D., Executive Vice President, Venn Strategies (moderator)
This event was made possible with support from Arnold Ventures.
Event Resources
Key Resources
(listed chronologically, beginning with the most recent)
“PDUFA VII: Fiscal Years 2023-2027.” Food and Drug Administration. Available here.
“Hearing on “FDA User Fee Reauthorization: Ensuring Safe and Effective Drugs and Biologics.” House Committee on Energy and Commerce. February 3, 2022. Available here.
“FDA Human Medical Product User Fee Programs.” Bodie, A., Sarata, A. Congressional Research Service (CRS). September 27, 2021. Available here.
“The Prescription Drug User Fee Act (PDUFA VII).” Pharmaceutical Research and Manufacturers of America (PhRMA). August 2021. Available here.
“Health Policy Handbook, Chapter 4: U.S. Food and Drug Administration.” Rawal, P., Whitlock, R. Alliance for Health Policy. July 20, 2021. Available here.
“Drug Pricing and Pandemic Preparedness in the 2022 FDA User Fee Reauthorization Process.” Sharp, J., Kenyatta, K. The Commonwealth Fund. July 23, 2020. Available here.
Additional Resources
(listed chronologically, beginning with the most recent)
“Statement for the Record: FDA User Fee Reauthorization: Ensuring Safe and Effective Drugs and Biologics.” National Health Council. February 3, 2022. Available here.
“This Week at FDA: Congress to Begin Work on User Fee Reauthorization Next Week.” Mezher, M. Regulatory Affairs Professionals Society. January 28, 2022. Available here.
“5 Things to Watch in Health Care in 2022.” Scarborough, S. Arnold Ventures. January 19, 2022. Available here.
“Prescription Drug Policy, 2021 and 2022: The Year in Review, and the Year Ahead.” Sachs, R. Health Affairs. January 7, 2022. Available here.
“Medical Trial Diversity Ignored by FDA in Fee Plan, Groups Say.” Castronuovo, C. Bloomberg Law. December 14, 2021. Available here.
“Reauthorization of the Prescription Drug User Fee Act; Public Meeting; Request for Comments.” Federal Register. August 24, 2021. Available here.
“PDUFA VII is Critical for Future Biopharmaceutical Innovation and For Patients.” Moscicki, R. Pharmaceutical Research and Manufacturers of America (PhRMA). August 23, 2021. Available here.
“Prescription Drug User Fee Rates for Fiscal Year 2022.” Food and Drug Administration. August 16, 2021. Available here.
“The Prescription Drug User Fee Act Fact Sheet.” Pharmaceutical Research and Manufacturers of America (PhRMA). May 7, 2021. Available here.
“To Improve Competition in Generic Drug Markets, the FDA Should Discount User Fees for Small Players.” Dotzel, M., Frank, R., McGuire, T. et al. Health Affairs. April 15, 2021. Available here.
“FDA Drug Approval: Application Review Times Largely Reflect Agency Goals.” U.S. Government Accountability Office. April 6, 2020. Available here.
“FDA User Fees: How Do They Work?” Hawana, J. The National Law Review. January 28, 2020. Available here.
“Extending the User Fee Approach to Pharmaceuticals.” Cutler, D. JAMA Forum Archive. September 5, 2018. Available here.
“Prescription Drug User Fee Act (PDUFA): 2017 Reauthorization as PDUFA VI.” Dabrowska, A., Thaul, S. Congressional Research Service (CRS). March 16, 2018. Available here.
“The Prescription Drug User Fee Act: Cause for Concern?” Gabay, M. Hospital Pharmacy. February 12, 2018. Available here.
“Speed, Safety, and Industry Funding- From PDUFA I – PDUFA VI.” Darrow, J., Avorn, J., Kesselheim, A. The New England Journal of Medicine. December 7, 2017. Available here.
“The National Health Council and NORD Commend House Passage of FDA User Fee Reauthorization; Urge Senate to Follow Suit.” Sencen, L. NORD. July 12, 2017. Available here.
“PDUFA Legislation and Background.” Food and Drug Administration. Available here.
“Prescription Drug User Fee Amendments.” Food and Drug Administration. Available here.
“PDUFA.” Pharmaceutical Research and Manufacturers of America (PhRMA). Available here.
Resources for Last User Fee Process in 2017
“PDUFA VI: Fiscal Years 2018-2022.” Food and Drug Administration. Available here.
“Prescription Drug User Fee Act (PDUFA): 2017 Reauthorization as PDUFA VI.” Dabrowska, A., Thaul, S. Congressional Research Service. March 16, 2018. Available here.
“H.R. 2430- FDA Reauthorization Act of 2017.” Office of Congressman Greg Walden. August 18, 2017. Available here.
“Senate Passes FDA User Fee Reauthorization.” Kacik, A. Modern Healthcare. August 3, 2017. Available here.
“FDA User Fee Reauthorization Poised to Pass House of Representatives.” Sachs, R. Health Affairs. July 12, 2017. Available here.
Experts
Speakers
Anna Abram
Akin Gump Strauss & Feld LLP, Senior Advisor
aabram@akingump.com
Emilia DiSanto, J.D. (moderator)
Venn Strategies, Executive Vice President
edisanto@vennstrategies.com
Jeremy Sharp
Waxman Strategies, Managing Director
jeremy@waxmanstrategies.com
John Stone, J.D.
BGR Group, Principal
jstone@bgrdc.com
Experts and Analysts
Andrea Noda, MPP
Commonwealth Fund, Vice President of Health Care- Drug Pricing
anoda@arnoldventures.org
Rachel Sher, J.D., MPH
Manatt Health, Phelps, & Phillips, LLP., Partner
rsher@manatt.com
Lovisa Gustafsson, MBA
Commonwealth Fund, Vice President, Controlling Health Care Costs
ilg@cmwf.org
Jeromie Ballreich, Ph.D.
Johns Hopkins Bloomberg School of Public Health, Assistant Scientist
Jballre2@jhu.edu
Josh Rising, M.D., MPH
Rising Health Strategies, Principal
josh@rhstrategies.com
Government
Sarah Despres, J.D.
U.S. Department of Health and Human Services, Counselor for Public Health and Sciences
Sarah.Despres@hhs.gov
Eric Stone, MBA
Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Director, Office of Management
Eric.Stone@fda.hhs.gov
Patrick Zhou, M.A.
Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Operations Research Analyst
Patrick.Zhou@fda.hhs.gov
Beena Alex, MPH, MBA
Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Lead Management Analyst
Beena.Alex@fda.hhs.gov
Stakeholders
Abigail Lore, MPA
Pharmaceutical Research and Manufacturers of America (PhRMA), Director of Policy and Research
ALore@phrma.org
John A. Murphy, III, J.D.
Biotechnology Innovation Organization (BIO), Chief Policy Officer Deputy Counsel, Healthcare
Jmurphy@bio.org
Hillary Stires, Ph.D
Friends of Cancer Research, Science Policy Analyst
hstires@focr.org
Kirk Williamson, MPH
Arnold Ventures, Health Care Manager
KWilliamson@arnoldventures.org
Transcript
This is an unedited transcript.
0:04
Hello everyone, thank you for your patience as we started a little late. I’m Kathryn Martucci, director of policy and programs here at the Alliance, and for those who are not familiar with us and welcome, we are a non partisan resource for the policy community, dedicated to advancing knowledge and understanding of health policy issues, and thanks for joining today’s last installment of our Health Policy Roundup series. If you weren’t able to join us for previous three things in this series, please join our visit, excuse me, visit our website, where you can watch recordings of those events and access additional resources.
0:43
Today’s session, we’ll explain and explore user programs, And we must pass reauthorization legislation for Congress this year. And we have an excellent, an esteemed group of panelists gathered today has a range of experience within FDA, Congress, and HHS. So definitely leverage this opportunity to hear about this process from the experts themselves.
1:08
You can join today’s conversation on Twitter, as well, using the hashtag all health live, and join our community at all health policy, as well, as on Facebook and LinkedIn.
1:21
And we also will have a Q&A session after some words, from our keynote speaker and our panelists. And we want you to be active participants, Participants, excuse me, in the conversation today. So please, definitely get your questions ready. You should see a dashboard on the right side of your web browser that has a speech bubble icon with a question mark.
1:41
You can use that speech bubble to submit questions at any time. We will collect these and address them throughout the broadcast. You can also use that chat to discuss any technical issues may be experiencing and someone will attempts to help.
1:55
Before we dive in, I also want to recognize that this event was made possible with support from Arnold Ventures and we are grateful to have Kirk Williamson Healthcare Manager of Drug Prices at Arnold Ventures to get some quick remarks.
2:12
Great, thank you, Kathryn. Good afternoon, everyone, and we really appreciate all of you joining us today.
2:17
A special thank you to the entire Alliance team for pulling together a webinar today.
2:24
Our ventures are very happy to support the work of the alliance and I’m really looking forward to today’s important and timely discussion. I’ll keep my remarks brief because I know we have a lot to cover today.
2:34
Sarah mentioned, my name is Kirk Williams and I’m a manager with Arnold Ventures and our drug pricing portfolio, or no venture philanthropy dedicated to tackling some of the most pressing problem. In the United States, we invest in sustainable change.
2:48
Based on research and evidence driven policy, education and advocacy. Arnold Ventures focus the work and portfolios, including criminal justice reform, higher education, public finance, and, of course, healthcare.
3:02
Our objective in healthcare is to lower the cost while maintaining and enhancing access to kneaded high quality care.
3:09
Across the health care system, we focus on opportunities to achieve more affordable and equitable care, while securing better health outcomes.
3:17
These focus areas include reducing hospital and physician prices in car, identifying provider payment incentive, to accelerate the shift away from traditional paper service, to value based payment model, and improving the care for Americans with complex health conditions and needs, and, of course, the portfolio I work on, lowering prescription drug prices while maintaining incentives for meaningful innovation.
3:40
We do this by providing grants to many different organizations that conduct research, provide technical assistance, or engage in advocacy campaigns.
3:49
Our work examines a variety of issues, such as: anti competitive behavior, market distortions, unjustified price increases, ways to strengthen FDA approval standards for certain drugs and biologics, and clinical trial data transparency.
4:04
We hope today’s discussion of the upcoming reauthorization of the User Fee Act provide you all with useful background about the legislation and its potential effect on drug development and FDA regulatory review process.
4:16
So with that, I’ll turn it back over to Kathryn. Thank you all for joining, and I’m excited for this discussion.
4:23
Great, thank you, Kurt. And now I’m excited to introduce Emilia DiSanto, Executive Vice President of Venn Strategies, who will be leading our conversation today. I’m Amelia brings a wealth of experience more than three decades in federal service, both on Capitol Hill and various federal agencies. She spent most of her career working in oversight roles as Chief Investigative Council and Special Counsel to the Senate Committee on Finance. And she’s Investigative Council on a Special Committee on Aging. And after she left to Capitol Hill, she took a series of high level posts in the executive branch at the US Department of State and Federal Housing Finance Agency. So very excited to have you here with us today to guide this discussion. I’ll turn it over to you Amelia.
5:10
Well, thank you very much, Kathryn, and thank you everyone, for joining us today for the final session of the Alliance for Health Policy Health Policy Roundup series. Well, first, I’d like to welcome everyone. I’m delighted to be the monitor.
5:26
The moderator here today, for this critical and timely discussion regarding D the Prescription Drug User Fee Act, also known as to do. Today, will also be touching base with a few other of the user keys that deal with two Derek’s devices as well as biologics.
5:45
Well, let’s face it, 2022 is not only the year of the tiger and the 30th anniversary of the user Fee X, it is also the year that the user fee Act must be re-authorized by Congress.
6:02
And that reauthorization must be completed by September 30th, 2022, the end of the fiscal year.
6:10
With that in mind, I am pleased to introduce Andi Fristedt, Deputy Commissioner for policy, legislation, and international affairs for the Food and Drug Administration for some opening remarks.
6:26
Andy provides strategic policy direction to assist in advancing FTA’s mission and vision of protecting and promoting public health and oversees the agency’s engagement with Congress and its many global partners as well as state, local, territorial, and goal and tribal policymakers.
6:49
Andy joined the FDA in 2021 after nearly a decade in various capacities with the US Senate Committee on Health, Education, Labor and Pensions, fondly known as Help.
7:03
First as a senior advisor to Chairman Tom Harkin, and later two ranking member and chair Patty Murray. Andi, the floor is yours.
7:15
Well thanks so much Really appreciate that and appreciate the kind introduction. It’s an honor to be here today to join you all briefly to talk about the reauthorization of the Medical Products User fee programs. As this audience is likely aware of the programs which expire this year includes the user fees for prescription drugs or PDUFA.
7:35
Generic drugs are, could do for biosimilars or …, and medical devices or medusa.
7:43
We were told to spell out all our acronyms and for this talk, that when you really get started.
7:50
It’s hard to overstate the role These programs play in providing critical support for FDA’s mission to protect the public health and accelerate innovation, the benefits patients.
8:01
In FY 21, for example, 46% of FDA’s budget was funded by these programs.
8:07
Americans depend on the agency to review and approve medical products which meet our highest scientific standards.
8:14
FDA’s role in providing access to these products is critical to patients and healthcare professionals alike and to the entire healthcare market. I can say with certainty the FTAs, Medical Product Programs would not exist as they are today without these user programs.
8:30
In fact PDUFA FDA’s very first medical products user fee program, as you just heard was enacted 30 years ago.
8:37
A lot has changed in 30 years since then.
8:40
There’s no question that producer revolutionized the United States throughout the process.
8:46
It has reversed the lag and drug approvals that prompted its creation, providing Americans with more timely access to safe and effective medical products.
8:56
Before if it is the one the Media New Drug application, or NDA approval Time average 22.2 months.
9:03
Now in fiscal year 21, the median review time for original first cycle produced the applications was under 10 months.
9:12
More than that, PDUFA is not focused only on providing shorter review times for medicines, has also allowed FTA to provide more rapid feedback to companies.
9:22
This feature of the agreement, in and of itself, has resulted in faster approvals that give Americans access to drugs much sooner.
9:30
The producer was just the first of the FDA’s medical product user fee programs.
9:35
In the past 30 years, could do for the super medusa were enacted to address generic drugs, biosimilars, and medical devices, and there’s also drastically improved patients access to medical products that help enhance their lives.
9:50
…, for example, was enacted only 10 years ago.
9:54
Since then, close to 1001st generic medicines have been approved, offering patients access to drugs for which there was no previous generic competition.
10:03
FDA has also approved thousands of additional versions of generic medicines over this time, contributing to significant price reductions for consumers.
10:13
… was also enacted 10 years ago.
10:15
Since this is the one, the number of approved biosimilar products has grown to 33 biosimilars for 11 reference products, including two interchangeable biosimilars.
10:26
Together could do for embassy, has allowed FTA to provide access to affordable, safe, and effective medicines to millions of Americans who otherwise couldn’t afford them.
10:36
This equitable access to medical products is a top priority for FDA.
10:41
Since medusae wasn’t after 20 years ago, FTA has substantially reduce the median time to approve an investigational device exemption study.
10:50
Reduce the average time to reach a decision on a novel classification request, and has improved the medical device pipeline by ensuring that safe, innovative technologies are introduced in the US earlier than in the past.
11:03
These achievements across the medical products, user fees underscore the importance to the agency and to Americans, and the user fee agreements that we recently transmitted to Congress built significantly on FDA’s past successes.
11:17
I want to spend just a few minutes talking about what’s in those agreements.
11:22
seven focuses on ensuring FDA has capacity to review new and innovative products.
11:28
Specifically, the …
11:30
commitments include enhancing FTA’s capacity to support development, review and approval of cell and gene therapy products, and new allergenic extract products.
11:41
FDA has seen an 85% increase in Original Investigational new drug or IND receipts for these products, a 139% increase in IND Amendment Receipts, and 158% increase in formal meeting requests in the last seven years.
11:58
This commitment includes development of multiple guidances, public meetings, patient focused drug development meetings, and public outreach to facilitate product development and approval.
12:09
The commitments also include improving pre-market review processes and procedures, enhancements to the current system, and a new pilot program for certain efficacy supplements, but support review earlier, to expedite patient access to novel uses for existing therapies.
12:26
This also includes: new performance goals for pre-approval review of postmarketing requirements, and a new process for reviewing sponsor initiated requests.
12:37
Enhancing regulatory science to expedite drug development is another important focus of PDUFA seven.
12:43
This includes: continuing the successful model informed drug development and complex innovative design programs, advancing development of drugs for rare diseases, advancing real-world evidence for use in regulatory decision making, enhancing the use of digital health technologies to support drug development, and furthering the patient voice and drug development.
13:05
PDUFA seven would also enhance and modernize the drug safety system, including by modernizing and improving the risk evaluation and mitigation strategy assessments, and optimizing the Sentinel Initiative capabilities.
13:19
Under this agreement, FDA would also enhance product Quality reviews, chemistry, manufacturing, and controls approaches, and utilization of innovative manufacturing technologies.
13:31
Finally, PDUFA seven Commitments further review of combination products.
13:37
Does it is the three commitments focus on proposing new processes and procedures to achieve earlier cycle approvals and enhancing the free abbreviated new drug application, or pre and a program?
13:49
These are the three categories. First, Android assessments.
13:53
Here, the focus is on minimizing issuance of complete response letters, extending goal dates for minor issues, and timely regulatory feedback.
14:03
Second, designate a letter proposes expanding opportunities for early assessment of drug master files.
14:09
The agreement will also allow eligible generic drug facilities to request a post warning letter meeting to obtain preliminary feedback from FDA on corrective action plans, and third support for the pre and a program in regulatory science to help expedite complex generic drug development.
14:28
Finally, Pacific three expands upon and refines our current successful commitments and add some new features.
14:34
This includes enhancing pre-market review processes, procedures and performance by proposing new supplement categories, review timelines and performance goals, improving communication and feedback during the development process, and a new follow up opportunity for sponsors.
14:51
Pacific three would also enhance bio similar and interchangeable product development and regulatory science.
14:57
Under these commitments, the agency would issue guidance on use of alternative tools to assess manufacturing facilities and pending applications, notify sponsors in advance of facility inspections when there is a need to see a product being manufactured, An advanced review processes for file similar biological device combination products.
15:18
Importantly, collectively, these three agreements, but also improve hiring and retention of key, scientific, and technical talent.
15:25
That’s really key to all of these programs, enhanced information technology, and strengthen management of user fee resources across the board.
15:35
I’ll say Just a word about …, I know that many of you have been waiting for an announcement anarchy negotiations on Medusa five which are ongoing.
15:43
When those negotiations have been finalized, we’ll communicate with the public about the agreements reached through that negotiation process.
15:51
The user fee programs have revolutionized medical product review and approval without them hundreds of treatments or cures for life-threatening diseases may not be available to millions of Americans.
16:02
These programs are critical to FTA’s fulfillment of its mission and to the public health.
16:07
Critical to have been the many policies advanced by Congress, which have passed as part of user fee reauthorization packages over the years.
16:16
Many of these so-called policy writers have given us important authorities which further support FDA’s mission and getting treatments and cures to patients.
16:25
This includes many improvements to pediatric product development, the critical supply chain provisions, aphasia, or the enhancements to the generic drug programs and Fedora, FDA’s programs, and indeed, the public health, would look very differently today without these important policy advancements.
16:43
Finally, transparency and public engagement are important aspects of the user fee negotiation process.
16:50
Our success, both in the past and the future depends upon collaboration and communication with stakeholders, policymakers, and industry, which is why I am so glad to stop by this meeting to re-iterate FTA’s commitment to these programs. Thank you so much for allowing me to join you today as you begin this important conversation.
17:10
Thank you very, very much, Andy, for taking the time today and for your insightful comments. Your words are perfect in helping us frame today’s conversation. And we’ll now let you get back to your Jerry Jerry, busy schedule. Thank you again for joining us.
17:27
Thank you.
17:29
Now, I’d like to quickly introduce you to our esteemed panel of experts. Each of these individuals has had a front row seat when it comes to the FDA and the various user fee acts.
17:44
Our first panelist is Jeremy Sharp.
17:47
Jeremy is the Managing Director at Waxman Strategies and brings, to us, a vast, vast policy experience from both the Executive and legislative branches of the Federal Government. He has served as Deputy Commissioner for policy, planning, legislation, and analysis at the FDA, and both has counselor to the Secretary for Science and Public Health and Deputy Assistant Secretary for legislation at HHS. Before working in the executive branch, Jeremy served as the Legislative Director and Senior Health Advisor on the Senate Help Committee.
18:27
Our second panelist is John Stone, John is a principal at BGR Group and serves as co lead of the FDA and Public Health Working Group within BGR Health and life Sciences practice.
18:43
John joined the firm after spending nearly seven years, with the House Energy and Commerce Committee, first, as a Counsel on the Oversight and Investigations Subcommittee, and then, as a Senior Counsel on the Health Subcommittee, primarily handling issues under FDA’s jurisdiction.
19:03
Our third panelist is Anna Abraham, and is currently a senior advisor at Akin Gump.
19:11
Anna joined Akin Gump after serving as the Deputy Commissioner for Policy Legislation and International Affairs at FDA.
19:21
Prior to joining the FDA, Anna held roles within the Senate Help Committee and was appointed to serve as the get as the associate director of the Domestic Policy Council at the White House. Our panelists will now introduce the topic at hand, and after their presentations, we will get to a question and answer portion of today’s event. So, Jeremy, first, let’s start with you.
19:51
Thanks. Thank you to the Alliance for hosting this, and for all of you, for coming. I want to open with a little bit of an inside joke, which is to say, it’s fun to be on the panel where the two people that succeeded me in the job, I had an FDA are on the panel as well.
20:07
We have Andy, obviously, I’m going to speak who has the job presently, and Anna who replaced me in that job in 20 17.
20:15
So, John, I think this might give you a clue where your career’s going someday, and I think you probably may agree deputy commissioner policy at some point. But, but let me get on with what’s actually, you’re all here to talk and hear about and talk about.
20:30
Yeah. Exactly.
20:35
My role here is to just give a quick layout of what the user fees are, why they’re important, and how the administration goes about the process of using them. And then ultimately reauthorizing them to key up. So that congressional debate, this is, that is about to begin here in Congress. And it’s a great panel to be with, because we can all add a lot to that, all of that conversation. Andy covered some of this, so I’ll try to be efficient and not retread too much ground.
21:06
Very succinctly, user fees are the backbone of funding for FDA’s review of all medical products. Without the user fees, the modern FDA simply doesn’t exist or function. So there’s no there should be no question, but these huge fees must continue and they must keep: they must be funded and re-authorized by Congress by the end of this fiscal year.
21:29
Um, the resources that user fees provide are substantially impact FDA’s ability to be to be most effective on the regulation of medical products, and outside of the scientific strength or weakness of the applications FDA receives, or the strength of the science in the field that are backing up those applications. I think that the revenue streams that FDA receives from user fees and from appropriations are the single biggest indicator of how effective FDA can be, as they continue to carry out their mission to protect us, and to facilitate access, to needed drugs and devices.
22:10
And very succinctly, where FDA gets user fees, FDA can do substantially greater job.
22:16
In the areas where there are no user fees of FDA, those are the areas where the program staff and the program leadership has to prioritize more ruthlessly have to be faced with much harder choices about where they spend their time and energy.
22:30
Because because if they if without those user fees, without the revenue, they are sometimes not able to focus on that.
22:36
And so I think that’s something we need to keep in mind, as we’re thinking about these user fee programs in a reauthorization year.
22:44
And finally, the negotiations between industry and FDA.
22:49
And then ultimately between members of Congress and the stakeholder community are, are the place where everything is decided that hefty spends the money that comes from those, your user to use. And there’s a substantial push and pull that happens there.
23:04
And it’s important for people who want to understand what FDA works, to pay attention to those negotiations, and understand that pushing, because these resources are so central to FDA’s work, which is frankly an obvious statement.
23:17
They’re also central to what I think are the two biggest questions about FDA policy in general, and that have pervaded every FDA debate for the last decade that I’m aware of.
23:27
one is how much of a gatekeeper should the agency be, in terms of drugs and medical products? And how much, how should they balance the need to be protective of the public protective of public health?
23:40
Versus then, the consideration of what is the access to market of experimental products, with or without rigorous testing, those balances, and those questions are underscored by the user fee debate, and influenced by.
23:56
I will say, well, there’s, there’s some people who would suggest that the user, the user fees may create imbalances in that process. I don’t think the user fees are the cause of those questions, and, And, and that’s a distinction we need to understand. But I will talk more about a little bit about that in a little bit. I’ll skip over a little bit of the funding questions, because Andy cover them really well. But very succinctly, one thing that I want to make sure people are aware of is for the human drug regulatory operation, about 65% of the budget for the human drug process at FDA is funded by user fees. And that gets even bigger when you’re focusing on the Prescription Drug User Fee Act, where it’s, it’s more like 80%. So, there’s a substantial amount of revenue that is there a reason, And they are the core to the work.
24:46
As Andy said, these these program started in 19 92 with Purdue for. They’ve been expanded since then. They’re negotiated and renegotiate every five years.
24:55
And then, after those negotiations happen, the industries that are regulated by FDA, plenty of resources that FDA then uses to ensure that the products they’re reviewing are safe and effective.
25:07
The reason for the User fee agreements back in 19 92, and throughout the intervening years, has been because, frankly, Congress, and the administrations of both parties, have not been willing to appropriate the level of funding necessary to fund the agency.
25:22
And so it’s really important to understand that there is, this is not something where you can have a choice. Oh, should we have these risk, or shouldn’t we?
25:30
You can’t, because Congress won’t appropriate that level of funding to this agency to carry out its mission.
25:37
Beyond the basic medical product, user fees, FDA has user fees, a number of areas that this Webinar is not about. But I just want to make sure people are aware of them. Tobacco products, animal drugs, over the counter drugs. There’s a variety of places where FDA takes that revenue. But we’re here talking about the medical products and particularly the giants in this place.
25:56
Within the user fees, there are a few different kinds of user fees that are collected. As I said, these are negotiated …, five years. Structure changes, the amount changes from cycle the cycle. But ultimately, the categories of user fees that the industry pays, our application fees when they submit an application to FDA, There are a few different types of these program fees for when they have a development program.
26:20
Or they have a prescription drug program, and then established under facility fees that help resource FDA’s understanding of those of the manufacturing and the development facilities.
26:36
A few clarifications of some of what is, what is and isn’t covered by a user fee.
26:43
The expenditure of the user be funded by FDA is not specific to app to the applications they come and associated with. They are, they are aggregated into a pot of money for prescription drugs and aggregated into a pot of money for medical devices. You’re hitting into a pot of money for generic drugs and so forth. And they are used to fund the regulatory program the higher the staff. They pay for the IT.
27:05
Andy rattle off the various things that the goto, but they’re not tied to say pfizer’s application for extra. The Pfizer pays its fee for the fluid submits an application but it doesn’t get in compensation for that specific things that happen on its application, it gets no guarantees, but with that application, will meet all these goals.
27:26
That’s because this is also tied to aggregate goals, and an example of a goal, performance goal.
27:35
The FDA uses for this, is that they will complete review and final action on 90% of applications within 10 months.
27:41
So, that gives you the flexibility to decide, OK, this application is more complicated, or they’re real the real public health questions that we need to understand. Or the data here isn’t strong, so we’re going to choose a different path for that application, But it also ensures that FDA is moving along very rapidly and focused on overall performance. And making sure the management of this program is at a high level. The expenditures is also not associated with the approval product. It’s all about final actions, or it’s about process steps along the way.
28:14
This is not, there’s no guarantee that a drug company pays a user fee, that it will get a drug approved at the end of the process.
28:22
What it gets is process, and that’s, that’s a really important thing.
28:25
To understand about this, the user fees allow the industry to finance FDA’s activities, but they don’t, they are process oriented. They do not let that dictate FDA’s policy.
28:37
So with that kind of flexibility, FDA has a lot of ability to manage this. There’s one other caveat I wanted to flag about user fees that is sometimes not well understood.
28:48
They are tied to something called the appropriations trigger when Congress established this, this program that they thought it was really important in various sites, thought it was very important that that the user fee dollars not replace taxpayer dollars that were being appropriated to FDA. They didn’t want to see a backslide in this, and this has been important also in the new user fees that were created.
29:08
I know, especially on the generic Drug User Fee Program, in the second round of reauthorization, the industry is very concerned that they wanted to make sure that that trigger was high enough that they felt like they were not just paying the whole boat of the regulatory process.
29:24
And that appropriation trigger does increase from year to year based on inflation.
29:31
But even with that appropriation trigger, we have seen the user fees represent a greater and greater percentage of the revenue. that covers And the funding that covers the regulatory program.
29:40
And we’ve seen, and we’ve, frankly, seen some discussion about whether or not we should go one direction or the other.
29:48
It was at least rumored in the Trump administration that they were going to push to have the government, the FDA, completely funded by user fees and have no federal appropriations for it, I don’t know that actually ever got from proposing the budget. It clearly didn’t go anywhere in Congress. There’s also people pushing the other direction, wanting to lessen the role of VCs, and increase the appropriated spending. These are the kinds of debates that surround the use of the re authorization from year to year.
30:17
A quick note about what’s the process by which the administration develops the user fee agreements and brings them to Congress for the preparation for the re authorization process. Very quickly, every year, the FDA publishes annual reports on its performance metrics tied to the user fees and is expenditures times the user fees. These are really useful documents. For those of you who really want to dig in and understand what’s happening year to year, the wonks, on Capitol Hill, and in the stakeholder community who are paying attention to take a look at those reports every year and try to understand the direction of the program and the issues that are involved. Beyond that, we think of some of us on the outside think of the reauthorization process is being a one-year process on Capitol Hill about John’s gonna talk more about what’s involved in that but, but in truth, it’s really more like a three year process that has started two years before that.
31:11
In two years prior to the need to submit the the Agreements to Congress, the Administration start sorting through the agency. Start sorting through what are its priority is going to be and it’s upcoming negotiations with industry where does it need to expand the program? What does it need to make improvements, where they were, they had failings in the in the performance of the program. And what should they be hoping to ask the industry for? They’ll do some consultation with industry and stakeholder in that process. Then the, then after that happens, you start to have public meetings and open dockets so the end congressional briefings, so that FDA can receive input from various players about what those priorities should be. And then and FTA identifies its internal ratifiers, the senior officials who will sign off on or ask questions about the negotiations as they are progressing. And then, ultimately, the negotiations began.
32:01
And in a change that’s happened in the process of this over the years, partly because of work that Anna and others have done, is that those, the negotiation process is more public than it used to be. The meetings themselves are not public, their meetings between industry and FDA. But the minutes of those meetings are generally published shortly after the meeting, so the public can be aware of what’s happening in them. Alongside of those negotiations sessions, there now stakeholder engagement meetings where FDA breached the stakeholder community that’s interested in what’s going on in those negotiations, receives feedback on whether they are going to incorporate that feedback into their thinking.
32:34
It’s not a perfect process. They’re probably improvements that can be made from year to year. And they’re probably variances from cycle the cycle about how effective that process is, drawing in the feedback. But it is there, and there is a public engagement component to it.
32:49
Finally, once negotiation happens, the clearance process, FDA, HHS, and OMB, I’ll get there, say that can be a little harrowing inside. And then they’re publicly published for comment. And then they’re submitted to Congress in January, unless the medical device user fees, which are almost always published late and submitted late because that negotiations, always fraught.
33:11
Last couple of thoughts, and then I’ll hand it off.
33:15
When we’re thinking about user fees, I’ve highlighted one questions, should they Shouldn’t they exist? And that’s when it’s there. I think one question that comes up is, do user fees really lead to industry capture the agency, I think that’s an accusation is made.
33:28
And I will just put a finger on the scale and say, I don’t believe that they do lead to industry capture.
33:33
They are one element of the interaction between industry and FDA, but frankly, I think a much greater influence on the agency’s design Policies and thoughts about how they are going to, um, what standards are going to hold industry to in the applications they receive is, is where congressional and political pressures and congressional pressure. It has all, as, has for the entire time that I’ve been working on this, until the very heavily in the direction of more innovation.
34:01
more faster reviews, impatience with the process and steps, and that’s been also paired with somewhat, not consistently.
34:11
A very low tolerance for failure when FDA screws up and approves sending it shouldn’t or allows a drug on the market.
34:17
There are a couple of things that people should watch for, is there, is this process, symbols.
34:22
Watch out for the linkages between the user fees and approvals. That kind of linkage is where you start to get in trouble. Second, what happens to the timelines in these user fees? Are they getting shorter and shorter over time, or are they starting to stay consistent? That can add pressure to the process at FDA, and then what other issues or surface within them?
34:40
Like I said, usually won’t direct dictate FDA’s policy. But they’re focused on things like real-world evidence, the patient voice, breakthrough drugs, and devices, biomarkers, surrogate endpoints. The idea that the user if you say that those kinds of issues, we made an guidances.
34:55
Those that gives you some indication of what the direction the agency is being pushed to go.
35:00
And it is something to pay attention to if you want to consider how the user fees impacted. So, with that, I’m going to hand it off, sorry for talking.
35:08
Jeremy, thank you very, very much. Now just handing the solver to johnstown, John, all yours.
35:16
Great. Yeah, thanks for having me.
35:17
I’m I will be talking about how the sausage is actually made.
35:22
So once once industry and agency finalize the agreements and they go through the process that that Jeremy laid out, those those commitment letters will be sent to the Energy and Commerce Committee and the help Committee in the Senate, which are FTA’s authorizing committees.
35:41
Those committee staffers will have been briefed by FDA and industry have as those conversations play out.
35:50
But once they’re finalized, they’ll be sent to the Hill. They need to be transmitted by statute by January 15th.
35:56
But as has been discussed, medusa’s still outstanding.
36:02
Proposed legislative language is also sent to the Hill that would amend the statute.
36:07
Authorizing FDA to collect those fees for the next five years.
36:12
Setting out the new fee structure of the year every year amounts in any sort of reporting requirements that, that are included in the agreements.
36:21
It’s important to note that everything agreed to in the, in the negotiations, in terms of new activities, like pilot programs are, are new guidance documents that the agency agrees to issue must all be done within current statutory authority.
36:39
So once once those agreements are sensitive to how the legislative language is proposed, Congress doesn’t typically substantively alter the agreement’s or or the language.
36:51
Those four pieces, Purdue can do for the super amadou farwell become the first four titles of the legislative package that needs to be signed into law by by September 30th.
37:08
The legislative process always differs a little bit. Cycle after cycle.
37:14
The House Turley kicked off their process, an honest yesterday, with, with hearings on the three drug agreements, where the Cedar and Seabird Center, directors testified on a first panel on the hearings and stakeholders testified on the agreement’s on a second panel.
37:35
Help has been a lot, not entirely clear when they’re going to have there, Their kickoff hearing.
37:42
They typically have had all four agreements at one hearing where the center directors will testify, and then a subsequent hearing where stakeholders across the industry and patient and consumer communities will testify, but given that manure is outstanding, you know? I don’t know exactly how that’s going to play out over there.
38:09
So, I guess in parallel, or ongoing as, as the two committees are, are having hearings on the actual agreements, there’ll be: there’ll be FTA related bills that have been introduced.
38:25
They’ll likely be a hearing or hearings on these discrete FTA bills that members are trying to have included in this … package, right.
38:35
These are the so-called policy riders that, that folks have referenced, you know, in the house.
38:44
Historically, in order for policy writer to really be debated and considered as as as something that could be included into the user fee package. It needs to have been introduced on its own. Right.
39:00
So, a lot of folks have referenced curious to point out, there’s a lot of various provisions. Some touching, FDA, some unrelated to FDA and carriers to point out.
39:09
You know, for those FTA provisions to really be included or considered as something that could be included, they would need to be introduced separately. They really need to be bipartisan to be considered. I keep referencing the need for them to be FDA specific. That’s not necessarily a hard and fast rule.
39:30
There were some controlled substances related provisions. I recall in 20 12, for example, and they really need to be non controversial.
39:38
It doesn’t mean they need a stark without controversy or various, you know, without folks having various views on them.
39:47
Some, sometimes, you know, quite different views, but ultimately they need to get to the point where they’re relatively non controversial. And there’s no buy in on both sides of the aisle on both sides of the capital because you know that the first order of business is to get these user fee programs re-authorized well in advance of this september 30th deadline, Everything else is kinda icing on the cake, Anything that could get in the way of votes. And either Chamber is not going to ultimately be included.
40:21
So, again, the processes in the House and the Senate vary a little bit based on the rules, and just based on, you know, the cadence of either Chamber.
40:30
But, um, they’ll, there’ll be constant communication between the Energy and Commerce staff and the help committee staff about the issues that, that both sides are are working on.
40:42
Typically, they’ll be there’ll be a lot of overlap or there could be some priorities in the House that are not as big a priorities in the Senate and vice versa.
40:52
But, you know, typically these issues will either have been introduced as bells in both chambers or will be, you know, significantly discussed and and, and both chambers, um, then then the markups occur. And that’s where it really misses all packaged together.
41:10
So, on the house side, there’ll be a subcommittee markup and a full Committee markup date, Some, those TBD.
41:20
Know, we’ve heard that the House wants to, you know, have all of us on, on the floor around Memorial Day.
41:27
I think what we do for outstanding that timeline will likely slip, but we’ll see.
41:32
So, again, at the Subcommittee markup’s work is where they will pull those four, ah, user fee components together, first four titles of the bill.
41:45
And it could be an opportunity for there to be policy riders that are better pulled in that were discussed at previous markups, that might have to wait until full committee.
41:58
But by the time, Energy and Commerce reports out, it’s product from full committee for, for consideration.
42:08
A lot of, you know, what we call pre conferencing.
42:12
Well, it will have occurred between the House and Senate.
42:15
The goal here is four: No, both, well, for each chamber to have to only vote on this wants. Right?
42:23
You know, there’s always going to be a little need for clean up after the markups given, given no differences of opinion between chambers or just technical issues that have arisen, But all that will occur, prior to that, that house floor vote, which will, which will likely occur before the Senate.
42:45
The Senate doesn’t need for procedural reasons to help committee at least, and Anna can speak, you know, more to this, but they don’t typically need to have as much public facing process. As energy and commerce does, they’ll likely be just one markup.
42:59
That’s kinda pre orchestrated, for lack of a better term, Before they, you know, they vote their package out, and again, you know, both committees, product is kinda look pretty similar.
43:13
With the goal of just avoiding multiple votes, and in each Chamber of Alaska, around in 20 17, the whole package was voted off the house out House floor, with a voice vote.
43:25
On the suspension calendar, where you need two thirds vote of the chamber in need, even, to, you know, have it on suspension. I don’t think that’s going to happen this go around, the likely will need to be a recorded vote.
43:40
There are a lot of new members in the house, in particular who, who were not here five years ago. So, the floor vote is certainly going to be interesting. And I think that, you know, that dynamic will, certainly influenced the scope of the package.
43:55
I mean, the bigger the package gets, you know, beyond just the user fee components, the dicier that house for vote gets. So, I do think that this will be a relatively tight package with respect to ancillary matters being included.
44:12
That being said, there are a lot of, you know, policy writers that are already out there, some of which have been introduced, some of which likely will be introduced.
44:23
A lot of these issues were discussed at the energy and Commerce hearing yesterday, obviously a lot of noise around X potential accelerated approval reforms a lot of conversation around real-world evidence.
44:37
You know, building off of what was in 21st Century Cures Fedora Components of this this Paducah Agreement, lot of conversation around inspections and recall authority.
44:52
And then there are these these so-called super riders that folks are discussing, not not necessarily in the drug side, but the valid Act has certainly been, you know, a subject of significant activity and today that would that would comprehensively reform how diagnostic tests are regulated. Cosmetics, reform, dietary, supplements, reform. I mean, all of these are pretty big ticket issues. Each, each of which have a lot of different views.
45:24
You know, folks have a lot of different views on. So, yeah, that’s, that’s a lot of work for, for the committees to, you know, to, to get done between now.
45:34
And when they want to actually get, you know, best legislation to the President’s desk, not saying it can’t be done.
45:41
When there’s a will, there’s a way, and, you know, particularly with Senator Burr being the lead, um, Republican sponsor of the valid Act.
45:50
I certainly you know what wouldn’t dismiss that effort at all? So, so I’ll stop there.
45:59
You know, again, a lot of this is Crystal Balling, but based on how past cycles have gone, that’s, that’s more or less how I envision the next few months playing out.
46:10
Well, thank you very, very much, Jonna, we always like to hear about how you make sausage. And now I’m just going to turn this over to Anna.
46:22
Great, Thanks Amelia, for that introduction, and thank you to the Alliance for Health Policy for hosting today’s panel.
46:28
So I’ve been asked to kind of focus on reflection.
46:31
So what have we learned in the last 30 years since Congress enacted the Prescription Drug User Fee Act, which is commonly referred to as dupont as you’ve heard today.
46:41
And why does the reauthorization of Paducah matter?
46:47
Mission is to protect and promote public health. And the agencies either be funded medical product activity is an integral part of fulfilling a public health mission for patient and consumer.
46:57
And because of the critical role, the use of the program plan, supporting the timely review of medical products, they are understandably a great deal of interest and the reauthorization of the program, because of what they mean for patients, industry, the agency, and public health.
47:14
Congress is now considering produced seven or the reauthorization of the law. And for some of you, this may be the first time you’re learning about SBA programs and the role of the reauthorization played in the development and review of prescription drugs, generic drugs.
47:31
And by a similar biological product.
47:34
There is a lot at stake with the reauthorization of these programs, and that’s why they go through such an iterative process and receive the broad attention they do from so many stakeholders, Fashion industry, agency, Congress, and many other.
47:50
Over the years, Congress has enhanced the transparency of reporting and the opportunity for public input in the reauthorization process and respond to the high level of interest in the user fee program.
48:03
Herron to the use of the program and a process by which the program already authorized is a recognition that the time it takes to develop, review and approve.
48:15
They’ve been effective medicine, impacts public health.
48:18
Unnecessary delays in the approval of safe and effective products can adversely impact patient.
48:24
The patients are highly vested in the time it takes for safe and effective therapy to be developed, reviewed and made available.
48:32
There wasn’t part of response to concerns about the time it would take for FDA to review drug applications, and the level of staff and resources needed to support.
48:41
More timely and predictable revue was a direct response to the concern for patients of potentially promising drive for segregated and the experience of the aids crisis or the turning point in catalyzing reforms to address these concerns, as well as, the agency’s tool with respect to unmet medical needs.
49:02
The years that followed the enactment of the user key resources enabled a more modern and efficient approach to FDA review of new drug applications.
49:12
And in a manner, consistent with that, the gold standard, could put a finer point on that, before produced, by the time it was taking FDA, to review and approve new drug applications, with an average of more than 31 months.
49:25
And you can track that timeline to the performance goal in the Purdue for seven commitment level, which include the goal for FDA to review and act on that new drug applications, and pick one for Priority Drug 10 month for standard drive.
49:41
Per patient facing a difficult diagnosis, the difference between 39, 6 months.
49:48
It’s even more pronounced.
49:50
The one key takeaway as reauthorization considered is that produce a demonstrated that there was public health benefit to aligning resources to the agency workload.
50:00
And this came in the form of the user fees, supplementing, not supplanting general appropriation.
50:06
And with this experience in mind, 10 years ago, Congress enacted user fee program for generic drugs.
50:13
And biosimilars with the purpose of aligning resources to workload to support a modern and efficient review process for these products.
50:22
The fact that Congress is now considering produce 7 3 3 is a testament to the integral role of the CIP program and public health benefit they are provided.
50:35
Congress could have decided not to reauthorize the program the previous year and then pursue another legislative framework for supporting activities but that’s not what has happened.
50:47
And since the enactment of Paducah and the Super Congress has reauthorizing programs and in doing so also advance FDA related reform as part of the reauthorization legislative packages.
51:01
Another key takeaway is the importance of regulatory certainty, which is important for everyone involved in the development and review of medical products.
51:11
User fees have fostered enhanced regulatory certainty with respect to the review process, for industry, patients and stakeholders, as well as the agency.
51:20
And while user fees support review activity, as you’ve heard from others on the panel today, they do not determine FDA regulatory decision.
51:30
The path to bringing forward a safe and effective medicine is not just contingent upon the time at the correct date of review, a new drug application, biologics license application, abbreviated new drug application, or by a similar or interchangeable, by a similar application.
51:46
Drug development reflects scientific advancement, challenges and opportunities.
51:51
And one of the ways that FDA can help to enhance regulatory certainty is to issue guidance on various topics that can inform the development of products.
52:01
Commitment to issuing guidance to share the agency’s current and best thinking of various topics have been included in commitment letter.
52:08
Produce a performance goal for the review times of drug has decreased, stabilized over the course of the previous three authorization.
52:16
There has also been the integration of commitment regarding the timing and type of meeting FDA will hold with industry as part of the development and review process, as well as the incorporation of commitments to issue certain guidance document within a certain timeframe.
52:32
These types of actions can also help to enhance regulatory certainty to the process of bringing forward products.
52:40
one of the defining feature of the reauthorization process that the agency and the three patient stakeholders and Congress come together every five years to reauthorize the program versus other public health programs that do not have a similar mechanism for reauthorization.
52:58
The current program, being set to expire at the end of the fiscal year lends a particular urgency, a sense of must path to the reauthorization process.
53:08
The five year reauthorization cycle set up a regular cadence for the agency, industry, Congress, and stakeholders to come together and examine what is working well and where there is room for improvement with the goal of optimizing these programs for public health benefit.
53:24
These discussions are directly informed by prior year’s experiences with the program.
53:30
This theta is also very important because of the nature of scientific advancement.
53:35
The question of what modern and efficient development and review looks like today is in some ways different than what it looked like 30 years ago or will likely look like 10 years from now.
53:48
The agency has to be prepared for whatever comes before it, and that includes the ability to keep pace with the product, coming before the FDA for review, and having the workforce necessary to support the anticipated workload.
54:02
This is one reason that hiring is consistent, continual focus across users, the reauthorization.
54:09
The dynamics of a five year period in-between reauthorization, coupled with the pace at which science is advancing, enabled an ongoing dialog about how best to support the work in these areas as part of its public health mission on behalf of patient and consumer.
54:27
Because the re authorizations are considered, must pass.
54:31
The bill that carries them is often an attractive vehicle for advancing related policy writer.
54:38
Every member of Congress have patients as constituents.
54:41
How the final bill comes together is the reflection of member priority, as well as the need to enact the reauthorization before the current programs that fire in order to avoid any programmatic disruptions that could adversely impact.
54:57
So the process of reauthorizing youth programs is fundamentally about answering the question of how to have the most modern, efficient, highly development, and review of medical products, so that patients have the potential to benefit from safe and effective therapy as soon as possible.
55:15
And that will bring me to where I began, my remarks, which is to say that there’s a lot of things with each reauthorization, first and foremost, for patient and public health.
55:28
Thank you. Thank you very, very much, Anna, and I want to thank our panelists for their opening remarks.
55:38
We’re now coming to the question and answer portion of our webinar. Again, we encourage everyone in the audience to please submit questions in the chat feature, and as we’re compiling those questions, we’re just going to start the question and answer period period immediately, as questions come up.
56:01
And we’ll take them one at a time. Jeremy, this question is for you.
56:09
And then I think you could also contribute here, I think, actually, so can John.
56:13
Why should members care about, to do so, why does, why should they care about that? What’s the so what?
56:22
Yeah, The first thing, I think, and I think all three of us have touched on this, but just to underscore it. At a fundamental level, it is …, in particular, but it’s true of all uses.
56:34
This is this is the centerpiece of funding for the FDA and its work. And as Anna said, you know, that it has had dramatic impact and positive impact on the agency in terms of its ability to respond to applications that are coming in and facilitate products, making it to market, that are needed and wanted by patients and consumers. So at the most basic level, that’s really important.
56:56
But it’s also becomes a roadmap, or what the next few years and policy development is going to be, Where are the discussions going to be? Even though you don’t within the four corners of the user fees defined policy, you set direction and you set you, underscore the areas you want to focus on, and you tee up the legislative vehicle that John was talking about that, just curious so many other policy discussions with it. So this is going to be an engine that basically drives FDA policy for the next five years. And we will derive legislative discussion of that policy for the next eight months.
57:30
I know, would you like to add to what Jeremy just said?
57:34
Other than, to kind of further expand upon what I noted in my remarks, as, you know, ultimately, all this work comes down to what’s the impact patient, know, in terms of where there’s unmet medical need. Or the ability to have more competition, perhaps. Because you’ve got more products to be able to fund, depending upon which program you’re looking at.
57:54
And so, kind of coming back, I think, fundamentally, why does it matter?
57:58
For all the reasons kinda outlined before, But, again, it comes down to the time and path for those products that are going to meet FDA standard, the time it takes them for them to be potentially available to patients, and that’s, that’s a big deal.
58:15
That’s a very big deal.
58:17
And I would just further add, to put a finer point on this, and to build off of what Jeremy said around, you know, if you were to contemplate, suddenly not having these resources, and not having that piece of the intent, if you will, what would that look like? And when you start to contemplate that, it kinda comes back in my mind to why producer was established.
58:44
Now, on the 30th anniversary of that, it was to construct a process that lent itself, not the pre-determined outcome, but to greater certainty around the process, so that it could be more streamlined.
58:58
Well, let’s just take a moment and just also try and discuss the the elephant in the room.
59:03
We know that three decades ago, user fees were implemented And in 20 22, they generated almost $3 billion for FDA. That’s real money, OK? Critics?
59:19
Have and continue to raise concerns arguing that these funds by influence and as a result, FTA is not as independent as it could be. And drug safety and efficacy suffer as a result.
59:36
On the other side of the coin, proponents, as we know, proponents say that there will be untold delays for critical new therapies to get to market without these user fees, countless individuals would suffer unnecessarily as well as other alcohol undesirable downstream effects.
1:00:00
Is there a way or should we be considering better ways to balance these competing priorities And what measures are currently in place for balancing these tradeoffs. John, I’ll, I’ll go to you first.
1:00:21
Yeah.
1:00:21
I mean, you know, I think, regardless of how you get there, everyone benefits from a strong, efficient, nimble agency that can keep the pace of science, I think.
1:00:35
Irrespective of the funding mechanism, there is constant oversight from Congress of the agency. They get various viewpoints on what’s going, right.
1:00:46
What’s going orally, where there needs to be specific attention and discussion around the need for change, divorced from the user fee mechanisms, quite frankly.
1:00:57
And so, I do think the, You know, that the The oversight of the agency is strong, um, aye.
1:01:07
No, I think that conversation around, you know, regulatory capture or the right balance is you know, often.
1:01:20
Hyperbolic I haven’t seen it. I was at the Oversight Committee. I was on the authorizing side. It’s there, there’s a healthy tension there. There is robust and ample opportunity for congressional involvement, and I think everyone wants in Australia, FDA.
1:01:39
So, thank you, thank you, Jeremy, I think. Yes.
1:01:44
Yeah. I mean, first of all, I wanna say, I think, I don’t think there’s a culture at FDA being too close to industry, or, in general, I think, I think FDA is, is well designed as an institution to resist political and external pressure.
1:01:59
It is very, like, when my experience working there after working on Capitol Hill, was that the, the personnel there are deeply committed to reviewing science and evidence, and basing their decisions on that.
1:02:11
So, there, obviously, outliers are obviously things that happen. And I don’t think the user fees themselves alter that equation much.
1:02:20
Well, what I would say is, in addition, that is crucial.
1:02:23
Everyone knows that industry can’t afford to lose the user fee program either, right? So it’s not like industry has this stick that they can hold over FTE’s head and walk away from. So it’s not actually an effective tool for forcing FDA to behave in a certain way on an, on any given regulatory decision.
1:02:42
But the user fees do create a timeline, pressure, right? Which is, if FDA is trying to make sure that 90% of the applications that come in are completed within six months or 10 months, or whatever, the humans. That creates a mentality of, OK, we gotta move.
1:02:56
But generally, that’s not a bad thing like that, as Anna And John pointed out, that that accelerates the process progresses of treatments and medications to the public. But in specific cases, it can become a problem if FDA isn’t programed well to manage that.
1:03:13
And that’s where I think it is really important for Congress to balance its messages to, to FDA. between the yes. We want you to move quickly, yes. We don’t want you to be unnecessarily in the way, but we also don’t want you to get this wrong.
1:03:25
We want you to make sure you’re making the right decisions. And I’m not sure that balance is always been exactly right in the communications from Congress and from the political appointees in either in either party right now.
1:03:35
This is not a partisan pieces, both parties at the same message so and that alters things.
1:03:43
Thank you very much, Joel.
1:03:45
I’ll just jump in here To build off of my Jeremy touched upon and I also did, and my remark by opening remarks around.
1:03:54
If you look at the iterations and the evolution of the user be re authorizations and kind of a bygone, this cadence evolve as we evolve.
1:04:03
Over the past 30 years, a very thoughtful and concerted effort to expand upon: know the opportunity for public input, right? And, and transparency, and accountability. And reporting around that, to threaten, to know a bit open.
1:04:19
Aperture, if you will, around, you know, what are these?
1:04:22
What are these resources being put toward? What the agency’s performance? And I think that that’s something that we’ve seen, kind of each additional reauthorization, has built upon, the one that has gone before. And the spirit of is there more opportunity for that and what would that look like?
1:04:40
Thank you, thank you very much. Another question.
1:04:44
We heard a little bit earlier about medusa and the delay and Medusa and wanted to see if we could have a little bit, a few more details on. What’s causing that delay is, there’s just more controversy around with tufa.
1:05:00
Jeremy, I saw you shaking your head, so I’ll call you first.
1:05:06
So, with Kevin, I don’t really, I don’t know if either of my colleagues have any insight into what’s happening on the inside of the negotiation. Medusae in my experience has always been the problem child renegotiation.
1:05:18
It’s a more tense negotiations, I think the industry as a whole is less confident of the value of the user fees to them. And what I mean by that is, I think, I think the industry as a Whole is believes there’s value to paying these UBC. But, I think there’s sometimes disagreement about what’s the marginal value of each increase, in user. And I also think there’s some differences in the way, the Center for Drugs Center, for biologics and the center for devices have approached their negotiations in the past.
1:05:46
And I think, occasionally, the senator devices comes in with the, a longer list of asks that, that needs to be worked out between the industry and in the, in the center.
1:06:00
Lastly, I’ll say, I think, I think there may be less unanimity within the industry about where they want to go on this. Then there may exist in some of the other industries. We saw this a lot, actually, with the second line of …, where the industry had to sort of figure out where it wanted to be. The third round of Voodoo has gone to my mind sitting very, very smoothly. So, there’s, there’s differences in development and timeline for different industries, but, I’d love to hear what others say.
1:06:25
If they have any thoughts done.
1:06:29
Oh, I’m sorry, I had a call, please?
1:06:32
John? Go ahead.
1:06:33
I mean, I’m not privy to the state in the negotiations.
1:06:37
I would note that, uh, know, that the …
1:06:41
didn’t reach the ****, I don’t think until March, or maybe even April in 20 12, and, you know, at the end of the day, as well, it ended, well, I think everyone service there, but, you know, there’s time, it’s done.
1:06:57
I was actually going to share the same observation that there have been prior reauthorization cycle, where letters had not come up in January, and you know, carried off, carried forward.
1:07:08
But I would also make the observation reflected in the commitment letters that have gone to the Hill, that are, is that each of these programs, you know, prescription drug, generic drug, are similar. Then, you know, devices as Well, they’re each their own. And so there are considerations that may transcend across all of these programs. And you see those themes consistently around say, hiring and retention and from the reporting and the patient involvement the patient voice.
1:07:42
But you then also be individual commitment letter that are tailored to you and reflective of each of the programs in its own way.
1:07:52
Thank you very, very much. We just received a question from the audience that they would like a little bit of a further explanation On the appropriations triggers.
1:08:02
We kinda went through how the how the process works or how much work goes on prior to appropriations prior to Congresses and and involvement John, would you like to take that?
1:08:17
Think Jeremy Aranda may be better suited. Fine. That’s fine.
1:08:21
Of the Arabic very well and your remark.
1:08:27
Jeremy, it’s all yours. I don’t think any of us ever worked on the appropriations committee, so there’s some, there’s some chosen loosely. I think Operations trigger is based on sort of what’s the prior year’s program funding from the appropriations like, you know, so there’s a sort of baseline. Will. you don’t ever want to allow let fewer appropriations are less progress, right grammatical centers than the prior year. And then you have some sort of inflationary adjustments to it usually, then, I think depending on the anti cancer as well as if they’re very big differences between the programs and the and the and the individual letters.
1:09:02
So, depending on the program and depending on the industries’ interests, in what percentage they are willing to use to cover of the program, there may be a higher or lower target of the of the trigger versus versus the size and scale of the fees. Right.
1:09:22
So, how much you know how in Paducah a very high percentage of the, of the program is funded by the user fees themselves, and the appropriations trigger while increasing every year is still a lower and lower percentage of the program. And that’s because the prescription drug industry, the brand name, drug industry, is very, very confident of what the business valuable value is to them and to their customers, invest in these user fees.
1:09:50
Right, In, good, do phone use, in the super that’s less clear. Right, and those programs and the super program is far less developed than the Purdue for program. So I would guess over time you will see that percentage shift but the appropriations trigger will probably look pretty much the same as a sort of inflationary adjusted number over time.
1:10:12
OK, thank you, um, I think there was mention of possible policy writers and some questions from the audience around that issue.
1:10:22
And the question is, and this is posed to all three of you, all, what do you think are most likely to be discussed and possibly included in this process with regard to the policy writers?
1:10:40
Looking into your crystal balls, John, you typically look to the hearings and what members raise. And the hearings either, you know, in reference to bills, are there, as they’ve already introduced.
1:10:53
Storage is topics that are priorities, and, and, you know, judging by yesterday’s hearing and energy and commerce. You know.
1:11:00
As, as I mentioned, it’s clear that there’s, there’s a discussion to be had about accelerated approval and whether that pathway needs to be right. Sized or optimized, or reformed or whatever term you want to use.
1:11:13
Um, obviously a lot of noise around accelerated approval, you know, even at the hearing yesterday was, was on the, on the coverage side of the equation.
1:11:22
And, you know, CMS and coverage related reforms are really not germane to an FDA user fee package.
1:11:29
So, is there a conversation around FDA related reforms, the accelerated approval, likely, um, clinical trial diversity, and, and, you know, modernizing clinical trial structures, incorporating more real-world evidence.
1:11:47
A lot of this stuff doesn’t necessarily need new statutory authority, but, you know, will likely be discussed in legislative terms and, and I could see being riders.
1:11:59
Obviously, all of the FDA related provisions in 21st Century Cures to zero, and also in this health pandemic preparedness package, I think are, are ripe for consideration.
1:12:14
Pediatric, drug and device related regulatory reforms are, always issues that perennially pop up.
1:12:27
Um, I mentioned, you know, non drug related, big, non drug related, super riders, as they’re being called Valid Cosmetics Supplements.
1:12:38
I mean, I know it does involve a lot of crystal bawling and, again, um, for a variety of reasons.
1:12:45
I think the dynamics this go around when themselves to a tighter package, so how all of those issues are litigated at both committees and ultimately resolved, I don’t know, but just just just to name a few.
1:13:02
Thank you very much. Thank you very much, not. Well. I think the valid Act was mentioned as a possible rider on the user fee legislation is there bipartisan and bicameral by cameras support for that bill.
1:13:17
Uh, who would like to take that?
1:13:23
It’s bipartisan, obviously, it’s introduced in both chambers and there’s an app and, um, was looking pretty good, OK, OK. Bottom-up plus page piece of legislation right in and of itself.
1:13:39
So, yes, to answer your question with bipartisan and bicameral but, um, lot of lot of Feverish debate will certainly no perceived related to the valid.
1:13:53
We noticed earlier that the user fees are now, 30 years old, OK. And.
1:14:00
It, Just to summarize it, if we would identify what is working well, what is expanded, and what should be dropped, one item, what would you say? Ana?
1:14:18
That’s a good question.
1:14:23
Know, in my experiment, and Jeremy alluded to that, I think the transparency around this is really important. And opportunities for patients.
1:14:34
Input, right? And stakeholder input.
1:14:37
And I think, yeah, we each, as I mentioned before, each reauthorization, have come back to it.
1:14:43
Is there another element that can be built in, too?
1:14:46
Continue to foster really a collaborative conversation, right, and being able to capture that patient experience that that patient input and that currently extends event.
1:14:59
If Jeremy touched upon, it become a predictor here for area.
1:15:05
Priority and focus. Which isn’t to say that there won’t be other areas that are like that. But, you know, one of the difficulties in public health is, you know, with finite time and resources. How do you prioritize?
1:15:17
And so I think there is a value of people feeling like they’ve had an opportunity to weigh in. And of course, that can also come in the nature of the packages, moving through the Congress, right? You have elected official who represent you, you can meet with them and bring forward ideas.
1:15:37
But, no, oftentimes, when you, when you go to the event, and say there’s a lot of support for where you landed, it is the reflection of the consensus, right?
1:15:48
That these are areas that that are hard and meaningful, and or are worthy of being a commitment, right.
1:15:55
That this is going to be focused on versus, you know, the alternative of thing. Or we disagree that, we really think it should be other other areas.
1:16:02
And so, I don’t know that I have the perfect answer, right, to what that looks like, but I think that that’s an important element with each of the reauthorization to look at that and think through, could there be more done in that, Right, John, your thoughts on what’s working? Maybe what should be expanded and what should be dropped?
1:16:24
All right, that’s a hard, and that’s a hard question to answer. I’m sure there are a lot of.
1:16:32
Transparency is always a good thing, right?
1:16:34
Are there some reports that are probably really burdensome for FDA to know compiled.
1:16:42
That are kind of no, sign in. And then sit on a desk somewhere and collect dust probably.
1:16:50
So I’ve never worked at FDA, but if there are any FDA folks on the line, I’m sure there’ll be applauding me for saying that.
1:16:57
But there’s, there’s probably some underbrush that involves a lot of work that may not ultimately, you know, lead to bleed to anything.
1:17:05
So, if there’s any sort of ah antiquated reports that are required to be produced, maybe folks could give them a look.
1:17:14
Get rid of the dead yet, get rid of those mandated reports that take a whole lot of resources that no one reads. I got it. Jeremy, would you like to add to the conversation on it?
1:17:23
I mean, I think, look, I think the user, The process itself works pretty well, right. It is. It, is. It is a good mechanism. It’s process oriented by and large. So it is it, is it.
1:17:34
It keeps the, the focus away from policy, which needs to be much more public.
1:17:41
I do think there’s probably more room for the user fee negotiations to be a little bit more engage a bull by stakeholders, and besides industry. Because, I think there’s a little bit, at a fundamental level, they have to be closed doors. But, are there other ways to improve that visibility so that SQL, there’s can engage in them a little more directly? Because usually what happens SQL to sort of engage now, the agreements are already kind of done. So it’s, it’s a little I mean, there’s some that engage on?
1:18:12
I also like to just join my colleagues. And I think there are lots of reports that are of minimal value, their wonderful legislative compromises to, for Congress to direct these, to give them some information on something they need, because they can’t agree on something to do. But, but And the work that goes into, I mean, my biggest learning from being an FTA and I don’t know if this is John is observed FT very close to the outside and sort of the same. Transition. that I did.
1:18:39
Was that it takes a lot more time to do all of the work. The Agency does, than I anticipated, It takes more personnel, it takes more resources.
1:18:49
And that even with the budget it has, which is pretty good, it’s constraint.
1:18:53
And so I do think there’s a need for, for Congress to think about which things are is it asking the agency to prioritize. And spending time and energy on, which not.
1:19:03
We’ve seen user fee agreements have real issues in that front before, but.
1:19:09
But the process itself, I think, is mostly pretty good. And the legislative process is maybe the last vestige of pretty close to regular order.
1:19:16
I’m Just a Bill Lawmaking that that goes on in today’s Congress so I wouldn’t wouldn’t throw that out, either. There enough We’ve said, no throughout the conversation here, that these user Fee act are must pass legislation. OK, it’s must pass legislation.
1:19:38
What happens if the user fee program is not re-authorized by September 30th?
1:19:46
And what are the implications on pharma and payers providers?
1:19:53
What if it doesn’t happen? We’ve seen a lot of anomalies over the years. We’ve all been on the Hill for a lot of years. Strange things happen sometimes.
1:20:02
Who’d like to take that question?
1:20:06
So, when I two cycles ago, when I was on the hill trying to do one of these, we got very close to that moment, And one of the things that, one of the most immediate things that happens is, with, and I can’t remember how close you have to get to the deadline for this moment. But, there is a point where FDA is required by law And by agreement with its employees, to send out notices that there’s a high degree of danger that their job will have to be furloughed or ended.
1:20:32
If there is a halt and funding and the act of sending that out is highly disruptive to the efficient operation, of the people who are working with FDA, so we get that that deadline.
1:20:45
You’re going to start disrupting the way FDA works. If we get past that deadline and the user if funding is not gone, people are being for load and the entire review process will slow down to a crawl.
1:20:57
Right?
1:20:57
So there has to be funding if it’s temporary funding like a CR kind of thing. Right. Exactly.
1:21:05
But, but these were endless points about hiring are really important. You can’t do the hiring You need to do on this. You can’t retain personnel well, if there’s uncertainty about where this funding is coming from. That’s why the five year reauthorization is like this so good that you get this dedicated funding that you have. It’s a government shuts down, you know that there’s probably still getting funding for this, and for the people who take these jobs, working on, that’s of high value to the agency, to the public, into the industry.
1:21:32
I.
1:21:34
would also just add, too, know, what, we’re talking about, thousand of people, yeah, or an important city funds.
1:21:45
And so, I just think it, it’s worth noting. It’s not like you flip the way today upon tomorrow.
1:21:54
So as Jeremy mentioned, there’s the wind down and what’s on the other side, after you’ve come down, you gotta back up.
1:22:01
And so, you know, what could potentially be locked in, there could be deployed to production. If I use the term disruption. My remark right?
1:22:08
That, ultimately, it comes back to, if, if The goal is to try to have, as three lines a pathway as possible, for, again, those products that meet the standard.
1:22:19
Then, the impact of that is, it could be delayed to, ultimately, the time, then, for those that could get caught with that direction, to make their way through that process.
1:22:29
Understood, Well, thank you very much. Unfortunately, we’re about to come to the end of today’s event. So, with these last few minutes, I’d like to each ask each of you to provide it just gives you an opportunity for some brief closing remarks or key takeaways from this conversation on user fees. Jeremy.
1:22:54
I will just say, I sort of this is a great place for bipartisan policy making happens. It’s a great example of when you find ways to make compromise to make policy move forward. Sometimes the direction I like, sometimes in the direction I don’t like.
1:23:09
It’s it’s a really good thing to embrace and keep going and utilize as we go along.
1:23:17
And it’s a place where the common interests of the public, the industry, and the government line up nicely, even if there are disagreements about emphasis within them.
1:23:26
And I think that’s something for thank you, Jeremy Jonn.
1:23:33
Yeah, I mean, I agree with Jeremy. I really hope that hold Cisco around. I mean, I know in 20 17 it’s pretty politically contentious time and we got this done and again, off the House floor on the suspension calendar with a voice vote.
1:23:49
Aye.
1:23:49
I don’t see that happening this go around. I think obviously, you know, that this is going to pass, but a lot of education needs to occur.
1:23:56
There are a lot of members in both chambers where, you know, they’ll just start hearing about the ufa as you know over the next couple of months and that you know, you can’t just intuitively hope that these folks understand how sacrosanct and critically important these programs are to patients and consumers.
1:24:17
So, I would urge everyone to the extent they can, to just educate as many lawmakers as possible so folks understand what what this, otherwise, no esoteric package that has come into, and the House and Senate floor actually entails.
1:24:35
Thank you, Marina.
1:24:37
I think that I think we all consistently hit the theme about just how integral the program bar for FDA’s ability to fulfill its mission to protect and promote public health on behalf of patients and consumers. And so, they’re clearly very, very important. It has a real impact on patient.
1:24:54
We’ve seen over the course of 30 years, the impact the programs have had a public health, and as well as the potential that they could hold, you know, looking forward. And so I also view the reauthorization of these programs is a great opportunity, an opportunity for patients. It’s an opportunity to continue in a very collaborative dialog around how, how, how to optimize work that it’s so critical to patients and public health.
1:25:20
I’d like to thank our panelists today for their insights on no user fees And to our audience for joining us today and helping make this event a success. Unfortunately, this is all the time that we have today. Before you sign out, please take the time to complete a brief evaluation survey that you’ll receive immediately after the broadcast and via e-mail. Please keep an eye on the Alliance website for details about other exciting upcoming events. And last but not least, a recording of the webinar and additional materials will be available on the Alliance website.
1:26:02
So this concludes today’s Webinar on FDA user fees, Anna, Andy, Anna, Jeremy, and John.
1:26:11
Thank you so much for joining us today, sharing your insights, and have a great weekend, everyone.