Anyone who has spent any time around health care policymaking circles in Washington, D.C. has heard about the intense five-year cycle Congress goes through in order to reauthorize the biggest user fee programs at the Food and Drug Administration (FDA). Mintz previously published this wonderful explanation of the process complete with an estimated timeline for how this year’s reauthorization was likely to go. As a reminder, the current authorization for FDA’s various human product user fee programs – with the exception of the over-the-counter drug program that was freshly created in 2020 – will expire on September 30, 2022 with the end of the federal government’s fiscal year, unless they are renewed by Congress before that time.
Final prescription drug, biosimilar, and generic drug user fee agreements were submitted by FDA to Congress in January 2022, even though public negotiation meetings began a little bit later in calendar year 2020 due to the COVID-19 pandemic (those final PDUFA, BsUFA, and GDUFA agreements can be accessed here, here, and here, respectively). And although the medical device agreement negotiations took longer than usual and that final delivery to Congress was delayed until the end of March, it was ultimately completed (see here) and released in conjunction with a statement from the director of the Center for Devices and Radiological Health that emphasized the importance of modernizing the agency’s approach to devices as digital health technology rapidly advances.
House Version of the User Fee Legislation
Congressional panels have held a number of hearings related to the “UFAs” since FDA submitted the various negotiated agreements at the beginning of the year. During the first week of May, a bipartisan House bill reauthorizing all four of these big programs, named the “Food and Drug Amendments of 2022,” was introduced by leaders of the House Energy & Commerce (E&C) Committee. The proposed legislation currently being considered in the House includes more than 40 substantive riders that represent various reform or public policy priorities for FDA (a few of these substantive riders are summarized below). On May 18, following in the footsteps of a unanimous health subcommittee vote a week earlier, the E&C Committee marked up the lengthy bill and voted unanimously to send it along for consideration on the House floor. Although the House process so far has moved very quickly and without much rancor, the legislative process will likely slow down as a result of certain controversial policy changes being thrown into the mix more recently by policymakers on the Senate side (more on this below).
The House E&C Committee’s Food and Drug Amendments of 2022 (moving as H.R. 7667) includes the following proposed amendments to the Food, Drug and Cosmetic Act (FD&C Act) and FDA-related policy riders:
- Reforms to the agency’s accelerated approval pathway for drugs and biologics to allow for a more efficient withdrawal process by FDA when the clinical benefits of the product cannot be or are not timely verified;
- Creation of an affirmative labeling requirement for drug and biologic products marketed pursuant to an accelerated approval;
- Explicit recognition that “real world evidence” may be used to support or augment the post-approval verification study that sponsors of accelerated approval products are required to conduct under the existing framework;
- Directions to the Secretary of Health and Human Services and his delegates at FDA to evaluate how to enhance diversity in clinical trials, including how sponsors could be incentivized to do so, as well as a requirement for sponsors to submit a Diversity Action Plan to the agency as part of their clinical study proposals – the latter proposal is also included in a recent draft guidance issued by FDA (see also the April 13 press release announcing the publication of this draft guidance);
- Clarification of congressional intent related to the scope and effect of regulatory exclusivity that is granted to an orphan-designated drug or biologic, in response to a federal court ruling last year that rejected FDA’s longstanding interpretation of the current statutory provision (the agency recently posted its views on that court decision and emphasizes in its summary discussion that the ruling could decrease innovation and competition in the industry);
- Language intended to codify FDA’s Payor Communications Guidance through the addition of a new paragraph at Section 502 of the FD&C Act, which lays out various criteria for misbranded drugs and devices;
- Directions to FDA regarding a pilot program for conducting unannounced foreign inspections of drug manufacturers;
- Requirements for medical device developers to include cybersecurity in their device design and include information about their products’ cybersecurity in premarket submissions to FDA, as had been requested by the agency in its FY2023 legislative proposals that accompanied the Biden Administration’s proposed budget for the agency next year.
…and much more! E&C Committee leaders – in particular Committee Chair Frank Pallone (D-NJ) – have also signaled that they may be open to adding on further substantive reforms in order to align their bill more closely with the Senate’s version, most notably on the bipartisan subjects of diagnostic and cosmetic reform.
Senate Version of the User Fee Legislation
Not far behind lawmakers in the House are their Senate counterparts, who officially introduced the Senate Health, Education, Labor and Pension (HELP) Committee’s version of an FDA user fee reauthorization package on May 27. HELP Committee Chair Senator Patty Murray (D-WA) and Ranking Member Senator Richard Burr (R-NC) issued a joint statement in conjunction with their introduction of the bill, which has been named the “Food and Drug Administration Safety and Landmark Advancements Act” (FDASLA).
In addition to the core goals of reauthorizing PDUFA, MDUFA, GDUFA, and BsUFA, the Senate’s proposal includes many of the same reforms as the House bill, such as those related to strengthening FDA’s authority over drugs that get to market via the accelerated approval pathway. At the same time, however, the first iteration of FDASLA goes beyond the changes put forward in the House by requiring the creation of an intra-agency coordinating council “to ensure the consistent and appropriate use of accelerated approval” – and with the membership of such a council specifically defined in the draft statutory language. This 10-member Accelerated Approval Council would be tasked with meeting at least three times per year and also would need to be established by FDA within 180 days of the bill being signed into law.
Other significant legislative riders that are attached to the as-introduced FDASLA include:
- Wholesale reform of the legal framework for “in vitro clinical tests,” packaged as the VALID Act of 2022, which would take effect on October 1, 2027 and would also create a new user fee program at FDA (this newest version has several differences as compared to the 2021 version of the VALID Act, which we blogged about here);
- Modernized cosmetic regulatory requirements – to include things like good manufacturing practices for cosmetic products, mandatory facility registration and adverse event reporting, and listing of products in commerce – which has been a bipartisan goal for many years now and was also part of FDA’s FY2023 legislative request to Congress;
- Requirements for human dietary supplements that are in commercial distribution to be listed with FDA (another legislative proposal that was included in the agency’s budget request);
- A mandated timeline for FDA to finalize the over-the-counter hearing aid regulation that it proposed in October 2021 – specifically, the agency would be ordered to finalize the rule within 30 days of the bill being signed into law;
- A brand new process for the agency to make therapeutic equivalence determinations for drugs approved under a 505(b)(2) NDA within 180 days of such a drug’s approval, in light of the current situation in which only “true” generic drugs approved under the 505(j) pathway – i.e., the abbreviated new drug application process – are automatically evaluated for therapeutic equivalence to the reference listed product, such that complex generics or other follow-on products that come to market via a 505(b)(2) application are often not adjudicated one way or another for many years after initial approval;
- Directions to FDA for establishing a Rare Disease Endpoint Advancement Pilot that would culminate in agency guidance laying out best practices and strategies for developing efficacy endpoints for rare diseases, including surrogate and intermediate endpoints; and
- An amendment to the Public Health Service Act that would allow more than one interchangeable biosimilar to share exclusivity if they are approved on the same day (per current law, the “first” interchangeable biological product is eligible for one year of marketing exclusivity).
Leaders from both House and Senate committees with jurisdiction over FDA have stated publicly that they do not want the reauthorization to be held up and do not want FDA to have to notify any employees that they could be furloughed or fired as a result of a hold-up in the user fee legislative negotiations. The periodic FDA user fee reauthorization historically has been a bipartisan process and, unlike virtually every other expiring program, this one tends to resolve in Congress well ahead of the deadline. To that end, the Senate HELP Committee will likely mark up its version in June (as of this writing, the first Senate hearing on the bill had been scheduled for June 8), and both chambers will push to pass and reconcile their versions before the August recess. Some of these more controversial changes and the hyper-partisan nature of this particular Congress, however, may make the reconciliation process between the House and the Senate a bit more difficult.