FDA Keeps Its User Fees, but Fails to Gain Important Reforms

As expected, Congress reauthorized four key user fee programs last week, as part of a must-pass measure to fund the federal government for another two months. The Continuing Resolution (CR) signed on Sept. 30 averted a federal government shutdown by extending the Oct. 1 deadline for approving a federal spending plan for fiscal year 2023 to Dec. 16, 2022—after the mid-term elections in hopes that this will enable the legislators to reach a viable budget compromise.

The CR reauthorizes fees for drugs (PDUFA), generic drugs (GDUFA), biosimilars (BsUFA) and medical devices (MDUFA) for the next five years, 2023-2028.¹ In confirming FDA’s authority to collect fees from manufacturers, the agency avoids having to notify thousands of fee-supported staffers of looming layoffs. That prospect already had eroded agency morale and undermined efforts to recruit and hire needed staffers, as FDA struggled for months to maintain and expand its professional workforce during the added hardships imposed by the pandemic and in a very competitive labor market.

Medical product user fees generate some $2 billion in agency revenues, about 40% of FDA’s non-tobacco budget. While Congressional leaders on both sides of the aisle support this program to bolster key agency operations and innovations, the broader political debate over escalating government spending generated concerns about new fee-supported initiatives that extend the agency’s authority.

Even so, FDA officials, manufacturers, and research advocates gave a loud sigh of relief following the Congressional action. Commissioner Robert Califf praised the user fee reauthorization measure, while thanking agency staffers for their diligence in engaging in this “prolonged, demanding and, at times, frustrating process.” He noted that the bill avoids “a devastating impact” on FDA’s ability to carry out its “independent and transparent review of medical products.”

New initiatives

Califf added that FDA is committed to working with Congress to enact additional policy changes to benefit public health, but for now, is moving fast to implement multiple new fee-supported policies and programs. Similarly, Patrizia Cavazzoni, director of the Center for Drug Evaluation and Research (CDER), praised the new fee programs for supporting CDER’s commitment to monitoring the safety of marketed products, protecting patients in clinical trials, and facilitating review of treatments for life-threatening diseases.

In an effort to make up for lost time in meeting tight deadlines for implementing new PDUFA-backed initiatives, FDA quickly posted a new webpage outlining criteria for manufacturers to participate in the new Split Real Time Application Review (STAR) program to accelerate patient access to critical therapies.² The pilot aims to shorten the review time for efficacy supplements to approved drugs and biologics through earlier review of certain submissions before clinical study reports are final.³ FDA also provided information on a new Rare Disease Endpoint Advancement (RDEA) pilot to develop novel efficacy endpoints for critical treatments.⁴ Still to come are multiple new policies described in the PDUFA VII commitment letter.⁵

Enhancements in GDUFA III aim to reduce the number of assessment cycles for generics, support the development and approval of complex generics, and help manufacturers resolve quality issues more quickly. First off the line are new draft guidances on assigning review goal dates based on a manufacturing facility’s readiness for inspection⁶ and on early assessment of active pharmaceutical ingredients (APIs) to speed application review.⁷ There’s new guidance on expediting development of “competitive” generic therapies⁸ and on policies for reviewing complex generic products.⁹ More information on GDUFA III is available.¹⁰

And for biosimilars, BsUFA III priorities involve expediting the review of certain supplements and launching a new regulatory science program to advance biosimilars and interchangeable products, already posted in a new FDA webpage outlining new pilots to improve the efficiency of biosimilar development and for interchangeable products.¹¹ Further program revisions are listed out in the BsUFA commitment letter.¹²

Moving forward

The “skinny” user fee reauthorization dropped multiple high-profile proposals for revising how FDA regulates in vitro diagnostics, dietary supplements, and cosmetics, as well as the agency’s accelerated drug approval program and the regulation of artificial intelligence medical devices. Advocates for developing critical new antibiotics back the PASTEUR Act to establish a new funding mechanism to support the development and proper utilization of drugs to fight resistant diseases. There is bipartisan support for this “subscription” model for new cures, but its estimated cost of$11 billion over 10 years makes it a hard sell.

In past user fee reauthorization campaigns, a deadline to prevent fee expiration encouraged legislators to slip numerous pet proposals into this must-pass legislation. All parties recognized that this year would be much trickier, with a sharply divided Congress heading into mid-term elections and unwilling to agree on much in the policy arena. The short-term budget agreement avoids a government shutdown and funding crisis at FDA, but it dims prospects for enacting controversial or complex new measures.

References

1. https://acrobat.adobe.com/link/track?uri=urn%3Aaaid%3Ascds%3AUS%3A32756260-e9ae-43fd-869d-99fb4ed727ab&viewer%21megaVerb=group-discover
2. https://www.fda.gov/media/151712/download
3. https://www.fda.gov/drugs/development-resources/split-real-time-application-review-star
4. https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program
5. https://www.fda.gov/media/151712/download
6. https://www.fda.gov/media/162018/download
7. https://www.fda.gov/media/162019/download
8. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/competitive-generic-therapies
9. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/competitive-generic-therapies
10. https://www.fda.gov/industry/generic-drug-user-fee-amendments/gdufa-iii-reauthorization?utm_medium=email&utm_source=govdelivery
11. https://www.fda.gov/drugs/biosimilars/biosimilars-science-and-research
12. https://www.fda.gov/media/152279/download?utm_medium=email&utm_source=govdelivery