Jill Wechsler

As expected, Congress reauthorized four key user fee programs at the last moment, as part of a must-pass measure to fund the federal government for another two months. The Continuing Resolution (CR) signed on September 30, 2022 averted a federal government shutdown by extending the October 1, 2022 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 (Prescription Drug User Fee Amendments [PDUFA]), generic drugs (Generic Drug User Fee Amendments [GDUFA]), biosimilars (Biosimilar User Fee Amendments [BsUFA]), and medical devices (Medical Device User Fee Amendments [MDUFA]) for the next five years, 2023–2028. 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, approximately 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.”

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. 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.

 FDA also provided information on a new Rare Disease Endpoint Advancement (RDEA) pilot to develop novel efficacy endpoints for critical treatments.

 Still to come are multiple new policies described in the PDUFA VII commitment letter.

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 guidance documents on assigning review goal dates based on a manufacturing facility’s readiness for inspection

 and on early assessment of APIs to speed application review.

 There’s new guidance on expediting development of “competitive” generic therapies and on policies for reviewing complex generic products.

 More information on GDUFA III is available on FDA’s website.

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.

 Further program revisions are listedin the BsUFA commitment letter.

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 based 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 bi-partisan 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 generally unwilling to agree on much in the policy arena. This short-term
budget agreement avoids a government shutdown and funding crisis at FDA, but it dims prospects for enacting innovative, controversial or complex new measures.

This article originally appeared on Pharmaceutical Technology.

Split real time application review (STAR). Development resources, FDA. Accessed October 12, 2022. 

https://www.fda.gov/drugs/development-resources/split-real-time-application-review-star

Rare disease endpoint advancement pilot program. Development resources. FDA. Accessed October 12, 2022. 

https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program

PDUFA reauthorization performance goals and procedures fiscal years 2023 through 2027. Commitment letter. FDA. Accessed November 17, 2022. 

https://www.fda.gov/media/151712/download

Facility readiness: Goal date decisions under GDUFA, guidance for industry. Draft guidance. FDA. Accessed November 17, 2022. 

https://www.fda.gov/media/162018/download

Review of drug master files in advance of certain ANDA submissions under GDUFA, guidance for industry. Draft guidance. Accessed November 17, 2022. 

https://www.fda.gov/media/162019/download

Competitive Generic Therapies. Guidance document. Accessed October 12, 2022. 

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/competitive-generic-therapies

GDUFA III Reauthorization. Reference. FDA.Accessed October 12, 2022. 

https://www.fda.gov/industry/generic-drug-user-fee-amendments/gdufa-iii-reauthorization

Biosimilars–Science and research. Reference. FDA. Accessed
October 12, 2022. 

https://www.fda.gov/drugs/biosimilars/biosimilars-science-and-research

Biosimilar biologic product reauthorization performance
goals and procedures fiscal years 2023 through 2027. Commitment letter. FDA. Accessed October 12, 2022. 

https://www.fda.gov/media/152279/download

Articles

FDA keeps its user fees but fails to gain important reforms.

User Fees Reauthorized for FDA 

House and Senate leaders announced final agreement June 18 on legislation that authorizes industry user fees to support FDA regulatory programs. 

The bipartisan measure moved through Congress quickly with an eye to avoid getting tangled up in fallout from the imminent Supreme Court decision on health reform, as well as election-year politicking. The FDA Safety and Innovation Act (S. 3187) (FDASIA) reauthorizes the Prescription Drug User Fee Act (PDUFA), renews user fees for medical devices, and establishes new fees for oversight of generic drugs and for future biosimilar therapies. 

FDASIA reflects agreement on the need to spur development of new antibiotics and treatments for rare conditions. It includes a number of provisions sought by FDA to better secure the pharma supply chain, to block import of adulterated products, and to detect and prevent drug shortages. However, the legislators failed to agree on a national track-and-trace system for ensuring the integrity of drugs moving through the distribution system. Continued disagreement among wholesalers, manufacturers, and pharmacies now opens the way for the states to craft diverse drug pedigree requirements, and to FDA to propose national standards where appropriate. 

Several other contentious issues were dropped from the final bill. Generic drug-makers lost out on an effort to prevent brand manufacturers from using Risk Evaluation and Mitigation Strategies (REMS) to block access to innovator products needed to test and develop generic versions of drugs and biologics. And pharmacists scuttled a move to stiffen controls on hydrocodone-combination painkillers, proposed as a way to curb rampant abuse of prescription drugs. 

The legislation will keep FDA busy with a host of requirements for new guidances, new rules, and multiple reports. But more than $6 billion in user fees will roll in over the next 5 years to keep the agency going.    

House and Senate leaders announced final agreement June 18 on legislation that authorizes industry user fees to support FDA regulatory programs.