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To maintain smooth operations, FDA is relying on swift reauthorization of industry-paid user fee programs.
In the coming months, public health officials and biopharmaceutical companies face the task of addressing and mitigating the ongoing effects of COVID-19, while also continuing to develop and produce vital new vaccines and therapies. To maintain smooth operations, FDA is relying on swift reauthorization of industry-paid user fee programs, as well as Congressional action on policies affecting the agency and biomedical R&D. These ongoing imperatives, however, may be sidelined by mid-term elections in November 2022, where the pandemic response and access to medicines and healthcare services will be prominent issues. Drug pricing will remain a highly charged topic, as the debate continues over patent protections and the need for global vaccination efforts to control COVID-19.
A notable change at FDA will be the appointment of Robert Califf as commissioner. The Senate Health, Education, Labor and Pensions (HELP) Committee held a confirmation hearing for Califf in December 2021, setting the stage for his return to FDA after holding the top job at the end of the Obama administration. Califf will provide much-needed permanent leadership at the agency, where many experienced but exhausted staffers have struggled with uncertainty over FDA’s future and resources. A prime order of business for Califf is to advance a major staff hiring program to ensure the quality and stability of the agency’s workforce.
FDA benefitted in 2021 from the expert leadership of Acting Commissioner Janet Woodcock, but her temporary status limited the launch of new programs and policies. There are signs that Woodcock may stay at FDA under Califf to further implement reforms of the agency’s IT and management systems and other long-debated research and manufacturing initiatives. FDA and industry face ongoing difficulties managing clinical trials and supply chain disruptions that limit manufacturer access to necessary ingredients and supplies. The fast expansion of research on gene and cellular therapies has taxed biotech scientists and reviewers, as has mounting requests from sponsors for meetings to discuss the accelerated approval of cutting-edge therapies. FDA managed to maintain timely review and approval programs for new drugs and biologics during the pandemic, but it will be a challenge to continue those gains.
In addition to addressing difficulties related to medical product development and marketing, Califf will need to deal with a range of critical regulatory and policy issues. Misuse and abuse of opioids continues to escalate, while appropriate oversight of e-cigarettes and vaping products are in the spotlight. FDA needs to ensure the safety of much of the nation’s food supply, while also dealing with Congressional concerns about the regulation of cannabis-derived compounds in foods and dietary supplements.
Effective FDA oversight of new medical products requires timely reauthorization of user fee programs that are critical for maintaining FDA’s efficient regulatory processes for drugs and biologics (the Prescription Drug User Fee Act–PDUFA VII), for generic drugs (Generic Drug User Fee Amendments–GDUFA III), and for biosimilars (Biosimilar User Fee Act–BsUFA III). FDA officials have collaborated with industry representatives in crafting new “commitment letters” for implementing program and fee updates linked to product development, application vetting, and manufacturing oversight (1–3).
The need for Congress to approve these and other fee programs by Sept. 30, 2022, sets the stage for the legislators to enact additional measures affecting FDA, including the new Cures 2.0 measure that has been circulating on Capitol Hill. Building on the initial Cures program approved in 2016, the new bill aims to enhance biomedical research and FDA approval of new therapies, particularly those for serious and orphan diseases. The proposal also advances the use of real-world evidence and patient reported outcomes in drug development and supports efforts to approve more complex generic drugs and biosimilars.
During the past two years, biopharma companies adopted innovations in research and manufacturing to enable the quick scale up and expanded production of vital vaccines and therapies, in the United States and abroad. FDA has advanced these efforts with ongoing support for modern manufacturing systems and quality initiatives able to reduce the need for agency oversight and inspections. The pandemic greatly limited the agency’s field inspection process, and many of the resulting changes will continue, as FDA relies more on manufacturer records, inspection reports and regulatory decisions by trusted foreign regulatory agencies, and on remote facility assessments instead of on-site inspections.
These efforts further speak to the need for global harmonization in regulatory policies and for agreement on regulatory standards as the pandemic continues to highlight the need for the testing and production of medical treatments around the world. The International Council for Harmonisation provides support for such efforts as it further advances new guidelines to modernize the conduct of clinical trials, regulation of generic medicines, control of impurities in drugs, and quality risk
assessment by manufacturers.
At the same time, FDA’s process for quickly authorizing access to new COVID-19 vaccines and treatments through the Emergency Use Authorization (EUA) process will face added scrutiny, as the US and the world look to shift to a post-pandemic phase. FDA will urge sponsors to seek permanent approval of EUA products or phase out their production. At the same time, policy makers see a strong need to improve planning for future health emergencies, building on the lessons learned from COVID-19 at home and abroad.
Many of these regulatory and R&D goals may be sidelined, however, if the political debate over Biden administration proposals for expanding social and health programs delays approval of new user fee legislation, and other measures affecting FDA and its programs. As the midterm elections grow near, action on major legislation will face an unofficial deadline of enactment by mid-year or risk being left by the political wayside.
1. FDA, PDUFA VII: Fiscal Years 2023–2027, FDA.gov, accessed Dec. 8, 2021.
2. FDA, GDUFA III, FDA.gov, accessed Dec. 8, 2021.
3. FDA, BsUFA III: Fiscal Years 2023-2027, FDA.gov, accessed Dec. 8, 2021.
Jill Wechsler is BioPharm International’s Washington editor.
Vol. 35, No. 1
When referring to this article, please cite it as J. Wechsler, “Major Challenges Ahead for FDA and Pharma,” BioPharm International 35 (1) (2022).