Major Challenges Lie Ahead for FDA and Biopharma

In the coming months, public health officials and biopharmaceutical companies will need to address the ongoing effects of COVID-19, while continuing to develop and produce vital new vaccines and therapies. FDA is relying on swift reauthorization of industry-paid user fee programs to maintain smooth operations, as well as Congressional action on policies affecting the agency and biomedical R&D. These 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 patents and the need for global vaccination efforts to control COVID.

In a notable change, FDA will regain Robert Califf as commissioner, who held the top job at the end of the Obama administration. The Senate Health, Education, Labor and Pensions (HELP) Committee held a hearing last month to confirm Califf, who promises to provide much-needed permanent leadership at the agency. Many experienced staffers have struggled with uncertainty over FDA’s future and resources, making the first order of business for Califf a major staff hiring program to ensure the quality and stability of the agency’s workforce.

FDA benefitted last year from the expert leadership of Acting Commissioner Janet Woodcock, and she may stay at FDA under Califf to further implement reforms of the agency’s IT and management systems and long-debated research and manufacturing initiatives. FDA and industry face ongoing challenges in managing trials and dealing with supply chain issues that limit manufacturer access to ingredients and supplies. The fast expansion of research on gene and cellular therapies has taxed FDA scientists and reviewers, as have mounting requests from sponsors for meetings to discuss accelerated approval of cutting-edge therapies.

In addition to addressing difficulties related to medical product development and marketing, Califf will need to deal with many 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 also needs to deal 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 (PDUFA VII), for generic drugs (GDUFA III), and for biosimilars (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.

The need for Congress to approve programs such as these by Sept. 30, 2022, paves the way for additional FDA-related measures, including the Cures 2.0 bill that aims to enhance biomedical research and approval of new therapies, particularly for serious and orphan diseases. The proposal also advances real-world evidence and patient-reported outcomes in development, and supports the authorization of complex generics and biosimilars.

Post-pandemic planning

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. Such advances in modern manufacturing systems and quality initiatives have reduced the need for agency oversight and inspections, and these changes will continue. FDA looks to rely more on manufacturer records, inspection reports, and regulatory decisions by trusted foreign regulatory agencies, and on remote facility assessments, and less on real-time site inspections.

Global harmonization in regulatory policies will support such approaches, as the pandemic continues to illustrate the need for the testing and production of medical treatments around the world. The International Council for Harmonization (ICH) supports such efforts as it further advances guidelines to modernize the conduct of trials and quality risk assessment by manufacturers.

FDA’s process for quickly authorizing access to new anti-COVID vaccines and treatments through Emergency Use Authorization (EUA) also will face added scrutiny. The agency will further urge sponsors to seek permanent approval of EUA products or phase out their production. At the same time, policy makers will look to improve planning for future health emergencies.

Jill Wechsler is Pharm Exec’s Washington Correspondent. She can be reached at jillwechsler7@gmail.com.