Pandemic brings major changes to FDA and drug development in 2020
Manufacturers and regulators are accelerating R&D and production of new vaccines and therapies.
The imperative to prevent, treat, and cure the deadly COVID-19 virus has generated new research and manufacturing strategies, bolstered by unprecedented support from the federal government, private investors, and timely regulatory guidance and advice. Realizing the intense pressure and the great opportunity, the industry moved quickly to identify compounds that could treat sick patients and mitigate the lethal effects of the virus. The federal government has invested billions in the development and production of drugs and vaccines through its Operation Warp Speed (OWS) task force. At the same time, the FDA has struggled to respond to often conflicting and unclear requests and mandates from the White House and the Department of Health and Human Services (HHS), developments that have undermined public confidence in agency independence.
The FDA’s first crisis led to confusion over the development and approval of diagnostic tests for the COVID-19 pathogen, a situation that has resulted in notable delays in public access to products capable of reliably identify infected persons. While the FDA was not solely responsible for the situation, informed observers believe that more proactive leadership from the agency could have accelerated progress in this critical area.
The agency’s new commissioner, Stephen Hahn, has become even more entangled in the administration’s efforts to promote some unproven COVID-19 therapies and to demand that the FDA issue emergency use authorizations (EUA ) for potentially harmful treatments. Hahn’s initial support for such clearance alienated agency staffers and medical authorities and raised concerns about the agency’s ability to fully test and vet promising vaccines and other therapies. essential.
These issues came to a head in October as vaccine experts from the FDA and major medical institutions stressed the importance of obtaining enough data on the safety and effectiveness of any vaccine before approving its administration. to millions of people. This approach was seen as key to offsetting “growing public hesitancy towards vaccines” stemming from fears that companies and regulators are taking shortcuts to speed up the development of COVID-19 vaccines for political gain. Vaccine makers have backed the call for adequate test data to get results that could generate public support in the coming months.
The accelerated development of several COVID-19 vaccines has highlighted the essential role of the FDA’s Center for Biologics Evaluation and Research (CBER). In May 2020, CBER Director Peter Marks elected to stay with the FDA instead of moving to the administration’s OWS task force, as did Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), who moved temporarily to lead the development of OWS therapy. efforts. Under Marks’ leadership, CBER released key guidance in June clarifying the requirements for developing and testing new vaccines to protect against COVID-19 infection, emphasizing the need for large clinical trials. randomized placebo-based to provide adequate safety and efficacy. data, as well as initial manufacturing information (1). Other guidelines in October specified similar approaches to obtaining EUA from the FDA for a new vaccine (2).
Many of these clinical development programs have benefited from OWS funding, as has the early production of large quantities of test vaccines – a marked departure from the usual practice of delaying investment in scale-up. and production until clinical testing shows results. The goal was to provide sufficient amounts of any preventative as soon as tests showed an immune response. FDA guidelines have emphasized the need for early Chemistry, Manufacturing, and Controls (CMC) data, while allowing wiggle room in batch stability records (1). Vaccine sponsors have hired contract manufacturers and other companies to prepare for rapid scale-up once licensed. OWS has also supported the additional production of essential glass vials and injectors to vaccinate hundreds of millions of people.
The trains keep on rolling
While moving many staff members to operations involved in the development and review of COVID-19 products, the FDA has also been working overtime to maintain support and approval of new therapies for the usual wide range of serious medical conditions. Innovative biomedical R&D faced additional challenges due to delays in many new and ongoing clinical trials due to pandemic complications. Additionally, the FDA had to roll back regulatory inspections, especially those involving foreign companies. Instead, the agency sought to assess the quality status of an operation producing a new drug or vaccine based more on compliance history, production records, analysis of actual products and inspection reports from other trusted regulatory authorities.
Supply chain security for drugs and medical products has emerged as a critical issue as the global pandemic has raised concerns about reliable access to drugs and their components produced overseas. Shortages of certain therapies vital to treating hospital patients, including antibiotics and sedatives, have prompted the FDA to step up its efforts to detect impending shortages in advance and to support alternative production activities, including the broader drug composition and the administration’s “Buy American” campaign to expand drug production. and medical products in the United States. These developments have highlighted the importance of manufacturers’ ability to establish modern and more reliable pharmaceutical manufacturing operations capable of maintaining reliable, high-quality production with well-documented internal oversight.
The pandemic has slowed but not derailed a series of FDA initiatives to improve the regulatory process. CDER finalized the redesign of its Office of New Medicines, with an increased number of offices and divisions implementing a new team-based “integrated review” approach to evaluating new drug applications.
In addition to focusing on vaccine development, CBER’s Office of Tissue and Advanced Therapies (OTAT) continued to face an increase in proposals for the development of novel cell and gene therapies and regenerative drugs. Hundreds of applications for investigational new drugs flooded OTAT, many of them raising difficult and unique medical and technical issues, including concerns about product quality, purity and strength. In accelerating development programs, the FDA has focused on clarifying product characterization strategies, process validation, scale-up, and stability.
The FDA approved more than 40 innovative drugs last year and worked hard to meet user fee deadlines for prescription drugs, generics, and biosimilars. Those numbers could drop in the coming months as sponsors face continued challenges launching new clinical trials and documenting quality manufacturing operations. As the coronavirus once again spreads across the country and the world as this historic year draws to a close, the only certainty is that obstacles and opportunities will continue to challenge biopharmaceutical companies, regulatory authorities and the healthcare community. research in the coming months.
1. FDA, Development and Licensure of Vaccines to Prevent COVID-19, Guidance for Industry (CBER, June 2020).
2. FDA, Emergency Use Authorization for Vaccines to Prevent COVID-19 Guidance for Industry (CBER, October 2020).
About the Author
Jill Wechsler is Pharmaceutical technology Washington Editor, [email protected]
Flight. 44, No. 12
When referencing this article, please cite it as J. Wechsler, “Pandemic Brings Major Changes to FDA and Drug Development in 2020,” Pharmaceutical technology 44 (12) 2020.