A new presidential administration and administrator may impact the Food and Drug Administration and its approval of drugs and medications. The FDA faced serious challenges and pressure to authorize unproven drugs during the pandemic. It must address other issues involving biotech and pharmaceuticals in 2021.
The FDA authorized two new coronavirus vaccines last December. Soon, there may be three more vaccines with late-stage study results requiring approving. Testing is also ongoing for experimental antivirals and antibodies developed for keeping infected patients out of the hospital.
Like its approval process for the Pfizer-BioNTech and Moderna vaccines, the FDA may establish an advisory committee to review each clinically successful vaccine. This takes time but helps establish public trust.
Manufacturers that developed coronavirus treatments which were authorized for emergency use, such as Regeneron and Eli Lilly, may seek full approval for these treatments. Approval may become complicated because these companies increased the populations where these drugs can be used. Data from larger groups could change conclusions on their effectiveness.
Adapting clinical trial design
The expedited processes for speeding up the FDA’s authorization of coronavirus vaccines and treatment evaluation of hospital patients may pressure the FDA to reduce the time for multi-year clinical studies and provide more clinical trial design flexibility.
The FDA worked with manufacturers to design trials that can quickly determine the effectiveness of treatments for cancer and rare diseases. The FDA may need to consider ways to hasten the development of drugs for diabetes and cardiovascular diseases.
The FDA may have raised its standards for these medicines because of its unexpected rejection of BioMarin Pharmaceutical’s hemophilia gene therapy Roctavian and its scrutiny of manufacturing processes. Demands on the FDA will increase because over 1,000 investigational new drug applications for gene therapies were submitted.
BioMarin claimed that the FDA was concerned about early data from later-stage testing. This showed a weaker treatment compared to a smaller and early study which posed issues about the durability of the medication’s benefit. Hemophilia patients also have many drug options which may have changed the FDA’s risk-benefit calculation.
User fees partially fund FDA reviews under the law, known as PDUFA, which must be reenacted by Congress by Sept. 30, 2022. Under previous reauthorizations, the FDA had to set certain approval options and firmer deadlines for drug approval decisions.
The agency may have to expand activities on adaptive studies and other new clinical trials models that shorten the development time for experimental drugs. Manufacturers may seek to reduce paperwork for expedited reviews.
Attorneys can help assure business compliance. Lawyers may also assist with new drug applications.