On March 27, 2020, President Donald Trump signed into law a $2 trillion emergency relief bill to ease the economic impact of coronavirus (COVID-19) and support response efforts. The CARES Act^[1] included an allocation of $80 million in funding to the U.S. Food and Drug Administration (FDA) to continue its COVID-19 response efforts. The additional agency funding will be used, in part, for the development of medical countermeasures and vaccines. 

FDA’s current COVID-19 response efforts include Emergency Use Authorization (EUA) authority under §564 of the Food, Drug and Cosmetics Act (FD&C Act). According to §564 of the FD&C Act, the FDA commissioner may authorize unapproved medical products or unapproved uses of approved products in emergency situations to diagnose, treat, or prevent serious or life-threatening diseases or conditions once the Department of Health and Human Services has declared a public emergency exists. FDA has granted EUAs for diagnostic tests, sterilization and disinfectant devices, respirators, and ventilators.

Although there are no FDA-approved therapeutics or drugs to treat, cure, or prevent COVID-19, FDA has not used its EUA authority in this area. FDA Commissioner Stephen Hahn, M.D. stated, “We understand and recognize the urgency with which we are all seeking prevention and treatment options for COVID-19. FDA staff are working expeditiously on that front. … We also must ensure these products are effective; otherwise we risk treating patients with a product that might not work when they could have pursued other, more appropriate, treatments.” ^[2]

With the worldwide spread of COVID-19, academic medical research centers, public health agencies, pharmaceutical and biotechnology companies are all working to develop treatments and vaccines. Scientists are studying hydroxychloroquine, antiviral drug cocktails and convalescent plasma that contains antibodies from recovered COVID-19 patients.  To address the exigent need for COVID-19 drugs and therapeutics, FDA announced a comprehensive Coronavirus Treatment Acceleration Program (CTAP) to bring coronavirus treatments to market as fast as possible through new public-private partnerships. FDA claims study protocols are being reviewed within 24 hours in many cases. FDA is also streamlining efforts by collaborating with developers and researchers to create protocols that can be used across institutions and programs. Currently, there are 10 therapeutic agents in active trials and another 15 therapeutic agents in planning stages. ^[3]  Preventing the spread of COVID-19 through preemptive vaccination is the ultimate goal. According to the World Health Organization (WHO), 70 COVID-19 vaccines are in development globally, with some already beginning testing in human trials.^[4] It usually takes 10 to 15 years to get a vaccine to market. The pharmaceutical industry hopes to compress the time to within the next year.^[5]

To promote the availability of drugs as quickly as possible, FDA can accelerate the development and approval process for potential new drugs through:

• Orphan drug status;

• Priority review;

• Breakthrough therapy;

• Accelerated approval; and

• Fast track.

The Orphan Drug Act (ODA)^[6] provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan designation (or sometimes orphan status). To qualify for orphan designation, both the drug and the disease or condition must meet certain criteria specified in the ODA. Orphan designation comes with various development incentives, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription-drug-user fee for the rare disease or condition indication for which the drug was designated.

The priority review designation means FDA intends to take action on an application within six months. The breakthrough therapy designation is designed to expedite the development and review of drugs that may demonstrate substantial improvement over available therapy. The accelerated approval designation allows drugs for serious conditions that fill an unmet medical need to be approved based on a surrogate endpoint. The fast track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.^[7]

A drug that receives fast track designation is eligible for certain benefits, including more frequent communications with the FDA relating to the drug’s development plan and the collection of appropriate data needed to support drug approval. Depending on the circumstances, the drug may also be eligible for accelerated approval and priority review if the communications with the FDA indicate that it is appropriate.^[8] Moreover, a drug on the fast track can take part in rolling review where the drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, instead of waiting until every section of the NDA is completed before the entire application can be reviewed.^[9] These expedited review designations have been used in the past during viral outbreaks.

To understand how a COVID-19 drug may get approved, we considered the process FDA used to authorize the use of Regeneron’s drug REGN-EB3, an antibody cocktail treatment for Ebola virus infection. Regeneron requested and received orphan drug status and designation as a breakthrough therapy. FDA accepted priority review for a new Biologics License Application for REGN-EB3, which enables Regeneron to get an answer from FDA on whether it has won approval to market the Ebola drug by October of this year. ^[10] FDA’s expedited review of REGN-EB3 substantially accelerated the customary timeline to bring a new drug to market in the U.S.

Regeneron will start clinical trials in June for a COVID-19 drug. Hopes are higher for its antibodies based on the company’s extensive experience using antibodies to neutralize Ebola and other viruses. Although COVID-19 is caused by SARS-CoV-2, a different virus than Ebola, Regeneron’s COVID-19 program uses the same platform (i.e., discovery, development, and antibodies manufacturing processes), which may give it an edge and help earn approval for FDA priority review.

With development of several different types of treatments and vaccines currently underway, the FDA will continue to play a pivotal role in the response to this pandemic.. Additionally, as part of the government-wide effort to mitigate the spread of COVID-19, the U.S. Department of Health and Human Services (HHS) issued a request for proposals for research and advanced development and licensure of COVID-19 diagnostics, vaccines and medicines such as therapeutics or antivirals.^[11] For more information on how to submit a request for FDA Emergency Use Authorization or CTAP submissions for COVID-19 medical products, please contact one of the attorneys in Dinsmore’s Health Care practice group.