FDA Issues Emergency Use Authorization Guidance for COVID-19 Vaccine Sponsors

An Emergency Use Authorization (EUA) is a mechanism used to enable the accessibility and use of medical countermeasures, including vaccines, during public health emergencies.1 Under an EUA, FDA may allow the use of unapproved medical products to diagnose, treat, or prevent serious or life-threatening diseases or conditions when certain statutory criteria have been met.

During the COVID-19 public health emergency, three COVID-19 vaccines have been granted EUA. EUA was granted to the Pfizer-BioNTech COVID-19 vaccine on December 11, 2020 and to the Moderna COVID-19 vaccine a week later on December 18, 2020. Most recently, on February 27, 2020, Johnson & Johnson was given EUA on their Janssen COVID-19 vaccine. Several other companies are developing COVID-19 vaccines and expect to apply for EUA, including Novavax and AstraZeneca.

On February 22, 2021, FDA issued industry guidance to provide COVID-19 vaccine developers (“sponsors”) with recommendations and information needed to support the issuance of an EUA.2 The FDA guidance explains the statutory requirements to obtain an EUA for a proposed vaccine. The proposed vaccine must (1) be effective to prevent, diagnose, and treat COVID-19 based on the totality of scientific evidence available, including data from adequate and well controlled trials, and (2) demonstrate benefits that outweigh any known and potential risks of the proposed vaccine. There also must be no adequate, approved, and available alternative.

Logistically, FDA’s guidance recommends that sponsors create an early dialogue with FDA to discuss expectations, considerations, and the manufacturing of the particular vaccine. At least one-month prior to an EUA request, sponsors should submit a description of chemistry, manufacturing, and controls (“CMC”) data. Moreover, the sponsor is strongly encouraged to provide FDA with twenty-four hour notice of any interim analysis completion.

As far as information that should be included in an EUA request, FDA recommends sponsors submit certain regulatory, CMC, safety, and effectiveness information in order to properly evaluate whether the vaccine’s benefits outweigh the known the risks. Regulatory information includes proposed uses, doses, and method(s) of administration, need for the vaccine, identification of any approved alternatives, and information regarding the vaccines supply chain so that FDA may assess the vaccine’s expected availability. With regard to CMC information, FDA recommends submission of data regarding drug substance (“DS”) and drug product (“DP”) manufacturing sites, critical source materials used in manufacturing, quality control / validation systems, stability plan, filtration / sterilization processes, and storage conditions. Like the regulatory data, the CMC information should be provided to demonstrate the vaccine’s safety, quality, effectiveness, and consistency at all stages.

The sponsor’s safety and effectiveness information will guide FDA regarding the vaccine’s benefit-risk profile. Such efficacy data includes all nonclinical and clinical studies with associated safety data from Phase I, II, and III trials. Phase III studies should include a median follow-up duration of two months minimum so FDA may assess adverse events. For FDA to properly analyze a vaccine’s benefit-risk profile, all adverse events including sufficient cases of severe COVID-19 should be documented and submitted. The sponsor should include a plan to actively follow-up with trial participants to support continuation of a possible EUA.

After a sponsor makes an EUA request, FDA organizes an open session via FDA’s Vaccine and Related Biological Products Advisory Committee (“VRBPAC”) to discuss whether the proposed vaccine meets the statutory requirements needed for an EUA. Sponsors should submit briefing to the VRBPAC summarizing the proposed vaccine’s safety and effectiveness. It is likely that future sponsors, such as Novavax and AstraZeneca, will look to any publicly available FDA submissions and advisory committee minutes to guide their own EUA submissions. FDA’s EUA guidance will now serve to facilitate the EUA process for future vaccines to hit the market. The hope is that FDA’s guidance, outlined above, will expedite U.S. vaccination against COVID-19.

Overall, FDA’s EUA guidance gives transparency into the EUA submission procedure, not just for the COVID-19 public health emergency, but for any future public health emergency where there is no adequate, approved, or available medical countermeasures. For pharmaceutical companies developing and investigating new drugs, FDA’s EUA guidance allows pharmaceutical companies to assess, organize, and highlight FDA’s recommended CMC, safety, and effectiveness information prior to EUA submission. FDA’s EUA guidance simplifies the EUA process for sponsors and may lead to quicker EUA submissions and decisions.

1; see also § 21 U.S.C. 360bbb-3(b)(1))
2Emergency Use Authorization for Vaccines to Prevent COVID-19, FDA, February 22, 2021.