The Food and Drug Administration (FDA) issued guidance on emergency use authorization (EUA) for COVID-19 vaccines (press release). The guidance is intended to provide recommendations for vaccine sponsors regarding the scientific data and information that would support the issuance of an EUA for an investigational vaccine intended to prevent COVID-19.
Overall, the guidance establishes that a COVID-19 vaccine candidate authorized under EUA must meet the same safety and efficacy standards established in the “Development and Licensure of Vaccines to Prevent COVID-19” guidance. The FDA notes that while an EUA is a different standard than licensure approval, both pathways require the same submission of data demonstrating the vaccine’s safety and effectiveness.
Additionally, the agency emphasizes that EUA determination will be made on a case-by-case basis considering the target population, characteristics of the product, the clinical study data, and the totality of the available scientific evidence. To ensure transparency, the FDA details that the Vaccines and Related Biological Products Advisory Committee (VRBPAC) will convene an open session prior to any EUA issuance.
Additional considerations from the guidance follow:
- Criteria and considerations for the issuance of an EUA – The guidance establishes the following criteria for the issuance of an EUA for COVID-19 vaccine:
- Based on the totality of scientific evidence available, including clinical trials, it is reasonable to believe that the product may be effective to prevent, diagnose, or treat disease or conditions causes by COVID-19;
- The known potential benefits of the product outweigh the known an potential risks of the product; and
- There is no adequate, approved, and available alternative to the product for diagnosing, preventing, or treatment the disease of conditions.
- Key logistic recommendations for the request for an EUA – The FDA recommends that vaccine sponsors considering EUA contact the Center for Biologics Evaluation and Research’s (CBER’s) Office of Vaccines Research and Review (OVRR) early in development to discuss expectations. Additionally, the agency suggests that detailed information and data be included in the request, as well as notice when any interim analysis has been completed.
- Recommendations regarding information and data to be included in an EUA request – The guidance outlines the necessary regulatory; chemistry, manufacturing, and controls; and safety and effectiveness information that should be included in an EUA request. Highlights of this information follow:
- Safety and Effectiveness Information – In the EUA request, the FDA recommends that sponsors include the diagnostic bioassays that were used to assess the study endpoint of clinical studies, as well as a list of the nonclinical studies to support vaccine effectiveness and safety.
Additionally, the FDA acknowledges the potential to request an EUA for a COVID-19 vaccine based on an interim analysis of a clinical endpoint from a Phase 3 efficacy study, but the agency details that EUA issuance will be dependent upon meeting the safety and efficacy standards established in the “Development and Licensure of Vaccines to Prevent COVID-19” guidance. The FDA further explains that for EUA to be issued, a vaccine candidate must meet at least 50 percent efficacy and the data from Phase 3 studies should include a median follow-up duration of at least two months after completion of the full vaccination regime. The guidance also establishes additional Phase 3 clinical trial data requirements, such as detailed information on adverse reactions.
- Considerations for continuing clinical trials following issuance of an EUA – After an EUA is issued, the FDA requires that any ongoing clinical trials for a COVID-19 vaccine be completed to assess long-term safety and efficacy in sufficient numbers of subjects to support eventual vaccine licensure. The agency recommends that sponsors develop and implement strategies to ensure the ongoing clinical trials are completed.
- Considerations of an EUA for a COVID-19 vaccine by an FDA advisory committee – The FDA notes that the agency will convene an open session of the Vaccines and Related Biological Products Advisory Committee (VRBPAC) prior to the issuance of any EUA for a COVID-19 vaccine to discuss whether the safety and effectiveness data support authorization of an EUA. The guidance details that the public will have access to any briefing materials on the vaccine candidate after the conclusion of the meeting. Appendix 1 also includes the information to be prepared for a VRBPAC meeting.