On June 30, 2020 FDA issued a new guidance on Development and Licensure of Vaccines to Prevent COVID-19. The guidance sets regulatory expectations for the manufacture, non-clinical, clinical and post-licensure use of the vaccine(s). While the agency is cognizant of the urgent need for a COVID-19 vaccine, sponsors must demonstrate that the vaccine is at least 50% effective vs. a placebo control.
Currently the FDA asserts that traditional approval should be obtained, as there are currently no surrogate endpoints predictive of clinical benefit. Therefore the use of surrogate endpoints cannot be used as a means to accelerated approval. FDA does leave open the possibility though, if vaccine immunological responses can predict protection against COVID-19, then accelerated approval could be considered.
The FDA also allows for EUA (emergency use authorization), if the sponsor can demonstrate safety and effectiveness. However FDA cautioned that issuing an EUA prior to executing a large, randomized clinical trial could hamper the ability to demonstrate clinical effectiveness and impact licensure.
For clinical trials, FDA advised that late phase trials should include thousands of patients, including ethnically diverse populations, the elderly and patients with comorbidity. Trials should be randomized, double blind and placebo controlled.
For CMC, FDA expects the usual demonstration of product quality, development and control of the manufacturing process and characterization. If the sponsor is using a platform technology for its vaccine, and has existing marketed or investigational products, then development could potentially be accelerated and some aspects of the other products manufacturing process development could be leveraged. FDA indicated it may not be able to perform on site inspections due to the current COVID-19 pandemic.
For non-clinical, FDA expects safety studies for novel vaccines. If a platform technology is used, then existing toxicology and biodistribution data from commercial or investigational products could potentially be leveraged.
Food and Drug Administration (FDA). (2020). Development and Licensure of Vaccines to Prevent COVID-19. Retrieved from source.