Clearly, the FDA has acted too early on Emergency Use Authorizations (EUAs) for a few COVID-19 tests and products. The flurry of revocations of EUAs for tests that were deemed not reliable have cause a sputter in the onset of testing ramp up. Another specific example was the FDA’s EUA for hydroxychloroquine, which left many in the scientific community wondering how the Agency was making its decisions. Ultimately that EUA was revoked (not surprisingly). Lately, we have heard senior FDA officials opine that they would only issue EUAs or approvals (particularly those issued for vaccines) based on appropriate safety and efficacy data and not based on political pressure from above. While the administration has placed significant pressure “characterized as heat” on the Agency, I believe that the Agency will do the right thing.
The decisional memo (here) outlines the Agency’s determination that convalescent plasma “may be effective” for the treatment of patients suffering from COVID-19. The memo goes on to say, “Considering the totality of the scientific evidence presented in the EUA, I conclude that current data for the use of CCP in adult hospitalized patients with COVID-19 supports the conclusion that CCP meets the ‘may be effective’ criterion for issuance of an EUA from section 564(c)(2)(A) of the Act. It is reasonable to conclude that the known and potential benefits of CCP outweigh the known and potential risks of CCP for the proposed EUA. Current data suggest the largest clinical benefit is associated with high-titer units of CCP administered early in the course of disease. Adequate and well-controlled randomized trials remain necessary for a definitive demonstration of CCP efficacy and to determine the optimal product attributes and appropriate patient populations for its use.”
The “may be effective” designation is reminiscent of the “possibly effective” category in the Drug Efficacy Study Implementation (DESI) days, meaning that, while it might look like it works, the jury is still out about the specific mechanism underlying the efficacy. Clearly, in the time of a pandemic, it is incumbent on federal health authorities to have some latitude to allow patient access to treatments that look promising based on scientifically valid data. Early access can obviously help some COVID-19 patients based on the positive link between antibody production and the introduction of antibodies to ill patients to help them fight off the virus.
As a former FDAer, I wholeheartedly believe the Agency will rely on sound science to make future decisions (especially on a vaccine) as the consequences of making an error could be dire. Read the decision memo cited above and the FDA press announcement (here) for more details on the EUA for convalescent plasma.
