Should a Head-Spinning “Cure” and “Blessing From God” Abort Well-Established FDA Processes?

Whatever one’s political views, the COVID-19 diagnosis and treatment of the president, was certain to raise issues and questions. My original thoughts for this blog post were to discuss those. It is now focused on the potential effects and consequences due to the recovery of the president from his COVID-19 illness, if indeed that is what happened.

As most people know, the president was given an investigational drug cocktail from Regeneron, an antiviral drug remdesivir, as well as steroids and perhaps other treatments unknown at this time.

The world is still learning how to treat COVID-19 and a treatment course that includes multiple therapies, one of which is investigational, does not prove the safety and effectiveness of the therapy as a whole, or of any individual component. The president may have had the same course, or a different one with or without those treatments.

Everyone should be thankful that he appears to be getting better, and should hope that course continues in a positive direction. Causation science does not rest on one patient’s experiences (i.e., did the drug in question cause the president’s outcome?).

Still, the idea that just because one individual, even understanding that he is the president, may feel that he got better and ‘beat’ COVID-19 because he took an investigational drug cocktail and it should be made available for free and be delivered by the army to hundreds of thousands of our citizens is head-spinning. The effect (or not) on one individual does not demonstrate the safety and effectiveness of a drug across a population. This is the very definition of an n of 1. In addition, the president was reportedly given remdesivir and steroids (and perhaps other therapies) which also could have contributed to his recovery (if he has recovered).

Yes, the FDA drug and device approvals can be slow and cumbersome. However, there is a reason that controlled Phase-3 clinical trials are needed to prove the safety and effectiveness of a drug or combination of drugs before FDA can approve them. Clinical trials for the Regeneron cocktail drug are ongoing and the results, while preliminarily reported to be positive, are not completed.

To add the positive experience of one patient (n of 1) to preliminary promising results in a clinical setting―with dosages, timing, and other concurrent therapies undoubtedly not aligning with those in the clinical trials―and conclude that proves it is time for the army to deliver hundreds of thousands of doses to COVID-19 patients is worrisome.

After the president made his comments, Regeneron asked the FDA to authorize its investigational drug for emergency use. Maybe Regeneron read the political environment as positive for such an application given the president’s comments. Just a couple of days before, its CEO admitted that the ‘compassionate use’ granted to President Trump put Regeneron in a “very tough situation” and that the compassionate use exception is intended for small numbers of patients.

Of course, the FDA must decide whether to grant the emergency-use application based on the available data provided to it. One hopes that the n of 1, albeit an important one, does not interfere with the medical and scientific judgments that must be made about safety and effectiveness of a drug potentially helpful to our citizens stricken by the terrible virus causing this pandemic.

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