Hope, Not Hype For COVID Therapies

President Trump has fueled tremendous hype for the impact on COVID-19 of the combination of an antimalarial and an antibiotic (hydroxychloroquine + azithromycin, known by the brands as Plaquenil and Z-Pak).

As I've described, this is based on an uncontrolled study in 20 patients, only 6 of whom received the combination. The impact of the hype is that the drugs are in such short supply that there may not be enough available to conduct the clinical trial(s) necessary to know if the drug is safe and effective to use, and if so, in whom.

Perhaps I come across as negative/pessimistic about the potential impact of these drugs. That is not my intent. Rather, I know from a wide range of professional experience that until the clinical trials are performed with the right design, we can be misled by early results.

There used to be a statement ascribed to actor Christopher Reeve that I doubt he ever said: "If I were a laboratory rat, I'd be walking by now." That this became a statement I heard more than once is testimony to the amount of ground-breaking data from laboratories that never pans out. And it can be worse than that.

Those of us experienced in clinical research will easily recount treatments that appeared to be game-changers in early clinical trials and went on to be shown to hurt or kill people (encainide, flosequinan, milrinone, estrogen/progestin, cisapride, nitric oxide, tolbutamide, etc.). And the more subtle possibility that a drug provides neither harm nor benefit is also a risk, as it distracts providers from using better options, which in our current state includes enrolling patients into clinical trials.

This is why we need to conduct properly designed clinical trials starting last month. Doctors in Wuhan started to do this within days of the outbreak starting. In the US, we are still not doing so efficiently enough.

Neither hype nor hope is a strategy that leads to success. Instead, we need data generated in randomized, controlled clinical trials. In the coming days, I'll share a plan for doing exactly that.