At Genetic Literacy Project Ricki Lewis argues that we need to be cautious about COVID-19 treatments:
At ClinicalTrials.gov since last week, new registered studies have gone beyond China, with many from Italy and Spain, but also Egypt, Jordan, the UK, Canada, France, Germany, Denmark, Romania, Israel, and the US. In addition to more repurposing candidates are a sprinkling of studies on the social manifestations and consequences of the pandemic, such as “social media effect on knowledge dissemination.â€
[…]
Pharmaceuticals aren’t like noodles, flung against a wall to see what sticks.
But even though the “RCT†– a randomized controlled clinical trial – is widely recognized as the gold standard, many researchers, Dr. Fauci said, are uneasy at the prospect of giving a placebo in a study, even if it is an existing treatment. Is doing so a betrayal of their oath to “do no harm?â€
Fortunately, a novel clinical trial strategy, called an adaptive design, may help speed the drug approval process. Dr. Kalil describes it in his Viewpoint. It allows a clinical trial to test more than one treatment. That’s what the National Institutes of Health is doing in a trial of Remdesivir, which has been much talked about and is already through phase 2 to treat HIV/AIDS. It is an intravenous infusion.
In the end I think that Dr. Lewis fails in her argument. It amounts to “it isn’t good science”. So stipulated. Neither is issuing “stay at home” directives in the largest states in the country. When pharmaceuticals have been approved by the Food and Drug Administration for treating other conditions including conditions that are not “100% fatal”, and there is some clinical evidence and copious field experience albeit not in the U. S. it seems to me that resorting to something that isn’t pristine science is the lesser evil.
RCTs are very expensive. They are the “gold standard” but if they are underpowered (not enough trial patients) they can provide false information. People also still rely too heavily on p values. We have long used the p value of 0.05 when we should be looking 0.01 or 0.001. (This is why so many studies when repeated fail to find the same results.) So there are a lot of other ways to study stuff and a number of them are pretty good. study that is 98% as good but costs 1% of the RCT is pretty darn valuable. Just stay away from the tiny studies. I can think of multiple times when people changed practices based upon underpowered studies and we found that we were actually probably harming patients.
Steve
Dr. Fauci is acting like a bureaucrat. Bureaucrats tend to err on the side of caution because they don’t want to be blamed for anything going wrong as a result of their decisions or recommendations. No matter how much good might result from acting once a preponderance of evidence supports the action they’re resisting.
The perfect is the enemy of the good. The goal of eventual 99.99% safe and 99.99% effective ensures that anything not meeting those exalted standards cannot be used, regardless of the consequences. Would Dr. Fauci feel the same way if a loved one of his was sinking fast with Kung Flu? That’s a better standard to use IMO.
You’re being kinder than I would be. I think that the FDA and CDC are erring on the side of things that are patented or patentable.
I wouldn’t be surprised if a patent for hydroxychloroquine and azithromycin in combination is issued soon. A quick patent search didn’t reveal one but it’s a no-brainer.
The longer term question is whether the standards used by the Food and Drug Administration shouldn’t be revised. Should “safe and effective” be modified to “safe”? My support for a change in the standard for approval of medical equipment is already on the record. Presently, the standard is for approval of automated methods is that they must be better than a human practitioner. That should be changed to “no worse than”.