Basic Clinical Research

Basic Clinical Research
Leonard Zwelling

In 1995, when I became the Associate VP for Clinical and Translational Research at MD Anderson, I was thoroughly unqualified for such a title. I was a bench scientist who had done very little translational research and no clinical research beyond being a fellow in the Medicine Branch of the National Cancer Institute doing the scut work associated with the clinical research of others. I knew how a clinical study was supposed to be run, but little else. I had a lot to learn and many patient faculty members who were kind enough to teach me.

One of the great revelations I learned about a good phase 3 clinical trial was that the best of protocols had within it clear descriptions of when interim analyses were to be performed, what was to be examined during the analyses, and what would constitute the statistical rules for early stopping, like excess toxicity or overwhelming evidence of efficacy. I am going to assume the same must be true of the phase 3 clinical trials being done right now to identify a safe and effective vaccine against covid-19. But, according to The New York Times article above, these details are being kept from the public and from most scientists and that is giving many critics of the trials fresh ammunition with which to scare people, because our ability to get back to normal depends upon the development of a safe, effective vaccine that people actually take. It’s that last part that’s the catch. Right now, a lot of the left fears that political pressure will be brought to bear on the federal regulators to green light a vaccine before one is really ready. The right contains many anti-vaxxers who won’t take a shot no matter who says it works. Thus, there is a significant segment of the population who won’t participate in a broad-based vaccination effort even when a good vaccine is developed. That is really sad and it will adversely affect the acquisition of herd immunity.

Fortunately, there’s an easy solution. The CDC, the NIH and the FDA can ask President Trump to demand that the clinical protocols for the three most advanced phase 3 trials currently under way be widely published now or the results cannot be sent to the FDA for marketing approval. I assume the protocols have all been evaluated by a legitimate IRB and I see no reason to keep the plans a secret. Only then can the public have confidence that the trials are well-designed and well-audited and that the Data Safety Monitoring Boards doing the interim analyses are using widely-accepted criteria of safety and efficacy when evaluating the vaccines on trial. This would not compromise the trials as the results of the interim analyses would stay secret unless a vaccine is found to be too toxic or by contrast ready for use.

What I fear is a declaration by one of the pharmaceutical companies that it has a safe and effective vaccine just prior to Election Day and people refuse to take it because they don’t trust those declaring success.

This is in marked contrast to what I experienced as a child when we were all rushed to the Bridgeport, CT health department to get our Salk polio vaccines and eventually the Sabin sugar cubes as well.

It’s bad enough the trust has been so eroded in our many governmental institutions that the President of the United States had to downplay the threat of the coronavirus at a time when its toll on America could have been lessened. But, alas, that’s where we are. If we now find that science has risen to the occasion to develop a vaccine and the world walks the other way because it doubts the safety or effectiveness of the vaccine, that would be a double tragedy.

Let’s get the details of the studies out for everyone to see and when the data indicate a safe and effective vaccine has been developed, that data ought to be made public as well.

If we don’t do this now, we may be deep in the lockdown forever with the antidote for the lockdown in our grasp. Another tragedy!

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