An insider’s view of the process for approving new drugs like a COVID-19 vaccine

It appears that over the next three to 15 months, numerous new medications to treat and a vaccine to prevent contracting COVID-19 will be developed. It is imperative that the public understand the processes developed from experience over the years designed to assure the highest degree of safety and effectiveness of these drugs.

For 15 years, from 1987-2002, I served as a lay member on the institutional review board for the Mountain States Tumor Institute and St. Luke’s Hospital in Boise.

An institutional review board is charged with the obligation, among others, to review proposed new drugs, treatments and newly proposed therapies that seek to be approved for testing within the hospital. Drugs for treatment of diseases or vaccines for preventing them are tested in clinical settings for approval or disapproval, ultimately, by the Food and Drug Administration. Various “trials” occur in this process.

Phase 1 trials are generally the basic starting point. A substance or treatment has been shown to have some beneficial effects on a relatively small number of patients. Once shown to have some benefit, a Phase 2 trial may occur. Many proposed drugs do not get past Phase 1, and further testing is abandoned. In Phase 2 trials, larger groups are involved, and further study is done. Patients are studied for improvements in their condition or, in the case of a vaccine, to determine if, upon exposure to a germ or virus, the immune system keeps a disease from taking hold.

At this level, various doses of the drug may be used to determine how much of it is necessary to be effective without harm to the patient or what the lowest effective dose might be. There might also be tests using drugs in combination. Issues will be addressed as to the long-term use of a new medication and what harm it may do to patients. A drug might kill the disease but cause other diseases to arise or worsen other preexisting conditions.

A Phase 3 trial is generally the longest and largest to see the impact of the medication. A larger and more diverse group of patients is involved. A larger group may reveal problems not previously observed. A control group is given a placebo to make sure it is, in fact, the medication which is effective. Patients must give “informed consent” which includes advising them that side effects can result that have not been previously observed, up to and including death.

The institutional review board is advised of any “adverse events” in their own facility and in any facility which is taking part in the trial. The physician overseeing the trial may be asked to appear before the board and explain what occurred and his or her understanding and belief as to the cause of the adverse event or events that have occurred.

These procedures are in place for good reasons, protecting the health and safety of the patients and assuring that any new drug, vaccine, or treatment is fully vetted and safe for use in a large number of patients who will take it. If approved for general use, a new drug or treatment must be both safe and effective. Its benefits must outweigh its risks.

Institutional review boards include doctors who practice in the medical facilities involved in research, attorneys who regularly practice law in the community, researchers and lay people. Most volunteer their time and care deeply about both the welfare of patients and the work of reviewing proposed new medications, treatments and vaccines to assure that both risks and benefits are fully explained to anyone who takes part in these trials. Adverse events are reviewed on a regular basis and analysis provided of whether an adverse event, up to and including death, is related to the proposed drug or treatment or to other causes.

Trials are designed not only to determine whether a drug is suitable and safe for a given purpose but what the dose should be or if it might be effective alone or in combination with other drugs. Trials help determine how long a course of treatment should last. In the case of a vaccine, how long it might be effective or if repeat doses may be necessary, as is the case with flu vaccines.

There is no shortage of cases where a drug or treatment that shows promise is found to have unacceptable side effects and trials are shut down. There are also cases where the trial demonstrates a drug is so safe and effective that the drug is moved forward in the process so it can be made more rapidly available for its intended use.

These actions go on at sites around our country and sometimes in others. Their purpose is not to delay the introduction of new and effective drugs, treatments or procedures, but to avoid, to the extent humanly and scientifically possible, the introduction of something that is more harmful than helpful.

We want to avoid a return to the “good old days” when con men sold “Dr. Quack’s Miracle Elixir” out of the back of a horse-drawn wagon, a miracle potion that was guaranteed to “cure all that ails you.” People died as a result. That result is to be avoided.

We have come a long way since then because of rules learned the hard way. I would prefer to depend on scientists, doctors and trained chemists and researchers to rigorously test proposed new medicines or therapies as opposed to sideshow barkers or politicians (no matter their political party or gender).

That viewpoint comes from someone who has been involved in politics most of his life and whose life was saved at the age of 5 by large doses of such a drug, penicillin, which was developed for common usage over several years.

Joint efforts between the United States and Great Britain were required as well as efforts of more than one drug company to produce penicillin in quantity.

Science, not politics, perfected it for common use. Hopefully the same will be true for a COVID-19 vaccine and more effective treatments. It won’t happen in any politician’s office.