It's a famous story. Thalidomide is a sedative which was prescribed for morning sickness in pregnant women, starting in about 1960, in countries around the world. Not, however, in the U.S., because an official of the Food and Drug Administration named Frances Kelsey felt the application had insufficient data on safety, including whether the drug crosses the placenta. Turns out it does, and it can cause severe birth defects in which babies are born with flipper-like limbs.
At that time the FDA did not oversee clinical trials, and it pretty much took the drug companies' word for it about safety. As the linked article says:
The tragedy surrounding thalidomide and Kelsey’s wise refusal to approve the drug helped motivate profound changes in the FDA. By passing the Kefauver-Harris Drug Amendments Act in 1962, legislators tightened restrictions surrounding the surveillance and approval process for drugs to be sold in the U.S., requiring that manufacturers prove they are both safe and effective before they are marketed. Now, drug approval can take between eight and twelve years, involving animal testing and tightly regulated human clinical trials.
Drug companies don't necessarily like this. Some do, because they feel they depend on their reputation for safety, although as we have discussed here many times they frequently game the system by keeping unfavorable trial results hidden, and other unsavory practices. Recent efforts at reform have aimed at improving the system, by requiring registration of clinical trials and public availability of all results, among other reforms.
Comes now a reality TV star who wants to appoint as FDA commissioner a man with no research experience or academic background who is an investment adviser and member of pharmaceutical company boards. As Daniel Carpenter informs us in NEJM, "The Trump administration’s approach to the Food and Drug Administration (FDA) is guided by a libertarian belief in markets over science, and Gottlieb apparently shares this view." The idea is that bad drugs will be driven out of the market by consumer experience.
Of course, if your baby is born without arms it will be a bit too late for you, but that isn't the only problem with this philosophy. Lots of diseases and symptoms get better on their own. You can't necessarily tell whether the medication helped or not. Nor do you necessarily know that an adverse effect is attributable to the drug. You should read Carpenter's essay, but here's how he finishes:
The medical community and the Senate should greet this nomination with scrutiny. To this end, I propose some questions for Gottlieb. Perhaps the most important is one that can be answered only by his behavior: Will Gottlieb, if confirmed, listen more to FDA scientists or to his Trump administration superiors, corporate-board colleagues, and think-tank associates? At stake is not just the FDA, but the scientific regime of clinical pharmacology and the credibility of American therapeutics.
Questions for Scott Gottlieb.
- • You will have to recuse yourself from decisions about certain companies’ products because of conflicts of interest. How many companies, and which ones?
- • You have argued that there are “interim endpoints that can be used to more quickly gauge a medicine’s benefit.” Under your leadership, how would the agency commit to restricting the use of a drug or removing it from the market if later-stage evidence turns out to present a much weaker benefit profile?
- • You have argued that the FDA has an “unreasonable hunger for statistical certainty.” How, then, do you explain the fact that the FDA approves new drugs and devices more quickly than any other regulatory agency? Do you see accelerated approval, compassionate use, and breakthrough designations as inadequate, and if so, why?