The FDA failed the U.S. public by approving Biogen’s new Alzheimer’s drug against the recommendation of its expert advisory panel and its own statisticians. That event led me to explore whether this was an aberrancy or part of a pattern, and what I found disturbed me.
The FDA’s origins lie in the Food and Drugs Act, passed by Congress in 1906 and signed into law by President Teddy Roosevelt, which prohibited interstate commerce in misbranded and adulterated foods and medications. Much of the impetus for getting this bill enacted was the revelations by such “muckraking” journalists as Upton Sinclair, whose novel The Jungle exposed unsafe and unsanitary conditions in the meatpacking industry.
Probably the high point of the FDA was in 1961, when Canadian-born Dr. Frances Kelsey refused to allow the sale in the U.S. of thalidomide without further safety data, and her judgement was rewarded when the drug, widely prescribed in Europe for morning sickness, was found to cause serious birth defects.
This episode in turn led to passage of the Kefauver-Harris amendment to the Food and Drugs Act which required that all new drugs demonstrate “substantial evidence” of efficacy as well as safety. This is where the recent FDA decision failed to meet the intent of the law, but it is not the only time.
Under pressure from AIDS activists in the early 1980’s, the FDA developed an accelerated pathway to approve promising new drugs for otherwise poorly treated diseases, with the proviso that on-going studies be done to prove that the drugs fulfilled their promise. A study published this year in the British Medical Journal found that many drugs approved under this accelerated pathway stayed on the market for years or decades without the required studies being done and with major doubts about their efficacy persisting.
I am particularly concerned about the frequent approval of new cancer drugs, most with huge price tags and major side effects, based on “surrogate outcomes:” measures of changes in blood tests or imaging findings that might show the disease is responding, but which do not show meaningful results in terms of prolonging life.
The FDA is also responsible for regulating medical devices such as implants and pacemakers. Here too, as a ProPublica investigative report found, the FDA has often deferred to manufacturers and allowed dangerous products to stay on the market far too long.
How can these problems be improved?
First, as much as possible, the FDA, and the CDC, should be insulated from political pressure. The heads of these agencies should have terms that do not overlap with presidential terms and should be able to be removed only for malfeasance, so that they can make decisions based on science, not politics. Many public health decisions involve trade-offs but improving a president’s reelection odds should not be a factor when it comes to saving lives.
A much more “arms-length” relationship between the regulators at the FDA and the device and pharmaceutical industries must be enforced. No revolving door between agency and industry should color the regulators’ decision making.
The FDA should not be allowed to overrule the decisions of medical experts unless these decisions are close. If a vote is 6-5 with one abstention, there is room for the FDA to decide on approving a new drug based on their judgement. When there is a super majority vote, it should be respected.
Let us demand that the FDA uphold Dr. Kelsey’s standards.
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