One of the most infuriating aspects of the Trump Administration’s roll-back of federal regulatory powers is the implied assumption that those laws and regulations exist for no reason. That’s dangerous nonsense. Those laws were passed in response to real, serious risks.
The major revisions to the Food and Drug Administration laws and regulations in the 1960s were created in response to the thalidomide crisis of 1957-1964. Thalidomide was first marketed in 1957 in West Germany under the trade-name Contergan. The German drug company Chemie Grünenthal developed and sold the drug. Primarily prescribed as a sedative or hypnotic, thalidomide also claimed to cure “anxiety, insomnia, gastritis, and tension.” It was used against nausea and to alleviate morning sickness in pregnant women. Thalidomide became an over-the-counter drug in West Germany on October 1, 1957. Shortly after the drug was sold in West Germany, between 5,000 and 7,000 infants were born with phocomelia – malformation of the limbs. Only 40% of these children survived. Throughout the world, about 10,000 cases were reported of infants with phocomelia due to thalidomide; only 50% of the 10,000 survived. Those subjected to thalidomide while in the womb experienced limb deficiencies in a way that the long limbs either were not developed or presented themselves as stumps. Other effects included deformed eyes and hearts, deformed alimentary and urinary tracts, blindness and deafness.
The FDA refused to approve thalidomide for marketing and distribution. However, the drug was distributed in large quantities for testing purposes, after the American distributor and manufacturer Richardson-Merrell had applied for its approval in September 1960. The official in charge of the FDA review, Frances Oldham Kelsey, did not rely on information from the company, which did not include any test results. The FDA instructed Richardson-Merrell to perform tests and report the results. The company demanded approval six times, and was refused each time. Nevertheless, a total of 17 children with thalidomide-induced malformations were born in the U.S.Thalidomide was never approved in the Unite States. Frances Oldham Kelsey received the President’s Award for Distinguished Federal Civilian Service from President John F. Kennedy for blocking sale of thalidomide in the United States.
thalidomide is what happens when a drug is inadequately tested before it is used, or when it is used for something other than its recommended purposes. The FDA exists to try to prevent another thalidomide.
Which makes the Right to Try Act of 2018 an extremely dangerous law.1 The law cut out the FDA’s role in approving and overseeing the use of experimental drugs in patients with life-threatening diseases. Such patients will be able to work directly with a doctor and a drug company to gain access—outside of a clinical trial—to an experimental therapy that has only made it through early clinical trials and not obtained FDA approval. Incredibly, if Frances Olham Kelsey were alive today she could not say “No” to thalidomide if it were prescribed for someone with a “life-threatening condition.”
Okay, you say, what is a “life-threatening condition.” The new law uses a definition lifted from a regulation, 21 C.F.R. §312b, and means “Diseases or conditions with potentially fatal outcomes, where the end point of clinical trial analysis is survival.”
Folks, life is a condition with potentially fatal outcome and the point is survival. The definition is so all-encompassing as to be meaningless. In effect, the staged trials for approval of new drugs has been repealed. Drug companies are free to peddle their experiments to physicians and patients willy-nilly.
But wait, there’s more. The FDA is forbidden to use the outcomes of these experiments in deciding whether to approve the experimental drug. So if the stuff turns out to be as evil as thalidomide, that can’t be the basis for future FDA action. Nor can a patient poisoned by the untested gunk sue the drug manufacturer or his or her doctor.
In the case of real final stage diseases, the patient and the patient’s family are desperate. They will try anything. A dear friend of WC’s whose wife was dying of breast cancer, flew her to Mexico for laetrile treatments, long after laetrile – apricot pit pull – had been completely discredited. A metabolic byproduct of laetrile is cyanide. Persons given laetrile can develop symptoms of cyanide poisoning. Those are the person who will be going to these drug companies. Desperate people don’t always have the best judgment.
But the bigger concern is that the camel’s nose of the drug industry is now well and truly under the tent flap for FDA regulation, and given the hysterical reaction by the law’s sponsor, Senator Johnson (R, Wisconsin), to the suggestion that the FDA should have a role, the rest of Big Pharm is sure to follow.