Before a new drug is approved for use by the Food and Drug Administration in the US, say for cancer treatment, it has to go through quite a stringent process of at least three phases of clinical trials, with each phase having a different purpose.

• Phase I trials test if a new treatment is safe and look for the best way to give the treatment. Doctors also look for signs that cancer responds to the new treatment.
• Phase II trials test if one type of cancer responds to the new treatment.
• Phase III trials test if a new treatment is better than a standard treatment.
• Phase IV trials find more information about long-term benefits and side effects.

The Phase I trial stage is where a new treatment can get shut down quickly if it shows signs of causing harm. Phase II is meant to show that the treatment does work in the way advertised. Phase III can be a difficult bar to reach because you need to show that the new treatment is better than what is already available.

Nowadays most people think of these measures as reasonable precautions to prevent people from being harmed by untested drugs and other treatments. But as Dhruv Khullar writes, the idea that the government should be able to decide what people can put into their bodies has been, at least in the US, a controversial issue, and at one time there was nothing to stop people from doing whatever they liked.


The idea of medical freedom—that we should be allowed to decide what to do about our own health—has long had a special status in the United States. “A significant portion of Americans have embraced freedom of therapeutic choice as a constitutional value for most of the country’s history,” Lewis Grossman, a medical historian, writes in his book “Choose Your Medicine.” Benjamin Rush, a Founding Father and a physician, criticized the medical establishment for being closed-minded about treatments recommended by non-physicians. “Many of our most useful remedies have been discovered by quacks,” he argued. In the late nineteenth century, the psychologist and philosopher William James wrote, “If the word ‘sacred’ can be applied to any personal right, surely the right to treat one’s own body as one chooses may claim this title.” A few years later, the Postmaster General stopped delivering mail to the American School of Magnetic Healing, arguing that the organization was committing medical fraud. The school sued, contending that its methods were legitimate, and the Supreme Court ultimately ruled in its favor. “There is no exact standard of absolute truth by which to prove the assertion false and a fraud,” the majority opinion said.

In the twentieth century, however, scandals involving unregulated medical products helped swing the balance of power toward regulation. In the thirties, elixir sulfanilamide, an antibiotic mixed into a toxic solvent, gave patients intense pain, kidney failure, and neurological problems. After more than a hundred people, including many children, died across fifteen states, Congress passed the Federal Food, Drug, and Cosmetic Act of 1938, which authorized the F.D.A. to evaluate a medical product’s safety before it could be sold. The agency’s modern-day role was defined in 1962, after thalidomide caused birth defects in thousands of babies around the world. The Act was amended to include a much higher standard: drugmakers now needed to conduct controlled trials in order to demonstrate that their products were effective. The gap between the two standards was immense. Around two-thirds of drugs that met the 1938 safety requirement failed the 1962 efficacy bar.

The AIDS epidemic changed this dynamic. Large numbers of people were dying and approval of new drugs was taking a long time. Sufferers demanded the right to receive treatments that had shown some signs of efficacy but had not met the proper threshold for FDA approval. They argued that since they were going to die soon, what greater harm could befall them from some untested drugs? So the standards were loosened to allow limited use of new treatments in extreme cases such as AIDS.

But nowadays people are demanding the right to use treatments even when they are not in acute danger but want to alleviate some condition or just improve their sense of well being. Khullar writes that the latest movement is to inject oneself with peptides (short chains of amino acids), that have been promoted by so-called ‘wellness influencers’ as a solution to all manner of problems, such as a substitute for steroids in fitness regimens. Certain peptides are said to enhance strength, energy, endurance, and recovery, even though they have never been tested in humans. You can now order them from compounding pharmacies that can produce custom medications that differ slightly from the ones that require FDA approval, thus bypassing some of the safety rules. Some of these drugs are sold as nutritional supplements, which are not regulated as drugs. Anecdotal stories of their efficacy spread like wildfire on the internet.

In the era of Make America Healthy Again, the popularity of peptides has risen. Many compounding pharmacies are experiencing soaring demand; the Times reported that U.S. imports of gray-market peptides and hormones from China roughly doubled last year. The podcaster Joe Rogan has credited BPC-157 with healing a case of elbow tendonitis in two weeks. Beauty influencers who want deeper tans and enhanced libidos are taking Melanotan II, also known as the “Barbie peptide.” Even New York magazine recently published a freelance writer’s account of self-injecting peptides, titled “Life on Peptides Feels Amazing,” which failed to cite any peer-reviewed research or academic scientists. In February, R.F.K., Jr., said, on Rogan’s podcast, that he had taken peptides himself and that, under his leadership, the F.D.A. would stop restricting many of them. Kennedy, who has railed against the agency’s “aggressive suppression” of unproven treatments, has vowed to “end the war.”

It is not hard to see where this is heading in the Trump-Kennedy era.

It is becoming possible to imagine a future in which American regulators lose their grip on the safety and the efficacy of drugs. The venture capitalist Jim O’Neill, Trump’s former acting director of the Centers for Disease Control and Prevention and the current nominee to lead the National Science Foundation, has said that, as soon as drugs are proved to be safe, Americans should be able to “start using them at their own risk. . . . Let’s prove efficacy after they’ve been legalized.” Most states have enacted so-called right-to-try laws, which enable patients with life-threatening diagnoses to receive treatments that have completed Phase I safety testing. (More than ninety per cent of such treatments will not be F.D.A.-approved.) In recent years, Montana state laws have gone even further, granting all patients access to an even wider array of experimental treatments. The state may become a hub for medical tourism. Clinicians are shielded from many kinds of disciplinary action, and they can practice for up to twenty-one days a year without holding a Montana medical license.

The idea of injecting myself with untested drugs fills me with horror. But is is clear that I am an old fuddy-duddy, not in tune with the wild, regulation-free future where people view skeptically those things that have a long history of testing and approval of things like vaccines and drugs, and enthusiastically embrace ‘stuff they read about on the internet’.

This is a common sociological phenomenon. When some major catastrophe occurs, (as in past financial crises), strict regulations are put in place to prevent recurrence, even though it hurts corporate profits. We then enter a period of stability and calm. But that very calm seems to make people forget that it was those regulations that created it and they fall for the tale told by corporate types that we do not need regulations, that ‘the market’ will take care of itself, and the regulations are then ditched, leading to the next catastrophe, while the corporate types make windfall profits.

(See my review of the very entertaining and informative 2015 documentary Boom Bust Boom narrated by the late Terry Jones of Monty Python fame for more details on this phenomenon. He discusses economist Hyman Minsky’s theory that stability breeds instability because in times of prosperity, people forget that it was the regulations imposed after the previous bubble burst that produced the stability and growth, and they think that we have entered a new era where the old rules don’t apply and start relaxing all those controls leading to new bubbles.)

In the case of financial catastrophes, what results are massive financial hits for ordinary people in the form of losing jobs and homes and savings. In the case of vaccination denial and the ingestion of untreated drugs, the catastrophes are going to take the form of outbreaks of avoidable illnesses plus people requiring emergency care.

That is where the Trump-Kennedy Make America Healthy Again (MAHA) movement is taking us.