For the 15,000 boys in the U.S. afflicted with Duchenne muscular dystrophy (DMD)—a debilitating genetic disorder that leaves most of its victims paralyzed by their teens and dead by their twenties—a new drug called eteplirsen, which seemed to slow the progression of the disease in a clinical trial, could mean salvation.
However, the FDA has been skeptical of the long-term benefits and side effects of the drug and so has been reluctant to grant approval. In response, families of the ailing boys mounted a campaign to pressure the agency into reconsidering the decision to withhold approval, relying on petitions, legislative appeals and social media to get the message across that distribution of the drug could mean the difference between life and death for an entire generation of boys with DMD. The pressure seems to have made an impact; Sarepta Therapeutics, the company that developed eteplirsen, announced Monday that the FDA proposed a strategy for Sarepta to submit a new approval application and said it was willing to consider the drug for accelerated approval. This comes after the agency’s November decision that a 12-person trial was not sufficient to grant approval and that Sarepta would have to first conduct a larger trial, which could take years. The FDA’s turnaround doesn’t guarantee the drug’s approval, but it does give hope to the thousands of families facing heartbreaking eventualities.
While I’m glad that the FDA is now considering accelerated approval and that there may soon be an avenue for desperate families to try this treatment, it’s angering that our current guidelines for drug access require people to petition and plead for the ability to try and save their own lives. In the interest of “consumer protection,” these policies strip people of the right to make informed choices about what risks are worth taking to try to preserve the longevity and quality of their lives.
The current controversy with the FDA in many ways mirrors the situation with Ron Woodroof, whose battle with the FDA to be able to use and distribute unapproved drugs to treat HIV was the premise of the recent film, Dallas Buyers Club. Woodroof smuggled drugs for himself and other AIDS patients and even sued the FDA to be able to engage in consensual financial transactions with fellow victims of AIDS, a disease that, in the late 1980s, was basically a death sentence. As Forbes’s Kyle Smith said of Woodroof’s movie portrayal,
“Ron Woodroof’s story highlights how FDA policies (and, by implication, government itself) can wind up doing the opposite of what they are ‘designed’ (the word regulators and their defenders in the media have taken up to make their schemes sound more enticing) to do. The FDA is supposed to enhance American safety…. By denying potentially life-saving treatment?”
The argument for denying people the right to choose rests on the assumption that people are incapable of making informed choices to take risks, and perhaps unsurprisingly, that rational isn’t limited to medicine. Restrictions on food trucks, raw milk and a number of unlicensed services are based on the same premise that people need to be protected from making decisions for themselves.
To be sure, people have the right to make these choices based on accurate information (even if that information is that there is no available information), and if a drug company or any entrepreneur makes a false claim about a product they sell, consumers have every right to hold them accountable for fraud.
But this wasn’t the case for Woodroof and it isn’t the case for the boys afflicted by DMD. These people and their families simply want the ability to decide for themselves if it’s worth it to try unapproved drugs with unknown side effects in the interest of potentially preventing almost certain death.
If any of us found ourselves in the position of these people—or that of their loved ones—I think we would likewise want to be able to make this choice for ourselves rather than have some regulatory agency make it for us. Being able to decide what risks are acceptable and how one wants to live and die is a basic human dignity—not a privilege we plead for from the government.