The United States Food and Drug Administration has just approved the first drug aimed at treating patients who have the most common childhood form of muscular dystrophy. While this, alone, may be remarkable, it is also a marked example of the power that advocacy groups—and patient advocates, in particular—to encourage and motivate the federal government to evaluate and develop drugs.
Perhaps even more impressive, the FDA approval actually went against the recommendation of its experts. After all, the main clinical trial for this drug was very small: it involved only 12 boys who had been previously diagnosed with the disease known as Duchenne muscular dystrophy. And the study did not have the necessary control group of boys who had the disease but did not take the drug. As such, a group of independent experts met with the agency, this spring, to note there is not enough evidence to prove the vaccine is effective.
Furthermore, the approval came to the delight of those who advocated the drug and, with that notice, shares of Sarepta—who makes the drug—soared.
At the same time, this approval has also been viewed by drug policy experts as a fundamentally troubling sign that the FDA is too easily influenced—and has been for far too long—by patient advocates and drug companies. They argue that these measures have allowed the very delicate balance in drug approvals to favor quick decisions based solely on preliminary data and to deter from the more long-term conclusive evidence of safety and effectiveness.
Indeed, National Center for Health Research president Diana Zuckerman warns, “The agency has set a dangerous precedent. To prove something works, you have to compare it to something else — a placebo or a treatment. They didn’t do that.”
According to data, between 9,000 and 12,000 Americans, and most of them boys, have Duchenne muscular dystrophy. This is a rare genetic mutation, but those who have it actually lack the ability to make dystrophin. Dystrophin is a protein that acts as a kind of shock absorber that protects muscles from deterioration. As such, boys who have this condition tend to lose muscle function and require a wheelchair by their teen years and many die in their 20s.
FDA Center for Drug Evaluation and Research director, Dr. Janet Woodcock, comments”Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease. In rare diseases, new drug development is especially challenging, due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders.”
The drug is expected to cost a patient about $300,000 a year.