When we talk about disease, the big ones come to mind first. Various cancers and frightening infections rank high. And heart disease affects millions of people. The attention our ailments get from pharmaceutical companies is in direct proportion to the market size they represent.
Less common diseases don’t get the same attention - or the same research dollars. This makes September 19, 2016 a big day for individuals with Duchenne Muscular Dystrophy (DMD). That was the day the FDA granted Sarepta Therapeutics final approval for eteplirsen. This is the first and only drug on the market for the treatment of the underlying defect in DMD.
DMD is a genetic disorder. It affects boys almost exclusively. The disorder results in a lack of the protein dystrophin in their muscles cells. The absence of this protein leaves these muscle cells fragile. Most boys affected by DMD suffer from significant disability by their teens. Few live into their thirties as a result of deterioration of heart and respiratory muscles.
But few are born with DMD— only 1 in 3,500 to 6,000 males. Combine this low incidence with early age at death and the profit potential of this market is poor.. Or at least it’s not big — as it is for hypertension or cholesterol disorders. So you would expect the entry of eteplirsen to the market to be considered a big win for the little guy. Finally, a treatment for this uncommon but horrible disease!
But that wasn’t the case. After parents, muscular dystrophy groups, and Sarepta mounted a massive social media campaign and lobbied the FDA, the drug was approved. This was contrary to the recommendation of an independent advisory panel.
Further, approval was granted only a year after the New Drug Application was submitted. This process normally takes about two and half years. The FDA often requires more data from the drug manufacturer along the way. Instead the FDA decision was largely based on data from just 12 boys. The evidence is far from robust.
And while the FDA acknowledged these concerns, it was convinced that those affected by DMD deserved the chance at real treatment. It seemed like the compassionate thing to do.
But approval of the drug could be a poster child for “be careful what you wish for, you may get it.” Consider these issues:
First, eteplirsen will cost a patient $300,000 per year. Healthcare dollars are running a little short around here, so we have to consider the opportunity cost. What research or alternative treatments could those eteplirsen dollars fund instead? Will future DMD patients miss out on an opportunity for real treatment later because we are spending on an unproven treatment now?
Second, Sarepta is required, as a condition of approval, to conduct further research on the drug. But if my child had DMD, I wouldn’t sign him up for a study that might treat him with placebo. I would just get a prescription for the actual drug. This could make further research next to impossible to do well.
Finally, this will set the precedent for pharmaceutical companies to follow in the future. Will they now, as a matter of course, rally the sick and their families to social media and lobbying efforts in support of their profit motive? Of course they will.
Still, boys and young men are suffering from DMD today. Many of them could die or suffer disability waiting for a treatment to be approved tomorrow. This is why a Compassionate Use exception already exists for individuals who don’t have time to wait for the FDA approval process. Patients’ right advocates note that the Compassionate Use procedures are too cumbersome for many patients. The Right to Try movement promotes legislation to make that process simpler for those who need it.
Rather than expediting the approval of experimental treatments, the FDA should work with the Right to Try movement to improve access to these drugs for those with no other options. This will allow the FDA to keep experimental drugs off the market, while offering a chance to those who are truly desperate. We should be able to count on a drug approval process based on science, not emotion.
Amy Rogers MD is not a practicing physician and nothing written here should be taken as medical advice from either Amy or AssetBuilder. Medical decisions should be made with care in consultation with your health care provider.