News that the Food and Drug Administration (FDA) approved the first new drug to treat sickle cell in nearly 20 years last Friday understandably drew cheers from patients and advocates who have stood by with bated breath for a breakthrough. But as laudable as the milestone may be, the treatment's limited reach underscores just how hard it is to create groundbreaking medicines which do little more than staunch certain symptoms.
The FDA's latest approval is for Endari, a drug created by the under-the-radar biotech Emmaus Medical Inc. As the agency itself notes, Endari is no cure. Rather, it's approved to "reduce severe complications associated with the blood disorder," such as extreme side effects in the lungs and major pain. The drug also cut down patients' needs to go to a hospital for sickle-cell related pain compared with placebo in clinical trials.
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This is all welcome news for people who suffer from the genetic blood disorder, whose various forms afflict some 100,000 Americans at any given time, according to the National Institutes of Health. But there's still a long way to go when it comes to combating a lifelong illness.
The slow pace of progress is common in these kinds of diseases—one which are degenerative and afflict just a sliver of the population. In recent years, the FDA has ramped up its efforts to approve drugs for rare diseases. That includes some controversial decisions to clear treatments that tackle a smattering of symptoms or are basically unproven from a clinical standpoint (consider the agency's recent approvals for Duchenne muscular dystrophy medicines in the wake of patient group pressure).
The FDA has gone even further in some cases. Several months ago, regulators approved the first new amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, drug since 1995. In fact, the FDA actually asked the treatment's manufacturer to submit it for U.S. regulatory approval following promising results in Japan. Once again, this new drug, while historic, is not a cure.
That may be of little concern to patients and their families desperate for any treatment avenue. But the high costs of these drugs, which are particularly hard to test and manufacture given the limited number of patients, raise potential ethical and financial concerns. Some insurance companies have been reluctant to cover therapies like the newly-approved Duchenne drugs because they may not produce results in line with their prices.
And if medicines which simply swat back some of a disease's major symptoms, how much would an actual cure cost? At the moment, a number of companies—like Bluebird Bio—are trying to create gene therapies which may eliminate sickle cell disease. It may be unfair to put a price tag on good health. But it's an issue that patients and the health care system at large will have to address as more novel medicines hit the market.