DUCHENNE muscular dystrophy is a horrible disease. Afflicting mainly boys, it weakens their muscles and eventually confines them to wheelchairs. In the end, typically when they are in their 20s, it kills them. Patients and parents are understandably elated, therefore, at the decision taken last month by America’s drug agency, the Food and Drug Administration (FDA), to approve the first treatment for Duchenne. No one could fail to be moved by their campaign to win approval. At an FDA meeting this year one sufferer pleaded: “please don’t let me die early.” Nonetheless, the decision bodes ill for drug discovery in America. Sarepta Therapeutics, the firm behind the drug, did not meet the usual standards for approval to market it. Staff at the FDA’s drug-evaluation division are sceptical about the efficacy of Exondys 51 (also known as eteplirsen). They argue that the clinical evidence before them involved a flawed experiment on only 12 patients. But Janet Woodcock, the division’s director, overruled them. The FDA has asked Sarepta to conduct further trials to confirm that its drug works.

Shareholders in Sarepta do not have to wait that long to reap the benefits. After the news of the approval broke, the firm’s share price rose from $28 to $49. On September 28th a health insurer announced that it would pay for the drug in certain cases (others are not so keen). The treatment will not come cheap: its gross annual cost could be as much as $665,600 per patient. Ms Woodcock made the argument that Sarepta needed to be “capitalised” to fund more research. Her wish has now been granted. Sarepta looks a picture of corporate health: it has been doing deals, securing the rights to another anti-Duchenne drug on October 4th, and issuing new shares.

The judgment is the more worrying because it does not come out of the blue. Politicians have been urging the FDA to be more responsive to the needs of patients; the families of Duchenne sufferers did what anyone would, given the choice between the certainty of a bad outcome and the possibility of a better one, and campaigned for treatments to come to market. Exondys 51 was approved under a special programme that allows drugs to go on sale more quickly when they treat grave conditions with unmet needs. But that should not mean that standards for clinical trials are watered down.

Patients, not always a virtue

The pact between drugmakers and society is that innovative drugs win market exclusivity as a reward for the money that has been spent developing them. Weakening the FDA’s standards for approval has two pernicious effects. First, it creates an incentive for meaningless innovation: for drug companies to tilt investment towards drugs that may not work but which patients definitely want, and towards pursuing novelty at the expense of efficacy. Second, it passes more of the risk involved in drug development to consumers and taxpayers. Politicians in America routinely castigate the pharmaceutical industry for charging too much for its products. And now Sarepta’s post-hoc testing programme will, in effect, be paid for by the American government and by insurers.

Payers ought to dump this conundrum back into Sarepta’s lap and insist on a rebate arrangement if the drug proves to be ineffective. And in the future the FDA ought to stick to the clear standard that has served it well for decades: do not approve a drug unless it has been shown to work.