One Insurer Wants Real-World Evidence Before Shelling Out $1 Million for a Drug

Exondys 51 is being touted as a miracle drug for those suffering from the rare disease Duchenne muscular dystrophy. But, as the New York Times reports, insurers are balking at the exorbitant price for a medication whose long-term clinical benefits have yet to be measured. In fact, Anthem says it won’t cover the drug “because the FDA reserved the right to withdraw it from the market if future clinical trials fail to show it works.” Exondys 51 (eteplirsen) is made by Sarepta Therapeutics.

The price of “miracle drugs” that benefit only a relatively small number of people represents a growing headache for health plans. As the Times reports: “While insurers once covered drugs for rare diseases as a matter of course, that may be changing now that a wave of expensive drugs have reached the market.”

An estimated 30 million people in the United States live with about 7,000 rare diseases. In the case of Duchenne, between 9,000 and 12,000 people live with the disease. And there are patient advocates for many of these diseases who will not take kindly to the health plans’ hesitancy about coverage.

“The agency’s acceptance of Exondys 51 … followed a highly polished, passionate campaign by patient advocates,” the Times reports. Robert Califf, MD, a former FDA commissioner “ignored the agency’s career experts who viewed the drug as little more than a ‘scientifically elegant placebo.’”

Source: New York Times