DMD Advocates Lose Approval but Win One Step Ahead in Advocacy

In today’s New York Times, Andrew Pollack reports on the FDA Advisory Committee “no” vote for a new treatment for Duchenne muscular dystrophy. This is the second time in recent months that advisors to the FDA have refused to grant approval for a DMD treatment, despite patient advocate’s urgent pleas to bring needed therapies to market. “Some of the panel members had trouble reconciling the often compelling patient testimony with the FDA’s legal requirements,” wrote Pollack. “The controversy over eteplirsen is perhaps the most vivid example of how patients and patient advocates are playing a growing role in the FDA’s evaluation of drugs. This can result in intense pressure on the agency to approve drugs,” added

In rare diseases, there are often too few people with a disease to allow companies to enroll sufficient numbers of patients for large scale trials. As a result, regulatory requirements can be at odds with patient pleas for access to drugs that seem to work for them. “Patients have long pressed for approval of drugs that the FDA found only marginally effective, if at all,” adds Pollack. “Critics say that approving drugs that do not really work is offering false hope to patients and removing an incentive for drug companies to develop better products,” he adds. But, Pollack also notes that patient input is now intensifying due to social media and the FDA’s increasing consideration of patient input.

A big disappointment for patient advocates yesterday but one additional step forward in being heard by FDA.

Authored by Ann Moravick, president, Rx4good