OPINION — Following U.S. Food and Drug Administration Commissioner Marty Makary’s recent resignation, the incoming commissioner has a powerful opportunity to follow through on the agency’s promises to enact “common-sense” approaches to getting patients quicker, meaningful treatments. For rare-disease communities, there is no more urgent moment to do so.
My daughter Keira began life as a healthy baby. By age 3, her speech lagged behind her peers, and by 5 1/2, she was diagnosed with Sanfilippo syndrome. Now 16, she requires full-time supervision and can’t be left alone safely, even for a few minutes.
If quicker, common-sense treatments are the goal, regulators must align their decisions with these diseases. Promising therapies must be given a chance to reach patients when they need them.
