“Too often, regulatory tools authorized to speed patient access to breakthrough treatments are being used in ways that even prevent approval,” writes Kat Bryant Knudson.
When I was first diagnosed with limb girdle muscular dystrophy (LGMD), I was devastated to learn there wasn’t any treatment or cure. For those of us living with progressive diseases, time is not neutral. Every month a promising therapy is delayed, we will have irreversible loss of muscle function.
Rare disease drug development is in the midst of an extraordinary era of scientific innovation. Researchers are advancing gene therapies and targeted treatments unimaginable just a decade ago. The Food and Drug Administration has opportunities today with new leadership to modernize, including the utilization of innovative trial designs and tools so the United States continues to lead the world in rare disease drug development.