Charged with spurring greater R&D efforts on new drugs for rare diseases, the FDA's newly formed rare disease review group plans to meet today and tomorrow and will hear about a host of new ideas on a richer set of research incentives.
Conducting early studies on new therapies for rare diseases has been the subject of growing industry interest with the launch of special units devoted to the field at both GlaxoSmithKline and Pfizer. But BIO and other organizations say that an expanded period of market exclusivity--adding to the special provisions already in place--is needed to spur more work in the field.
According to a report in Reuters, roughly one in 10 of all Americans suffer from a rare disease, but there are treatments available for only 3 percent of them. "Even if you have very targeted and effective product development, it does cost real money and people will expect a return on investment," Jesse Goodman, chief scientist and deputy commissioner for science and public health at the FDA, tells the wire service.
Genzyme, of course, has been making a significant amount of money from rare diseases for some time, which has helped spur other biopharma outfits to follow in its footsteps. Back in February, the NIH and FDA announced that they would jointly work on new tools and regulations that would help spur rare disease drug programs. One of the suggestions that developers will pose to regulators this week is that fewer trials should be required to gain approval on a new therapy for a rare disease.
- here's the story from Reuters