WIKIMEDIA, TOM VARCOWith growing interest from the pharmaceutical and biotech industries in addressing diseases that afflict fewer than 200,000 Americans, the US Food and Drug Administration is going to ramp up its efforts to help drug makers develop treatments for rare diseases, the agency announced in a draft, five-year plan released last week. Starting late next year, the FDA's Center for Drug Evaluation and Research will bolster its Rare Disease Program (RDP) by providing pharmaceutical and biotech companies specialized training on the development of drugs for rare, or orphan, diseases and improving its outreach to rare disease patient organizations. According to the draft plan, the agency will also add five new positions to the staff of the RDP, retool the way it evaluates the success of the program, and hold public meetings to hear concerns and suggestions from stakeholders in rare disease drug development. On October 14th of this year, the FDA will hold a public meeting to hear feedback on the draft recommendations.
The 44-year-old patient has Hunter syndrome, which doctors hope to treat using zinc finger nucleases.