The Food and Drug Administration has issued draft guidance for industry on drug development for rare disease. The goal of the guidance is to "assist sponsors of drug and biological products intended to treat or prevent rare diseases in conducting more efficient and successful development programs through a discussion of selected issues commonly encountered in rare disease drug development." The selected issues include the question of what constitutes an adequate amount of natural history, the selection of endpoints and use of biomarkers.
My initial take on the guidance is that the majority of the content is devoted to describing elements of drug development that are the same for both common and rare disease. At the end of each major section there are a few comments about the difficulties of developing drugs for rare disease, but little accommodation. Important issues such as the use of surrogate markers for slowly progressive disease and trial design for small and/or heterogeneous populations are not addressed in detail. Although a good documentation of the status quo, it will seemingly take legislation such as the 21st Century Cures Act, which passed out of the House of Representatives in July, to creatively solve the challenges that are particular to drug development in rare disease.
You can download a copy of the guidance here, and you can submit comments on the guidance at the same site before October 16, 2015. MDF, in consultation with industry and academic partners, will plan to submit a comment on this guidance in the context of specific issues relevant to myotonic dystrophy.
If you are interested in working with MDF to submit comments on the draft guidance, please email me by September 17th.