On Tuesday, November 2, the National Institutes of Health Center for Translational Research announced its first solicitation for collaborative research projects by NIH’s new Therapeutics for Rare and Neglected Diseases (TRND) program. Entities interested in partnering with NIH to develop clinical candidates in this category must submit initial applications by December 6, 2010.
About two weeks ago, the FDA and NIH also co-sponsored their first workshop on Accelerating Therapies for Rare Diseases, which was aimed at supporting industry efforts to develop orphan drugs. (Even though there is no official categorization of “orphan devices,” the process applies to these as well.) I attended this innovative partnership meeting, which included detailed presentations and discussions with agency officials about IND requirements, clinical trial design, endpoints and standards, and use of biomarkers and surrogates for orphan drugs; as well as how applications will be evaluated, and how to communicate with FDA.
The meeting also included a presentation on the venture community’s perspective on funding orphan drug development. Currently, there is also a federal Orphan Grants Program which provides some limited support to researchers involved in orphan drug development, up to $200,000 per year for Phase 1 and up to $400,000 per year in phases 2 and 3.
Accelerated orphan drug (and device) approval is available for previously marketed therapies with a newly determined orphan indication, or for an orphan drug which may also have potential therapeutic benefit in non-orphan indications. An orphan drug designation is not required for orphan drug grant funding, but there should be enough data to support the research hypothesis.
The application for collaborative research projects may be found here: http://bit.ly/8WWLiU
Information on the Orphan Drug Grants Program is here: http://bit.ly/lTAu7
Current grant-funded orphan drug and device research is listed here: tp://bit.ly/byrWvL
For questions about the regulatory process in this area or related intellectual property issues, please feel free to contact me.
Deanna L. Baxam
Baxam Law Group, LLC
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