Regenerative Medicine Therapies: Manufacturing Changes May Not Impact RMAT Designation
Final guidance notes that preliminary clinical evidence may come from studies conducted outside the US, says historical controls may be considered in clinical trial designs.
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Product characterization questions and partial or inconsistent clinical data are among the more common reasons why the US FDA denies, or seeks additional information about, a request for regenerative medicine advanced therapy designation, FDA’s Bryan says; sponsors should consider why they are seeking designation when deciding whether to apply for RMAT, breakthrough therapy or both.
The framework includes two draft guidance documents and two final guidances. One of the drafts, which focuses on the regulation of devices used with regenerative medicine, implements a provision of the 21st Century Cures Act and strongly suggests that most of these products will be regulated via class II.
Draft guidance suggests comparing therapies for a rare disease to each other and active control, offers examples of novel efficacy endpoints and what therapies may qualify for breakthrough and RMAT designations.