Very Brief Blog: FDA Releases Two Draft Guidances re Biomarkers for Rare Subsets and IDE IVDs in Clinical Trials

One December 18, 2017, the FDA released two draft guidances (both open for 60 days comment) on precision medicine.

• The FDA's press release is here.
• Coverage at Genomeweb here.
• The FIRST guidance, on biomarkers for rare disorders in clinical trials, is here (8pp).
• This codifies some of the principles used in the May 2017 approval of Keytruda in all solid cancers with high MSI (here).  The pivotal labeled study involved 90 colorectal cancer patiens and 59 other patients, such as 11 biliary, 9 gastric, and as few as 1 or 2 for a number of cancers.  
• It was far from conventional "proof" that Keytruda works in "all" solid cancers.
• This guidance is paired with and timed to an FDA article in Nat Rev Drug Devel (12/2018) by Schuck, Pacanowski, Woodcock, and Zineh, here.  The FDA guidance and the Schuck et al. article are mutually explanatory.
• The SECOND guidance, on requirements for Investigational Device Exemption when biomarkers are used in clinical trials, is here (26pp).  For example, an FDA approved KRAS biomarker used off label in a different tissue trial may require IDE.
• To my eye FDA has already offered this rigorous view of the need for IDE status in clinical trials, but FDA pointedly notes that perhaps not all IRBs and investigators got the point.
• FDA immediately tags this upgraded regulatory enforcement by adding it is looking to "streamline the review of oncology products and [oncology] IVDs" through additional guidance and in the near future.