FDA issues guidance on genomic biomarker submissions

Lack of standards is often cited as a reason biomarkers are slow to travel from lab to FDA approval. Perhaps in an answer to that criticism, the FDA has released a new guidance document on qualification of genomic biomarkers. It was developed by a group with the rather windy title of the Efficacy Working Group of the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use. As Antoinette Konski, a Foley & Lardner partner who focuses on life sciences IP, notes on the firm's Personalized Medicine blog, "The guidance is provided to facilitate a consistent format for the submission of data to the end of easy review and exchange of assessments."

The document says that while biomarkers have "great potential for drug development," the FDA guidance lays out parameters within which a biomarker can be relied on to accurately reflect what's going on in the body and can be used from drug discovery through post-approval of a drug. The FDA document, which involves recommendations only and nothing legally enforceable, is based on the agency's previous experience with submissions that involve biomarker data. That means either standalone biomarkers or ones used as a piece of a larger product-marketing application. Konski points out that the document focuses on genomic biomarkers, but the principles can be applied to other biomarker categories such as genomics, proteomics, imaging and a combination of biomarkers.

However, the document notes that while the structure of biomarker submissions for FDA approval should be consistent "regardless of the context proposed," they also need to be "flexible enough to deal with the specific attributes of each submission." Here is where we get into some of the difficulties inherent in biomarkers, since they can mean different things within the contexts of different diseases or in combination with other biomarkers. That is why, the FDA guidance says, "It is therefore only possible to provide general guidelines on data format for a biomarker qualification submission."

So, to be clear, the FDA is beginning to think about standards for biomarkers. But, in the same breath, acknowledges what many in biomarker research already know: that coming up with standards is extremely difficult.

- read Konski's synopsis in the Personalized Medicine Bulletin
- or download a PDF of the FDA's guidance on biomarker submissions