On August 10, 2011, the Food and Drug Administration (FDA) released a guidance document describing recommendations regarding context, structure and format of regulatory submissions for qualification of genomic biomarkers. The guidance document was developed within the Efficacy Working Group of the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). 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 guidance document notes that qualification is a conclusion that within the stated parameters, a biomarker can be relied upon to adequately reflect a biological process, response, or event, and support use of the biomarker during drug or biotechnology product development, from discovery through post approval.

Background and Scope

The guidance document notes that biomarkers have great potential for drug development. For example, they can guide dose selection and provide safer and more effective therapies. A qualification submission can include information for a single genomic biomarker, or for multiple genomic biomarkers used as classifiers. The guidance document focuses on genomic biomarkers, but it is noted that the principles described in the guidance document are applicable to other biomarker categories such as genomics, proteomics, imaging and a combination of biomarkers.

5 Recommended Parts or Modules

Five sections or modules should be included in the submission. Briefly, the five sections should contain the following information:

  1. Regional Administrative Information: The submission should contain applicable forms and/or cover letter. The guidance document indicates that the relevant regulatory authorities can specify the content and format of this section
  2. Summaries: A summary that discusses the strengths and weaknesses of the submitted data should be provided. In addition, it should describe the key characteristics of the biomarker and the context of use, e.g., the general area of use (pharmacology, toxicology, safety, efficacy), the purpose or use of the biomarker and the critical parameters of the context of use. A high level summary of the methods and results should be included, as well as a critical assessment and appraisal of the overall results. This section should also contain a conclusion that provides an assessment of expected benefits, any issues encountered during the qualification studies, and any unresolved issues and why they should not be barriers to qualification for the proposed context of use. A synopsis of the individual studies should be included, which can include information from scientific publications.
  3. Quality: The guidance document notes that drug or biotechnology product quality and manufacturing data would not be expected in a stand alone biomarker qualification submission independent from an New Drug Application (NDA), Biologics License Application (BLA) or Market Authorization Application (MAA).
  4. (Clinical) and 5. (Nonclinical): These sections should provide full study reports, including raw data if requested. The rationale for the the selection of the population sample as well as other variables related to the phenotype that was studied should be provided.