Biomarkers are essential to rigorous decision making in therapy development programs. Yet, funding for technical validation of assays and intra- and inter-site validation necessary for their use has not been readily available. A to-be-released NINDS program is a potential source of funding for studies to validate DM biomarkers.
NINDS has released a Notice in the NIH Guide indicating that Funding Opportunity Announcements will be released: Analytical and Clinical Validation of Candidate Biomarkers for Neurological Diseases (https://grants.nih.gov/grants/guide/notice-files/NOT-NS-18-015.html).
The Notice indicates that these Funding Opportunity Announcements will likely come out in April 2018, with applications due in June.
The NINDS Notice points out that biomarker discovery has prospered, but few candidate biomarkers have been subject to rigorous analytical and clinical validation that is essential to their use in interventional clinical trials.
The Notice of Intent suggests that research grant opportunities as well as funding for workshops will be included. NINDS indicates that this opportunity will include awards that will be milestone driven—for NINDS, this typically takes the form of annual, quantitative go/no-go milestones, although details are not specified in the Notice.
NINDS references FDA-developed biomarker guidance documents—a non-inclusive list of such documents generated by MDF is below. This reference suggests that competitive applications for the NINDS initiative likely will need to take into account FDA guidance on biomarker development and qualification.
FDA Biomarker Guidance Documents and Publications
E16 Biomarkers Related to Drug or Biotechnology Product Development: Context, Structure, and Format of Qualification Submissions, August 2011, https://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm267449.pdf
Qualification Process for Drug Development Tools, January 2014, https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM230597.pdf
Biomarker Qualification: Toward a Multiple Stakeholder Framework for Biomarker Development, Regulatory Acceptance, and Utilization. Amur S1, LaVange L2, Zineh I3, Buckman-Garner S1, Woodcock J4. Clin Pharmacol Ther. 2015 Jul;98(1):34-46. doi: 10.1002/cpt.136. https://www.ncbi.nlm.nih.gov/pubmed/25868461
FDA-NIH Biomarker Working Group: BEST (Biomarkers, EndpointS, and other Tools) Resource, April 2016, http://www.biostatsolutions.com/wp-content/uploads/2016/11/Bookshelf_NBK326791.pdf
Framework for Defining Evidentiary Criteria for Biomarker Qualification, October 2016, https://fnih.org/sites/default/files/final/pdf/Evidentiary%20Criteria%20Framework%20Final%20Version%20Oct%2020%202016.pdf
FDA Case Study: BIOMARKER QUALIFICATION—DEVELOPING AND OBTAINING REGULATORY ACCEPTANCE OF A NEW BIOMARKER, November 2016, https://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/UCM531522.pdf
Updated Process for Qualification of Drug Development Tools Under New FD&C Act Section 507, June 2017, https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm561587.htm