The U.S. Food and Drug Administration (FDA) on issued draft guidance explaining its qualification process for drug development tools (DDTs) in line with the 21st Century Cures Act. The draft guidance came ahead of the deadline set in the Cures Act and fulfills some of FDA’s Prescription Drug User Fee Act (PDUFA VI) commitments to enhance its DDT qualification pathway for biomarkers. Section 507 of the Federal Food, Drug, and Cosmetic Act, as amended by the Cures Act, defines DDTs as biomarkers, clinical outcome assessments and “any other method, material, or measure that the Secretary determines aids drug development and regulatory review.”
Guidance
As FDA notes, DDTs are methods, materials, or measures that can aid drug development and regulatory review. Under new section 507 of the FD&C Act, qualification and qualified mean a determination that a DDT and its proposed context of use (COU) can be relied upon to have a specific interpretation and application in drug development and regulatory review. A qualified DDT used within the COU may be used to support or obtain approval or licensure (as applicable) of any drug or biological product, provided the qualification has not been rescinded or modified.
FDA further states that encouraging the identification and use of reliable DDTs can significantly advance the development of new, safe, and effective drugs. Qualified DDTs allow integration of innovative technology and approaches to conditions or diseases that may create opportunities in new areas of drug development as knowledge of disease and pathogenesis advances. For example, using a DDT to enrich a study population with individuals exhibiting certain characteristics may help to reduce the size of the study population and may shorten the duration of the study. Qualifying a DDT is a stepwise process; if at any stage a DDT is determined to be not accepted or not qualified, a requestor may take into account the input from the FDA and subsequently resubmit.
The guidance addresses many areas, such as communication between the DDT requestor and CDER or CBER. As the agency describes, the purpose of these communications may be to identify challenges and opportunities, guide the collection of data, request input on a proposed COU, identify the level of detail appropriate for a given stage of submission, or obtain clarification on considerations and recommendations. Requestors should contact the appropriate qualification program for additional information on meeting type and scheduling and submission of pre-meeting materials, if applicable. A requestor may submit a request for a teleconference or other meeting type at any time. Once an FDA project lead has been identified for the project, all communications and exchanges of information related to the project should be directed to that project lead to facilitate the review process.