FDA Issues Draft Guidance for Qualifying Drug-Development Tools

The US Food and Drug Administration issued a draft guidance last week on the Qualification Process for Drug Development Tools. According to the document, drug-development tools (DDTs) include biomarkers and patient-reported outcome instruments, among other things. Specifically, the guidance offers a framework for DDT submitters to interact with officials at FDA’s Center for Drug Evaluation and Research (CDER) regarding data needed to support qualification of a DDT. The draft guidance also offers a mechanism for formal review by CDER to qualify the DDT.

“Qualification” in the document is defined as “a conclusion that within the stated context of use, the results of assessment with a DDT can be relied upon to have a specific interpretation and application in drug development and regulatory decision-making.”

FDA is issuing the draft guidance now because it has recognized that “the process of drug development and the availability of new therapies have not been as strongly affected by recent advances in biomedical science as might be possible,” states the document. Furthermore, “CDER anticipates that this guidance will encourage individuals and companies with an interest in these tools to advance their development.”

CDER has not had a qualification process for DDTs in the past. Instead, DDTs have been reviewed on a case-by-case basis, which, says the draft guidance, can inhibit the DDTs' development and potentially delay a drug-development program. A new process may provide “some degree of generalizability for use of the tool, such as use across multiple clinical disorders, multiple drugs, or drug classes. Having a qualified DDT that many sponsors will be able to use will aid in advancing therapy development and evaluation in multiple cases, and can more widely benefit patients.”

In the draft guidance, FDA proposes stages for the qualification process, including consultation and advice with CDER (e.g., letter of intent, briefing, meeting, investigation), and review for the qualification decision. The agency intends to make public its information regarding qualified DDTs.