Slide 1

Hello. I’m Marianne Noone with the FDA’s Center for Drug Evaluation and Research, also known as CDER.

In this module, I’ll explain the biomarker qualification process and discuss the submission roadmap for biomarker qualification requesters.

Slide 2

The Biomarker Qualification Program was established to encourage the development of biomarkers for use in drug development, to facilitate an efficient review process, and to make information on qualified biomarkers publicly available.

So, why qualify a biomarker? A qualified biomarker can be used in multiple drug development programs without a need for CDER to reconfirm the suitability of the biomarker’s qualified context of use, and a qualified biomarker has the potential to advance public health by streamlining the drug development paradigm.

Slide 3

The process for biomarker qualification is being modified in response to legislation passed in 2016. This module is an introductory guide through the submission stages involved in the biomarker qualification process.

Under the 21st Century Cures Act enacted on December 13, 2016, the new Section 507 Qualification of Drug Development Tools was added to the Federal Food, Drug, and Cosmetic Act and formally establishes an updated, multistage process for biomarker qualification. This updated process includes three submission stages: the Letter of Intent, the Qualification Plan, and the Full Qualification Package.

FDA can decide whether to accept a qualification submission at any of these stages based on a number of factors, including the scientific merit of the qualification submission.

Slide 4

Let’s take a closer look at the three submission stages. For each of the stages, FDA will either accept or decline the submission. If FDA makes a determination to accept the submission, this means that the requester can proceed to the next stage.

For the first stage, a requester submits a Letter of Intent, or LOI. The LOI provides the initial information about the submission and may include general information about the biomarker, the proposed context of use, information on how the biomarker will be measured, and the drug development need the biomarker is intending to address. The LOI submission will be reviewed to determine if the biomarker has the potential to address an unmet need in drug development. If accepted, the requester may submit a qualification plan when ready.

The Qualification Plan, or QP, describes the proposed development plan to generate the necessary supportive data to qualify the biomarker for the proposed context of use in drug development. The QP should include detailed information on the suitability of the biomarker measurement method, as well as summary data on completed studies and study designs of planned future studies to confirm the usefulness of the biomarker in drug development. The QP submission will be reviewed to determine the acceptability of the plan to support the qualification of the biomarker. If accepted, CDER will provide the requester with the instructions for the final submission stage along with potential information requests.

The third and final stage is the submission of the full qualification package, or FQP. The FQP contains all accumulated data to support the qualification of the biomarker for the proposed context of use. FDA will make a final decision about whether the biomarker is qualified based on a comprehensive review of the FQP.

Once a biomarker is qualified, FDA will publicly post the qualification determination on the Biomarker Qualification Program website. Upon qualification, the biomarker may be used under the context of use for which it is qualified in any CDER drug development program to support the regulatory approval of a new drug.

Slide 5

The 21st Century Cures Act includes new transparency provisions for biomarker qualification submissions. In keeping with these requirements, FDA will make public information about biomarker submissions that are in the Biomarker Qualification Program. This information will be available on the Biomarker Qualification Program website.

FDA will post information with respect to each biomarker qualification submission under the qualification process, including the stage of the review process, the date of the most recent submission, the summary data and evidence contained in the submission, and the FDA’s formal written determination at each stage.

Upon issuing a qualification determination, FDA will post summary reviews that document the assessment of the submission and any rescissions or modifications to the qualification determination. Information will be updated at least twice a year.

Slide 6

This module introduced you to CDER’s updated process for biomarker qualification. For more information on the Biomarker Qualification Program, please see the program’s website. For more information on biomarker qualification submissions, please see the website on the updated drug development tool qualification process.

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