Hello.
I’m Dr. Kylie Haskins with the FDA’s Center for Drug Evaluation and Research, also known
as CDER.
In this module, I will highlight some of the many opportunities that biomarker developers
interested in qualifying a biomarker have for interacting with CDER’s staff.
These interactions can help guide biomarker development from the early phase of development
through qualification of the biomarker.
Biomarker development can be a long and resource-intensive process.
CDER offers multiple opportunities for biomarker developers to engage with FDA staff, which
can help to clarify the path and ensure that activities stay on track.
FDA offers biomarker developers interested in seeking qualification three primary opportunities
for engagement: the Critical Path Innovation Meeting, the Letter of Support Program, and
the Biomarker Qualification Program.
An initial opportunity for biomarker developers, in the early stage of development, to engage
FDA is the Critical Path Innovation Meeting.
These meetings cover innovative strategies that aim to address a broad range of challenges
in drug development.
Among these innovations are biomarkers in the early phase of development that are not
yet ready for CDER’s Biomarker Qualification Program.
A Critical Path Innovation Meeting offers an opportunity for CDER and the biomarker
developer to explore the potential of a proposed biomarker.
The meeting is not a forum for obtaining regulatory decisions and it is not a formal part of the
biomarker qualification process.
Instead, the intent of the meeting is to help developers understand some of the important
regulatory questions FDA may have about the proposed biomarker and obtain general advice
early in the development process.
Topics for meetings held to date are publicly available on the program’s website.
The second opportunity is for biomarker developers with preliminary data to engage with CDER
staff through the Letter of Support Program.
Biomarker developers can request a Letter of Support from CDER for promising biomarkers
that have a potential application in drug development.
CDER will evaluate the acceptability of the request and determine whether to issue a letter.
A Letter of Support is meant to enhance the visibility of a biomarker, demonstrate the
FDA’s support for the development of the biomarker, encourage collaborative efforts,
and stimulate additional studies.
The letter briefly describes CDER’s thoughts on the potential value of a promising biomarker
across multiple drug development programs and encourages further evaluation.
The letter does not connote qualification of a biomarker and does not endorse a specific
biomarker test or device.
CDER has issued 14 Letters of Support for promising biomarkers and these letters are
publicly posted on the program’s website.
A third opportunity for biomarker developers to engage with FDA is the Biomarker Qualification
Program, which requires a greater level of data than the Letter of Support Program.
The qualification process includes three submission milestones that have increasing levels of
evidence required: the Letter of Intent, the Qualification Plan, and the Full Qualification
Package.
Specifically, at each submission milestone, the qualification requester will receive a
formal written determination letter from the FDA accepting or declining the submission.
This letter will also include the rationale for the decision, advice, and requests for
additional information, as appropriate.
Formal written determinations will be publicly available on the FDA website and will be updated
on at least a biannual basis.
Throughout the biomarker qualification process requesters can engage the program to seek
clarification regarding the content of a determination letter or to obtain additional information
about the qualification process, as needed.
After qualification, if additional data is generated for the biomarker that supports
an expanded context of use in drug development, the biomarker developer can seek qualification
of the biomarker for the expanded COU and may engage with the FDA throughout the new
qualification process.
Throughout biomarker development there are opportunities to interact with FDA to enhance
the prospects for successful development and qualification of a biomarker for a specified
context of use.
This module served as a brief introduction to opportunities for engaging with FDA about
qualification during biomarker development through the Critical Path Innovation Meeting,
the Letter of Support Program, and the Biomarker Qualification Program.