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US agency will be more 'proactive' in engaging with outside groups interested in using the biomarker qualification process using the new resources and directives of the 21st Century Cures Act and the pending Prescription Drug User Fee Act reauthorization.


The 21st Century Cures Act made some small but potentially significant changes to FDA’s process for reviewing Biomarker “Qualification” requests – and the results should be a more “proactive” approach by the agency, Biomarker Development Regulatory Science Team Director Christopher Leptak said during FDA’s annual science forum May 31.


A key change derived from the Cures law signed in December is the creation of a new step in the qualification process: the submission of a formal biomarker qualification plan.


The existing biomarker qualification process begins with a letter of intent, and then a series of interactions to develop evidence and approaches to qualify the biomarker, leading (hopefully) to a formal biomarker qualification request. That process can be “lengthy” and “a bit sloppy,” Leptak said, since it is “hard to stay on the same page” as the iterative discussions drag on.


The enhanced approach will help ensure that FDA and the biomarker development group “understand what the overall game plan is and then execute it,” he said.


The Cures law also includes transparency provisions that will make all biomarker qualification requests public, along with the development plan and FDA’s eventual decision. Before Cures, qualification requests are publicized only with the permission of the requestor, and without much information beyond the basic idea. “Now, you can see our advice and use that to your advantage” in the context of other potential biomarker qualification programs, Leptak said.


Enhancing the biomarker qualification process was an early point of consensus in the Cures legislative drafting. It is also the subject of several commitments negotiated as part of the PDUFA VI agreement. (Also see "The Evolution Of 21st Century Cures Legislation" - Pink Sheet, 29 Nov, 2016.)


Both agreements also include enhanced funding for the qualification efforts – arguably the most important change of all. (Also see "Biomarker Qualification Costs As Much As Application Review, FDA Says" - Pink Sheet, 20 May, 2015.)


“There is not a preferred pathway from an FDA perspective,” Leptak stressed. In fact, “many times they are synergistic.”


Collectively, the result will likely be to put “FDA in a proactive stance around biomarker development efforts,” Leptak said. The existing qualification pathway, he said, places FDA more in a reactive position: “bring us your best idea and we will give you our advice,” and then “go away and come back” with more information.


The formal biomarker qualification pathway remains a relatively sparingly used process – but, according to Leptak, the types of biomarkers under development are broadening over time.


There are currently 27 biomarker qualification processes under way at FDA. The agency has only granted six qualifications, but as Leptak noted, many of those are for several different biomarkers. He also suggested that the range of projects has broadened. By his breakdown, eight of the projects are for “response” biomarkers, seven are for preclinical safety, six are for clinical safety, five are for patient selection and one is for a monitoring biomarker.


In addition, Leptak stressed, there are three different pathways to biomarker development. In addition to the formal qualification process, FDA also routinely reviews proprietary biomarker programs in the context of individual drug development programs. Finally, FDA also recognizes biomarkers that are identified and developed by scientific organizations.


“There is not a preferred pathway from an FDA perspective,” Leptak stressed. In fact, “many times they are synergistic.” FDA may be working with a single sponsor on an IND program at the same time a consortium is pursuing a qualification request; data developed on those separate pathways “can work together and help each other.”

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