On July 15, a Duke-Margolis workshop showcased an early deliverable of an ongoing Biotechnology Innovation Organization-sponsored research project: a preliminary policy roadmap to improve biomarker utilization in product submissions. The event offered a forum for live reactions and ideation from industry and regulatory perspectives, and AgencyIQ has highlights.
Background: Challenges in biomarker development
- Biomarkers are a vital tool for disease diagnosis, patient risk assessment and drug development. The FDA describes biomarkers as defined, measurable characteristics of the body that indicate biological and pathogenic processes, or the response to an intervention such as a medical product. While clinical assessments can capture how a patient feels, functions or survives, biomarkers provide researchers with a valuable window into the body’s inner workings. These tests can be molecular, histologic, radiographic or physiologic.
- The FDA validates biomarkers in two principal ways. First, biomarker use can be validated for an application-specific use through the agency’s review and approval of a new drug or biological product. Second, biomarkers can be validated for a given context of use through the agency’s formal Biomarker Qualification Program, which was established in 2007 and formalized in 2016 through the 21st Century Cures Act. The BQP uses a three-stage pathway for groups to submit prospective biomarkers for FDA verification within a specific context of use. Once qualified this way, a biomarker can be incorporated into any drug development program within its qualified context to support regulatory approval.
- The FDA has qualified only 11 biomarkers through the BQP, spurring criticism that it may not be as effective as intended. Research published by the Friends of Cancer Research in October 2025 found that the FDA’s review of letters of intent and qualification plans far exceeded goal timeframes, and some stakeholders have attributed the underperformance to a lack of dedicated resources. [See AgencyIQ analysis here for additional background.] In practice, this has left many sponsors with no choice but to pursue validation through a product application.
Workshop discusses biomarker policy roadmap
- The July 15, 2026, event was driven by an ongoing Duke-Margolis research project that aims improve the use of biomarkers in individual product submissions. An important note on scope: the project does not pertain to the FDA’s formal BQP. Rather, this work is sponsored by the Biotechnology Innovation Organization trade group.
- The project has generated an early deliverable, referred to as the Preliminary Policy Roadmap, which bills itself as a “preliminary inventory of policy opportunities rather than a final, prioritized set of recommendations.” This document is organized around four goals: (1) operationalizing fit-for-purpose biomarker evidence development, (2) improving transparency and consistency in FDA review, (3) making FDA biomarker feedback earlier and more actionable and (4) building shared infrastructure and incentives for biomarker evidence generation and reuse. For each goal, the roadmap assigns more specific actions to stakeholders such as the FDA, sponsors, consortia, assay developers or providers.
- The July 15 event provided an overview of the roadmap, followed by presentations of two use cases (minimal residual disease in multiple myeloma and Roche’s NeuroToolkit). The subsequent sessions of the event were devoted to moderated panel discussions of the recommendations provided to accomplish these goals. A final panel synthesized the feedback received and looked ahead toward next steps. AgencyIQ has distilled highlights below.
- In service of the roadmap’s first goal, operationalization, panelists for the workshop’s first session reacted to a Modular Acceptability Framework for biomarker development presented by Sanofi’s PUJITA VAIDYA. According to Vaidya, the framework was developed by a “small regulatory policy working group” of personnel from Biogen, BioMarin and BridgeBio. The resource presents endpoint archetypes across risk modules. For each module, the risk level was determined by considering the ability to leverage prior work and the level of residual uncertainty. Vaidya explained, “We propose a structured acceptability framework that can tell sponsors and regulators exactly what evidence is needed for what type of endpoint in what regulatory context, and the purpose really is to focus our resources – regulators and sponsors’ resources – on generating the most critical evidence that’s required to establish novel endpoints across diverse regulatory scenarios by creating clear linkages between scientific objectives that we have and the regulatory requirements that are there.” She acknowledged that this framework is in early stages, but it’s “designed to provide greater regulatory predictability and efficient development, support both sponsors and FDA efficiency, provide expectation guardrails, and encourage leveraging prior work to have it be more of a scalable model overall.”
- Panelists representing both industry and regulatory perspectives weighed in on the proposed framework and other recommendations in the preliminary roadmap. ISSAM ZINEH, director of the Center for Drug Evaluation and Research’s Office of Clinical Pharmacology, said: “I love this idea of residual risk. I think it’s a very much an important concept, and evidentiary standards or requirements should really be driven by tolerance for residual uncertainty calibrated to the question that the biomarker is intending to answer.”
- Zineh pushed back on a few elements of the broader roadmap. He spoke to calls for regulatory consistency that have arisen when a biomarker is accepted in one context but not another. “What we’re really driving for is consistency in the thought process,” he said. “The outcomes may be different, and maybe they need to be appropriately contextualized. But it’s the consistency in sort of the evidence assessment.” Zineh also noted that the preliminary roadmap contains “a huge inventory” of FDA-directed tasks. While biomarker development is a priority, he explained his view that the roadmap underestimates the operational costs and feasibility of the agency completing this work. From Zineh’s perspective, overreliance on biomarker development as a regulatory issue can obscure independent bottlenecks, such as scientific maturity and sponsor fragmentation and inconsistency.
- The workshop’s second session focused on shared infrastructure, incentives and evidence-reuse mechanisms – the roadmap’s fourth goal. The panelists described the tensions and opportunities of collaboration in the precompetitive space. The Foundation for the NIH’s STEVE HOFFMAN said the first-mover burden cannot be overcome by smaller companies but, rather, must be addressed through partnerships or larger philanthropic funding sources. To enable these types of partnerships, NICOLE GORMLEY, former director of the FDA’s Division of Hematologic Malignancies 2, emphasized the importance of early alignment and data standardization. Hoffman weighed in: “I think there’s a misconception that data reuse is often a legal problem, right? Much of it is really a planning problem, the old adage of ‘a failure to plan is a plan for failure.’ If you’re not thinking about future use and your consents, reusable governance, standardized agreements, and these interoperable data standards, then you’re having an issue.”
- The third core session of the workshop integrated the second and third goals of in the preliminary roadmap, focusing on biomarker-related review clarity and engagement opportunities. Friends of Cancer Research President and CEO JEFF ALLEN spoke to the need to increase transparency around context-specific biomarkers that have not gone through the agency’s formal qualification programs. Allen acknowledged that this information can sometimes be found in summary basis-of-approval documents made available for initial approvals but noted that biomarker utilization sometimes occurs in supplemental applications. “I think we’re seeing interest in a greater level of transparency for some of these review documents,” he said, adding that the agency’s push to publicize complete response letters could provide further insights with appropriate redaction. [See AgencyIQ’s analysis of the FDA’s July 2026 CRL release here.] ALLYSON BERENT from the Foundation for Angelman Syndrome Therapeutics emphasized the need to provide visibility into the FDA’s feedback on biomarkers outside the individual product application. She later proposed an incentive for sponsors to share data with advocacy groups or forums like the Critical Path Institute or NIH, suggesting that sponsors who share could receive something akin to a priority review voucher.
- The workshop concluded with a look toward refining the roadmap, and Duke-Margolis’ VALERIE PARKER offered four takeaways. First, she described the consensus that a framework is needed that is not a universal checklist, but a product that “calibrates evidence to the biomarker’s intended decision, context of use, prior evidence and residual uncertainty.” Second, Parker emphasized the need to “treat biomarker development as a shared infrastructure problem, not primarily an FDA problem.” Third, she stressed the importance of prioritizing prospective harmonization and evidence reuse by assigning “complementary roles to FDA sponsors, consortia, patient groups, assay developers, researchers, neutral conveners, philanthropists and government funders.” Last, Parker highlighted the need to “make regulatory learning and engagement more structured and actionable.”
- Zineh provided thoughts on how the risk-based credibility or evidentiary framework for biomarkers could be created. He pointed to the International Council for Harmonisation’s M15 guideline on model-informed drug development, describing it as “completely portable” and noting its origins in engineering principles. “There are all kinds of sectors that we can sort of borrow and synthesize this idea of what are the dimensions and domains of credibility around a biomarker, depending on what we’re hoping to use it for. So, I think that would be a worthwhile consortial effort,” he said. Zineh also reiterated that the FDA faces “a bandwidth problem,” and suggested an action item: “I would encourage, maybe an action item is to take a look at the universe of resources that not just FDA but EMA, PMDA, other regulators globally put out there that try to speak to this question of biomarker science. And then see, OK, well, are there gaps that then we can begin to fill either from a knowledge management and dissemination standpoint or an engagement standpoint?”
Analysis
- The policy project is poised to continue refining its preliminary roadmap based on the feedback received during the workshop. While a specific timeline is not provided, the next phase of the project is set to include workshop analysis and survey analysis prior to revision and publication of the roadmap.
- While the FDA recently announced a new “Biomarker Incubator” program, this effort was not a focal point of the workshop’s discussion. Revealed through a May 12 request for information related to a pilot project for aggregating data on biomarkers of drug-induced kidney injury, the incubator is meant to “complement” the FDA’s existing qualification process. The agency requested input on the “scope and direction” of the incubator, and the comment period closed July 13. [Read AgencyIQ analysis here.]
Featuring previous research by Ned Pagliarulo.
To contact the author of this item, please email Amanda Conti ( aconti@agencyiq.com).
To contact the editor of this item, please email Jason Wermers ( jwermers@agencyiq.com).