FDA Drug Development Tool qualification program

FDA's Drug Development Tool (DDT) qualification programmes let a requestor ask the agency to accept a biomarker, clinical outcome assessment or animal model for a defined context of use, so that sponsors can use it in any drug development programme without re-arguing it each time. The process was set in section 507 of the FD&C Act by the 21st Century Cures Act of 2016 and has three stages: Letter of Intent, Qualification Plan and Full Qualification Package. Throughput has been slow: by mid 2025 only eight biomarkers were qualified, seven of them under the older legacy route. Nothing opened for this entry shows a prion disease biomarker in the programme. It matters to prion work mainly as the formal route for any future surrogate or enrichment marker, distinct from the Rare Disease Endpoint Advancement pilot.

Key facts

  • The DDT qualification programmes cover three tool types: biomarkers, clinical outcome assessments and animal models used under the Animal Rule.
  • The qualification process rests on section 507 of the FD&C Act as set up by the 21st Century Cures Act of 2016.
  • Qualification means FDA concludes a tool can be relied on for a specific interpretation and application within a stated context of use; the biomarker is qualified, not the measuring assay.
  • Stage 1 is a Letter of Intent describing the need, biomarker, context of use and measurement; stage 2 is a Qualification Plan summarising evidence and gaps; stage 3 is a Full Qualification Package on which FDA decides.
  • A qualified tool is publicly available and can generally be used in IND, NDA or BLA submissions without FDA reconsidering its suitability for that context of use. FDA posts submission summaries, determinations and a list of qualified biomarkers.
  • The review targets of about 3 months (LOI), 6 months (QP) and 10 months (FQP) come from FDA's November 2020 final guidance, not from statute; FDA pages opened here do not state them.
  • A July 2025 analysis found 61 of 99 listed biomarker projects accepted; 30 sat at Letter of Intent, 18 at Qualification Plan and 8 at Full Qualification Package.
  • In that analysis median LOI review took 6 months against the 3 month target, median QP review 14 months, and building a QP after LOI acceptance took a median 32 months (47 for surrogate endpoint projects).
  • The same analysis counted eight qualified biomarkers, seven under the pre-Cures legacy process, with the latest qualified in 2018.
  • FDA announced on 2025-12-08 the qualification of AIM-NASH, an AI-assisted liver biopsy scoring tool for MASH trials, describing it as the first AI drug development tool qualified.
  • A Letter of Support is a lighter instrument: CDER's view on a promising biomarker not yet in the programme. It does not qualify the biomarker or endorse a test.
  • Plasma neurofilament light is the main neurodegeneration precedent: FDA accepted an FNIH Biomarkers Consortium Letter of Intent on 2024-05-16 for early detection of impending symptomatic familial FTD in GRN mutation carriers, for trial selection. NfL also received a Letter of Support for progressive MS in June 2021 (excerpt only).
  • Collaborative requestor groups that include at least one non-profit and one for-profit organisation are encouraged.

Timeline

  1. 2014Earliest Letter of Support date on FDA's list (last listed 2024-08-19).
  2. 201621st Century Cures Act enacted; section 507 FD&C Act sets the three stage qualification process.
  3. 2020FDA final guidance on the qualification process sets the 3, 6 and 10 month review targets.
  4. 2021NfL receives a Letter of Support for progressive MS trials, per a charity page seen as a search excerpt.
  5. 2024FDA accepts the FNIH Biomarkers Consortium Letter of Intent on plasma NfL for familial FTD.
  6. 2025Data cutoff of the Friends of Cancer Research timeline analysis (published online 2025-10-26).
  7. 2025FDA announces qualification of AIM-NASH for MASH trials.

Last checked 1 November 2026

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