The FDA released today a new draft guidance for comment on individualized therapies: "Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause"
With the recent announcement that the Agency would accept a single pivotal trial, this draft guidance fits well within that framework and the press release noted this is for "targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations"
The Introduction lays out what is needed for a 'plausible mechanism framework' that will achieve approvals for these therapies.
"The purpose of this guidance is to describe considerations for generating substantial evidence of effectiveness and evidence of safety for individualized therapies based on a plausible mechanism framework. The plausible mechanism framework outlines a set of recommendations to help developers of individualized therapies generate sufficient clinical safety and efficacy data to demonstrate that a drug or biological product is safe and effective for the intended use, and that the product can be manufactured to regulatory quality standards. These data are used to support approval or licensure of an individualized therapy for a specific indication. This includes a careful evaluation of the results of nonclinical and clinical data and chemistry, manufacturing, and controls (CMC) data necessary to support product quality. Application of the plausible mechanism framework involves:
• Identifying a specific genetic, cellular, or molecular abnormality with a clear connection between specific alteration and disease indication
• Developing a therapy that targets the underlying or proximate pathogenic biological alterations
• Relying on a well-characterized natural history of the disease in an untreated population
• Confirming that the target was successfully drugged or edited or both
• Demonstrating improvement in clinical outcomes or course"
Considerations for the use of the Plausible Mechanism Framework
| U.S. Food and Drug Administration |
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| Considerations for the use of the Plausible Mechanism Framework |
| This is the draft guidance Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause |
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#fda #indivdualizeTherapy #PlausibleMechanismFramework
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Mark Arnold Ph.D., FAAPS
Westampton, NJ
[email protected]Bioanalytical Solution Integration
LinkedIn:
https://www.linkedin.com/in/markearnoldphd/Website & Blog: Bioanalysis & Biomarkers <bioanalysisandbiomarkers.blogspot.com>
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