Considerations for the Use of the Plausible Mechanism Framework To Develop Individualized Therapies That Target Specific Genetic Conditions With Known Biological Cause; Draft Guidance for Industry; Availability

Streszczenie

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled "Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause." The draft guidance document describes considerations for generating substantial evidence of effectiveness and evidence of safety of individualized therapies based on a plausible mechanism framework.