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USFDA Guidance: Multiple Endpoints in Clinical Trials & Gene Therapy for Neurodegenerative Diseases

Earlier on 21 October 2022, the USFDA released two finalized guidances "Multiple Endpoints in Clinical Trials" and "Human Gene Therapy for Neurodegenerative Diseases".

Efficacy endpoints are measures designed to reflect the intended effects of a drug. They include assessments of clinical events, symptoms, measures of function, or surrogate endpoints that are reasonably likely or expected to predict a clinical benefit.

Using this guidance, sponsors and review staff can understand how the Agency approaches the analysis and interpretation of study results if there are multiple endpoints, and how these problems can be handled in clinical trials for human drugs, including biological products that are licensed for use in humans.

This guidance discusses a number of strategies for grouping and ordering endpoints, as well as using some well-recognized statistical methods for managing multiplicity within a study in order to limit the risk of making erroneous conclusions about a drug's effects.

Neurodegenerative diseases are a heterogeneous group of disorders characterized by progressive degeneration of the structure and function of the central nervous system or peripheral nervous system.

The guidance provides recommendations to sponsors developing human gene therapy (GT) products for neurodegenerative diseases affecting adults and children and covers the design of product development, preclinical testing, and clinical trials.


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