On March 12, 2024 the FDA updated early Alzheimer’s drug development guidance to add support for biomarkers, surrogate endpoints.
Background
FDA is announcing the availability of a revised draft guidance for industry entitled “Early Alzheimer's Disease: Developing Drugs for Treatment.” This draft guidance is intended to assist sponsors in the clinical development of drugs for the treatment of the stages of sporadic AD that occur before the onset of overt dementia. This draft guidance revises the draft guidance for industry entitled “Early Alzheimer's Disease: Developing Drugs for Treatment” issued February 16, 2018 (83 FR 7060), and reflects FDA's consideration of public comments on the draft guidance. This revision describes FDA's current thinking regarding the use of biomarkers for the selection of participants with early stages of AD for enrollment in clinical trials, the selection of outcome measures for clinical trials in early AD, and the use of effects on characteristic pathophysiological changes of AD to support approval in these populations.
https://www.federalregister.gov/documents/2024/03/12/2024-05178/early-alzheimers-disease-developing-drugs-for-treatment-draft-guidance-for-industry-availability
A general section on outcome measures has been changed in the latest revision, now incorporating descriptions surrounding
the use of co-primary endpoints and cognition originally found in the background section the 2018 draft guidance. The outcome measures section contains subsections describing clinical endpoints, time-to-event analysis, and surrogate endpoints. “Sponsors considering the use of a biomarker as the primary measure of effect should discuss their plans with FDA early in development,” FDA wrote. “In general, even if accelerated approval is considered as the initial approval pathway, clinical outcome assessments should be included in clinical trials for early AD to assess early clinical changes that may potentially provide support for any changes observed on biomarkers.”