FDA, Hard-To-Treat Diseases
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AI could review data sets to assist clinical scientists
FDA Will Drop Two-Study Requirement for New Drug Approvals, Aiming to Speed Access U.S News February 18, 2026 https://www.usnews.com/news/business/articles/2026-02-18/fda-will-drop-two-study-requirement-for-new-drug-approvals-aiming-to-speed-access
The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay Prasad in November, was criticized for lacking detailed guidance. Agency leaders elucidated on the pathway for personalized medicines on Monday.
The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.
Plasmalogen precursors for Alzheimer’s are named as first program candidate There are nutrients and dietary compounds
Drugmakers are urging the FDA to reassess the regulatory process of switching medications from prescription to over-the-counter, a rigorous procedure that must demonstrate a patient no longer needs a doctor’s supervision to access a treatment.
The decision follows a pattern of policy shifts, including the rollback of COVID-19 recommendations and cancellation of mRNA funding.
The drug would need to be taken long before symptoms first appear—but it could be used to help protect at-risk individuals.