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Real World Evidence

All articles tagged with #real world evidence

FDA approves leucovorin for ultra-rare CFD-FOLR1 subset without trial
health4 hours ago

FDA approves leucovorin for ultra-rare CFD-FOLR1 subset without trial

The FDA updated the leucovorin label to treat cerebral folate deficiency caused by FOLR1 mutations (CFD-FOLR1), a ultrarare condition linked to autism features, approving the drug based on published case reports and mechanistic rationale rather than a randomized trial. The decision narrows the target population to CFD-FOLR1 after reviewers found the highest-quality data in this subgroup; the move relies on real-world evidence and a plausible mechanism pathway. GSK will stop manufacturing Wellcovorin, but generics exist and importation is allowed; broader autism use remains controversial and calls for more research.

via Fierce Pharma|
#cfd-folr1#fda#health
FDA Adopts One-Pivotal-Trial Path for Drug Approvals
health19 days ago

FDA Adopts One-Pivotal-Trial Path for Drug Approvals

The FDA will default to approving drugs based on a single well-controlled pivotal trial, while allowing supporting confirmatory evidence such as mechanistic data, related indications, animal models, or real-world data. The move, framed by FDA leaders as a shift from an outdated ‘two-trial dogma,’ aims to speed development and cut costs, but has drawn criticism from some policymakers and former FDA officials who warn it could weaken patient protections. The policy preserves discretion to require additional studies in certain cases, and reactions are mixed among analysts and industry figures, with Pazdur expressing concerns before his retirement.

via BioSpace|
#clinical-trials#confirmatory-evidence#fda-approvals