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- Pediatric rare cancer research draws steady investment despite biotech market volatilityThe article notes that pediatric rare cancers continue to attract investment because they address the greatest unmet medical needs and allow clear measurement of patient impact. Even as the biotec…
- Experts call for modernizing rare disease evidence models using FDA plausible mechanism frameworkA recent paper in The Journal of Clinical Investigation argues that the evidentiary approach for rare diseases needs updating. The authors suggest applying the FDA’s plausible mechanism framework,…