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#RareDisease #ClinicalTrials #DrugDevelopment #MarketAccess #HTA #RegulatoryAffairs #PatientAdvocacy #Biotech #EvidenceGeneration #HealthEconomics Seventy-seven percent of drugs for rare diseases with pivotal efficacy data have orphan designations, and most are approved with Phase III randomized controlled trials (Domike et al, 2024)

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