Discordance in orphan drug approvals between the U.S. Food and Drug Administration and the European Medicines Agency: A retrospective observational analysis.
BACKGROUND: The United States (US) and European Union (EU) have long-established orphan drug regulations to incentivise the development of medicines for rare diseases. Our findings suggest that orphan incentives are not the primary driver of commercial EU-launch decisions and that recent EU regulatory reform of these incentives may not achieve their goal of improving access to therapy for rare diseases.
BACKGROUND: The United States (US) and European Union (EU) have long-established orphan drug regulations to incentivise the development of medicines for rare diseases. Our findings suggest that orphan incentives are not the primary driver of commercial EU-launch decisions and that recent EU regulatory reform of these incentives may not achieve their goal of improving access to therapy for rare diseases.
What the study was
- Study design
- Retrospective study
- Category
- Drug Development
- Maturity
- Validated
- Journal
- PLoS medicine
Why it surfaced
Relevant Rare diseases with high unmet need study (high-unmet-need population coverage) meets standard-priority threshold.
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