Presentation (CTI)

OBJECTIVES: Objective: To assess the clinical benefit of orphan drugs approved in China and the United States and to evaluate the net health benefit of orphan drugs approved in China.
METHODS: Methods: Clinical trial studies of orphan drugs approved in China and the United States were systematically identified through multiple databases. Key efficacy outcomes were extracted, and meta-analyses were conducted when appropriate. Pooled results were evaluated against minimal clinically important differences (MCIDs) or relevant clinical thresholds to determine meaningful clinical benefit. For orphan drugs approved in China, net health benefit was assessed by estimating quality-adjusted life-years (QALYs) forgone based on incremental costs and health opportunity costs.
RESULTS: Results: A total of 92 orphan drug-condition pairs approved in China and 316 approved in the United States were included. Clinical trial evidence was identified for 60 Chinese drugs (226 trials) and 151 U.S. drugs (369 trials). In China, 47.8% of orphan drug-condition pairs demonstrated significant clinical benefit, 12.0% showed limited benefit, 5.4% showed no clear benefit, and 35.9% lacked clinical evidence. In the United States, corresponding proportions were 38.9%, 8.9%, 5.1%, and 36.7%, respectively. Economic evaluations of 19 Chinese orphan drug-condition pairs showed that 42.1% demonstrated positive net health benefit.
CONCLUSIONS: Conclusion: Only half of orphan drugs in China and one third in the United States demonstrated significant clinical benefit. Fewer showed positive net health benefit in China, underscoring the need for stronger policy consideration of drugs lacking clinical or societal value and addressing substantial evidence-policy gaps.