Reconceiving orphan drug market exclusivity as a conditional public-law entitlement: a sustainable regulatory governance model with comparative lessons for China.

Background: Rare disease patients, who collectively number over 400 million worldwide, remain systematically underserved within existing universal health coverage frameworks. Orphan drug market exclusivity is a widely adopted regulatory incentive designed to stimulate pharmaceutical innovation for rare diseases. Recent regulatory reforms in China, the United States, and the European Union have introduced conditional mechanisms, signaling a shift from static proprietary protection toward a conditional regulatory governance model. Japan, while not undertaking legislative reform in 2026, offers a distinct reference point through its re-examination system. To date, no study has systematically integrated these latest legislative reforms into a comparative regulatory analysis or examined how the institutional design of orphan drug exclusivity can be calibrated to balance innovation incentives with equitable patient access. Methods: This study employs an integrated multi-method approach combining comparative legal analysis, doctrinal interpretation, evidence-based policy evaluation, and case analysis. A structured analytical framework is constructed around four key regulatory dimensions-eligibility criteria, exclusivity duration, scope of protection, and exception mechanisms. Using this framework, the study compares the orphan drug exclusivity regimes of the United States, the European Union, and Japan, and assesses the institutional risks embedded in China's newly enacted legislation. Results: This article proposes a novel theoretical model that reconceptualizes orphan drug market exclusivity as a conditional public-law entitlement whose legitimacy is premised on the continuing fulfillment of public health objectives. Applying this framework, the study identifies four core structural deficiencies in China's current regime and, drawing on mature comparative practices, develops targeted institutional design proposals for each regulatory dimension. Conclusions: Reconceiving orphan drug market exclusivity as a conditional public-law entitlement, rather than as an intellectual property right, offers a sustainable governance pathway for reconciling innovation incentives with the equity and financial protection goals of universal health coverage. Embedding conditional constraints into pharmaceutical exclusivity rules ensures that regulatory incentives serve long-term public health objectives and carries implications for addressing market failures and access challenges in other areas of global public health.