Global Justice and Access to Orphan Drugs: A Bioethical Analysis

Abstract

Orphan drug policy was designed to correct a market failure -- pharmaceutical firms had little commercial incentive to develop treatments for very small patient populations -- by offering regulatory exclusivity, tax incentives, and expedited review.
Four decades on, the policy has produced hundreds of approved treatments but has not closed, and arguably has widened, the gap between drug approval and actual patient access, particularly outside a small number of wealthy jurisdictions. This analysis examines the ethical architecture of orphan drug incentives, the health-technology-assessment and pricing mechanisms that mediate access once a drug reaches the market, the documented scale of disparity between high-income and low- and middle-income countries, and
the particular affordability challenge posed by curative but extraordinarily expensive gene therapies. We argue that the accident of being born with a rare disease and the accident of being born in a country without a functioning reimbursement pathway compound in ways that a global-justice framework should treat as morally arbitrary rather than acceptable. We conclude by reviewing proposals -- pooled procurement, dedicated multilateral financing, price transparency, and equity-by-design manufacturing -- that treat global access, not domestic approval alone, as the relevant measure of policy success.