Ethical and Legal Challenges in Resource Allocation for Rare Disease Patients
Keywords:
Rare diseases, Resource allocation, Medical ethics, Orphan drugs, Health equityAbstract
Rare diseases, defined as conditions affecting fewer than 5 per 10,000 individuals, present unique ethical and legal challenges in healthcare resource allocation. Despite legislative advancements like the Orphan
Drug Act (ODA), systemic barriers persist, including high treatment costs, limited access, and inequitable distribution of resources. This paper examines the ethical and legal dilemmas in allocating resources to
rare disease patients, evaluates global frameworks, and proposes actionable solutions to balance equity, cost-effectiveness, and justice. A comprehensive review of literature, legislative policies, and economic
evaluations was conducted, focusing on rare disease definitions, orphan drug development, and resource allocation models. Key challenges include economic disincentives for pharmaceutical investment, ethical conflicts between individual rights and collective welfare, and fragmented international classification systems. Solutions such as multi-criteria decision analysis (MCDA), expanded orphan drug legislation, and centralized reference networks are discussed. A hybrid approach integrating ethical principles, economic pragmatism, and global collaboration is essential to ensure equitable care for rare disease patients.
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Copyright (c) 2025 Kebria Farzaneh, Saman Eskandari, Hamid Mohammadikojidi (Author)
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