Year: 2025
Type: editorial
Abstract: Background/Objectives: Orphan drugs (ODs) provide transformative treatments for rare diseases but raise ethical and policy challenges due to high costs and unequal access. This review explores these dilemmas (balancing access, affordability, and innovation across income settings), and proposes practical, actionable strategies to translate ethical principles into implementable solutions.Methods This narrative review and policy analysis explores global OD frameworks, emphasizing regulatory definitions, incentives, health technology assessment (HTA) practices, pricing, reimbursement, and delivery-system needs. It integrates policy tools, payer models, and real-world cases, alongside an ethical evaluation grounded in justice, beneficence, and equity, with a focus on LMICs. The analysis is based on peer-reviewed English-language studies from Scopus, PubMed, and Web of Science.Results We identify persistent inequities despite innovation: <5% of ∼7,000 rare diseases have approved therapies; high prices limit uptake, especially in LMICs. Structural barriers span affordability, diagnostics capacity, and fragmented governance. The review outlines an actionable package: (i) context-sensitive value-based tiered pricing blended with outcomes-based agreements; (ii) regional pooled procurement to aggregate demand and bargaining power; (iii) transparency via HTA benchmarks to guide negotiations; (iv) inclusivity enablers: registries, ORPHA/ICD coding, decentralized/hybrid trials; and (v) delivery models (in-country build-out, regional hubs, or cross-border referral) tied to readiness criteria.Limitations As a narrative synthesis drawing from secondary sources across varied country contexts, the findings lack quantitative generalizability. Emerging proposals like solidarity funds remain supported by limited prospective data, and their feasibility depends on local legal, fiscal, and health-system capacities.Conclusion An ethical, sustainable OD ecosystem is achievable by pairing innovation incentives with equity-first operations. Priority next steps are to (i) expand outcomes-linked access and pooled-procurement pilots for high-cost therapies, (ii) accelerate rare diseases data visibility through ORPHA-code adoption, and (iii) embed patient co-leadership across research and policy so that geography and income no longer determine access to life-saving treatments.
Source: Journal of Medical Economics
Authors: Esther Ugo Alum, Joseph Enyia Ekpang II, Patience Owere Ekpang, Christine Ainebyoona, Kingsley Ekene Nwagu, Olisa Alfred Nwuruku, Kibuuka Muhammad
Language: English
Subfield: Economics and Econometrics
Domain: Social Sciences
Open Access status: diamond
OpenAlex ID: W4415381665