The importance of the policy framework on orphan drug accessibility

Purpose As orphan drugs are agents for treating rare diseases, the pharmaceutical industry usually has little interest in developing them without government incentives due to the lower sales potential. In addition to a cross-national overview of existing legal instruments, we identify and evaluate key regulatory areas based on the systematically available literature that significantly affect accessibility. We also highlight further research needs and policy implications. Subject and methods To achieve this objective, we conducted a systematic literature review. Results In addition to external factors such as medical technological advances, market size, strategic decisions of pharmaceutical companies in bringing products to market, and a country’s wealth, the regulatory system has a strong impact on the accessibility. In this context, we identified and evaluated six other key areas of government regulation in addition to the size and income strength of a country: the pricing and reimbursement policy, the health insurance regime, the supranational orphan drug policies, the duration of market exclusivity and subsidies, the approval process, and the type and degree of collaboration. This is the first study to provide a systematic overview of the research to date on the importance of policy frameworks for accessibility of orphan drugs, while identifying seven areas where this research is taking place. Conclusion To efficiently treat more people with different rare diseases with orphan drugs, an appropriate regulatory system with appropriate incentives at both the national and supranational level, complemented by collaboration, is needed. Our analysis indicates that in addition to a country’s size and income strength, the design of the regulatory framework in the areas of pricing and reimbursement policies, health insurance system, supranational policies for orphan drugs, the duration of market exclusivity and subsidies, the approval process, and the nature and extent of collaboration determine accessibility to orphan drugs. Our research also shows that previous literature has not systematically and holistically presented country-specific incentive models and their impact on accessibility in terms of a comprehensive consideration of national and supranational frameworks. As a result, there is a need for research in this area to draw better overall conclusions about accessibility. The implications gained can serve as a basis for economic policy recommendations.