20 Years of Orphan Drug Policy Evolution in the Netherlands: The Role of Institutional Entrepreneurs
Summary
This academic research explores the historical evolution of orphan drug policy in the Netherlands, with a focus on the role of institutional entrepreneurs in influencing these developments. Rare diseases, which affect a significant number of individuals in the European Union, pose unique challenges that require tailored responses, due to, among others, differences in healthcare systems, patient demographics, and organizational capacity between countries. Using an institutional theory and institutional entrepreneurship framework, the study examines the impact of enabling conditions in driving institutional change. The research adopts a qualitative grounded theory approach, including literature review, document analysis of orphan drug regulations and media articles, and semi-structured expert interviews. The findings highlight the significant role of institutional entrepreneurs in catalyzing disruption and reshaping the policy landscape, leading to improved access and pricing of orphan drugs. However, challenges remain, requiring continued collaboration to address affordability and access challenges. The study highlights the importance of social status and public discourse in facilitating institutional change, and underscores the importance of innovation, international collaboration, and societal engagement in fostering a patient-centered and economically viable healthcare landscape in the Netherlands.