Mapping the rare disease stakeholders in India

Rare diseases (RD) are not rare collectively, affecting around 300 million people globally and 96 million in India. These diseases have not been prioritized in most low- and middle-income countries’ health policies. India launched its first functional RD policy in 2021. Successful policy implementation requires the active participation of diverse stakeholders. In the context of rare diseases, such collaboration has been particularly instrumental in driving policy execution and systemic transformation. RDs are not well researched in India and there are no studies on mapping and analysis of RD stakeholders. Thus, this study aims to comprehensively map all stakeholders in the RD ecosystem in India, to understand their power, positions, influence, and needs. In-depth analysis of stakeholder perspective was done through semi structured interviews and news-media analysis. This is an exploratory study aimed to map all RD stakeholders and present their perspectives without drawing conclusive inferences. We found that stakeholders such as local and international patient organizations, think tanks, research communities, policymakers, local and multinational companies engage extensively with RD activities. However, high influence is limited largely to policymakers, and a few rare disease specialist physicians, with some participation of other groups. A significant lack of awareness and knowledge about RDs was found among general healthcare professionals and allied health professionals. This places a disproportionate burden on a limited pool of specialized doctors, predominantly concentrated in a few cities. Thus, for better implementation of RD policy it is crucial to encourage diverse stakeholder engagement and participation. The study highlighted stakeholders with high and low engagement. Highly engaged stakeholders should be leveraged for policy implementation, while awareness and training programs need to be targeted towards low engagement groups.