Indian Rare Disease Stakeholder Mapping

Abstract

Rare diseases aren't rare collectively, affecting around 300 million people globally and 96 million in India. In low- and middle-income countries like India, policies addressing these diseases have only recently been enacted. In 2021, India launched its first functional rare disease policy. This study comprehensively maps all stakeholders in the rare disease ecosystem in India to understand their power positions, influence, and needs, thereby enabling better implementation strategies for the rare disease policy. We conducted in-depth interviews with various stakeholders to understand their perspectives and supplemented the study with media analysis to reach those who did not respond to interview invitations. Our findings suggest a lack of awareness and knowledge about rare diseases among healthcare professionals who do not specialize in RDs. Encouraging and formalizing the involvement of rare disease patient organizations in policy-making is crucial due to their high knowledge, interest, and constructive critical capabilities despite their low power. Another important stakeholder group, local companies, can drive innovation and make treatments accessible for rare diseases but have much lower power than multinational companies, potentially leading to policies that do not favor local needs.