Protecting the Right to Health for Rare Disease Patients: Legal Frameworks, Policy Gaps, and the Need for Reform in India

Abstract

Right to Health is a fundamental human right recognized by various international and regional treaties, and governments worldwide have a duty to ensure its protection. However, several impediments exist for the full realisation of this right which includes economic, social and political factors. This requires coordinated efforts from governments, international organisations and civil societies.
Access to healthcare and medicines is central to this right. In the case of certain diseases, the full realisation of this is burdensome. This issue is reflected for individuals suffering from rare diseases, where orphan drugs, for their treatment are often unavailable due to the lack of incentives for pharmaceutical companies to invest in their development. Orphan diseases presents a complex dynamics due to the numerous diseases, varied and unidentifiable symptoms and difficulties in diagnosis.
Orphan drugs face significant hurdles in research, development, and commercialization, primarily due to the small market size and high costs involved. These barriers leave patients without access to life-saving treatments, thereby increasing health inequities. Several countries have introduced incentives for orphan drug development, such as market exclusivity, tax credits, research grants, fee waivers, accelerated approval etc. However, accessibility remains restricted due to high costs and insufficient governmental support.
India, with its large population suffering from rare diseases, lacks a robust regulatory framework to promote orphan drug development and ensure access for patients. Despite the introduction of the National Policy for Rare Diseases (NPRD) in 2021, there are significant gaps in incentivizing research and providing affordable treatment options. This paper examines the scope of right to health as a human right within both international and Indian contexts, analyzes the challenges associated with orphan drug development, and reviews the effectiveness of existing policies. It also explores global initiatives for development of orphan drugs, emphasizing the need for policy reform to ensure equitable healthcare for all.