Published Online:April 2025
Product Name:The IUP Journal of Law Review
Product Type:Article
Product Code:IJLR020425
DOI:10.71329/IUPLR/2025.15.2.14-26
Author Name:Ritwika Mazumdar and Bhawna Tyagi
Availability:YES
Subject/Domain:Law
Download Format:PDF
Pages:14-26
India, home to an estimated 70 million individuals affected by rare diseases, faces significant challenges in addressing the needs of this vulnerable population. Despite the critical importance of orphan drugs, the lack of awareness, a comprehensive regulatory framework, and sustainable support systems continue to hinder progress. This paper explores the current state of orphan drug development in India, highlighting key obstacles such as limited awareness among medical professionals, absence of a national rare disease registry, and inadequate incentives for research and development (R&D). The paper advocates for a multifaceted approach to strengthen orphan drug policies in India. Recommendations include establishing a clear legal definition of orphan drugs, offering targeted financial and regulatory incentives for R&D, enhancing procurement and distribution mechanisms, and fostering public awareness through educational initiatives. Additionally, strategies for intellectual property (IP) protection, including robust patent filing and data exclusivity provisions, are discussed as critical enablers for fostering innovation. The role of partnerships between government, academic institutions, and non-governmental organizations (NGOs) is emphasized in driving sustainable progress. By addressing these gaps and implementing a cohesive strategy, India can create a supportive ecosystem for orphan drug development, improve access to life-saving treatments, and position itself as a global leader in rare disease innovation.
India is the manufacturing hub for global pharma, which boasts the ability to manufacture generic drugs on a mass scale at relatively low prices. However, orphan drugs for diseases such as Duchenne Muscular Dystrophy, Gaucher’s Disease, and Huntington’s Disease cannot said to be a successful story for India.