Rare diseases affect approximately 70 to 96 million individuals in India, representing 6 to 8% of the population. The development and delivery of orphan drugs for these conditions present unique pharmaceutical challenges including limited patient populations, high research and development costs, complex formulation requirements, and inadequate regulatory frameworks. This review examines the current landscape of orphan drug development in India through a pharmaceutical sciences perspective, analyzing formulation challenges, drug delivery innovations, regulatory pathways, and policy frameworks. We evaluate advanced drug delivery systems including nanoparticle-based formulations, liposomal carriers, and targeted delivery mechanisms specifically designed for rare disease therapeutics. The National Policy for Rare Diseases (2021) and its implications for pharmaceutical manufacturing, pricing regulations, and research incentives are critically assessed. Key findings reveal that while India manufactures active pharmaceutical ingredients for approximately 450 orphan drugs, domestic availability remains severely limited due to formulation complexities, regulatory gaps, and economic constraints. Novel drug delivery technologies including lipid nanoparticles, polymeric carriers, and gene therapy vectors show promise in addressing bioavailability and targeting challenges specific to rare disease treatments. The review identifies critical pharmaceutical interventions including establishment of specialized orphan drug formulation facilities, implementation of fast-track regulatory approval processes, and development of cost-effective manufacturing protocols. This comprehensive analysis provides evidence-based recommendations for pharmaceutical scientists, regulatory authorities, and policymakers to enhance orphan drug accessibility while maintaining quality standards.
Keywords: Orphan drugs, rare diseases, Drug delivery systems, pharmaceutical formulation, Nanoparticles, Regulatory framework, National Policy for Rare Diseases, India, Gene therapy, Lipid nanoparticles
How to cite this article: Swathi G, Sundaram A, Jeyaprabha B, Orphan Drug Development and Delivery Systems for Rare Diseases in India: Pharmaceutical Challenges, Regulatory Frameworks, And Innovative Solutions. Int J Drug Deliv Technol. 2026;16(4s): 963-970; DOI: 10.25258/ijddt.16.4s.113
Source of support: Nil
Conflict of interest: None