As rare disease regulation moves from the margins to the mainstream of global health policy, India faces both a challenge and an opportunity: how to align with international best practices while designing solutions suited to its vast population and limited resources. This article — authored by experts from Power In Me Foundation, the Indo-US Organization for Rare Diseases, JSS Academy of Higher Education and Research, MTT Consulting LLC, and 4Biosolutions Consulting — was originally published in the October 2025 edition of DIA’s Global Forum magazine. Together, the authors examine how the United States, European Union, and India can close regulatory gaps to foster innovation, improve access, and deliver equity for the world’s 440 million rare disease patients.
The Urgency for Rare Disease Regulatory Harmonization
The World Health Assembly’s May 2025 resolution designated rare diseases as a global health priority, mandating the WHO to coordinate a 10-year plan for better diagnosis, registries, and access. Yet the gap between aspiration and on-the-ground reality remains wide.
In affected Indian households, rare diseases are not only medical emergencies but financial catastrophes. Treatments, when available, can cost upwards of ₹1 crore (over 110,000 USD) per year, far beyond their reach. Families rely on crowdfunding or on government-provided financial support of ₹50 lakh (USD 57,000) per patient. The system fails not because India lacks ingenuity or talent, but because of structural policy, regulatory, and reimbursement inefficiencies.
The key question arises: Can India adopt global best practices—like exclusivity incentives, tax credits, and registry-driven policymaking—while designing solutions tailored to its vast population and constrained resources?
Facts and Takeaways
Global Frameworks: US and EU
Policy innovations in the United States and European Union show what is possible.
- The US FDA Orphan Drug Act (1983) grants seven years of market exclusivity, tax credits for R&D, fee waivers, and pathways like Fast Track and Accelerated Approval.
- The EU EMA Orphan Regulation (2000) provides 10 years of exclusivity (12 years for pediatric therapies), major fee reductions, and centralized approval through the EMA. Applicants and their products benefit from advice by the Committee for Orphan Medicinal Products (COMP).
These frameworks fostered a thriving ecosystem: almost 7,500 orphan designations and 1,352 FDA approvals to date. Importantly, both regions link policy to coordinated public funding and research networks such as NIH NCATS, the Rare Diseases Clinical Research Network, and the EU’s ERDERA and European Reference Networks.
The reliance on patient registries, natural history studies, and innovative trial designs (basket, platform, Bayesian adaptive) has accelerated research. Both FDA and EMA now accept digital health technologies (DHTs), wearables, and in silico trial models as part of regulatory decision-making [FDA MIDD; EMA AI].
India’s Progress and Persistent Gaps
India’s rare disease ecosystem is nascent but growing.
- Policy base: The National Policy for Rare Diseases (2021) and the New Drugs and Clinical Trials Rules (2019) offer some reforms, including the establishment of a National Consortium for Research and Development on Therapeutics for Rare Diseases (NCRDTRD) to promote domestic orphan drug R&D; Promotion of Research and Innovation in Pharma MedTech sector (PRIP) to accelerate domestic therapeutic discovery, development, and commercialization; and Clinical Trial Waiver for orphan therapies approved by the FDA, EMA, and other specified agencies.
- Infrastructure: 13 Centers of Excellence (CoEs) were designated to deliver diagnostics, treatment, and care for rare diseases.
- Regulation: A fast-track 90-day approval mechanism for rare disease drug applications was announced in 2024.
Yet multiple challenges persist:
- Delayed diagnosis: Many patients wait 10–20 years before identification, due to limited newborn screening, minimal genetic counseling, and rural inequities.
- Funding shortfalls: Only four of 12 CoEs utilized NPRD funds in recent years.
- Few regulatory incentives: India offers neither exclusivity nor structured tax credits, leaving limited industry participation.
- Fragmented registries: NRROID and CTRI remain underdeveloped and lack global interoperability.
- Reliance on philanthropy: Corporate Social Responsibility (CSR) and crowdfunding substitute for national insurance-based support.
