On Oct 29 and 30, 2023, the Indo U.S. Bridging RARE Summit was held at George Mason University in Arlington, VA. This Summit brought together key leaders representing all stakeholders of rare diseases from across the world with a specific focus on the U.S., and the Indian subcontinent.
At this Summit, the U.S.-based nonprofit Indo US Organization for Rare Diseases (IndoUSrare) committed to combating rare diseases, and aimed to catalyze the potential cooperation and harmonization between the FDA and Indian regulators to create a pathway for biopharmaceutical sponsors to start commercially launching orphan therapies in India.
Dr. Peter Marks, Director of Center for Biologics Evaluation Research (CBER), FDA, keynote speaker at the Summit, announced a the Creation of Operation Warp Speed for Rare Diseases, a pilot program called Support for Clinical Trials Advancing Rare Disease Therapeutics (START). This program aims to accelerate the pace of development of therapeutics for very small populations with very high medical need.
Globally, rare diseases are defined by The World Health Organization as a disorder that affects less than 6.5 to 10 people out of 10,000. According to a report by RARE-X, a research program of Global Genes, that provides a collaborative platform for global data sharing and analysis to accelerate treatments for rare disease, there are over 10,897 known rare diseases affecting over 400 million people worldwide. The FDA has approved about 1,100 orphan drugs to treat about 5-7% of rare diseases. Hence, a majority of rare diseases remain without treatment options.
In the United States, more than 30 million people suffer from rare diseases, many of which are life-threatening. India, being a home to the largest population in the World, faces a particularly high burden with an estimated 70-96 million people there living with a rare disease.
Dr. Marks said,”India is an incredible opportunity in terms of capacity and capability. By leveraging them and working together, we can achieve some wonderful things for people with rare diseases.”
A few Summit’s key takeaways:
- Sponsors want to have clear guidance on how clinical development can go hand-in-glove with commercial launch. To go beyond the usual launch sequence of U.S., EU, Japan, small Sponsors are disadvantaged in scale and complexity compared to big pharma. Smaller biotech companies are innovating but lack the resources and scale to make long-term commitment to commercialize an orphan therapy in a new country such as India. Sponsors are viewing India as grounds for compassionate use of orphan therapies, and not as a market. Only a small number of about 1,100 FDA approved orphan products are accessible in India. What does it take to include India in the execution of accelerated decentralized clinical trials paving the way for commercial launch of orphan products in India?
- To minimize the bias of training the AI models on patient data from less than 5% of the global population, we need to urgently setup patient registries, natural history studies, and digitize the data for rare diseases in highly populous countries like India.
- Dr. L Swasticharan, Director General of Health Services, Ministry of Health and Family Welfare, during his keynote address from India on a live video. said that India has now budget allocation and has created over 11 centers of excellence across the country to combat rare diseases. Additionally, India’s policy makers have put in place a National Policy for Rare Diseases 2021.
Last updated: December 26th, 2025
