IndoUSrare: Catalyzing Crucial Cross-Border Collaborations and Innovation to Address Unmet Needs in Rare Diseases

For the rare disease community in India, accessing available treatments is particularly challenging due to regulatory and economic hurdles. In 2021, the Government of India established Centres of Excellence (CoEs) under the National Policy for Rare Diseases to treat 63 identified conditions.

There are now 12 such CoEs nationwide, with more under consideration. The Union Ministry of Health and Family Welfare (MoHFW) has allocated funds to these CoEs to cover up to 50 lacs INR per patient with rare diseases towards one-time treatment. A recent ruling by an Indian court has directed the Indian government to establish the National Fund for Rare Diseases (NFRD), recommending an allocation of INR 9.74 billion ($115.8 million) for the financial years 2024-25 and 2025-26, pending approval from the MoHFW. The court also instructed the Drug Controller General of India (DCGI) and the Central Drugs Standard Control Organisation (CDSCO) to monitor local and global clinical trials to ensure more patients can be enrolled.

Dr. Harsha Rajasimha, founder of the Indo-US Organization for Rare Diseases (IndoUSrare), welcomed recent advancements that signal positive momentum in the prevention, screening, and diagnosis of rare diseases, as well as in the research, development, and commercialization of orphan drugs. With over 8000 global clinical trials for rare diseases underway, hardly 80 (<0.1%) of them have a site in India. A recent circular from the DCGI has shown a progressive approach by invoking Rule 101 of the New Drugs and Clinical Trials Rules, 2019. This may enable waiver of local clinical trials for rare disease drugs approved by countries like the U.S., U.K., EU, Japan, Canada, and Australia and grant permission to conduct clinical trials for rare diseases.

“Just as the Indian government recently collaborated with the US FDA to create Asha, a pathway for cancer clinical trials to include India, there’s an opportunity to extend that program to include these orphan drug trials,” Rajasimha explained. Such efforts could help millions of Indians with early access to novel therapies during clinical development. As Rajasimha noted, “This is the best hope for patients with no better options.”

Inclusion of the geographic and cultural diversity of the Indian population will require innovative strategies and solutions to engage rural populations. Inclusive digital health platforms that can support local Indian languages, telemedicine, SMS, emails, and other channels of digital patient engagement and decentralized clinical trials can be instrumental in achieving this goal. Getting rare disease-specific national patient registries that can be inclusive of rural populations can be a game changer for India.

In line with these goals, the Indo-US Bridging RARE Summit 2024 is forging crucial international partnerships in advancing clinical research and access to critical therapies. The three-day summit to be held at the prestigious Indian National Science Academy (INSA) campus in New Delhi brings together over 200 members of the rare disease ecosystem for immersive workshops, keynote talks, panel discussions, and the formation of working groups. This year’s theme is “Combating Rare Disease by Fostering Cross-Border Collaborations, Clinical Trials, and Data Sharing.”

The Summit organized by IndoUSrare in collaboration with the All India Institute of Medical Sciences (AIIMS), New Delhi offers an exciting opportunity to engage with the global rare disease community in advancing clinical research and data sharing. The event will be held in a hybrid format including an excellent online networking platform.