The second annual Indo US Bridging RARE Summit received an overwhelming response from all stakeholders of rare diseases and orphan drugs development from Nov 16-18, 2024 at the Indian National Science Academy Campus, New Delhi, India. The Summit's robust agenda featured four workshops, eleven plenary sessions, a hackathon, pitch4rare contest, posters session, virtual networking platform, and four keynote addresses highlighting challenges, innovation, and solutions in addressing unmet needs for persons affected by rare diseases.
NEW DELHI and HERNDON, Va., Dec. 4, 2024 /PRNewswire-PRWeb/ -- The Indo US Bridging RARE Summit 2024, held from November 16-18 at the Indian National Science Academy, New Delhi, India, successfully concluded its mission to advance global partnerships in rare disease advocacy, research, and innovation. The organizing committee was led by Dr. Harsha Karur Rajasimha, Executive Chair, Indo US Organization for Rare Diseases (IndoUSrare), a US-based nonprofit committed to combating rare diseases, and Dr. Madhulika Kabra, Professor and Head of Department, Pediatrics, AIIMS New Delhi. The three-day Summit brought together over 400 delegates in-person and online, including patient advocates, researchers, policymakers, and industry leaders, for a transformative three-day event.
Key Highlights
- Patient Stories: Individuals affected by specific rare diseases highlighted their lived experiences with Prader Willi Syndrome (PWS), Fibrodysplasia Ossificans Progressiva (FOP), Ichthyosis, Myositis, Duchenne Muscular Dystrophy, Spinal Muscular Atrophy (SMA), CDKL5 deficiency disorder, Sickle Cell Disease, GNE Myopathy, Rett Syndrome, Inflammatory Bowel Disease, MNGIE disease, and many more.
- Keynote Addresses: "It was a pleasure to attend and present on global initiatives on behalf of the International Rare Disease Research Consortium (IRDiRC). Much can be learned from what is being done in India on research and advocacy for people living with rare diseases." says Dr. David Pearce, Chair, IRDiRC. The regulatory keynote address was delivered by Peter Marks, MD, PhD, Director, CBER, U.S. FDA, highlighting various cross-border initiatives of the FDA particularly focused on affordable cell and gene therapies. "India is making positive strides to advance and address therapies for rare diseases and meet the needs of the rare disease community. The time is ripe for India and the U.S. to collaborate in this space and expedite the process of bringing safe and effective solutions that would benefit those that are in need," says Dr. Mathew Thomas, Former Country Director for the U.S. FDA India office and member of the Board of Directors, IndoUSrare. Biopharmaceutical executive and rare parent, Amrit Ray, MD, delivered an inspiring keynote address that emphasized early diagnosis as the ticket to enter through the door of healthcare systems and receive care and set ambitious yet achievable goals for IndoUSrare.
- Epidemiology and Digitization: Experts from AIIMS and the Centers for Disease Control and Prevention (CDC) emphasized the need for comprehensive national patient registries to understand the prevalence, incidence, and foster epidemiological research to shape policy frameworks.
- Screening and Diagnosis: Discussions explored strategies for scaling newborn screening programs and ensuring quality clinical genomic diagnostics. Policymakers from India and the U.S. shared insights on cross-border data integration for rare disease diagnosis.
- Novel Therapies and Care Pathways: Sessions delved into emerging therapies like CAR-T treatments, drug repurposing, and gene therapy, with panelists presenting success stories and affordable innovations in India.
- Access to Orphan Drugs: A focus on diversity, equity, and inclusion showcased pathways to address global disparities in orphan drug availability, particularly in low- and middle-income countries.
- Policy and Regulation: Regulatory experts discussed frameworks including India's New Drugs and Clinical Trials Rules, and initiatives such as the U.S. FDA's START program for cancer trials, exploring their applicability to orphan drugs.
- Novel models for public-private partnerships between Industry, Academia, Government, and Patient Groups were discussed.
Innovative Initiatives and Recognitions
- Abbey Meyers Khushi Bridging RARE Awards 2024 honored two initiatives, one from the U.S. and another from India. (1) Frank Sasinowski, JD, MPH was honored for his lifetime contributions towards the implementation of the Orphan Drug Act at the U.S. FDA in the 1980s and his decades of contributions towards the regulatory approvals for hundreds of orphan therapies. (2) Dr. Rahul Purwar, Professor at IIT Bombay and Founder of ImmunoACT, and Dr. Hasmukh Jain, hemato - oncologist and Principal Investigator from the Tata Memorial Centre were jointly honored for their pioneering affordable CAR-T therapy.
- At the Pitch4RARE Startup Contest, seven startups addressing unmet needs in diagnostics, therapeutics, digital health, and accessibility sectors pitched solutions to a panel of international judges, fostering innovation in the rare disease space. The winners, CrisprBits and Kaiteki Innovations were announced at the closing ceremony. The contest was supported and advised by James A. Levine, MD, PhD, Professor at Mayo Clinic and President of Fondation Ipsen, France. Dr. Levine also generously donated a copy of his book for newbie biotech entrepreneurs: Entrepreneurs Have the DACMAR Advantage: 6-Steps for Successful Biotech Startups, to all registered in-person attendees of the Summit.
Advancing India's Role in Rare Diseases
The Summit highlighted India's advancements in rare disease policies, including the establishment of Centres of Excellence (CoEs), many of whom were represented on a panel discussion of government funding initiatives, progressive clinical trial frameworks that aim to integrate India into global research networks, emerging policies facilitating international clinical trials and local drug development, and digital health innovations to connect rural populations and improve patient outcomes.
The Summit Chief Guest, Hon'ble Minister of State for Rural Development and Communications, Dr. Chandra Sekhar Pemmasani in his keynote address lauded the Indo-US Bridging RARE Summit 2024, celebrating India's National Policy for Rare Diseases 2021 and the health sector transformation with a mission to tackle treatable rare diseases. Pemmasani lauded "the most brilliant minds of two great nations, representing a billion dreams and countless possibilities. Rare Diseases know no borders and neither should our efforts to combat them." Dr. V K Paul, Member of NITI AYOG, presided as the Guest of Honor, and stated, "Our rare children are truly special to us. In my experience as a pediatrician, it has always been a profound, sincere, and heartfelt guilt that we need to do so much more for these families, children, and adults, and to challenge the ever-evolving modern science to find solutions. The recent unveiling of four formulations (Nitisinone, Eliglustat, Trientine, and Cannabidiol) and CAR T cell therapy shows that India has the scientific and industrial capabilities to develop treatments for these disorders."
"This Summit represents a critical step toward addressing global inequities in rare disease research, clinical trials, and access to therapies. The power of patient advocacy, creation of high-quality patient registries, decentralized clinical trials, and regulatory innovation are critical pillars for engaging India and similar countries in the orphan drug revolution," says Rajasimha, who is also the Founder and CEO of Jeeva Clinical Trials.
Looking Ahead: A Vision for the Future
The Summit concluded with all participants signing up for working groups to collaborate throughout the year to identify actionable opportunities for advancing U.S.-India strategic collaborations in research, advocacy, clinical development, and market access. Plans for the 2025 Indo-US Bridging RARE Summit are already underway to expand on this year's resounding success. "The Indo-US Bridging RARE Summit demonstrates the power of international collaborations in combating rare diseases. As we plan the agenda for Nov 10, 11, 2025 in the Washington, DC area, we invite mission-driven organizations and decision makers to join us" says Rajasimha.
The Summit was made possible through the generous support from the Gold sponsor, Soleno Therapeutics; Silver sponsors BridgeBio and Shivanka Research; Bronze sponsors āshibio, Entrada Therapeutics, Jeeva Clinical Trials, Lal Pathlabs, Prince William County, VA, Sanofi India; along with other sponsors and exhibitors including, Fondation Ipsen, Amicus Therapeutics, AstraZeneca India, Dhiti Omics, JSS Academy of Higher Education and Research, Medgenome, Premas Life Sciences, Strand Life Sciences, and Takeda India.
For the complete Indo US Bridging RARE Summit 2024 program and to get involved in the #BridgingRARE Summit 2025, visit https://summit.indousrare.org.
About the Indo-US Bridging RARE Summit:
The Indo-US Bridging RARE Summit 2024 was the second annual Summit organized with the mission of "combating rare diseases through fostering cross-border collaborations, clinical trials, and data sharing." The 2024 Planning Committee led by Dr. Rajasimha and Dr. Kabra included Anish Bhatnagar, MD (IndoUSrare and Soleno Therapeutics), Neerja Gupta, MD, DM (AIIMS, New Delhi), Reena Kartha, MS, PhD (IndoUSrare and University of Minnesota), Dayaprasad G. Kulkarni, MBBS (Arogya Seva), Ashwin Dalal, MD, DM (CDFD), Juhi Naithani, MBA (IndoUSrare and bGlobal Consulting), Ratna Dua Puri, MD, DM (SIAMG and Sir Ganga Ram Hospital), Mathew T. Thomas, MBBS (IndoUSrare and Former USFDA India Office Country Director), and K. Thangaraj, PhD (CSIR–CCMB). Indo US Organization for Rare Diseases, a non-profit organization based in the Biohealth capital region, collaborated with the All India Institute of Medical Sciences (AIIMS), New Delhi, Doctors for Seva AarogyaSeva Foundation, the Society of the Indian Academy of Medical Genetics (SIAMG), Sir Ganga Ram Hospital, IKP Knowledge Park, George Mason University, Prince William County Virginia, BioHealth Capital Region Forum, Virginia BIO, Centre for DNA Fingerprinting and Diagnostics (CDFD) India, Fondation Ipsen, Rare Disease Diversity Coalition, Beyond The Diagnosis, Raregivers Global, Tamahar Trust, DakshamA Health, and other collaborators and sponsors. The Summit will alternate in Washington DC in Nov 2025. For speaking and sponsorship opportunities visit https://summit.indousrare.org.
About All India Institute for Medical Sciences (AIIMS), New Delhi:
All India Institute of Medical Sciences (AIIMS), New Delhi, is a public medical research university and hospital in New Delhi, India. The institute is governed by the AIIMS Act, 1956, and operates autonomously under the Ministry of Health and Family Welfare. For more information, visit: https://www.aiims.edu
About Indo US Organization for Rare Diseases (IndoUSrare):
IndoUSrare is a humanitarian nonprofit 501(c)(3) tax-exempt public charity organization based in the United States with the mission of "Bridging Nations, Empowering Rare Lives". Founder and Executive Chair Dr. Harsha Rajasimha, who lost a child to a rare disease in 2012, has been a rare disease advocate for more than 12 years. To address the massive unmet needs of persons with rare diseases globally, the leadership team of experienced professionals from research, advocacy, regulatory, and drug development is fostering cross-border collaborations among stakeholders in low- and middle-income countries such as India, with their counterparts and clinical researchers in the United States and the European Union (EU). By doing so, IndoUSrare, a member of Global Genes, a Platinum Member of the National Organization for Rare Disorders, and a collaborator of the EveryLife Foundation for Rare Diseases serve as a key catalyst in accelerating the development of affordable diagnostics, therapeutics, and equitable access to life-saving therapies to all patients and families affected by rare diseases. To learn more, visit https://indousrare.org.
Media Contact
Harsha Karur RAJASIMHA, Indo US Organization for Rare Diseases (IndoUSrare), 1 5402390465, [email protected], https://indousrare.org
Nisha Venugopal, IndoUSrare, 91 9886793939, [email protected], https://summit.indousrare.org
SOURCE Indo US Organization for Rare Diseases (IndoUSrare)
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