IndoUS2024The Indo US Bridging RARE Summit 2024 will spotlight the urgent need for cross-border collaborations to address disparities in rare disease treatment by bringing together patient advocates, clinicians, researchers, and policymakers. The summit will focus on expanding access to orphan drugs, facilitating international clinical trials, and streamlining regulatory pathways. By honoring the pioneers of the orphan drug regulatory review process and the development of affordable CAR-T cell therapies, the summit aims to inspire affordable innovation and foster global partnerships for equitable healthcare in rare diseases.

HERNDON, Va. and NEW DELHI, Nov. 5, 2024 /PRNewswire-PRWeb/ -- The World Health Organization (WHO) defines a rare disease (RD), or orphan disease, as affecting fewer than 65 per 100,000 people. Although individual RDs are uncommon, collectively, over 10,000 rare diseases impact about 400 million people globally—roughly 5% of the world's population. Almost 95% of these diseases remain without a single approved treatment. Orphan drugs for about 500 rare diseases have received U.S. FDA-approval till date, yet most of these orphan drugs remain inaccessible to almost 90% of the global population. Diagnosis and treatment for RDs often lag due to limited provider familiarity, leading to frequent delays or errors in care. Patients endure an average six-year journey to diagnosis, with marginalized groups facing even longer waits. This prolonged diagnostic odyssey requires numerous specialist visits, tests, and hospitalizations, leaving many patient families feeling isolated and unsupported.

Even after diagnosis, RD patients often face limited or unavailable treatment options, with existing therapies frequently unaffordable. The U.S. Orphan Drug Act (ODA) of 1983 accelerated drug approvals for RDs, and by 2022, the FDA had granted 882 orphan drug designations, leading to at least one approval for 392 RDs. In the last two years alone, the FDA approved over 90 new orphan drugs.

Despite incentives of ODA, drug development for rare diseases lags when compared to other therapeutic areas, with 90% of RDs lacking approved treatments and a 6% success rate in clinical trials. The 2022 Inflation Reduction Act (IRA) adds challenges by exempting single-indication orphan drugs from price negotiations but removing this exemption if a new indication is added, subjecting them to Medicare negotiations. The U.S. Biosecure Act of 2024 prohibits entities that receive federal funds from using biotechnology that is from a company associated with a foreign adversary [06]. The ORPHAN (Optimizing Research Progress Hope And New) Cures Act, currently before the U.S. Congress, aims to extend the negotiation exemption to multi-indication orphan drugs, though legislative uncertainty remains as the current Congressional calendar dwindles and the 2024 elections will bring a new Congress to power.

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 across the country, 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 a rare disease 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 for granting 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 drugs 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.

The conference will include a diverse lineup of pre-conference workshops, keynote addresses, panel discussions, poster exhibits, and interactive breakout sessions, all designed to promote collaborative approaches to the complexities of rare diseases. This year, we are also introducing a start-up pitch contest (Pitch4RARE) in collaboration with Dr. James Levine from Fondation Ipsen from France, to encourage entrepreneurship and affordable innovation in the rare diseases space. Through this contest, IndoUSrare has attracted collaboration with life sciences innovation hubs within India, such as IKP Knowledge Park, and aims to create a US-France-India triad for rare diseases.

Chimeric Antigen Receptor T-cell (CAR-T) therapy is a promising treatment for various rare cancers and autoimmune diseases. Since 2017, the U.S. FDA has approved six CAR-T therapies for adult and pediatric patients with B-cell lymphomas and leukemias. In October of last year, India's CDSCO approved the country's first CAR-T cell therapy, developed by ImmunoACT, a company incubated at the Indian Institute of Technology (IIT) Bombay, for the treatment of leukemia and refractory or relapsed lymphoma.

This innovative therapy was created by a team led by Dr. Rahul Purwar at IIT Bombay and Dr. Hasmukh Jain at the Tata Memorial Centre. Impressively, the Indian CAR-T therapy not only matches the efficacy of existing treatments but is also more affordable and comparatively well-tolerated than similar U.S. therapies.

In recognition of their groundbreaking work, the summit will be honoring Dr. Rahul Purwar and Dr. Hasmukh Jain with the Abbey Meyers Khushi Bridging RARE Awards for their contributions to CAR-T therapy for leukemia and B-cell lymphoma.

Additionally, Frank Sasinowski, JD, MPH, will be honored for his lifetime of contributions to the regulatory review process for orphan therapies, fostering patient advocacy and global cross-border collaborations, particularly between India and the U.S. in the rare disease sector. Through his support of sponsors and patient organizations, Frank has been instrumental in the implementation of ODA and securing FDA approval for hundreds of new molecular entities targeting serious rare diseases.

"Dr. Abbey Meyers is a living legend who was instrumental in the enactment of the ODA, which has had an immense impact in the U.S. and kicked off a global crusade against rare diseases. By honoring these legends who have innovated in the development of affordable therapies and implementation of regulatory frameworks, we seek their vast wisdom and guidance to carry forward our mission." Says Rajasimha.

The organizing committee invites all interested individuals to the Summit and thanks the Gold Sponsor, Soleno Therapeutics; Silver Sponsors BridgeBio and Shivanka Research; Bronze sponsors ashibio, Entrada Therapeutics, Jeeva Clinical Trials, Prince William County, VA, Lal Pathlabs, Sanofi India; along with other sponsors and exhibitors including, AstraZeneca India, JSS Medical College, Medgenome, Premas Life Sciences, Dhiti Omics, Strand Life Sciences, Takeda India, and Amicus Therapeutics.

For the complete Indo-US Bridging RARE Summit 2024 program and to get involved, visit https://summit.indousrare.org.

About the Indo-US Bridging Rare Summit 2024:

The Indo-US Bridging Rare Summit 2024 is the second annual Summit organized with the mission of "combating rare diseases through fostering cross-border collaborations, clinical trials, and data sharing." Indo-US Organization for Rare Diseases, a non-profit organization based in the Biohealth capital region, is collaborating 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, George Mason University, Virginia BIO, BioHealth Capital Region Forum, Centre for DNA Fingerprinting and Diagnostics (CDFD) India, Fondation Ipsen, Rare Disease Diversity Coalition, and other collaborators and sponsors. For the full agenda, keynote speakers, Gala, Registration, and Sponsorship opportunities, visit https://summit.indousrare.org

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 Chairman Dr. Harsha Rajasimha, who lost a child to a rare disease in 2012, has been a rare disease advocate for more than 10 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 seal member of the National Organization for Rare Disorders, and a collaborator of the Everylife Foundation for Rare Diseases, serves 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.

References:

Media Contact

Harsha K Rajasimha, Indo US Organization for Rare Diseases (IndoUSrare), 1 5402390465, This email address is being protected from spambots. You need JavaScript enabled to view it., https://summit.indousrare.org

Nisha Venugopal, Ph.D., Indo US Organization for Rare Diseases (IndoUSrare), 91 9886793939, This email address is being protected from spambots. You need JavaScript enabled to view it., https://summit.indousrare.org

SOURCE Indo US Organization for Rare Diseases (IndoUSrare)