The FDA’s Commissioner, Dr. Robert M. Califf, recently visited India and shared his opinion on India’s Unique Opportunity and Important Responsibility as the Pharmacy to the World. The Inaugural 2-day Indo US Bridging RARE Summit was held on Oct 29 and 30, 2023 at George Mason University in Arlington, VA. The organizing committee led by Dr. Harsha Karur Rajasimha at the Indo US Organization for Rare Diseases (IndoUSrare), a US-based nonprofit committed to combating rare diseases, are aiming to catalyze this 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. The effect can be far reaching, saving or impacting millions of lives. Dr. Peter Marks, Director of Center for Biologics Evaluation Research (CBER), FDA, keynote speaker at the Summit, announced the Creation of Operation Warp Speed for Rare Diseases, a pilot program called Support for Clinical Trials Advancing Rare Disease Therapeutics (START).
(HERNDON, Va.) October 24, 2023: The launch of Operation Warp Speed in 2020 to develop COVID-19 vaccines was a resounding success, developing and making available the vaccines to the general public in record time and saving an estimated 3.2 million lives and $1.15 trillion in the U.S. alone.1 As a result of that achievement, the U.S. Food and Drug Administration (FDA) is launching a pilot program — Support for Clinical Trials Advancing Rare disease Therapeutics (START) — this year to accelerate the pace of development of therapeutics for very small populations with very high medical need. 1 Globally, rare diseases are defined by The World Health Organization (WHO) as a disorder which affects less than 6.5 to 10 people out of 10,000.2 According to a report by RARE-X, there are over 10,897 known rare diseases affecting over 400 million people worldwide. Since the Orphan Drug Act of 1983, the FDA has approved about 1100 orphan drugs to treat about 5-7% of rare diseases. Hence, a majority of rare diseases remain without treatment options. At the recently concluded Indo US Bridging RARE Summit 2023, Dr. Peter Marks, director at the FDA’s Center for Biologics Evaluation and Research (CBER), spoke about the START program, the India opportunity, and stated, “We have fifteen FDA approved gene therapies till date. We are very committed to working with the rare disease communities for gene therapies and with our colleagues globally. 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.”
Accelerating the diagnosis and treatment of rare diseases
In the United States, more than 30 million people suffer from rare diseases, many of which are life-threatening.3 But the U.S. is not alone in this fight or the worst off. 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.
”There are gene therapies, antisense oligonucleotide (ASO), and other orphan drug therapies that are ready to begin their development. Developing these therapies can be a matter of life or death for many and patients and their families are desperate.” says Frank Sasinowski, a leader recognized by the Vatican and NORD for his lifetime of achievements in patient advocacy, and the founding board director of IndoUSrare. “If we can create the same sense of urgency that we had around COVID-19 for every single rare disease, that would be huge.” Says Sasinowski.
“If we can do a COVID-19 vaccine in nine months, why not an orphan drug in nine months?” Dr. Rajasimha says, that’s the idea! It still takes five, 10, 12 years or sometimes much longer to get an orphan drug to market. If START is successful what we want to do is explore the possibility of concurrent collaborative review of applications with global regulatory partners in a manner similar to that being undertaken through Project Orus in the FDA's Oncology Center of Excellence.
To ensure the success of the FDA’s new endeavor, Rajasimha believes it is essential for all stakeholders to view each rare disease as a global public health problem and not just an American one. Since the FDA will not be able to fund it in the same way they did COVID-19 research, to increase the success of the START program it will be critical to engage biopharmaceutical Sponsors and patient advocacy groups to make it scalable and affordable. “It is critical that we engage the Sponsors of orphan drugs in this process together with patient advocacy groups.” Says Dr. Anish Bhatnagar, CEO of Soleno Therapeutics, and a member of the IndoUSrare Corporate Alliance Program.
Advancing the cause at the Bridging RARE Summit 2023
The key highlights and takeaways from the Summit include:
1. With about 360 registrations (in-person + virtual) for the inaugural Indo US Bridging RARE Summit including speakers from some of the top leaders in the industry, FDA, Indian ministry of Health, physicians, researchers, patient advocates, investors, BioHealth innovators, and data scientists, the two-day summit spanned 8 lively sessions.
2. Letters of support for the Summit’s goals poured in from US Congressman Gerald Connolly, a member of the rare disease congressional caucus, Senator Mark Warner, Co-chair of the India Senate caucus, Indian Ambassador to the US, Taranjit Singh Sandhu, and a video recorded message by Senator Cindy Hyde-Smith, also a member of the bilateral and bicameral, Rare Disease Caucus.
3. The "one-and-done" nature of Gene Therapies offers unique opportunities and challenges needing global cooperation to make them affordable. There was excitement among stakeholders to foster Cooperation between the US FDA and Indian regulators to accelerate the development of affordable gene therapies.
4. Desperate patients and caregivers are seeking rapid development of orphan drugs. They are no longer willing to accept the speed or costs of the past.
5. 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 US, 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 1100 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?
6. Non-profit Patient organizations focused on particular diseases such as Prader Willi Syndrome, Rett Syndrome, Sickle Cell Disease, and others are curious to scale their outreach and patient registries globally but need collaboration with organizations such as IndoUSrare to connect in other countries such as India.
7. AI is not to be seen as a be-all, end-all, or as a replacement for humans. It’s a means to an end. To minimize the bias of training the AI models on patient data from <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.
8. Engaging the policy makers to influence critical public health legislation has been a hallmark in the US-led rare revolution that was set off in 1983. We need a similar revolution is now unfolding in India with the national policy for rare diseases (NPRD) 2021. This was highlighted by Dr. L Swasticharan, Director General of Health Services, Ministry of Health and Family Welfare, during his keynote address from India on a live video. India has now budget allocation and has created over 11 centers of excellence across the country to combat rare diseases.
9. Vikram Karnani, EVP & President, Global Commercial Operations and Medical Affairs, Horizon Therapeutics (now Amgen) in his Keynote talk at the evening awards Gala said “If this platform can achieve awareness about rare diseases among the Indian Diaspora leading to faster diagnosis of patients, that alone would be a huge success!”
10. Recipient of the 2023 Abbey Meyers Khushi Bridging RARE Award, Dr. Ishwar Chander Verma opined that the entrepreneurship of America leveraging the scientific expertise in biochemistry in India can be a powerful combination to accelerate orphan therapy development.
11. Another recipient of the same award for 2023, Dr. William A. Gahl, for his lifetime of achievements and contributions to improve lives of those affected by undiagnosed and rare diseases, urged the next generation to be bolder, take bigger risks, and try newer approaches.
12. To celebrate the 40th anniversary of the Orphan Drug Act and to honor Dr. Abbey S Meyers and the awardees, the Summit featured a unique Indo US fusion dance production by Dr. Mani Sreenivas and Anagha Sreenivas during a black-tie Gala event.
The Indo US Bridging RARE Summit 2023 thanks the Gold Sponsors Exela Pharma Sciences and Soleno Therapeutics; Silver Sponsors BioMarin, Larimar Therapeutics, and Synergy BI Solutions. The Summit attendees wish to create a Blueprint for US-India cooperation to combat rare diseases for the next 10-40 years before next year’s Summit scheduled for Nov 16-18, 2024, in New Delhi.
About Indo US organization for rare diseases:
IndoUSrare is a humanitarian nonprofit 501(c)(3) tax-exempt public charity organization based in the United States. 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 unmet needs of diverse patients with rare diseases globally, the leadership team comprised of experienced professionals from research, advocacy, regulatory, and drug development seeks to build cross-border collaborations connecting stakeholders of rare diseases in low- and middle-income regions such as India, with their counterparts and clinical researchers in the United States to improve the diversity of clinical trial participants, accelerate research and development, and improve equitable access to life-saving therapies to diverse populations of rare disease patients. Visit https://indousrare.org.
References:
1 Mulero, Ana; “CBER to Launch Operation Warp Speed for Rare Diseases by Year’s End”; BioSpace; April 24, 2023; biospace.com/article/cber-to-launch-operation-warp-speed-for-rare-diseases-by-year-s-end/.
2 Drayeh, Simin, Alireza Kazemi, Reza Rabiei,* Azamossadat Hosseini, and Hamid Moghaddasi;
“National information system for rare diseases with an approach to data architecture: A systematic review”; Intractable Rare Diseases; August 7, 2018; ncbi.nlm.nih.gov/pmc/articles/PMC6119672/.
3 Eisenman, Daniel; “Operation Warp Speed for Rare Diseases: Expected Boom in Drug Development and Approval”; Advarra; August 10, 2023; advarra.com/blog/operation-warp-speed-for-rare-diseases/.
4 “Understanding Rare Diseases”; Invest India; October 14, 2021; investindia.gov.in/team-india-blogs/understanding-rare-diseases.
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