A social entrepreneur on a mission to bridge the clinical research divide between the U.S. & India
June 15, 2021
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Even as the world awaits a vaccine to tackle the global pandemic instigated by the novel coronavirus, there are over 22,000 known medical conditions many of which are treatable with drugs, devices, biologics, or other therapies. About 7000-9000 of these conditions are considered rare disorders because taken one at a time, they affect fewer than 200,000 Americans. About 80% of these diseases are genetic in origin, 50% of them affect children and 30% of them do not live to see their 5th birthday. Taken together, rare diseases affect an estimated 350 Million people worldwide. That’s approximately the same number of patients living with Diabetes worldwide. However, a majority of these rare diseases continue to be neglected and most countries are yet to recognize them as a public health concern. After losing my own child to one such rare congenital disease in 2012, I decided to apply my years of postdoctoral R&D experience at the National Institutes of Health and startups towards accelerating clinical research, as 95% of rare diseases and the millions of people who have them remain undiagnosed or without any treatment options. I became a social entrepreneur.
Learning about the world of rare disease patient advocacy originating in the 1980s with the U.S. Orphan Drug Act (ODA), National Organization for Rare Disorders, Global Genes, Every Life Foundation for Rare Diseases, EURORDIS, and the NIH office of rare disease research, it also pointed the obvious lack of awareness, education, policy frameworks, or investments in India and among the Indian diaspora globally. Even the generally educated population misunderstand genetic diseases, consanguineous marriages, and social stigma. Within a short period of time, I reached out to hundreds of stakeholders of rare diseases in India and brought together a team of co-founders to form the national umbrella organization for rare disorders for India. The results of this research were published in Cambridge journal Genetics Research and set the roadmap for the Organization for Rare Diseases India. While I served as co-founder and co-chair of the organization from 2013 until Nov 2019, national and international visibility and awareness was raised about the millions of people living with rare or undiagnosed diseases in India, leading to the convergence of global leaders in this space at the 7th undiagnosed diseases network international conference at New Delhi, IN.
Realizing that over 98% of global clinical research happens outside of India, with a majority Biotechnology investment in the US, I founded the Indo-US Organization for Rare Diseases to build collaborative bridges between the various stakeholders in the US and India. There are over 600 orphan drugs approved so far by the US FDA in the last 37 years whereas only 3 orphan therapies could be traced to Indian origin during the same period. As a result, Indian diaspora worldwide rely almost entirely on orphan therapies approved by FDA or EMA. Hence, informing the Indian diaspora about global clinical research as a care option will lead to significant acceleration while helping individual patients gain access to innovative new therapies decades before they are approved and become affordable. Including Biotechnology, gene therapies, and orphan drugs as a focus area in the US-India bi-lateral relationship can play a critical role in achieving acceleration of clinical research leading to affordable therapies for thousands of rare diseases that remain without any FDA approved treatments. My hope is to see that the rare disease community can collaboratively achieve routine approval of orphan drugs for simultaneous commercialization in the USA and India at price points that are affordable to all patients who need them. IndoUSrare is seeking community engagement, partnerships, and collaborations with all stakeholders to realize the vision at IndoUSrare (www.indousrare.org).
Harsha Rajasimha on the closing Keynote panel at the Global Genes RARE Summit in Sept. 2019 at San Diego, CA, with Lara Bloom of Ehlers Danlos Society, Dr. Stephen Kingsmore of Rady’s Children’s Hospital, and Razelle Kurzrock.
Despite significant advances in digital health technologies, travel and logistical burdens associated with participating in clinical research have long remained unsolved. To address this gap and drive the adoption of remote touchless patient recruitment and faster data collection, we developed a cloud-based software platform that works on any of the patient’s mobile devices. The clinical research industry has been slow to adopt mobile and digital health technologies to optimize trial operations. COVID-19 created an urgent need for conducting touchless recruitment, remote monitoring, and flexible hybrid decentralized clinical trials. Jeeva is offering free 30-day trial licenses to support clinical researchers, coordinators, academic medical centers, hospital systems, and patient groups planning their upcoming trials at Jeeva Trials (www.jeevatrials.com/solutions).
Harsha Rajasimha is a rare disease social entrepreneur globally recognized for his work in genomics data science, persistent advocacy, and technology innovations addressing grand challenges facing the estimated 350 Million people suffering from rare diseases world-wide. Harsha earned his bachelor’s degree in computer science and engineering from Bangalore University, a master’s degree in computer science and a doctorate in the interdisciplinary program genetics, bioinformatics, and computational biology from Virginia Tech. He can be reached on LinkedIn,Twitter, and Facebook.
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MS. KANAN LATHI
Kanan Lathia is an Ambassador and scientific consultant at Indousrare.She has spent 20+ years as a research scientist focusing on neurodegenerative diseases, cancer, and HIV. She has also worked as a project manager supporting teams in building brain maps of the first 1000 days of babies. Kanan’s passion for rare diseases is driven by a personal cause. Her work with Dup15q Alliance strengthened her passion for giving back to rare disease families. Kanan hopes to scale the organization’s efforts by securing funding and building a patient alliance membership program. Kanan loves yoga and spending time between the US and India.
Dr. Krishna Ramaswamy
Dr. Krishna Ramaswamy is a life science professional with experience in research, management consulting, and business development in academia and industry. She has a Ph.D. in Biological Sciences from University at Buffalo, SUNY, and pursued postdoctoral research in cell biology from the University of Southern California, Los Angeles. She has worked as a consultant for the biotech/pharma industry and worked with Dr. Harsha and ORDI in the past for about 2 years. Dr. Krishna currently resides in Hyderabad India and has a keen interest in the rare diseases space and would like to bring awareness about the various diseases, diagnoses and treatments that are available and those in the pipeline. She first began working in the rare disease space during her postdoctoral training and has worked on peroxisome biogenesis disorders under the guidance of Dr. Joe Hacia at USC.
Ms. Neeraja
Neeraja is in her junior year of high school, studying at a School in Gurgaon, India. She has completed all 8 grades in a set of vocal examinations conducted by Trinity College as the captain of the Student Council in her school, currently serving the post of House Captain. Neeraja aims to find solutions to the problems associated with the fields of healthcare, technology, and sustainable development. She is passionate about teenage welfare and has founded a youth-centric organization called ‘Discover Yourself’ through which she aims to inspire, and collaborate with young people across the world and wants to bring awareness regarding various teenage and adolescent issues
