The real epidemiology of most rare diseases is unknown, and it is hard to assess the prevalence and incidence of a rare disease particularly in resource constrained countries. “We are working together with IndoUSrare to advance epidemiology research on rare diseases under a Memorandum of Understanding (MoU) which calls for out of the box thinking and non-traditional approaches
We were honored to collaborate with IndoUSrare conducting a feasibility study addressing the need for patient-owned data collection programs in India and what is needed to support this type of initiative. Our report was published in April 2021. Now, we are announcing our expanded support of patient-centered data collection and international data governance for patient-owned and stewarded data supporting patients/patient advocates of Indian descent
Every country needs a national organization advocating for patients with rare disorders. We saw enormous progress in the US in the last 38 years after Orphan Drugs regulations were adopted. IndoUSrare is connecting resources and expertise across international borders by building the much-needed collaborative bridges between silos
We commend the mission and enormous undertaking of IndoUSrare in educating the Indian diaspora about rare diseases and connecting them with experts, resources, and access to global clinical trials. We need more collaboration and engagement with our international partners. No country or organization can do it all.
The next generation of basic and translational researchers need to align with the paradigm of patient focused drug development (PFDD). Practice of consanguinity and endogamy are the primary cause for large number of rare genetic disorders in India /South Asia. Population-based genetic studies would help in identifying a genetic cause and support evidence for family counseling. The genetic diversity of the Indian population may hold the key to developing therapies for many rare diseases