Rare and orphan diseases represent a major public health challenge in India, collectively impacting an estimated 70 to 96 million lives. Since the majority of the diseases are progressive, leading to debilitating conditions, this places an immense burden on the healthcare infrastructure, and on families, especially as there are no treatments for most of the diseases. Rare disease drug discovery represents the absolute frontier of modern drug development—encompassing some of the most modern technologies such as cell and gene therapies and RNA therapeutics. Increasing numbers of clinical trials for rare disease drugs are taking place and there is a gap in the number of trained professionals in this area.
Standard academic curricula train students in classical, large-scale (Phase I-IV) clinical trial designs optimized for high-prevalence indications (e.g., diabetes, cardiovascular diseases). Since traditional clinical trial frameworks are often mathematically, logistically, and ethically unfeasible for rare diseases, there is a need to organise tailored workshops and training programs pertaining to clinical development of rare disease drug candidates.
Rare disease drug development demands an entirely different operational blueprint. Small patient populations require adaptive statistical designs. The geographic dispersal of patients requires decentralized logistics. And severe physical disability requires digital health monitoring. This workshop is specifically designed to equip the next generation of clinical trialists, scientists, regulatory managers and others with these highly specialized, future-ready competencies.
Towards this end, we in collaboration with Medanta Institute of Education & Research (MIER) and World Without GNE Myopathy (WWGM) and the Indian Society for Clinical Research as knowledge partner is organising a workshop ‘Rare Disease Drug Development: Science, Regulation & Patients’ . The aim of the workshop is to strengthen national capacity for the design, conduct, oversight, and management of clinical trials involving rare disease drug candidates through multidisciplinary training and stakeholder engagement.
📅 12-14 November 2026
🕑 Indian National Science Academy, New Delhi
