Express Pharma

Challenges and strategies for rare diseases trials in India

Krutikesh Age, Co-founder, DPHS, sheds light on the challenges that Indian pharma companies and research organisations face while conducting clinical trials for rare diseases, and the strategies to deal with these

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Every year, about 7,000 new rare diseases are discovered, affecting over 400 million people worldwide. The number of approved studies still stands at only around 500. It is a milestone for patients with rare diseases to receive a diagnosis; however, the lack of proven treatment options often leaves their prognosis unchanged. Clinical trials for rare diseases generally present unique challenges, with no precedents or a doubtful commercial outlook. Despite these challenges, the pharma industry is making its way through research and development. Many drug companies place a high priority on these patients, but progress is slow.

India currently conducts very few clinical trials for rare diseases. Despite its top 30 ranking, India is underrepresented in clinical research due to its large population of rare disease patients. Compared to prevalent chronic diseases such as diabetes and hypertension, less than three per cent of rare disease clinical trials include investigational sites in India. Clinical trial sponsors now have the opportunity to recruit patients outside of the usual hotspots.


Longer time to recruit: Due to the difficulty in diagnosing rare diseases, clinical trials for these patients enroll at a slower rate than for others. Clinical sites enroll an average of 0.7 patients per
month for non-oncology rare diseases, which is three times slower than the average across all non-oncology diseases (approximately two patients/site/month). Due to this, rare disease trials typically enroll for longer periods of time, adding to the cost burden of R&D.

Increasing study costs: The investigational site remains the focal point of traditional clinical research. Clinical trials with rare diseases require either a greater number of clinical sites or fewer participants in order to compensate for the sparse number of eligi