Why rare-disease drugs could become India’s next pharma frontier

Rare diseases are quite uncommon, but there are over 8,000 such diseases globally, impacting hundreds of millions. India reports 1,004 rare genetic disorders. Early on, the small patient base made drug development unattractive to pharmaceutical companies, earning them the label "orphan drugs." However, governments worldwide have taken steps to incentivize rare-disease drug development, including tax credits, market exclusivity, and grants.

This has led to a surge in approvals – from 38 in the U.S. pre-Orphan Drug Act to 1,122 by 2022. High costs, often exceeding $100,000 per year, limit access, prompting crowdfunding efforts. India offers up to Rs 50 lakh in support, but this often falls short. Most rare-disease drugs target only 5% of cases, leaving many without adequate treatment.

Developing orphan drugs involves hurdles like finding patient cohorts for trials and establishing endpoints. India's large population and high rates of endogamy create opportunities for patient advocacy and more localized rare-disease prevalence. Foreign drug companies can collaborate with local organizations for clinical trials and manufacturing.

Yet, Indian patients have not traditionally participated in international trials. The government could encourage this by partnering with foreign companies, similar to its approach in chip manufacturing. Additionally, setting drug prices within India or the Global South could encourage local access to these expensive treatments.