India is home to a diverse population, with over 4,600 population groups, thousands of which are endogamous, potentially contributing to a high prevalence of rare genetic diseases. An estimated 96 million people in India live with a rare disease, often facing limited or nonexistent access to treatment.
In response to this growing need for therapies, India introduced its first National Policy on Rare Diseases in 2021. The policy aims to lower the high cost of treatment for rare diseases, boost indigenous research, and establish a national hospital-based registry to collect sufficient epidemiological data for defining and conducting requisite R&D. It also emphasizes the creation of Nidan Kendras for early screening and prevention, and proposes strengthening existing secondary and tertiary health facilities at designated Centres of Excellence.
In recent years, the Central Drugs Standard Control Organization (CDSCO) has made provisions to fast-track applications for rare disease drugs under the New Drugs and Clinical Trials Rules, 2019. These provisions include expediting drug trials and experimental therapies and exempting application fees for potential drug candidates.
And now, the government is reportedly considering a proposal to eliminate local clinical trials for drugs used to treat rare diseases, aiming to speed up their availability. The Drug Controller General of India is expected to discuss this proposal with state regulators soon, with the availability of rare disease drugs being a key agenda item.
While the fast-tracking of processes offers hope, plans to do away with local trials also raise concerns, particularly among families of children with rare diseases. Jins Antony from Ayur, Kerala—whose 10-year-old son suffers from Duchenne muscular dystrophy (DMD)—is one such parent.
DMD is a genetic disorder that is characterised by progressive muscle degeneration and weakness.
The condition affects various muscles, including the diaphragm—the muscular partition separating the thorax from the abdomen and aiding in breathing. In India, DMD patients rarely survive beyond the age of 20, and Antony is acutely aware that time is running out to save his son. Coming from a middle-class background, he believes local clinical trials are one of the few lifelines available to most Indian parents striving to save their children. Antony told THE WEEK that many parents depend on local clinical trials due to the exorbitant cost of these drugs if they finally come out to the open market.
“Enrolling in a clinical trial is already challenging,” Antony said. “If the government plans to eliminate local clinical trials for rare disease drugs, it should also take steps to ensure these drugs are available at affordable prices.”