One Year of Treatment, Then Nothing: India’s rare disease policy and why patients are left to fight alone
India's rare disease policy sets a Rs. 50 lakh ceiling. Roche's Risdiplam costs Rs. 72 lakhs a year. A generic alternative exists at a fraction of the price, but is tied up in patent litigation. Amidst this, rare disease patients are left to fight their own battles.
Anand Grover
Published on: 7 April 2026, 04:22 pm
THERE ARE APPROXIMATELY 7,000 known rare diseases, often referred to as ‘orphan diseases’, affecting around 4 to 6 percent of the world’s population, that is nearly over 400 million people of the 8.2 billion people in the world. 75 percent of rare disease patients are children. In the United States (‘USA’), a disease is classified as ‘rare’ if it affects less than 200,000 individuals. In the European Union (‘EU’), they are defined as affecting less than 1 in 2,000 people.
Because rarity is defined proportionally, even a small percentage translates, in a country of India’s size, into hundreds of thousands of affected individuals. Merely because they represent a small percentage does not mean their concerns and health can be ignored.
Why rare disease drugs remain out of reach
Rare diseases include Hutchinson-Gilford Progeria Syndrome, a genetic disorder causing rapid aging in children; and Spinal Muscular Atrophy, caused by a mutation in the SMN1 gene that results in insufficient production of the Survival Motor Neuron protein. The deficiency progressively weakens the muscles of the spine, impairing posture and breathing, and is ultimately fatal.
An estimated 3,112 infants with SMA are born in India every year. A medical genetics clinic covering Uttar Pradesh (home to 16.5% of India's population as per the last census) and neighbouring states found an SMA carrier frequency of 1:38 among individuals with no prior family history of the disease, a finding with significant implications for policy.
Because rarity is defined proportionally, even a small percentage translates, in a country of India’s size, into hundreds of thousands of affected individuals.
It is well-known that medicines for treating rare diseases are not a priority for the pharmaceutical industry. The few treatments that do exist are largely developed by Western and Japanese Multi-National Companies (‘MNCs’). On the pretext of high costs of discovery and development costs, these companies price their drugs at levels entirely beyond the reach of ordinary patients..
Western countries and Japan cushion their citizens through national health systems that cover rare disease treatments. In developing countries, no such safety net exists which makes access to costly rare disease medicines a persistent and often insurmountable challenge.
