Roche's Risdiplam Profits Spark Pricing Debate in India

Pharmaceutical giant Roche is said to have earned profits exceeding a hundredfold the estimated price of taking its spinal muscular atrophy (SMA) medication, risdiplam, to India. Public health organization Knowledge Ecology International (KEI) puts the cost of Roche's clinical trials at an estimated $50 million, as compared to overall risdiplam sales in 2021-2024 at $5.8 billion.

Roche justified its pricing by saying that the costs and risks of unsuccessful projects also have to be taken into account. KEI pointed out that when accounting for US tax credits for trials of rare diseases, Roche's net trial expenses could be as low as $11–25 million. The firm also gained a Rare Pediatric Disease Priority Review Voucher, worth approximately $100 million, further balancing development costs.

Roche licensed rights to risdiplam via a $30 million purchase agreement with PTC Therapeutics, which had the program developed in collaboration with $100 million financing from the Spinal Muscular Atrophy Foundation (SMAF). Manufacturing is cheap with less than 2 grams of risdiplam annually required per patient costing less than $50,000 per kilogram to make, KEI indicates.

In India, treatment per adult patient costs about ?72 lakh every year. Roche mentioned that as of October 2024, 56 of the 168 Indian SMA patients got the drug for free under its Compassionate Use Program, 53 were sponsored by government health programs, and 59 accessed it through Roche's Patient Access Program, with one free bottle given per purchase.

Cure SMA has 1,800 registered SMA patients in India, whereas the government's rare disease portal lists 700.