Rs 72 lakh/year rare-disease drug can be made for Rs 3,000/year, HC told

A drug to treat Spinal Muscular Atrophy (SMA) that costs about Rs 72 lakh per patient per year because of patent protection and absence of local production could be produced for as little as Rs 3,000 per year if India would allow local manufacture . This opinion from a drug costing expert was part of an affidavit filed by a 24-year-old SMA patient in Kerala high court.

Dr Melissa Barber of Yale University, who has worked as a consultant for WHO, World Bank and Médecins Sans Frontières (MSF) and has specialised in developing and refining methods for estimating the cost of production of medicines, conducted a cost analysis for risdiplam on the request of the petitioner. “Even a markup of 1,000% (unit price $12.83 per 60mg/80mL vial) would result in a 99% decrease relative to current prices in the United States ($11,170 per vial),” stated Dr Barber on the price of active pharmaceutical ingredient (API) required for making risdiplam.

In her submission to the court, Dr Barber pointed out that risdiplam was the preferred treatment for SMA in many health systems contexts for both cost and ease of administration. It can be administered orally by family members, while other SMA treatments require infusion or injections using lumbar punctures, she added. “Risdiplam is a small-molecule medicine, with less complex manufacture than other treatments for SMA. Production in small volumes is both feasible and cost-effective,” she stated. The costing Dr Barber submitted to the court included costs of materials (active pharmaceutical ingredient, excipients, vial), formulation and secondary packaging costs, a 20% mark-up, and tax on profit.

While the health ministry affidavit filed in Sept stated that financial assistance for patients with rare diseases has been increased from Rs 20 lakh to Rs 50 lakh, the petitioner pointed out that it would not be adequate unless indigenous production was made possible. The ministry stated that a digital platform for crowdfunding has been started to facilitate donors to contribute to the treatment costs of patients with rare diseases. The petitioner’s affidavit pointed out that the digital platform has been a non-starter with just Rs 3.5 lakh collected through it when the number of patients registered as of Oct 14, was 2,340.

Her affidavit pointed out that the ministry did not respond to the court’s specific query about how the supply of medicines for the patients could be continued, considering that financial assistance and crowdfunding were insufficient. The ministry merely stated that the department of health research was set to launch a programme developing 12 indigenous drugs for rare diseases without stating whether these 12 drugs included risdiplam.

National Rare Diseases’ Committee was constituted through an order in May 2023 for implementing National Policy for Rare Diseases, 2021. The ministry response stated that the short-term goal of the committee is to look after procurement of drugs, while the mid-term goal is indigenisation of drugs. However, there were no details regarding what steps it had taken for creating a conducive environment for indigenous manufacture of drugs for rare diseases at affordable prices as stated in the policy, the petitioner’s affidavit stated.