Patients of rare disease welcome prospect of dramatic slash in price of drug

Parents of children with spinal muscular atrophy (SMA), a rare disease, welcomed the announcement by the Indian pharma company, Natco, that it would be providing the generic version of the SMA drug risdiplam at a fraction of the price currently being charged by the multinational pharma company Roche. From roughly Rs 72 lakh per year to treat an adult patient with SMA, the cost could fall to about Rs 5 lakh per year with generic production.

Natco revealed in a “legal update regarding Risdiplam launch in India” that the “company has decided to price the product (Risdiplam) at Rs 15,900” per 60 mg bottle. An individual with a body weight of over 20 kg needs roughly 2.5-3 bottles per month, or about 30-36 bottles per year. However, Natco being able to manufacture risdiplam depends on the outcome of a case filed by Roche against a single bench judgement in the Delhi high court on March 24. The high court had denied Roche’s plea for an injunction against Natco making the drug.

“Making this medicine available at a reduced price without compromising efficacy and safety will increase accessibility and be a game changer. It will make a significant difference for patients,” said a parent of a child with SMA.

The price in China for Roche’s risdiplam is approximately Rs 44,700 per bottle and in Pakistan about Rs 41,000 per bottle, pointed out an SMA patient Seba PA in her affidavit in the Supreme Court. She pointed out that these were 80% lower than the price in India, where Roche supplies risdiplam to the Centres of Excellence for Rare Diseases at more than Rs 2 lakh per bottle. A drug pricing expert from Yale university has calculated that with local production, the cost of making risdiplam could be as low as Rs 3,000 per year.

The central government’s rare disease policy provides one-time financial assistance of up to Rs 50 lakh for individuals registered with its rare disease portal. At the current price being charged by Roche, Rs 50 lakh would not cover the cost of risdiplam even for a year. With the reduced price that Natco announced, Rs 50 lakh could help a patient with SMA access medicines for almost ten years.

There are over 750 SMA patients registered with the government portal. Cure SMA, a Trust formed by parents of children with SMA has 1,800 patients registered. However, only two or three SMA patients have got any financial assistance from the government, according to the Cure SMA intervention application in court. In 2018-19, 2019-2020 and 2020-21, out of Rs 200 crore that had been budgeted by the Centre for expenditures related to rare diseases, only Rs 7 crore were spent.

If the government was to provide Rs 50 lakh to 1,800 patients of SMA, it would cost Rs 90,000 crore and yet not afford them even a year’s worth of medicines at the drug’s current price. To put that in perspective, the annual budget of the union health ministry for 2024 was just over Rs 90,000 crore. “The only option before the government is to facilitate local production as envisaged in the National Rare Diseases Policy. That coupled with bulk procurement by the government could bring the price down further and make the financial assistance meaningful,” said a public health activist.