How a Rs 72 lakh drug for a rare genetic disease could cost just Rs 3,000

The staggering cost of life-saving drugs in India often means that patients and their families face crippling financial burdens. A 24-year-old patient of a rare genetic disorder called spinal muscular atrophy (SMA) filed a petition about how the price of the drug Risdiplam can actually cost Rs 3,000 if it is produced locally.
Risdiplam is a self-administered liquid medicine taken orally by adult SMA patients. The petition featured an elaborate cost analysis of Risdiplam by Dr Melissa Barber from Yale University.
Dr Barber, who has worked as a consultant on pharmaceutical issues for the World Health Organisation (WHO) and the Médecins Sans Frontières (MSF), has been developing and refining methods for estimating the cost of production of medicines since 2016.
At the request of the petitioner, Dr Barber analysed the cost of production of Risdiplam and, according to her calculation, this drug can be priced as low as Rs Rs 3,024 per year.
In an interview with IndiaToday.in, Dr Barber said that the low-cost estimate accounts for India’s generic drug landscape, where manufacturers typically operate on a 10% profit margin compared to the inflated prices set by patent holders.
Locally producing Risdiplam could drastically reduce its price for patients, as India’s pharmaceutical industry has shown the capacity to produce affordable alternatives to costly drugs.

At the request of the petitioner, Dr Barber analysed the cost of production of Risdiplam and, according to her calculation, this drug can be priced as low as Rs Rs 3,024 per year.

For example, in 2012, India’s compulsory licensing policy enabled Natco Pharma to make a generic version of Bayer’s cancer drug Nexavar, cutting the price by over 90%.
Dr Barber highlighted that implementing similar licensing for Risdiplam would likely yield comparable results, adding that generic production aligns with government goals under the Patents Act of 1970, which permits third-party manufacturing if it serves public health.
The active pharmaceutical ingredient (API) is the most important determinant in the cost of a production of a medicine, said Dr Barber.
“Risdiplam is a single molecule, whereas alternative treatments are gene therapies which are far more complex. Risdiplam production in small volumes is both feasible and cost-effective, and API costs for even relatively small quantities (20kg) are estimated to cost $8700/kg,” added Dr Barber.
She stated that given the low dose of this treatment (not more than 5 mg/day) a favourable annual treatment cost per-patient is likely achievable even at a cost of $40,000/kg for the API.
“Even a markup of 1000% (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),” the expert said.

Risdiplam is a single molecule, whereas alternative treatments are gene therapies which are far more complex. (Photo: Getty Images)

Risdiplam should be stored at 20°C to 25°C but can tolerate 15°C to 30°C without refrigeration.
After reconstitution with purified water, it can be kept in a refrigerator or withstand temperatures up to 40°C for five days. Its oral administration makes it easier and more cost-effective than other SMA treatments.
This could make treatment more accessible, especially in rural areas where the healthcare infrastructure is limited.
Despite this potential, Roche, the manufacturer of Risdiplam, has raised legal challenges in Indian courts, blocking domestic production.
Yet, Dr Barber pointed out that Risdiplam’s relatively simple formulation makes local manufacturing viable within months, providing regulatory support and infrastructure.
Efforts to make Risdiplam affordable through crowdfunding have also fallen short.
While the government has launched initiatives to raise funds for patients with rare diseases, these resources are far from sufficient. Although the government raised financial caps from Rs 20 lakh to Rs 50 lakh, the petitioner mentioned that this is not enough.
Till now, the total number of patients registered for Risdiplam is 2,340, and the total money collected through crowdfunding is Rs 3,49,832, which is not enough.
Dr Barber criticised the dehumanising process families must endure by publicly sharing personal stories to seek funding, often leaving them vulnerable to data misuse.

Till now, the total number of patients registered for Risdiplam is 2,340, and the total money collected through crowdfunding is Rs 3,49,832, which is not enough. (Photo: Getty Images)

“Unless Roche offers substantial price reductions, domestic manufacture will be necessary to ensure sustainable access to treatment for most patients in India,” she said.
For Dr Barber, a sustainable solution lies in government intervention. India’s laws already support compulsory licensing as a means to protect public health, allowing third parties to manufacture drugs when companies fail to provide accessible pricing.
“To my knowledge, no generic company has yet applied for a compulsory license on Risdiplam. The government should consider initiating a government use license under section 100 of the Patents Act, 1970,” said Dr Barber.
The expert emphasised that such measures could save lives, ensuring that drugs like Risdiplam reach patients who would otherwise face life-threatening consequences.
According to Dr Barber, solutions include patent buyouts, where the government pays a fixed sum to patent holders, or compulsory licensing, allowing others to produce drugs.
In India, non-working patents mean companies may not market essential drugs, limiting access for patients.
The petition mentioned that while the health research department announced that it is going to launch a programme to develop 12 indigenous drugs for rare diseases, it is not specified if Risdiplam is among them.
The National Rare Diseases Committee implemented the National Policy for Rare Diseases, 2021, aiming for drug procurement in the short term and promoting indigenisation in the mid-term.
However, there are no specific details on measures taken to support local manufacturers in providing affordable treatments for rare diseases as stated in the policy, the petition mentioned.