Patients and their families often face severe financial burden due to the high cost of life-saving medicines in India. A 24-year-old patient of a rare genetic disorder called Spinal Muscular Atrophy (SMA) filed a petition asking how the drug Risdiplam can actually cost Rs 3,000 if it is produced locally.

Risdiplam is a self-administered liquid medication taken orally by adult SMA patients. The petition features a detailed cost analysis of risdiplam by Dr. Melissa Barber of Yale University.

Dr. Barber, who has worked as a consultant on pharmaceutical issues for the World Health Organization (WHO) and Médecins Sans Frontières (MSF), has been developing and refining methods for estimating the costs of producing medicines since 2016.

At the request of the petitioner, Dr. Barber analyzed the cost of production of Risdiplam and as per his calculations, the price of this drug could be less than Rs 3,024 per year.

In an interview with IndiaToday.in, Dr Barber said the lower costs are projected for India’s generic drug landscape, where manufacturers typically operate at 10% profit margins compared to inflated prices set by patent holders .

Producing risdiplam locally could significantly reduce its cost to patients, as India’s pharmaceutical industry has shown the ability to produce affordable alternatives to expensive drugs.

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 more than 90%.

Dr. Barber highlighted that implementing similar licensing for risdiplam is likely to yield comparable results, adding that generic production aligns with government goals under the Patent Act of 1970, which are to serve the public health. Allows third party creation.

The active pharmaceutical ingredient (API) is the most important determinant of the cost of producing a drug, Dr. Barber said.

“Risdiplam is a single molecule, whereas alternative treatments are gene therapies that are far more complex. Producing risdiplam in small quantities is both feasible and cost effective, and even for relatively small quantities (20 kg) the API cost is $8700/kg. is estimated to be,” Dr. Barber said.

They said that given the low dosage of this treatment (no more than 5 mg/day), there is potential to achieve a favorable annual treatment cost per patient even at a cost of $40,000/kg for the API.

The expert said, “Even a markup of 1000% (unit price $12.83 per 60mg/80mL vial) would result in a price reduction of 99% relative to current prices in the United States ($11,170 per vial).”

Risdiplam should be stored at 20°C to 25°C, but it can tolerate 15°C to 30°C without refrigeration.

After reconstituting with pure water, it can be stored in a refrigerator or withstand temperatures up to 40 °C for up to 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 health care infrastructure is limited.

Despite this potential, Roche, the manufacturer of risdiplam, has mounted legal challenges in Indian courts, blocking domestic production.

Nevertheless, Dr. Barber pointed out that risdiplam’s relatively simple formulation makes local manufacturing feasible within months, providing regulatory support and infrastructure.

Efforts to make Risdiplum affordable through crowdfunding have also failed.

Although the government has launched initiatives to raise funds for patients with rare diseases, these resources are not enough. Although the government increased the financial limit from Rs 20 lakh to Rs 50 lakh, the petitioner said it was not enough.

Till now, the total number of patients registered for Risdiplam is 2,340, and the total amount collected through crowdfunding is Rs 3,49,832, which is not enough.

Dr. Barber criticized the dehumanizing process that families have to endure by publicly sharing personal stories to receive funding, which often leaves them vulnerable to data misuse.

“Unless Roche offers substantial price reductions, domestic manufacturing will be essential to ensure sustainable access to the treatment for most patients in India,” he said.

For Dr. Barber, a permanent solution lies in government intervention. India’s laws already support compulsory licensing as a means of protecting public health, allowing third parties to manufacture drugs when companies fail to provide accessible pricing.

Dr. Barber said, “To the best of my knowledge, no generic company has yet applied for a compulsory license on Risdiplam. The government should consider introducing a government use license under Section 100 of the Patent Act, 1970 ”

The expert stressed that such measures could save lives, ensuring that medicines like risdiplam reach patients who would otherwise face life-threatening outcomes.

According to Dr. Barber, solutions include buying out patents, where the government pays a certain amount of money to patent holders, or compulsory licensing, allowing others to produce drugs.

In India, non-functioning patents mean companies cannot market essential medicines, limiting access to patients.

The petition noted that while the Department of Health Research has announced that it is going to launch a program to develop 12 indigenous medicines for rare diseases, it has not specified whether Risdiplam is among them or not. .

The National Committee on Rare Diseases implemented the National Policy for Rare Diseases, 2021, which aims to promote drug procurement in the short term and indigenization in the medium term.

However, as stated in the policy, there is no specific detail on the measures taken to support local manufacturers in providing affordable treatments for rare diseases, as noted in the petition.