In a country where healthcare costs can quickly become unbearable, the cost of life-saving drugs often brings enormous burdens on patients and their families. Imagine having a rare disease that has a treatment, yet that treatment is so costly it seems out of reach. This is the stark reality faced by many in India who suffer from spinal muscular atrophy (SMA), a genetic disorder that gradually weakens muscles, impacting movement and, in severe cases, breathing. Currently, the drug Risdiplam, used to treat SMA, is priced at an unattainably high rate. However, recent research suggests that if manufactured domestically, the cost of Risdiplam could drop drastically, giving hope to thousands

One SMA patient in India, only 24 years old, recently took a bold step to bring attention to this issue. This young person filed a petition highlighting that Risdiplam could be produced locally for as low as Rs 3,000 per year. This isn’t just a random guess; it’s backed by an in-depth cost analysis performed by Dr. Melissa Barber from Yale University. Dr. Barber, an expert in pharmaceutical pricing, concluded that if India’s robust generic industry produced Risdiplam, it could be available at a fraction of its current international price. Her findings have sparked new discussions on making the drug accessible for SMA patients across India.

Risdiplam is a relatively simple medicine in terms of formulation. It’s an oral liquid treatment that can be self-administered, which is a significant benefit for patients who rely on a consistent medication regimen. Dr. Barber’s analysis reveals that the primary cost factor lies in the active pharmaceutical ingredient (API). Risdiplam’s API, which is a single molecule, is comparatively cheaper to produce than the components of other, more complex SMA treatments like gene therapies. Producing small amounts of this molecule is feasible and affordable.

To put it in perspective, even with a substantial mark-up, a vial of Risdiplam could be available at Rs 1,000, which is still about 99% cheaper than the U.S. price of approximately Rs 10 lakh (around $11,000) per vial. Such a price reduction would make this life-changing treatment accessible to a broader population in India.

India’s pharmaceutical industry has a remarkable history of producing affordable generic drugs. One of the best examples of this is the case of Bayer’s cancer drug Nexavar. When the cost of Nexavar proved unaffordable, India used its compulsory licensing policy to allow a domestic company, Natco Pharma, to manufacture a generic version, which cut the price by over 90%. The impact was significant and helped thousands of cancer patients gain access to this vital treatment. Dr. Barber believes that if Risdiplam follows a similar path, its generic production could be equally transformative for SMA patients.

While the potential for locally manufactured Risdiplam brings hope, Roche, the drug’s manufacturer, has taken legal steps to block any such production in India. The company’s legal challenges in Indian courts aim to protect its patent and control over pricing, making it difficult for domestic companies to step in and produce an affordable version.

This has placed patients and advocates in a difficult position, as Roche’s control over the pricing means that without local production, the cost remains unattainably high. To counteract this, Dr. Barber and other experts are calling on the Indian government to consider compulsory licensing, which would allow third-party companies to manufacture Risdiplam under India’s Patents Act, 1970, if it benefits public health. Such intervention could be a lifeline for SMA patients who cannot afford the high prices set by Roche.

India’s laws already allow for compulsory licensing in cases where public health is at risk, as demonstrated in the Nexavar case. However, to date, no Indian pharmaceutical company has applied for a compulsory license for Risdiplam. This hesitation might stem from the complexity of navigating legal challenges or from the uncertainties around the government’s stance on the matter.

Dr. Barber advocates for the government to take a proactive role in this situation. One approach could be initiating a government use license under Section 100 of the Patents Act, 1970, which would enable third-party manufacturing of Risdiplam. Other potential solutions include patent buyouts, where the government purchases the patent to make the drug accessible, or negotiating directly with Roche for substantial price reductions.

In India, patients often turn to crowdfunding to raise money for costly treatments. Yet, the amounts needed are so high that even collective community efforts fall short. Since the inception of these crowdfunding campaigns, only about Rs 3,49,832 has been raised which is far less than the total amount required for comprehensive treatment coverage for SMA patients. Additionally, families are often forced to share deeply personal stories to appeal to potential donors, a process that can be emotionally exhausting and invasive.

Risdiplam’s case is a part of a larger discussion on how India addresses the treatment of rare diseases. The National Policy for Rare Diseases, launched in 2021, includes provisions for short-term drug procurement and long-term indigenization. However, the policy lacks specific details on how to support local manufacturers in producing affordable treatments for rare diseases, leaving gaps in accessible care for patients.

Although there have been government efforts to increase financial assistance for rare diseases, including raising the cap from Rs 20 lakh to Rs 50 lakh, this is still insufficient for long-term treatment requirements. The policy’s lack of specific measures for promoting domestic production of rare disease treatments remains a barrier for patients who need consistent, affordable access to life-saving drugs.

Beyond the financial implications, the current situation raises ethical questions. Patients and their families are forced to navigate a system that seems more concerned with profit than with providing life-saving treatments. The SMA community, like so many others, is left in limbo, torn between legal restrictions, limited financial support, and an inaccessible healthcare system.

Dr. Barber has been vocal about the “dehumanizing” experience that patients endure as they publicly share personal details to obtain crowdfunding. She points out that patients should not have to “plead” for help in such a manner, especially when there are viable solutions that could make treatment affordable and accessible.

There are feasible solutions to address this crisis, but they require a commitment to policy change and government intervention. For instance, compulsory licensing could be implemented to override the patent and allow local companies to produce Risdiplam. The government could also consider a patent buyout model, paying a set amount to the patent holder in exchange for the rights to produce the drug domestically.

India could also take inspiration from other countries that have successfully negotiated with pharmaceutical companies to reduce drug prices. By working collaboratively with companies and regulatory bodies, the government could make a significant impact in ensuring affordable access to life-saving treatments like Risdiplam.

Another approach involves growing local manufacturing capabilities, which could be instrumental in the long-term strategy to provide affordable treatments for rare diseases. By investing in local pharmaceutical infrastructure and fostering partnerships between public and private sectors, India can create a more sustainable path to affordable healthcare for all.

The case of Risdiplam is emblematic of a broader issue facing India’s healthcare system: the need to balance pharmaceutical innovation with accessibility and affordability. The high cost of life-saving drugs shouldn’t force patients to choose between their health and financial stability. As the world’s largest supplier of generic medicines, India has the capability and expertise to bring about change. For patients with rare diseases like SMA, the wait for affordable treatment is ongoing.