In a fresh appeal that underscores the widening gap between medical innovation and public access, persons living with Spinal Muscular Atrophy (SMA), along with caregivers and advocacy groups, have written to Prime Minister Narendra Modi seeking urgent intervention to make a life-saving drug widely accessible through government channels.
At the heart of their demand is Risdiplam, a drug that has transformed SMA treatment globally but remains out of reach for most Indian patients due to its staggering cost.
SMA is a rare genetic neuromuscular disorder that progressively weakens muscles, often leading to severe disability and, in many cases, premature death.
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Crucially, treatment delays can result in irreversible deterioration, making timely access to medication critical.
India’s National Policy for Rare Diseases (NPRD), introduced to support such patients, offers a one-time financial assistance of ₹50 lakh. But patient groups argue that this support falls dramatically short when matched against the cost of the innovator version of Risdiplam, priced at ₹5.44 lakh per bottle.
For adult patients who may require up to three bottles a month, the entire assistance is exhausted in less than a year, even after discounts.
The urgency of the letter stems from a recent development in the availability of an Indian-manufactured generic version of Risdiplam.
Priced at around ₹15,900 per bottle and available for roughly ₹12,790, the generic drug represents a dramatic 97% reduction in cost. Patient groups say it is not only significantly more affordable but also clinically effective, with some patients already benefiting from accessing it.
Yet, paradoxically, access remains limited.
According to the representation, many government-designated Centres of Excellence under the NPRD have not begun prescribing the generic version, citing technical reasons. This has effectively denied treatment to several patients, especially adults, despite the existence of a far more affordable alternative.
The letter makes a strong economic argument that if the government were to procure the generic drug centrally, costs could drop even further by more than 40% below the listed price due to bulk purchasing.
This, patients argue, could transform the NPRD from a stopgap funding mechanism into a sustainable treatment pathway. The existing ₹50 lakh support, which currently lasts less than a year for many, could instead cover treatment for over a decade if aligned with generic pricing.
Beyond cost, the appeal also highlights logistical inequities. Limited prescribing centres mean that many patients must travel long distances to access care, an added burden for individuals already dealing with severe physical limitations.
The signatories, comprising individuals with SMA, families, and organisations such as Families of Spinal Muscular Atrophy, I'M POSSIBLE and SMAART, and MIND Trust, have made a demand that there be an immediate direction to all Centres of Excellence to prescribe generic Risdiplam to all clinically eligible patients, irrespective of age or SMA type.
Centralised government procurement and distribution of the drug across Centres of Excellence and district hospitals, ensuring free and equitable access nationwide.
The appeal raises broader questions about how India’s healthcare system responds to rare diseases, particularly in an era where generics can dramatically alter the economics of treatment.
While the NPRD marked a significant step in acknowledging rare diseases within public health policy, patient groups say its current design does not account for long-term treatment needs or rapidly evolving pharmaceutical landscapes.
The availability of low-cost generics, they argue, presents a rare opportunity to move from limited, high-cost interventions to scalable, equitable care.
“The issue is no longer just about affordability,” a caregiver involved in the representation said. “It is about whether the system can respond quickly enough when solutions become available.”
Copies of the letter have also been sent to Union Health Minister J.P. Nadda and senior officials in the Ministry of Health and Family Welfare.
For thousands of families navigating SMA, the stakes are immediate. With each passing month, delays in treatment can mean irreversible loss of function, making policy inertia not just a bureaucratic concern, but a deeply personal one.
Whether the government moves to integrate generic Risdiplam into the NPRD framework could determine if a breakthrough in affordability translates into real-world access or remains out of reach.