Funding Rare Disease Care, Why the Government Must Ensure Patients Aren’t Financially Stressed
Spinal Muscular Atrophy is an inherited disease that affects nerves and muscles, causing them to become increasingly weak. It mostly affects infants and children, but can also develop in adults. There are four types of SMA, the first two being life-threatening, and types 3 and 4 impact the quality of life severely. Type 1 typically takes away the life of a child within two years, and type 2 patients usually live up to 25 years with difficult handicapped situations.
It is classified as one of the rare diseases, yet it affects approximately one in 7,744 live births in India. There is no cure for SMA; it can only be managed, and the progression can either be stopped or slowed down. For families facing this diagnosis, the word “rare” offers little comfort. The disease is real, the suffering is real, and the costs are astronomical.
The High Cost of Treatment
The cost of treatment for SMA in India is astronomically high. While the gene therapy costs ₹17 crore (a one-time treatment), Evrysdi, the oral solution, produced by Roche, costs ₹72 lakh a year. Gene therapy is only for children below two years, and the only medical remedy for SMA patients that was available till recently was Evrysdi.
It is only in the last month, after winning a patent litigation, that an Indian company Natco started manufacturing the molecule in India, bringing down the cost to ₹15,000 per vial from ₹6 lakh. Yet, the annual cost for the medicines alone would come to around ₹5 lakh.
This is progress, but it is not enough. ₹5 lakh per year is still beyond the means of most Indian families. And for those who need the one-time gene therapy, ₹17 crore is simply unimaginable.
The National Rare Diseases Policy
There exists a National Rare Diseases Policy, 2021, initiated by the Union government. The policy classifies rare diseases into three groups and designates a few hospitals as Centres of Excellence for the treatment of these diseases. NRDP also grants ₹50 lakh as a one-time financial support for a patient under the policy.
This amount is thoroughly insufficient in view of the exorbitant prices of the life-saving medicines, especially for group 3 rare diseases. ₹50 lakh might cover ten years of treatment with the generic version of Evrysdi, but what happens after that? And what about patients who need the gene therapy, which costs more than three times that amount?
The policy is a recognition of the problem, but it is not a solution. It acknowledges that rare disease patients need help, but it does not provide enough help to actually save them.
The Legal Battle
Patients have approached the government and the courts seeking treatment under the fundamental right to life. Seba, a 24-year-old SMA patient from Kerala, also approached the High Court seeking the invocation of Section 100 of the Patent Act, 1970, which empowers the government to use inventions for non-commercial public use.
Section 100 was inserted into the Act in 2005 as a legislative safeguard against patent monopoly abuse, particularly for medicines. Unfortunately, not once has the Section been used by any government, catering to the right to life of the citizens. The provision exists, but it has never been activated. It is a tool that has remained in the toolbox, unused, while patients have died.
The case filed by Seba has reached the Supreme Court along with cases of several SMA patients. The Supreme Court is yet to decide whether ₹50 lakh can be the ceiling for the funds for the SMA patients, and how Seba can be given further treatment, etc.
The Scale of the Problem
There are 4,230 patients registered with the government on the NPRD website. The fund in the crowdfunding account of the government was ₹3,95,189 as on November 28, 2025. Even under NPRD, only a few patients are granted ₹50 lakh. The Centres of Excellence are not yet prescribing the generic version, despite the same being much more affordable. Yet, even if the generic version of Evrysdi is used for treatment, ₹50 lakh will cover only 10 years.
Per the NRDP website alone, 911 SMA patients are awaiting financial aid. The gap between need and available resources is staggering. ₹3.95 lakh in a crowdfunding account against 4,230 registered patients and 911 awaiting aid. The numbers do not add up.
The Constitutional Obligation
Article 21 is a right against deprivation of life. If the government has the tools to ensure the preservation of lives through provisions under the Patent Act, the inaction amounts to deprivation. Therefore, in these cases, the government and the constitutional courts carry not just a ‘social burden’ but a ‘constitutional and legal burden’ to ensure the rights of the citizens.
This is not charity. It is not benevolence. It is a constitutional obligation. When the state has the power to act, and fails to act, it is violating the fundamental rights of its citizens.
The Real Solution
The real solution would be for the government to procure the generic version of Evrysdi and distribute it for free to the SMA patients. The government must also allocate greater funds towards research and development for the indigenous manufacturing of these medicines. The apex court can issue necessary directions in that regard.
Procurement at scale would bring costs down further. Distribution through the public health system would ensure that no patient is denied treatment for want of funds. Investment in R&D would reduce dependence on foreign patents and create domestic capacity.
Conclusion: A Matter of Life and Death
The SMA patients of India are not asking for charity. They are asking for their constitutional rights to be honoured. They are asking for a policy that actually works, for funds that are actually available, for treatment that is actually affordable.
₹50 lakh is not enough. ₹3.95 lakh in a crowdfunding account is not enough. 4,230 registered patients and 911 awaiting aid is not a statistic; it is a measure of the state’s failure.
The Supreme Court has an opportunity to change this. It can order the government to use Section 100 of the Patent Act. It can direct that generic versions be procured and distributed. It can ensure that the right to life means something for those with rare diseases.
For Seba and thousands like her, this is not an academic debate. It is a matter of life and death.
Q&A: Unpacking the Rare Disease Funding Crisis
Q1: What is Spinal Muscular Atrophy and how common is it in India?
SMA is an inherited disease affecting nerves and muscles, causing progressive weakness. Types 1 and 2 are life-threatening; types 3 and 4 severely impact quality of life. It affects approximately one in 7,744 live births in India. There is no cure; progression can only be slowed or stopped.
Q2: What are the treatment costs for SMA in India?
Gene therapy costs ₹17 crore (one-time, only for children under two). Roche’s Evrysdi costs ₹72 lakh annually. After a recent patent litigation, Indian company Natco now manufactures the molecule, bringing cost to ₹15,000 per vial—annual cost around ₹5 lakh. While improved, this remains unaffordable for most families.
Q3: What does the National Rare Diseases Policy, 2021 provide?
The policy classifies rare diseases into three groups, designates Centres of Excellence, and provides ₹50 lakh as one-time financial support per patient. This amount is insufficient—it covers only about 10 years of generic Evrysdi treatment, and falls far short of the ₹17 crore needed for gene therapy.
Q4: What is Section 100 of the Patent Act and why is it important?
Section 100, inserted in 2005, empowers the government to use inventions for non-commercial public use as a safeguard against patent monopoly abuse, particularly for medicines. It has never been used by any government. Patients argue that invoking it could allow procurement of affordable generic versions for SMA patients.
Q5: What is the current status of patients and funding?
4,230 patients are registered on the NPRD website. The government’s crowdfunding account held only ₹3,95,189 as of November 28, 2025. Only a few patients receive the ₹50 lakh grant. 911 SMA patients are awaiting financial aid. Centres of Excellence are not yet prescribing the more affordable generic version. The gap between need and resources is staggering.
