“Hey, is everything okay? Which doctor are you going to today?” I heard this while I was shifting from my wheelchair to the car on a day I was actually looking forward to a road trip with friends. For a second, I just sat there, frozen. A pit formed in my stomach as it hit me, yet again, that my disability is the first, and often the only thing people see when they look at me. Not the excitement I felt about the trip. Not the rare moment of freedom. Just my condition, my weakness. This is due to the genetic disorder resulting in Spinal Muscular Atrophy (SMA).
In many Indian households, even basic healthcare expenses can push families into debt and poverty. The situation becomes even more critical in cases of chronic and rare diseases such as SMA, where long-term treatment costs are astronomical. Without intervention, life-saving treatments are available only to a privileged few, leaving the vast majority to suffer or perish prematurely. This disparity undermines the very essence of healthcare.
What is SMA?
SMA is a genetic condition that affects motor neurons in the spinal cord, leading to progressive muscle weakness and atrophy. It is caused by mutations in the Survival of motor neuron 1 (SMN 1) gene, which result in a deficiency of the SMN protein – a crucial component for nerve function. Without this protein, the nerve cells responsible for muscle movement begin to deteriorate, causing a gradual loss of voluntary muscle control. This primarily affects movement and posture, and in severe cases, it can also impair breathing and swallowing. While SMA does not affect cognitive abilities, its complications, including scoliosis and respiratory issues, can have a profound impact on overall quality of life.
Unlike the majority of rare diseases, SMA has at least three medicines/treatments. First, gene therapy for SMA type 1 in children below two known as Zolgensma, can cure the disease. Second, therapies modify the diseases, especially slow down the progress of the disease, such as Spinraza and Risdiplam for all types of SMA. Those above two years of age have left with therapies in the second category. However, all these therapies are expensive which make them out of reach for a majority of the patients. While Zolgensma costs Rs 18 crore the cost of Spinraza and Risdiplam are around Rs 1.5 crore per dose and Rs 6.20 lakh per bottle.
These three treatments – Zolgensma, a one-time gene therapy effective only for infants; Spinraza, an injection into the spine every three months; and Risdiplam, a daily oral medication that is the most convenient and effective for people like me. Both Spinraza and Risdiplam require lifelong use, but their exorbitant costs make them inaccessible to most patients. Gene therapy and Spinraza aren’t even available in India.
How does SMA plague the life of a family?
SMA is stripping away my muscle strength bit by bit, making even the most basic tasks impossible without help. I rely on someone for everything – getting out of bed, brushing my teeth, eating, drinking water, adjusting my posture, and even scratching an itch. Every little action that most people don’t even register as effort is something I need assistance with. There is no such thing as privacy or independence when your body refuses to cooperate.
The only thing I can still do on my own is work, thanks to my right hand retaining just enough function to use a smartphone and a computer. I work from home as a professional, something that gives me a sense of purpose, and a reason to wake up every morning. But with SMA’s constant progression, even that feels like it’s slipping away. Every keystroke, every scroll on my phone is a reminder that time is running out. I don’t know how much longer I’ll be able to do the one thing that still makes me feel productive, and that uncertainty is terrifying.
SMA doesn’t just affect me – it dictates my entire family’s life. Every plan, every decision, no matter how small, has to revolve around my condition. There’s no such thing as spontaneity. A simple outing isn’t just about deciding where to go – it’s about accessibility, about who will be there to lift me, adjust my posture, or help with things that most people never even think twice about.
My parents, my siblings – they don’t get to take a break from SMA, just like I don’t. The hardest part? Knowing that as my condition worsens, their sacrifices will only grow.
Should I hope?
For years, hope wasn’t even part of my vocabulary. But then I go to know about Risdiplam – a drug specifically designed for people with SMA. This wasn’t just another experimental treatment; it was the real deal. It worked. It promised to slow down the progression of the disease, giving me a chance to hold on to what little independence I had left.
But then came the gut punch: Risdiplam costs more than Rs 72 lakh a year for an adult if the company gives two bottles free with every bottle we purchase. It was like dangling salvation in front of me, and then locking it behind an insurmountable paywall.
For people like me, Risdiplam could mean the world. It could mean not watching yourself slowly fade away. It could mean going on that road trip without needing ten things planned in advance. It could mean finally feeling like you’re not just surviving but actually living.
Adding insult to injury, I found that Risdiplam is available at one-sixth of the price in our neighbouring countries compared to what we’re charged here in India. How is this fair? How is it acceptable for the same drug to remain so outrageously out of reach for those who need it the most?
Implementing policy
India has the tools to fix this. Indian pharmaceutical industry has the capability to produce Risdiplam. In fact, an Indian generic company submitted an application for marketing approval in India and the USA. However, the patent protection on Risdiplam will prevent the marketing of the generic version. The patent holder (Roche) has filed suit seeking an injunction from the Delhi high court to prevent the marketing of the generic version of Risdiplam.
The Patents Act, specifically Section 100, gives the government the power to step in and allow the production of generic versions of patented drugs in public interest. It is a critical provision that empowers the government to intervene in matters of public interest. This includes authorising the manufacture, use, or distribution of a patented product, without requiring prior permission from the patent holder.
Over the years, Indian pharmaceutical companies have played a vital role in breaking monopolies on expensive drugs and providing affordable options for conditions like HIV, cancer, and hepatitis. These contributions have saved countless lives – not just within the country but across the globe. So why can’t it be done for SMA as well?
Producing a generic version of Risdiplam would bring the price down drastically, making it accessible to thousands of SMA patients. India has some of the best pharmaceutical companies in the world – companies that can take this drug and make it affordable. All we need is for the government to give the go-ahead. According to news report, Dr Melissa Barber analysed the cost of producing Risdiplam, and the results were staggering. The annual production cost of this lifesaving drug? Barely Rs 3,024 per patient per year. A drug priced at Rs 70 lakh costs just Rs 3,024 with a 20% markup.
Prioritise life over profits
I’ve heard the argument: “If you allow generics, pharmaceutical companies won’t innovate.” To that, I say – enough. Most of these “innovations” are built on decades of research funded by taxpayers, and by the public. The companies refine these treatments, slap on a patent, and then act like they built everything from scratch.
Don’t get me wrong. Profit has its place. I get that pharmaceutical companies invest heavily in research and deserve returns. But Rs 70 lakh for something that costs Rs 3,000 to produce? And that too after many years of production. That’s not profit. That’s exploitation.
What makes it worse is that SMA isn’t even that rare globally. Thousands of people could benefit from this treatment. And yet, because it doesn’t impact millions, the system lets us slip through the cracks. It’s heartbreaking and infuriating.
Medicine is not just a commodity; it is a basic human right. A robust and accessible healthcare system requires policy frameworks that prioritise public health over profit, leveraging tools like compulsory licensing or government authorisation under Section 100 of the Patents Act to ensure life-saving medications are accessible and affordable.
This isn’t just about policy. It’s my life, my story. This is about people like me – people who are tired of watching their world shrink while being told to “be brave”. What I want is basic human right to live with dignity.
Every day that this drug remains out of reach, someone with SMA gets weaker. Someone like me loses another piece of their independence. And someone loses hope.
But beyond the physical challenges is the emotional toll. It’s knowing that while I fight to stay afloat, decision-makers argue over geopolitics, innovation, and profit.
The Union government has the power to change this. They can invoke Section 100, authorise generic production of Risdiplam, and ensure that people like me are not left behind. Even after the demand for the use of Section 100 within and outside the parliament, the Union government does not provide any explanation for its reluctance to use Section 100. They can look at us – not as burdens or statistics, but as individuals who deserve a chance to live with dignity as enshrined under Article 21 of the constitution.
Saifullah Khalidi has SMA and he is an advocate of access to treatment.
