Article 21 of the Indian Constitution promises a non-negotiable Right to Life, but for families like mine that are living with a rare disease, it is still not being implemented in practice. When access to treatment depends on delays, fragmented systems, and decisions that arrive too late, Article 21 becomes an aspiration instead of a guarantee and I learned this the hard way.
In August 2025, my son passed away, and this tragedy was shaped by time lost while we waited for access, approvals, and execution. The treatment he did get started very late and that too was through the charitable program of a pharma company. While nothing can undo that loss, what I definitely can do is fight so other parents do not lose their children while systems take their time.
A rare disease1 is a medical condition that affects a small proportion of the population. These are often serious, chronic, life-threatening, and frequently require specialist diagnosis and long-term care. Rare diseases include MPS II (Mucopolysaccharidosis Type II), also called Hunter Syndrome, Gaucher disease, Fabry disease, Pompe diseases, among others.
Sadly, for families living with MPS II (Hunter Syndrome), Article 21 often feels less like a guarantee and more like an aspiration. We can all remember one grim moment in our constitutional history when the enforcement of Article 21 was formally suspended during the Emergency between 1975 and 1977 through a Presidential Order under Article 359. That period left a scar on India’s democratic conscience and taught us what it means when life and liberty are made negotiable.
Today, Article 21 cannot be suspended, even during a proclaimed emergency, yet for children with MPS II daily life can feel like an undeclared emergency due to prolonged inaction, fragmented systems, and decisions that never seem to arrive in time.
The first rare disease case in India was highlighted in 2014, and we are now almost towards the end of 2025. Eleven years is a very long time in any policy cycle, but it is an eternity in the life of a child with progressivel worsening health parameters. The disease burden of any rare disease is far reaching and impacts entire families, caregivers. Every delay compounds not only medical risk, but emotional exhaustion, financial ruin, and a lingering despair that quietly steals hope from the next family still waiting.
Organisations like the Rare Diseases India Foundation (RDIF), and our National Hunter Alliance chapter, exist because caregivers have had to hold each other up just to survive. Our collective voice is not a request for charity but a plea for timely access to care within the framework of the policies already announced, the commitments already made, and the constitutional principle already settled.
For MPS II, treatment and disease-management pathways exist. What does not exist is access. Under the National Policy for Rare Diseases (NPRD) 2021, Bone Marrow Transplant (BMT) is supported with financial assistance up to ₹50 lakh per patient. Bone marrow transplantation (BMT) can improve outcomes in some rare diseases, but in MPS II (Hunter syndrome) evidence suggests its benefit—particularly for neurocognitive outcomes—is greatest when performed very early, with outcomes worsening when undertaken after about age three, effectively excluding many patients by age at diagnosis2.
Even when a child qualifies by age and clinical condition, the pathway is constrained by another harsh reality of there being only a handful of specialists in the country with meaningful experience managing MPS II in the transplant setting. In such a fragile scenario, infrastructure alone cannot substitute for expertise, multidisciplinary collaboration, and protocolized follow-through.
Funding under NPRD 2021 is routed only through designated Centers of Excellence (CoEs). In principle, that sounds like good governance but in practice when expertise is missing, centralization becomes another bottleneck. In one of the most prominent rare diseases CoE, tragically lost a child in May 2025 due to complications.
I write this to underline the painful truth that if we do not urgently strengthen clinical capability for MPS II through trained experts, collaborative care models, and real-world experience, even the best buildings and the highest reputations will not guarantee outcomes.
India needs to explore mechanisms that convert public money into saved lives like structured Memorandums of Understanding (MoUs) with experienced centres, direct financial support to expert clinicians, mentorship models across institutions, and international collaborations where needed.
Globally, gene therapy trials for MPS II are underway in Europe and the United States, offering genuine hope for long-term solutions3. India has the talent and the patient populations to participate meaningfully in this next chapter, but right now we are not positioned to do so. We have no clear roadmap, limited investment, and too few training pathways.
Enzyme Replacement Therapy (ERT) remains the only established treatment currently available in India for MPS II. Expensive? Yes, but it is also life-extending and life-improving. MPS II is a recognized rare disease under Group 3(a) of the National Policy for Rare Diseases 2021 with financial aid of up to 50 lakh per patients, and ERT is the available option to help MPS II patients lead quality lives.
In 2024, the Ministry of Health and Family Welfare invited pharmaceutical companies to submit reduced price quotations, and companies responded. Patient groups have followed up repeatedly since then, because for caregivers, implementation is the only silver lining.
The judiciary has become a frequent destination for families who have run out of options. Over the past decade, parents have approached courts not because they enjoy litigation, but because they are trying to keep their children alive.
The Delhi High Court directed the release of approximately ₹974 crore5 to address pending rare disease cases, but the matter is now before the Supreme Court. In early 2025, the Union of India filed a Special Leave Petition (SLP) clarified in its affidavit that this amount was a budgetary direction that was stayed, and therefore never constituted an existing fund.
While files move and arguments continue, children continue to wait for the inevitable with each passing day. The truth we rarely say out loud is that no matter how compassionate, courts are not meant to be the primary gateway to healthcare. A functioning administrative system should spare families that indignity.
The social safety net, too, remains full of holes. Rare disease patients are largely outside traditional insurance coverage. Ayushman Bharat provides ₹5 lakh for BPL families4 which is helpful but inadequate for lifelong treatment needs. Families above the BPL threshold face the cruelest gap of being too “well- off” to qualify for aid, yet nowhere near wealthy enough to sustain recurring costs for years. State announcements such as monthly support in Rajasthan and West Bengal seemed to offer hope, but the absence of disbursement has left families living a life of uncertainty.
India has the institutional strength, the clinical talent, and the policy intent to lead with compassion and precision. What we lack is decisive coordination and timely execution. Right to Life is non-negotiable and rarity does not reduce responsibility. For rare disease families, this has been an emergency stretching across 11 years.
If Article 21 is truly a promise that every life matters, we must prove it to those who are unseen, unheard and running out of time that every life matters.
India has the diagnostics, the specialists, the identified children, ERT, and NPRD support on paper, so why are children with MPS II still dying before treatment reaches them? Until the approved therapy reaches a patient in time, just availability does not solve the purpose. The government needs to ensure all patients have access, else Article 21 will just remain a constitutional right on paper.
The article is written by Saurabh Singh, Founder of Rare Diseases India Foundation (RDIF).
(DISCLAIMER: The views expressed are solely of the author and ETHealthworld.com does not necessarily subscribe to it. ETHealthworld.com shall not be responsible for any damage caused to any person/organisation directly or indirectly)
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