Balancing Health Rights and Resource Constraints: The Dilemma of Rare Disease Treatment in India
Newsletter Edition #260 [The Files In-Depth]
Hi,
The disconnect between countries’ multilateral commitments in Geneva, and practices in national capitals is not new. Therefore, efforts at the international level continue to hold significance and meaning, if only to highlight, and understand certain policy challenges on the field.
Take the case of a resolution on Rare Diseases being considered by WHO member states. To understand the relevance of such initiatives, read our detailed report on an ongoing litigation unfolding in India that shows how, implementing the commitments made internationally lie within countries.
The case also encapsulates the many objectives of ongoing negotiations in global health including, the accent on local production, the use of legally sanctioned policy space to protect public health as enshrined by international trade law, and an overall emphasis on universal health coverage.
My colleague Vineeth Penmetsa has put together a clear analysis on the Indian context specifically looking at a rare disease condition and the lack of effective institutional response so far. He also discusses judicial approaches to address the implementation gap by referring to precedents, and cites case law in various developing countries. Vineeth is a India-based legal researcher with a strong interest in health law and global health policy.
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I. ANALYSIS
Balancing Health Rights and Resource Constraints: The Dilemma of Rare Disease Treatment in India
By Vineeth Penmetsa
The right to healthcare is an essential component of human dignity, and its realization is often at the centre of legal and policy battles. In India, where millions struggle to access high-cost treatments for rare diseases, the role of the judiciary in enforcing healthcare rights has become increasingly significant. The Union of India v. Seba P. A. case exemplifies the complex intersection of health rights, resource allocation, and pharmaceutical access in developing countries.
While India maintains a robust constitutional framework protecting the right to health and advocates for access to medicines in international forums, its domestic implementation reveals significant shortcomings.
This disconnect is particularly evident in the treatment of rare diseases, where prohibitive drug pricing creates an insurmountable barrier for patients despite available legal mechanisms to enhance affordability.
Background: The Case and Policy Context
At the centre of this dispute is Seba P.A., a 24-year-old patient with Type 2 Spinal Muscular Atrophy (SMA) who requires ongoing treatment with Risdiplam (marketed as Evrysdi) at approximately Indian Rupees (INR) ₹18 lakhs [USD 21,000] per month.
Having already received the maximum Indian Rupees ₹50 lakh [USD 58,000] lifetime benefit permitted under India's National Policy for Rare Diseases (NPRD) 2021, Seba petitioned the Kerala High Court to mandate continued treatment coverage.
The court's interim order directing continued treatment has prompted the Union of India's appeal to the Supreme Court, contending that such judicial directives constitute overreach into executive policy domains, would create unsustainable financial burdens, and establish precedents affecting broader health resource allocation.
While the Supreme Court has temporarily stayed the High Court's order, the case highlights fundamental tensions in India's approach to rare disease management. (The matter is tentatively scheduled for consideration before the Supreme Court in April.)
According to a statement by the India-based, Working Group on Access to Medicines and Treatments:
“Since early 2021, the High Courts have been addressing petitions related to SMA, calling for local production and generic competition to address challenges like the high cost of medications caused by patent monopolies. Effective therapies, such as risdiplam, have proven to be lifesaving, but remain out of reach for many due to their prohibitive cost. Despite the availability of funding under the National Policy for Rare Diseases, the high price of risdiplam – priced at Rs 6.2 lakh per bottle [USD 7100] by Roche – continues to limit access. For patients weighing under 20 kg, one bottle is required each month, and those above 20 kg may need up to three bottles per month, making the treatment unaffordable.
Following a special leave petition filed by the Union of India, the Supreme Court on 24 February 2025 stayed the Kerala High Court Division Bench’s order that had previously directed the government to provide the medication as an interim measure due to Seba’s critical condition and also directed the Single Judge to address the issues raised in Seba’s writ petition. In her writ petition, Seba has called for the continuation of her treatment with risdiplam beyond the Rs 50 lakh cap under the National Rare Disease Policy, emphasizing that the drug’s exorbitant cost demands a sustainable solution, including compulsory licensing for low-cost generic supply.
This decision has effectively halted the provision of the drug to Seba till the SLP is heard by the Supreme Court.”
The Working group is a network of patient advocates, patient groups, civil society organisations, academics, lawyers and journalists who work on affordable healthcare, especially medicines and treatments.
The NPRD categorizes rare diseases into three groups based on treatment availability, cost, and effectiveness. For Group III diseases like SMA, which require lifelong therapy costing between INR ₹50 lakhs [USD 58,000] to INR ₹8 crore [USD 925,000] annually, the policy imposes a cap of INR ₹50 lakhs per patient lifetime. This cap represents the government's attempt to balance individual patient needs against broader public health priorities within fiscal constraints.
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