Rare Diseases: Justice Delayed, Treatment Denied

On 7 November 2025, the Supreme Court took up the Union government’s appeal concerning the Delhi High Court’s rare-disease funding judgment and set the matter down for final hearing in March 2026. The Court did not issue any interim directions on financing. For families whose children have exhausted the INR 50 lakh ceiling at Centres of Excellence (CoE) under the National Policy for Rare Diseases (NPRD) 2021, the immediate effect is continuity of the status quo. While hundreds of patients await the outcome of the legal process, treatment schedules that cannot be paused result in inevitably foregone care.  During these proceedings, organisations such as the Organisation for Rare Diseases India (ORDI) and Lysosomal Storage Disorders Support Society (LSDSS) have documented deaths of petitioners and other rare-disease patients, underscoring how judicial or administrative delay can be fatal for time-sensitive therapies.

The costs of judicial and administrative delays in the health sector are perhaps even more serious, as they are paid in organ damage, disability, and often death.

In September, Sanjeev Sanyal, a Member of the Prime Minister’s Economic Advisory Council (PMEAC), told a gathering of legal professionals that India has a narrow window to complete its structural transformation and that delays in the legal system have become a binding constraint. His remarks concerned contracts and commercial mediation. The costs of judicial and administrative delays in the health sector are perhaps even more serious, as they are paid in organ damage, disability, and often death. The longer a case drifts between filings and listings, the greater the chance that those who sought relief will not live to see it.

Process as Punishment

The current momentum in rare disease policy is primarily catalysed by judicial action. Responding to a growing need for rare disease treatment in the country, and in light of ongoing litigation, the National Policy for Rare Diseases (NPRD) 2021 was finalised and operationalised by the Government of India. In October 2024, the Delhi High Court, in Master Arnesh Shaw v. Union of India, directed the Union Government to establish a National Fund for Rare Diseases and to create flexible pathways for financing beyond the INR 50-lakh cap in appropriate cases.  Months later, the High Court recorded that the Supreme Court had granted an interim stay while admitting the Union’s appeal. The 7 November hearing, the latest in a series of court proceedings, has not changed ground realities for any patient.

Patient groups have documented the human cost more directly. They report dozens of deaths over the past three years among people with different rare diseases, resulting from therapy interruptions or non-initiation.  Furthermore, more than 50 out-of-treatment Lysosomal Storage Disorders (LSDs) patients have been identified; having reached the funding ceiling, they now require urgent restoration of therapy. In this context, where petitioners die while proceedings are prolonged, a recent commentary argued that the judiciary should treat the present situation as a humanitarian emergency and order implementation of the High Court’s blueprint.

The Elusive Promise of Crowdfunding

With the extremely high costs of treatment, many patients with rare diseases have turned to private crowdfunding platforms for assistance, and the government has also launched its own platform.  An earlier analysis revealed critical gaps in the government’s rare-disease crowdfunding portal. While verified cases listed by CoEs indicate aggregate annual treatment requirements of about INR 9,100 crore, total donations routed through the portal since its August 2021 launch amount to only around INR 3.9 lakh—even as private platforms routinely mobilise crores for single campaigns. It was argued that, despite being legitimate and auditable, the portal’s weak user experience, limited storytelling, lack of seamless pathways for recurring and CSR/FCRA contributions, and modest outreach have constrained its traction.

To resolve this challenge, the Delhi High Court took an institutional step. Last week, the Court constituted a supervisory committee chaired by Rajiv Bahl (Secretary, Department of Health Research and Director-General, ICMR), to oversee the government’s rare-disease crowdfunding portal.

To resolve this challenge, the Delhi High Court took an institutional step. Last week, the Court constituted a supervisory committee chaired by Rajiv Bahl (Secretary, Department of Health Research and Director-General, ICMR) with V. K. Paul (Member, NITI Aayog), a Joint Secretary from the Ministry of Corporate Affairs, and a Joint Secretary from the Department of Public Enterprises, to oversee the government’s rare-disease crowdfunding portal, build donor awareness, channel Corporate Social Responsibility (CSR) contributions from public sector undertakings, and liaise across ministries to effectuate NPRD-2021. The committee has been directed to meet monthly and file a status report, signalling judicial intent to convert the portal from a passive listing site into an active financing conduit.

A Forward Path for Rare Disease Patients

Even as Courts continue to drive rare disease policy, the pace of litigation often seems to defeat its purpose. The rare-disease funding case in the Delhi High Court began as a batch of petitions in 2020 led by Master Arnesh Shaw v. Union of India, seeking uninterrupted treatment and a predictable public financing pathway. The 7 November order gives a timeline towards the final hearing in March 2026, yet it does not change the fact that children initiated on therapy are falling off treatment when they hit the funding ceiling.

The legal process must reach a logical conclusion soon, and the NPRD architecture must be strengthened and streamlined to better meet patient needs—lest judicial and administrative delays continue to drive the human toll to unacceptable levels.

India has, over the last few years, developed the pieces of a workable rare-disease financing pathway, including a national policy and registry, CoEs, duty and tax relief, early State models supporting affordability, and a judicially articulated pooled-fund architecture. The legal process must reach a logical conclusion soon, and the NPRD architecture must be strengthened and streamlined to better meet patient needs—lest judicial and administrative delays continue to drive the human toll to unacceptable levels. 

Oommen C. Kurian is Senior Fellow and Head of the Health Initiative at the Observer Research Foundation.

• Healthcare
• India
• national policy for rare diseases
• national policy for rare diseases
• NPRD 2021
• NPRD 2021
• rare disease crowdfunding
• rare disease funding
• rare disease policy india
• rare disease portal
• rare diseases funding judgment

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