Rare disease policy falls short where it is needed most: Public policy often fails not when it is announced, but when it must endure contact with real life. India’s rare disease policy is in danger of failing in exactly that way. The National Policy for Rare Diseases, 2021, was meant to bring relief to families facing impossible medical odds and impossible treatment costs. It did create hope. But for many patients, that hope now ends at an administrative ceiling of Rs 50 lakh. Once the money is exhausted, treatment often stops. The disease does not.
That is the central flaw in the current rare disease policy. India has acknowledged the problem of rare diseases, but it has not yet built a credible framework for continuity of care. For disorders that require lifelong therapy, a one-time financial cap is not a solution. It is a pause button.
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System breaks when patients need it most
Recent discussions with patient groups, doctors and former public officials, the reality came through with distressing clarity. Families described a familiar sequence. Diagnosis is delayed. Awareness among frontline doctors is patchy. Treatment pathways are hard to navigate. And once therapy finally begins, it is interrupted when the approved financial limit runs out.
This is not merely an issue of financial burden. It is an issue of public credibility. The state cannot say it supports treatment and then look away when treatment must continue to remain effective.
One example that struck me deeply was the broader desperation surrounding these diseases. Even parliamentarians have found themselves scrambling to mobilise funds for individual patients with conditions such as SMA, where therapies can cost several crores. If elected representatives are reduced to piecing together financial support case by case, the position of an ordinary family is self-evident.
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Funds exist, but access does not
The discussions on the rare diseases policy draws attention to a troubling contradiction. Patient representatives cited figures showing that substantial sums allocated for rare disease treatment remain underutilised, even as patients lose access to therapy after exhausting their individual ceiling. They referred to an allocation of around Rs 299 crore in 2025-26, of which only a fraction had been utilised, along with earlier parliamentary data showing unspent balances with Centres of Excellence.
This is not a minor accounting issue. It goes to the heart of policy design and implementation. If funds are available in the system, but children already on treatment are denied continuity because their personal cap is exhausted, then the problem is no longer scarcity alone. It is also one of governance, coordination and administrative rigidity.
A rare disease policy that funds treatment only until the patient shows improvement, and then withdraws support, does not produce savings. It produces reversal. In many rare disorders, interrupted treatment can mean renewed deterioration, loss of function, and permanent damage. Several patient advocates made precisely this point. Months of treatment gains can be undone by months of policy delay.
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This is not charity. It is state responsibility.
Rare disease treatment is often discussed in language that invites pity rather than policy seriousness. That is a mistake. What came through in the consultation was that many patients, if diagnosed early and treated consistently, can study, work and live productive lives. Examples were shared of patients becoming doctors, engineers and professionals.
That changes the frame completely. This is not about exceptional compassion for a few tragic cases. It is about whether the public system is capable of preserving lives that can remain socially and economically meaningful. In any serious republic, that should matter.
Rare disease policy response must shift
The first requirement for the rare disease policy is conceptual clarity. The government must recognise that continuity of care is not a secondary matter. For several rare disorders, it is the policy itself. A one-time financial ceiling may suit budget files. It does not suit lifelong conditions.
Second, the Ministry of Health must examine why Centres of Excellence are not functioning in a more timely and coordinated manner. At the consultation, patient groups pointed to weak coordination between the ministry and the centres, uneven onboarding of eligible patients, and delays in treatment decisions. If this is accurate, then the problem lies not only in financing but in execution.
Third, India needs better data. A former senior health official at the consultation made an important point: the country needs systematic evidence on incidence, prevalence and regional concentration. Without that, rare disease policy will remain reactive, driven by scattered cases and courtroom battles rather than public health planning.
Fourth, the Centre must create a structured framework for states to participate. Some states, including Kerala, Karnataka and Rajasthan, were cited as having moved to support patients through their own mechanisms. This federal pathway deserves to be developed, not left to activism and litigation alone.
Fifth, India must push harder on lower-cost treatment pathways, including domestic manufacturing and generic alternatives where scientifically and regulatorily feasible. The long-term sustainability of rare disease care depends not only on public finance, but on reducing the cost of treatment itself. Participants pointed to encouraging movement in this direction for SMA.
Parliament must push the system to act
The immediate lever for change is parliamentary oversight. A parliamentary question can reveal underutilised funds or highlight distress. But information alone does not change policy. It must be followed by institutional scrutiny.
That is why the Parliamentary Standing Committee on Health is the right forum for this issue. Once the committee takes it up formally, the ministry, the Centres of Excellence, ICMR and other authorities can be asked direct questions in one place. That is how a matter moves from sympathy to accountability. The consultation underlined this clearly, and rightly so.
In rare diseases, time is not an administrative variable. It is the difference between stabilisation and decline.
India has already accepted the principle that rare disease patients deserve public support. It must now decide whether that support is meant to save appearances or save lives. A rare disease policy that stops at the funding cap has not finished its job. It has stopped halfway.