India’s rare disease policy has crossed the first threshold. The National Policy for Rare Diseases, 2021 gave formal recognition to a class of patients who had long remained outside the practical reach of public health financing. That mattered. It signalled that the Union government was willing to engage with conditions that are medically complex, financially punishing, and numerically small. But the policy’s central weakness is now clear. It can help some patients begin treatment. It cannot reliably help them stay on it. That distinction is no longer technical. It is the core policy question.
For a large part of the health system, one-time support can still produce a meaningful result. A surgery is completed. A course of treatment ends. A health event is resolved. Ultra-rare disorders do not fit that template. In conditions such as Gaucher disease, Pompe disease, Fabry disease and some forms of mucopolysaccharidosis, treatment is not episodic. It is continuing, sometimes lifelong. Once therapy is initiated, interruption is not an administrative inconvenience. It can mean clinical deterioration, lost functionality and, in some cases, irreversible damage.
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Rare disease policy needs a relook
This is where the current framework begins to fray. Group 3A patients are eligible for one-time financial support of up to Rs 50 lakh. That may be enough to start treatment in some cases. It is plainly inadequate for therapies that must continue over years. A policy designed around entry into treatment, rather than continuity of treatment, creates a false sense of resolution. It allows the state to say support exists, while leaving families exposed to the far harder question of what happens after the ceiling is exhausted.
That design flaw goes beyond budget arithmetic. It reflects an incomplete understanding of the nature of these diseases. The value of therapy in such cases lies not in initiation alone, but in sustained administration. Once that is accepted, continuity of care stops being a moral appeal and becomes a policy requirement.
The issue is often described as one of high cost. That is true, but insufficient. The real problem is that India’s public health financing architecture is still built mainly for high-volume, lower-cost burdens. Rare diseases sit outside that instinct. They force the system to confront a category it does not handle well: very small patient populations with very high and recurring needs. That is why the present debate matters well beyond rare disease policy. It asks whether the Indian state can design institutions for medical conditions that do not fit majoritarian budgeting logic.
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Continuum of care is the real health system test
The right way to think about ultra-rare disorders is not as a funding event, but as a chain of care. Diagnosis, referral, clinical assessment, treatment approval, procurement, financing, administration, monitoring and follow-up are all linked. The chain fails at its weakest point. A delayed approval, a stalled procurement process, a break in fund flow, or the absence of state-level implementation support can each destroy the value of treatment already begun.
That is why the language of access, though important, is not enough. Access without continuity is partial relief dressed up as policy success. Affordability without dependable financing is fragile. Quality without sustained administration is moot. Equity without implementation capacity remains rhetorical.
The burden of this failure does not fall evenly. It falls first on children, whose developmental losses cannot simply be recovered later. It falls on caregivers, who are pushed into constant financial and emotional crisis. It falls on clinicians, who must explain why a therapy that works cannot be reliably sustained. And it falls on patient groups, which are left to do what public systems should have learned to do by now: keep the issue alive, case by case, year by year.
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Centre-state coordination holds key
The next phase of reform cannot be left to the Union health ministry alone. Rare disease policy will remain fragile if it stays confined to a central document and a small set of tertiary institutions. The implementation burden sits elsewhere too: in hospitals, procurement systems, state health departments and legislative oversight.
This is where Parliament matters. Not because MPs can produce clinical solutions, but because they can force administrative attention. They can ask whether allocated funds are being fully used. They can examine why approvals are delayed, why treatment centres struggle with procurement, and why patients fall through after an initial sanction. They can also give political legitimacy to a difficult but necessary argument: that some categories of public health spending cannot be judged by patient volume alone.
States matter for an equally practical reason. Health delivery is never purely central. Even where financing comes from Delhi, treatment continuity depends on local institutions. A national policy without state-level ownership becomes another well-worded framework with uneven outcomes. Rare disease care needs cooperative federalism in a literal sense, not as a slogan. It requires shared responsibility between the Centre, states, hospitals and clinicians.
Use of funds matters as much as larger funding
It would be easy to treat this debate only as a demand for more money. That would be incomplete. Before expanding the financial envelope, the system must examine how efficiently existing support is being used. Are sanctioned funds reaching patients in time? Are hospitals able to draw and deploy allocations without procedural breakdowns? Are there avoidable bottlenecks in approvals, procurement or coordination? A policy that under-utilises what it has will struggle to justify what it asks for next.
But the reverse is also true. Administrative efficiency cannot substitute for structural reform. Better fund utilisation is necessary, not sufficient. If the financing design remains one-time while treatment needs are continuous, smoother administration will still leave the central mismatch intact.
A more credible framework would begin with risk prioritisation. Patients already on therapy, especially children and those facing imminent interruption, should be protected first. That is where policy triage is most defensible. The second step is to recognise continuity of care as a legitimate public objective in itself. Until that principle is embedded, rare disease financing will continue to oscillate between sympathetic rhetoric and episodic support.
Rare disease care needs new policy imagination
India does not need to decide whether rare diseases are worthy of policy attention. That question has already been answered. It needs to decide whether it is serious about designing a care model that reflects the clinical reality of such conditions.
The state has already acknowledged the patient. It must now acknowledge time.
That is the missing element in the present framework. One-time grants address cost at the point of entry. They do not address the continuity on which outcomes depend. If treatment is interrupted after initiation, the system does not merely pause care. It wastes prior expenditure, deepens family distress and weakens trust in public policy itself.
The test of rare disease policy is not whether it can announce support. It is whether it can sustain treatment where interruption is medically destructive. That is a harder standard. It is also the only serious one.
Dr K Madan Gopal is a Senior Health Sector Expert. He is a former Senior Consultant with NITI Aayog.