Funding support for rare disease patients: how to claim Rs.50 Lakh Government grant

Background: India’s Rare Disease Policy

India’s journey with rare disease management has been shaped by the sheer complexity and scale of the challenge. Although each rare disease affects only a small number of people, collectively they impact an estimated 6–8% of the population, with 6000–8000 such conditions identified globally and new ones still emerging. ¹

In India, around 450 rare diseases are known, many of them genetic and disproportionately affecting children. But gathering accurate data and ensuring timely care remain difficult because of demographic diversity, varied disease presentation, gaps in awareness, limited research capacity, and restricted diagnostic access. Treatment is often delayed and expensive, with high costs and the absence of clear payer guidelines affecting affordability. ¹

It wasn’t until 2017 that the Government of India formally recognized rare diseases as a priority, leading to the release of the National Policy for Rare Diseases (NPRD), later revised in 2021. The policy acknowledged these long-standing hurdles and emphasized the need for strategic planning, improved access to quality and affordable care, and stronger coordination across stakeholders.¹

^{Which Patients Qualify for the Rs.50 Lakh Support}

Under the NPRD 2021, patients can qualify for financial support of up to ₹50 lakh through the RAN scheme, but only if they meet the eligibility norms of the Pradhan Mantri Jan Arogya Yojana, covering roughly 40% of India’s population. This assistance is available solely for treatments carried out in government tertiary hospitals and designated Centres of Excellence listed under NPRD 2021, and does not apply to care sought in private hospitals. While the scheme extends beyond BPL families, access remains shaped by restrictive criteria, slow approvals, and administrative delays, which continue to limit timely and equitable use of the ₹50 lakh support despite the heavy lifelong costs associated with rare disease care.^2,3

Documents Required for Application

To apply for the ₹50 lakh financial support, the patient or their parent/guardian needs to submit the official application form (Annexure-IV) at the Centre of Excellence (CoE) where they are seeking treatment. This form must be filled in the prescribed format and signed by the treating doctor using Annexure-V. Once submitted, the application is reviewed by the Rare Disease Committee set up at each CoE, with the Nodal Officer for Rare Diseases acting as the Member Secretary. The CoE may also bring in an external expert if needed during this evaluation.⁴

^{Step-by-Step Process for Submitting a Claim}

To submit a claim under the ₹50 lakh support:⁴

1. The process begins with the patient, parent, or guardian filling the prescribed application form (Annexure-IV) at the Centre of Excellence (CoE) they are receiving treatment from, which must be signed by the treating doctor (Annexure-V).
2. After this, the CoE’s Nodal Officer scrutinizes the application and places it before the Rare Disease Committee, which must decide on treatment and fund allocation within four weeks.
3. Once approved, the CoE sends a fund request to the Ministry of Health & Family Welfare along with the application, the doctor-signed Annexure-V, and the Committee’s recommendation.
4. The Ministry’s Rare Disease Cell then releases the funds as per procedure, after which the CoE is responsible for proper utilization under NPRD-21.
5. If more funds are required later, either after full use of the initial amount or for new patients, the CoE can request additional support by submitting a utilization certificate signed by the Nodal Officer.

^{How Government Committees Review Applications}

Each CoE has its own Rare Disease Committee, led by the Nodal Officer as Member Secretary, which may include an external expert if needed. The Nodal Officer first scrutinizes the application and then places it before the Committee, which must take a decision on treatment and fund allocation within four weeks. Based on the Committee’s recommendation and the Director’s approval, the CoE sends a fund demand to the Ministry of Health & Family Welfare along with all required documents.⁴

^{Common Challenges Patients Face}

Even though the NPRD 2021’s RAN scheme promises financial assistance of up to ₹50 lakh, many patients still struggle to access this support because of bureaucratic delays, lengthy approval steps, and strict eligibility rules. ²

On top of this, most Centres of Excellence, around ten across India, are clustered in major metros like Delhi, Mumbai, Bengaluru, and Chennai, forcing patients from rural and remote regions to travel long distances at high cost. ²

While plans to expand these centers exist, concrete steps are still early. Add to that the lack of a centralized rare disease registry, and it becomes even harder to shape effective policies, track patient needs, or improve research. A strong, unified registry could help streamline decision-making and ultimately improve access and outcomes for patients.²

^{Role of NGOs and Rx4U in Assistance}

When a family is suddenly faced with a high-cost medical emergency, even a small bit of support can take a huge weight off their shoulders. That’s where NGOs and platforms like Rx4U step in. By connecting patients to verified assistance options, guiding them through documentation, and helping them navigate available schemes or funding channels, these groups play a crucial role in bridging the gap between need and access. Their involvement often brings clarity, reduces confusion, and makes the entire process of seeking up to ₹50 lakh support feel a little less overwhelming for those already going through a difficult time.

Success Stories from Families Who Got Support

In India, where treating Gaucher disease (GD) can cost an average of ₹84.85 lakh per patient each year, the National Policy for Rare Diseases (2021) has become a crucial lifeline.

A Mumbai-based study on 30 patients highlights just how overwhelming this burden can be, disease-specific treatments like enzyme replacement therapy accounted for nearly 99% of total expenses, with treated patients costing 157 times more than those who remained treatment-naïve. Yet it also shows what timely support can achieve: early diagnosis, expert-guided care, and access to funded therapies can prevent severe complications, reduce hospitalizations, and significantly improve quality of life. Against this backdrop, financial assistance of up to ₹50 lakh per patient under India’s rare disease framework is more than a policy, it’s a direct intervention that makes long-term, life-altering treatment possible for families who would otherwise be priced out of care.

Conclusion

India’s rare disease ecosystem is evolving, but the challenges remain immense, from high treatment costs and limited diagnostic access to uneven distribution of Centres of Excellence. The NPRD 2021 and the RAN scheme offer a critical financial pathway, especially for conditions like Gaucher disease, where annual treatment costs can exceed ₹84 lakh per patient.5 With clearer processes, stronger infrastructure, and better coordination across stakeholders, the ₹50 lakh support can become more accessible to those who need it most. Continued policy refinement, expanded CoE networks, improved data systems, and active collaboration with NGOs will be essential to ensure that rare disease patients and their families receive timely, equitable, and sustainable care.^1-5

^FAQs

1. Who is eligible for the ₹50 lakh support under NPRD 2021?
   Patients who meet PM-JAY eligibility norms and receive treatment at designated government Centres of Excellence qualify for the assistance.^2,4
2. Where do patients submit their application?
   The prescribed form (Annexure-IV), signed by the treating doctor (Annexure-V), must be submitted at the Centre of Excellence where treatment is being taken.⁴
3. How long does the approval process take?
   The Rare Disease Committee at the CoE must review the application and decide on treatment and fund allocation within four weeks.⁴

References

1. Mishra S, Bhat D, Venkatesh MP. Navigating health policies and programs in India: exploring opportunities to improve rare disease management and orphan drug research. Orphanet J Rare Dis. 2024;19(1):446. Published 2024 Nov 29. doi:10.1186/s13023-024-03377-6
2. Danish S, Rasool I. Evaluation of the National Policy for Rare Diseases(NPRD) 2021 and Ayushman Bharat in Addressing Financial Barriers, Infrastructure Gaps, and Healthcare Accessibility Challenges Journal of Academy of Hospital Administration. 2025;36(2):50-61. doi:10.4103/jaha.jaha_17_24
3. Rare Diseases India. Big relief: Union Health Ministry hikes financial grant for rare disease treatment from Rs 20 lakh to Rs 50 lakh (applies to all categories). RareDiseases.in. Published May 19, 2022.
4. Rashtriya Arogya Nidhi Guidelines
5. Mhatre SP, Muranjan M, Gogtay NJ. Economic burden of Gaucher disease at a tertiary care public hospital in Mumbai. Indian J Pediatr. 2023;90(12):1234-1240. doi:10.1007/s12098-023-04740-4

Note:

This information is provided for educational purposes only and is not a substitute for professional medical advice. Lokelma should only be taken under the guidance of a qualified healthcare provider. Patients should always consult their doctor or pharmacist for advice on diagnosis, treatment, and medication use, and should not make changes to their prescribed therapy without medical supervision.

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