Crowdfunding for Rare Diseases: How Families in India Can Seek Financial Support
How Much Does Rare Disease Treatment Cost in India?
Rare disease treatment in India can cost several lakhs to crores of rupees, depending on the condition and therapy required. For instance, the Ministry of Health estimates the one-time cost of Zolgensma gene therapy for Spinal Muscular Atrophy (SMA) at around ₹16 crore. Annual Enzyme Replacement Therapy costs for a 10 kg child with conditions such as Gaucher disease and Pompe disease can range from approximately ₹40 lakh to ₹50 lakh or more.
Rare Disease Treatment Costs in India
| Disease | Published Cost Estimate |
|---|---|
| SMA – Zolgensma gene therapy | ₹16 crore (one-time) |
| SMA – Risdiplam | ₹1-2 crore annually |
| SMA – Nusinersen | ₹3-5 crore annually |
| Gaucher disease | ₹30 lakh annually for a 10 kg child |
| Pompe disease | ₹45-50 lakh annually for a 10 kg child; ₹2.7 crore for a 50 kg patient |
| MPS II | ₹44 lakh annually |
Note: Costs are indicative and may vary depending on dosage, patient requirements and treatment duration.
Why Is Rare Disease Treatment So Expensive in India?
Rare disease treatment can be expensive because developing medicines for small patient populations involves significant research costs, while specialised therapies may require complex manufacturing and imported ingredients.
- Limited patient populations: Pharmaceutical companies have fewer potential users to recover the costs of research, clinical trials and drug development.
- High cost of specialised therapies: Treatments such as gene therapy and Enzyme Replacement Therapy involve complex development and manufacturing processes.
- Dependence on imported medicines: Some rare disease drugs are not manufactured locally, making patients dependent on imported medicines.
- Limited treatment alternatives: When only one or a few therapies are available, patients have fewer options to manage treatment expenses.
- Long-term treatment requirements: Conditions requiring lifelong medication, regular infusions or continuous monitoring create recurring expenses for families.
One-Time Treatment vs Lifelong Treatment: How Do the Costs Differ?
Rare disease treatment costs depend largely on whether a patient needs a one-time intervention or ongoing medical care. Some treatments involve a large upfront expense, while others require families to pay for medicines, therapies and monitoring over several years or throughout the patient’s life.
- One-time treatments: Gene therapies and certain stem cell or organ transplants may offer long-term benefits through a single treatment procedure. For example, Zolgensma for Spinal Muscular Atrophy (SMA) is a one-time gene therapy that can cost several crores.
- Recurring medication and enzyme replacement therapy: Conditions such as Gaucher disease, Pompe disease and Fabry disease may require regular Enzyme Replacement Therapy (ERT). Patients need repeated infusions, making treatment a recurring annual expense. Some conditions, such as SMA, also involve regular medication such as risdiplam.
- Lifelong dietary and supportive care: Certain metabolic disorders, including Phenylketonuria (PKU) and Maple Syrup Urine Disease (MSUD), require specialised diet or medical nutrition. Patients may also need regular monitoring and supportive care to manage their condition.
Note: A one-time treatment does not necessarily mean that all future medical expenses end. Patients may still require follow-up consultations, rehabilitation and supportive care. Similarly, recurring treatment costs can vary with dosage, body weight and treatment duration.
What Additional Expenses Do Families Face Beyond Treatment Costs?
The cost of treating a rare disease may extend beyond medicines or a specific procedure. Families may also need to pay for tests, hospital visits, supportive care and day-to-day assistance. These expenses can add up over time, particularly when treatment requires regular monitoring.
- Diagnosis and genetic testing: Identifying a rare disease can involve multiple consultations, specialised laboratory tests and genetic testing. Families may need to visit different specialists before receiving a confirmed diagnosis.
- Hospitalisation and regular consultations: Some conditions require repeated hospital admissions, specialist appointments, blood tests and other investigations to monitor the patient’s health and response to treatment.
- Rehabilitation and supportive therapies: Physiotherapy, occupational therapy, speech therapy and other supportive services may be needed to manage symptoms, maintain mobility and improve daily functioning.
- Medical equipment and home care: Depending on the condition, families may need wheelchairs, respiratory support devices, feeding equipment, mobility aids or assistance from trained caregivers.
- Travel and caregiving expenses: Patients may have to travel to specialised hospitals or Centres of Excellence, sometimes in another city. Transport, accommodation, food and time away from work can add to the family’s financial burden.
These costs vary by disease, the patient’s needs and where treatment is available. They may continue even when the main treatment is subsidised or covered.
Does the Government Cover Rare Disease Treatment Costs in India?
The Government of India provides financial assistance and certain import-duty exemptions to help families manage rare disease treatment costs. The main framework is the National Policy for Rare Diseases (NPRD), 2021, which provides financial support of up to ₹50 lakh per patient for eligible treatment at notified Centres of Excellence (CoEs). However, this assistance does not necessarily cover the full cost of treatment, particularly when therapies cost several crores.
National Policy for Rare Diseases (NPRD), 2021:
Introduced by the Ministry of Health and Family Welfare in March 2021, the policy aims to improve the diagnosis, prevention and treatment of rare diseases. It also supports research and the development of affordable treatment options in India.
The policy classifies conditions into three groups:
- Group 1: Disorders that can be treated through a one-time curative intervention, such as certain stem cell or organ transplants.
- Group 2: Diseases requiring long-term or lifelong treatment, generally involving relatively lower treatment costs. These may include special dietary formulas, food for special medical purposes (FSMP) or specific medicines.
- Group 3: Diseases for which treatment is available but involves very high costs and lifelong therapy, with challenges in identifying patients who are likely to benefit.
Eligible patients can receive financial support of up to ₹50 lakh for treatment at a notified CoE. The assistance was increased from the initial ₹20 lakh limit. It is important to note that ₹50 lakh is the maximum assistance available, not a guaranteed amount for every patient.
The government also provides full exemption from Basic Customs Duty (BCD) and Integrated Goods and Services Tax (IGST) on eligible imported drugs, medicines and food for special medical purposes for personal use or through CoEs. The exemption applies subject to the prescribed conditions and documentation. In February 2026, seven additional rare diseases, including cystinosis, hereditary angioedema and primary hyperoxaluria, were added to the customs exemption list for personal imports.
Limitations of government assistance
While these measures can reduce the financial burden, they may not cover the entire cost of treatment. For example, a therapy costing several crores can exceed the ₹50 lakh assistance limit. Families may therefore still need to arrange funds for treatment expenses that exceed the available assistance, as well as related medical and caregiving costs.
How Can Families Manage the Financial Burden of Rare Disease Treatment?
Rare disease treatment can involve expenses that continue for months or years. Families may need to combine government assistance, medicine support programmes and fundraising to manage these costs. The options available depend on the patient’s diagnosis, prescribed treatment and eligibility.
- Government financial assistance: Under the National Policy for Rare Diseases, eligible patients can receive financial assistance of up to ₹50 lakh for treatment at a notified Centre of Excellence (CoE). Families can contact a CoE to understand the registration and assessment process. This is a maximum assistance limit, not a guaranteed payment to every patient.
- Pharmaceutical company patient assistance programmes: Some pharmaceutical companies offer patient assistance programmes (PAPs) that help eligible patients access specific medicines at reduced prices or through other forms of support. Families can ask their treating hospital or the medicine manufacturer whether a programme is available for the prescribed treatment and check its eligibility requirements.
- Private medical crowdfunding: Families can also raise funds through private medical crowdfunding platforms. A fundraiser can explain the patient’s condition, treatment plan and financial requirement, supported by medical records and hospital estimates. Sharing it with relatives, friends, employers and wider communities can help reach potential donors. Donations are voluntary, and a fundraiser does not guarantee that the required amount will be raised.
These options can be explored together. Government or medicine assistance may cover part of the treatment, while crowdfunding can help families address the remaining expenses.
How Milaap Supports Families Raising Funds for Rare Disease Treatment
Rare disease treatment can involve high upfront expenses, recurring medicine costs and long-term care. Explaining these needs to potential donors and keeping a fundraiser active over time can be challenging for families. Milaap provides support at different stages of the fundraising process.
- Presenting complex treatment needs clearly: Families may need help explaining a rare disease, the prescribed treatment and its cost to people unfamiliar with the condition. Milaap’s campaign team can support them with campaign storytelling and help communicate the family’s financial needs.
- Managing fundraising over time: Some rare disease treatments require repeated payments rather than a single hospital bill. A dedicated campaign manager can guide families through the fundraising process and help them share updates as treatment progresses.
- Accessing funds when needed: With fast withdrawals, families can access donations as they come in, subject to document verification and withdrawal processing. This can be useful when medical expenses arise at different stages of treatment.
- Reaching a wider donor network: Families can share their fundraiser with friends, relatives, colleagues and other potential supporters. Employer matching benefits may also help eligible campaigns receive additional contributions through participating employers.
- Raising funds without platform fees: Milaap charges zero platform fees, allowing families to raise funds without a platform commission being deducted from donations. Payment gateway charges may still apply depending on the payment method.
Start a medical fundraiser on Milaap today.
Frequently Asked Questions
Rare disease treatment costs in India vary widely depending on the condition and therapy required. Some patients need medicines costing a few thousand rupees, while advanced treatments such as gene therapy can cost several crores.
Zolgensma, a gene therapy used to treat Spinal Muscular Atrophy (SMA), is among the most expensive treatments, with a reported price of around ₹16-18 crore for a single dose.
The government does not provide free treatment for every rare disease. Under the National Policy for Rare Diseases, 2021, eligible patients can receive financial assistance of up to ₹50 lakh for treatment at notified Centres of Excellence.
The policy covers a defined list of rare diseases across three groups. These include conditions such as Spinal Muscular Atrophy, Gaucher disease, Pompe disease, Fabry disease, various Mucopolysaccharidoses (MPS), Phenylketonuria and several metabolic disorders. The list and classification have been updated over time.
Yes. Eligible patients can seek assistance under the National Policy for Rare Diseases through notified Centres of Excellence.
Yes. Families can use medical crowdfunding platforms such as Milaap. They can share medical records, treatment plans and hospital estimates to explain the financial requirement and seek voluntary contributions.
You can start a fundraiser on Milaap by providing details about the patient’s condition, treatment requirement and estimated medical expenses. Relevant medical documents and hospital estimates help explain the need to potential donors. Milaap also offers support with campaign storytelling, fundraising guidance and donor outreach.
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Rare disease: what will ongoing treatment cost as the patient grows?
For therapies dosed by body weight, the cost rises with weight. Project it year by year, and see how long the ₹50 lakh NPRD maximum would last.
Use the hospital's quote for the current dose.
Only approved or sanctioned amounts, across the whole period. This is optional.
This assumes cost rises in proportion to body weight. Ask the hospital how your dose and price are calculated, as dosing and prices can change. The ₹50 lakh NPRD figure is the maximum assistance per eligible patient at a notified Centre of Excellence. It is not a guaranteed amount, so treat the bars as showing the most it could cover. For reference, the Ministry of Health's indicative estimates put Pompe disease at ₹45–50 lakh a year for a 10 kg child and ₹2.7 crore for a 50 kg patient.
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