India is entering a phase where medical science can save lives that were once beyond hope. But for patients with rare diseases, survival is increasingly becoming tied not to public healthcare, but to crowdfunding campaigns and online visibility.
Take the case of a 16-month-old girl suffering with Spinal Muscular Atrophy (SMA) Type 1 from West Bengal. In June 2025, she received Zolgensma, among the world’s most expensive gene therapies, after nearly Rs 9 crore was raised through a private crowdfunding platform with support from close to six lakh donors.
The drug has a global price of about Rs 16 crore. The child accessed it at a reduced cost under the manufacturer’s treatment access programme. Zolgensma received regulatory approval for commercial launch in India in August 2025.
Such stories highlight public generosity. Medical crowdfunding has grown into a parallel financing system for rare diseases in India. Around 2021–22, one major platform mobilised over Rs125 crore for rare-disease patients in just 14 months. Several individual campaigns have crossed nine-figure sums, sometimes driven by a single large donor, according to reports.
In sharp contrast, the government’s own attempt to create an institutional crowdfunding mechanism has made little impact. Under the National Policy for Rare Diseases (NPRD), 2021, the Centre launched a national crowdfunding portal to supplement the one-time Rs 50 lakh assistance provided per patient for rare conditions such as LSDs — including Gaucher, Pompe, Fabry and Mucopolysaccharidosis (MPS) which are caused by enzyme deficiencies that lead to toxic build-up in cells. The disorders result in progressive organ damage beginning in infancy. Without uninterrupted Enzyme Replacement Therapy (ERT), children deteriorate rapidly.
Since its launch in August 2021, the Government portal has raised less than Rs 4 lakh for 12 Centres of Excellence (CoEs) combined — a figure patient groups describe as “symbolic, not supportive”.
Against this backdrop, pressure on the government is mounting.
Recently, a group of 45 doctors-turned-Members of Parliament under the umbrella of the Indian Medical Parliamentarians’ Forum (IMPF), in a representation to Prime Minister Narendra Modi, said dozens of children who had earlier stabilised on therapy are now relapsing after treatment was stopped once the Rs50 lakh cap under NPRD’s Group 3(a) was exhausted.
“Children who were earlier improving are now collapsing back into disease — becoming bedridden or dying — only because therapy has stopped despite an existing policy,” said IMPF chairperson and Rajya Sabha MP Anil Bonde. A separate submission has also been made to Union Health Minister JP Nadda.
According to doctors, even brief gaps in treatment can cause irreversible organ damage and sudden medical emergencies. Nearly 60 patients have already crossed the Rs 50 lakh annual limit, leaving their therapy unsupported, while another 100 are at risk of imminent discontinuation, said Bonde in the representation.
A month ago, Delhi BJP MP Praveen Khandelwal too had flagged the issue, highlighting the plight of children with rare genetic disorders. He has also written to the Prime Minister seeking urgent approvals for treatment of LAMA2-related Congenital Muscular Dystrophy (LAMA2-CMD), another condition requiring lifelong and extremely costly care. Several therapies for such conditions remain unapproved despite the policy.
Families, however, say they have heard assurances before — with little change on the ground. “Justice delayed is justice denied. We have already lost many children while waiting for funds,” said Saurabh Singh, co-founder and director of the Rare Disease India Foundation (RDIF).
“Treating a patient does not just save a life; it also restores hope and stability to the caregivers.” Talking to The Health Outlook, he said, CoEs across the country still lack a stable and predictable funding mechanism for long-term therapy.
Singh also questioned why families, already under extreme emotional and financial strain, should be forced to turn into or to fundraisers, racing against disease progression, online algorithms and time. He also pointed out that only families who are tech-savvy or have access to effective fundraising networks stand any real chance of mobilising such support, leaving many equally needy patients invisible and unsupported. Experts note that while the National Registry for Rare and Other Inherited Disorders (NRROID) has enrolled over 17,500 patients across India, only about 3,800 are registered on the government’s crowdfunding platform — underscoring its limited reach.
Since donations remain voluntary, Singh suggested that both public funding and private corporate social responsibility (CSR) contributions must be structured, coordinated and mandatory. Without this, he warned, deaths will continue despite available treatments.
Parents are also demanding that the government negotiate harder with pharmaceutical companies to cap drug prices for at least three years. Advocates say without price control, even public funding cannot stretch far enough. In this connection, an order by the Division Bench of the Delhi High Court on October 9, 2025 has come as a ray of hope for many in the sector particularly those with SMA.
It upheld a ruling of a single judge of the Delhi High Court, Justice Mini Pushkarna in March 2025, that allows Indian pharma firm Natco to manufacture and sell a generic version of lifesaving risdiplam, rejecting a patent infringement claim filed by Swiss drugmaker Roche.
Natco has already received approval from the Drugs Controller General of India (DCGI) and announced that it will launch the drug at comparatively cheaper than Roche’s product.
Welcoming the court order, Dr. Mayank Nilay, Associate Professor and Head of the Medical Genetics Department at the Post Graduate Institute of Child Health, Noida, said that States such as Kerala and Rajasthan are already supporting access to Risdiplam, and others could follow suit.
While Risdiplam is significantly cheaper than other therapies, he noted that it remains unaffordable for many middle-income families. The court order may help ease many families. Access is usually possible only when the manufacturer provides the drug at a lower rate or free of cost when government agencies step in to fund treatment, he said.
Dr. Nilay explained that rare diseases can occur due to marriages between close relatives, or as a result of defects in a single gene or chromosome, or from disruptions in the interaction between genes and the environment.
He also stressed the need to strengthen research and development in the public sector to ensure the availability of affordable generic medicines for patients who depend on lifelong therapy.
At the same time, Parliamentarian Praveen Khandelwal told The Health Outlook that after the current session of Parliament concludes, he plans to take up the issue more forcefully. He said he would also urge the government to allow clinical trials of medicines that are not yet approved in India but are already available abroad, a move he believes could help reduce costs and improve patient access.
