Thiruvanathapuram: Kerala, the first state in the country to provide high-cost treatment free of charge for certain rare diseases for children, is set to expand coverage to all patients under the age of 18, senior state authorities told ThePrint.
The initiative, soft-launched in 2022 to provide life-saving drugs free of cost to children with Spinal Muscular Atrophy (SMA), was rolled out as the Kerala United Against Rare Diseases (KARE) programme last year, offering access to approved medicines and specialised treatment for children with SMA under the age of 12.
Under the KARE project, the state procures risdiplam from Swiss pharma giant Roche, one of the three drugs available to treat the condition, at a negotiated but undisclosed price, far below the market cost of Rs 72 lakh annually for the first two years and Rs 56 lakh annually from the third year.
Kerala Health Minister Veena George said the government was now set to offer the medicine to all up to the age of 18 years. “We believe that no patient should be deprived of an opportunity to live a healthy, dignified life, however complex or rare the disease may be,” George told ThePrint. “We are proud of being the first Indian state to set up a sustainable and innovative model of care for patients with rare conditions.”
SMA is a rare and often fatal genetic neurodegenerative condition—depending on the severity—that affects muscles used for movement, and patients need lifelong therapy, which can be prohibitively expensive.
Risdiplam, available under the brand name Evrysdi, is most effective if administered at birth or within the first two years of age, but older patients can also benefit as it arrests the progression of muscle degeneration. The drug is a survival motor neuron-2 (SMN2) mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.
Widening coverage
Building on the SMA model, KARE has been expanded to other rare conditions, including Lysosomal Storage Disorders (LSDs), such as Gaucher and Pompe diseases, as well as Growth Hormone Deficiency in paediatric patients requiring long-term hormone therapy.
Aimed at preventing and providing comprehensive care to children suffering from rare diseases, the project focuses on early detection, treatment access and providing financial assistance through government funding, public-private partnerships, corporate social responsibility (CSR) initiatives and crowdfunding.
More than 100 patients have benefited so far and the state government is preparing a registry of children with rare diseases.
According to World Health Organization (WHO) estimates, rare diseases affect fewer than 1 in 1,000 people, but collectively impact about 6-8 percent of the population in any country. Nearly 80 percent of these diseases are genetic.
Of nearly 7,000 rare diseases identified, drugs for less than 5 percent are available. Many are unaffordable for most families, often forcing people in countries like India to resort to measures like crowdfunding.
As part of its National Policy for Rare Diseases (NPRD), the health ministry in 2021 announced a one-time financial assistance of Rs 50 lakh for certain categories of rare diseases.
It was also decided that all rare disease patients in India would be eligible for this one-time grant, provided the special committees at the hospitals designated as Centres of Excellence (COEs) approved their applications.
But multiple medical practitioners from the state ThePrint spoke to concede that COEs are struggling to implement the initiative and it has not impacted a significant number of patients so far. Also, the beneficiaries in most cases are only young children or those who need comparatively less expensive drugs, as they are prioritised for treatment.
Kerala’s unique experiment
ThePrint had earlier reported that the Union health ministry is working on a major initiative to procure in bulk high-value drugs for some rare diseases—which can be life-threatening and chronic, with prohibitively expensive medicines—at an estimated cost of Rs 900 crore.
The plan followed a nudge by the Delhi High Court to ensure that patients with rare treatable diseases are not denied treatment and to fast implement the Centre’s rare disease policy of 2021.
But this initiative remains in limbo, mainly on account of financial hurdles.
Kerala, however, has taken the lead in forging collaborations with patient advocacy groups like CureSMA to understand patients’ needs as well as industry partners like Roche to secure better pricing and make treatment accessible to more patients, said Dr Rahul UR, state nodal officer for child health and rare diseases.
The authorities did not reveal the purchase price of risdiplam under KARE, citing a confidentiality clause, but stressed that it was significantly lower than the market price.
For the first time in the country, a hub-and-spoke model was implemented in the treatment of rare diseases and the state also set up an SMA patient registry for better monitoring and improved patient care, authorities said.
Independent experts welcomed the initiative, saying that Kerala’s pooled procurement initiative was a commendable and pragmatic step that leveraged economies of scale, as bulk negotiations can cut costs, making treatment viable.
“It creates a framework for improving patient access, reducing prices, and ensuring the rational use of public funds for rare diseases,” said Chetali Rao, a legal researcher with Third World Network, an international non-profit that works on development issues.
Rao, however, also noted that many rare disease drugs are patent-protected and available only from sole global suppliers, reducing bargaining power and leaving access vulnerable to negotiating prices.
In such cases, pooled procurement alone cannot secure big discounts, and the high cost of rare disease therapies that can reach crores a year, with certain drugs like risdiplam costing Rs 72 lakh a year, can strain state budgets if beneficiary numbers increase.
Funding mostly relies on state budgets and the central National Policy of Rare Disease grants, but a Rs 50 lakh limit per patient puts a significant constraint on the amount a patient can receive.
For example, risdiplam from Roche is priced at Rs 6.2 lakh per bottle, and under the NPRD, the grant can barely cover eight months for an SMA patient. However, the same drug from an Indian generic manufacturer, if available, could cover the same patient for many years.
Without parallel reforms such as local manufacturing, compulsory licensing (when the government ends a drug’s patent under exceptional circumstances before patent expiry), and broader central government funding, state-driven pooled procurement alone may not sustainably widen access, Rao said.
She said pooled procurement at the national level and then supplied to the Centres of Excellence across the country would have been a better option. “National-level pooling and cost-sharing with the NPRD would have yielded better outcomes,” she added.
(Edited by Sugita Katyal)