Rare disease patients seek inclusion in BJP’s manifesto for funding

Patients and caregivers have requested equal weightage and priority to all notified disease conditions under the National Policy for Rare Diseases, 2021

“Any delay in treatment poses serious risk to (rare disease) patient lives,’’ Manjit Singh, president, Lysosomal Storage Disorders Society of India, said. Photo: Facebook/LSDSS

Rare disease patients’ advocacy groups have appealed to Prime Minister Narendra Modi, and Defence Minister Rajnath Singh, the chairman of the Bharatiya Janata Party’s (BJP) manifesto committee, seeking inclusion for sustainable funding support in the party’s election manifesto.

In a two-page letter, patients and caregivers said that rare diseases, especially chronic and ultra-rare conditions, including Lysosomal Storage Disorders (LSD) such as Gaucher disease, Pompe disease, MPS I and II, Neiman Pick disease, and Fabry disease, present a unique set of challenges.

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The group noted that the National Policy for Rare Diseases, 2021 has been instrumental in providing a framework for support, leading to life-saving interventions and improved outcomes for affected individuals.

“These efforts have not only helped patients start their life-saving therapies but also provided hope and reassurance to countless families across the nation. However, the journey toward sustainable treatment support for chronic rare diseases remains a crucial next step,’’ they said in the communication, adding that it was imperative to ensure that patients with chronic conditions had access to ongoing treatments based on clinical parameters, enabling them to lead fulfilling lives.

The group has also highlighted that there is an immediate need to prioritise and ensure sustainable funding support for all notified ultra-rare diseases — conditions that are chronic but have approved therapies with excellent clinical outcomes.

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“The current budgetary provision of up to ₹50 lakh is only one-time, thereby hindering the process of saving lives for patients diagnosed with these conditions. Several patients across the Centres of Excellence have once again been put off life-saving therapies after the one-time support was exhausted,’’ the group said.

The patients and caregivers have requested equal weightage and priority to all notified disease conditions under the National Policy for Rare Diseases, 2021. “Any delay in treatment poses serious risk to patient lives,’’ Manjit Singh, national president, Lysosomal Storage Disorders Society of India, said.