Include funding support for rare disease in Poll Promises: Advocacy group to PM

Advocacy groups for rare disease patients have urged Prime Minister Narendra Modi and BJP manifesto committee head Rajnath Singh for including sustainable funding support for such patients in the party’s Lok Sabha poll promises.

The appeal has been made by the Lysosomal Storage Disorders Support Society (LSDSS), the Rare Diseases India Foundation (RDIF) and the Niemann Pick Disease Charitable Trust (NPDCT).

In a letter to Mr Singh and Mr Modi, they said for rare disease patients and their families, “seeing their issues addressed in a manifesto” will “instill hope for positive change and improvement in their circumstances”. It will also show that their voices matter and can make a difference in shaping policies, the groups said.

The National Policy for Rare Diseases-2021 (NPRD) has been instrumental in providing a framework for support, leading to life-saving interventions, and in improving outcomes for affected individuals, they said as they acknowledged the government’s efforts in addressing the unmet needs of rare disease patients.

Rare diseases, especially chronic and ultra-rare conditions such as Lysosomal Storage Disorders (LSDs) like Gaucher disease, Pompe disease, Mucopolysaccharidosis (MPS) type I and II, Neimann Pick disease and Fabry disease, present a unique set of challenges, they said.

“The government’s commitment to rare disease patients is evident through its transformative measures such as the provisioning of funding support for group-3 conditions, in particular. These efforts have not only helped patients start their life-saving therapies but also provided hope and reassurance to countless families across the nation,” the letter said. However, the journey towards sustainable treatment support for chronic-rare diseases remains a crucial next step, it said, underlining that it is imperative to ensure that patients with chronic conditions have access to ongoing treatment based on clinical parameters, enabling them to lead fulfilling lives.