Groups urge BJP to include sustained funding support for rare disease patients in manifesto

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 Singh and 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.

Drawing the attention to the current operational challenges, the groups in the letter highlighted the immediate need for prioritising and ensuring sustainable funding support for all notified ultra-rare disease conditions that are chronic but have approved therapies with excellent clinical outcomes. The current budgetary provision of up to Rs 50 lakh is only one-time, thereby hindering the process of saving lives for patients diagnosed with these conditions, they said.

”Several patients across Centres of Excellence (CoEs) have once again been put off life-saving therapies after the one-time support got exhausted,” the groups said in their letter.

They stressed on providing equal weightage to all notified disease conditions under the NPRD-2021.

Though notified as Group 3(a) condition in the NPRD 2021, patients diagnosed with Pompe disease, Fabry disease, MPS I and II are still not getting the same priority at the CoEs, thereby delaying treatment and posing serious risk to their lives, they said.

”For the marginalised or under-served communities like rare disease patients and their families, seeing their issues addressed in a manifesto can not only instill hope for positive change and improvement in their circumstances but would also show that their voices matter and can make a difference in shaping policies,” the letter said. It also said that a mention in the election manifesto would also reflect the priorities of the BJP and would inspire confidence, mobilize support, and drive positive change on the ground, the letter said.