Rare disease patients write to new health minister, demand sustained funding & policy action

New Delhi: Patients suffering from rare genetic disorders, along with their caregivers, have penned a heartfelt appeal to JP Nadda, the newly appointed Union Minister of Health and Family Welfare.

The letter, endorsed by advocacy groups, acknowledges the strides made during the previous government’s tenure, particularly the introduction of the National Policy for Rare Diseases 2021. However, it underscores critical gaps that continue to jeopardize the lives of patients, particularly children.

Chief among their concerns is the issue of sustainable funding. Despite the policy’s promising provisions, patients with chronic ultra-rare conditions like Lysosomal Storage Disorders (LSDs) are facing uncertainty due to the inadequacy of ongoing funding. The current one-time allocation of up to INR 50 lakh per patient has proven insufficient, leaving many without access to life-saving therapies once the initial support is exhausted.

The appeal also emphasizes the unequal prioritization of certain rare diseases at the Centres of Excellence (CoEs) designated under the policy. Patients diagnosed with Pompe disease, Fabry disease, MPS I, and II, classified as Group 3(a) conditions, report delays in treatment compared to others. This disparity poses serious risks to their health and underscores the need for equal treatment prioritization across all notified conditions.

Advocates further call for enhanced accountability and efficiency within the CoEs, urging expedited processes to ensure the effective utilization of allocated funds. Many CoEs have struggled to maximize the funds provided by the Ministry of Health and Family Welfare (MoHFW), thereby limiting access to critical therapies despite repeated appeals.

While expressing gratitude for the government’s past support, especially in addressing rare diseases impacting children, advocates urge Minister JP Nadda to prioritize their community’s voice and needs with the urgency they deserve. They appeal for the inclusion of rare diseases as a top priority in the government’s 100-day action plan, emphasizing that such recognition would highlight the government’s commitment to this critical health issue and prompt swift action and resource allocation. The plea from rare disease patients and caregivers comes amidst hopes for renewed government attention under the new leadership, aiming to improve the lives of thousands affected by rare genetic disorders across India.