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Advocating for Rare Disease Patients: Priorities and Challenges Ahead

Rare disease patients and caregivers have expressed their gratitude to JP Nadda, the newly appointed Union Minister at Union Health Ministry, following the government's third consecutive term win. They commend the progress made in the previous term, particularly the introduction of the National Policy for Rare Diseases 2021.

However, they also highlight significant gaps in policy implementation that continue to impact the lives of patients, especially children with chronic rare genetic disorders such as Lysosomal Storage Disorders (LSDs). A major concern is the lack of sustainable funding and delays in utilizing allocated funds, which lead to uncertainties and hinder access to life-saving treatments provided through Centres of Excellence (CoEs).

Patients, caregivers, and advocacy groups have outlined several recommendations to address these challenges:

Build a continuum of care with sustainable funding support for Group 3a conditions: It is crucial to prioritize and ensure continuous funding for all notified ultra-rare diseases with approved therapies. The current one-time budgetary provision of up to INR 50 lakh hampers ongoing treatment for patients at CoEs.

Provide equal priority to all notified disease conditions under NPRD 2021: Despite being classified under Group 3(a), diseases like Pompe disease, Fabry disease, MPS I and II lack equal priority at CoEs, resulting in delays in crucial treatments.

Enhance accountability and efficiency: Direct CoEs to expedite fund utilization to promptly improve access to life-saving therapies. Many CoEs have not effectively utilized funds allocated by MoHFW, despite repeated appeals.

While acknowledging the government's support, particularly for children affected by rare diseases, they urge the new health minister to prioritize the needs of this often-overlooked community with the urgency they require.

They call for rare diseases to be a top priority in the government's 100-day action plan, emphasizing that this commitment will not only demonstrate governmental dedication but also catalyze crucial actions and resource allocation to enhance the lives of rare disease patients nationwide.

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