Patients with rare diseases in India are still waiting for change, days after the Delhi High Court’s order to the Centre to create a ‘National Fund’ for their treatment. The National Policy for Rare Diseases (NPRD), launched in 2021, aims to provide financial support to patients suffering from rare diseases ¹. However, the implementation of this policy has been slow, and many patients are yet to receive the necessary treatment.

Five out of 12 Centres of Excellence (CoEs) formed to treat patients suffering from rare diseases have utilized less than 35% of the funds allotted to them by the Centre over the last three years ². This has resulted in a significant delay in the treatment of patients with Group 3a conditions, which include diseases such as Lysosomal Storage Disorders (LSD’s) that require lifelong therapy.

There are currently around 454 eligible rare disease patients with LSD’s in India, but only 98 are receiving government-approved treatment ². The delay in the commencement of treatment has led to the loss of 28 lives in the last few months alone. Patients and advocacy groups are urging the government to direct CoEs to ensure efficient utilization of funds and expedite the treatment process.

The Ministry of Health and Family Welfare has identified 63 rare diseases under the NPRD and provides financial support of up to Rs. 50 lakhs per patient for treatment at notified CoEs ¹. However, the lack of awareness and inadequate infrastructure remain significant challenges in the diagnosis and treatment of rare diseases.

The government must take immediate action to address these issues and ensure that patients with rare diseases receive the necessary treatment and support. This includes increasing funding, improving infrastructure, and streamlining the treatment process to prevent further delays and loss of life.