Patient Advocacy Group Urges Govt Action on INR 974 Cr Rare Disease Fund

This appeal was made during a virtual press conference held to discuss treatment challenges, state-level models, and other critical concerns.

As the deadline to establish an INR 974 Cr national fund for combating rare diseases nears, CureSMA India, a patient advocacy organization for Spinal Muscular Atrophy (SMA), has urged the Indian government to initiate the procurement process within the court-mandated 45-day period.

This appeal was made during a virtual press conference held to discuss treatment challenges, state-level models, and other critical concerns.

Archana Panda, founder of CureSMA, expressed optimism about the new budget allocation for rare disease treatment.

“This is a new lease on life for SMA patients. SMA is a progressive disease, and every day of delay denies our warriors their right to good health. Life-saving medicines can halt disease progression and preserve essential functions in SMA patients,” she said.

SMA, a severe genetic neuromuscular disease, affects basic physical abilities such as walking, eating, and breathing.

Recognized as a rare disease under India’s National Policy for Rare Diseases (NPRD), SMA falls under Group 3b, requiring long-term, high-cost treatment.

Expanding Care Infrastructure

Currently, India has 12 Centers of Excellence (CoEs) where individuals with rare diseases can seek financial aid under existing government schemes.

However, CureSMA emphasized the need for multidisciplinary care centers to improve patient outcomes. Moumita Ghosh, a representative from Kolkata, stressed that SMA management extends beyond medication.

“An integrated care model, including physical therapy and psychological support, is vital for long-term benefits,” she said.

The group also highlighted the success of Kerala’s state-specific KARE program, which offers free access to life-saving therapies and holistic care for SMA patients, including adults.

The advocacy group called for replicating this model nationwide by utilizing the NPRD funds.

High Court’s Mandate

Last month, the Delhi High Court directed the Union government to create a national fund for rare diseases and allocate INR 974 Cr for 2024-25 and 2025-26, based on the recommendations of the National Rare Diseases Committee (NRDC).

The court also criticized the existing INR 50 lakh cap for treatment under the NPRD, deeming it inadequate for some rare diseases.

“The Centre must reconsider the upper limit fixed under the policy,” the court stated, emphasizing the financial strain on families battling rare diseases like SMA.

High Costs of SMA Treatment

Globally, three drugs are approved for SMA treatment: Spinraza (Nusinersen), Zolgensma, and Risdiplam.

In India, only Risdiplam, an oral medication developed by Swiss pharmaceutical company Roche, is available. However, its cost remains prohibitive at INR 7-8 lakh per month per patient.

The advocacy group reiterated the urgency of expediting fund allocation and procurement processes to provide timely access to life-saving treatments.

CureSMA India concluded the virtual meeting by emphasizing the critical need for a structured, nationwide approach to rare disease management.

From creating more CoEs to adopting holistic care models, the group called on the government to address the unmet needs of patients with rare diseases such as SMA.

“Delays in implementing policies not only deny patients their basic rights but also increase the burden of disease progression. Immediate action is essential to ensure better outcomes for our SMA warriors,” said Panda.

The establishment of a national rare disease fund, coupled with policy revisions and state-level models, could mark a significant step forward in addressing the challenges faced by India’s rare disease community.

Stay tuned for more such updates on Digital Health News.