Kolkata: A baby girl of only twelve months old, Asmika, whose name means beauty, has recently gone viral as a crowdfunding movement is underway for her treatment.
Asmika Das was born in the district of Nadia, West Bengal, on January 9, 2024, at Ranaghat’s Swami Vivekananda Sarani. Her parents, Lakshmi and Shuvankar Das, were married in 2022. Asmika’s birth brought immense joy to the family. However, when Asmika was four months old, her parents noticed that she did not move like other children her age. Alarmed, they sought medical advice, traveling from their district to the state capital, Kolkata, and even to Southern India. After undergoing several tests, she was diagnosed with Spinal Muscular Atrophy (SMA) Type-1, a rare and severe genetic disorder.
The High Cost of Hope: Zolgensma and Crowdfunding Challenges
All the excitement turned into despair. Asmika suffers from SMA Type-1, the most severe form of the disease, which manifests in infancy with symptoms such as muscle weakness, poor muscle tone, and difficulty breathing and swallowing. Without early treatment, it often leads to life-threatening respiratory complications. SMA is a rare genetic condition that affects motor neurons—nerve cells that control voluntary muscle movements.
Gene replacement therapy is the only viable treatment to save Asmika. A single dose of Zolgensma, a one-time-only gene therapy for children under two years of age with SMA, costs $2.1 million, making it the world’s most expensive drug. Manufactured by Novartis, Zolgensma is not available in India and must be imported from abroad, taking nearly 20 days to reach India after an order is placed.
For Asmika’s lower-middle-class family, raising such a massive amount is impossible. While some government assistance is available, it is insufficient. Crowdfunding has become their only hope, but it is a time-consuming and arduous process.
Two years ago, on January 7, 2023, another child, Nirvaan, the son of Sarang Menon and Adithi, was diagnosed with SMA Type-2. The family, originally from Kerala but settled in Mumbai, also required a similar amount for treatment. They launched campaigns on crowdfunding platforms Milap and ImpactGuru. Fortunately, an anonymous donor contributed ₹11 crores to their Milap account. However, in Asmika’s case, raising such funds seems far more challenging.
Rare Diseases in India: Policy Gaps and High Stakes
Rare diseases are health conditions with low prevalence, affecting a small portion of the population compared to more common illnesses. Globally, an estimated 6,000 to 8,000 rare diseases exist, with approximately 80% of patients affected by only 350 of these. Rare diseases are lifelong conditions, and around 95% of them have no approved treatments. Where treatments are available, they are often prohibitively expensive.
To address this, the Ministry of Health & Family Welfare (GOI) launched the National Policy for Rare Diseases (NPRD) in March 2021. The policy classifies rare diseases into three groups:
1. Group 1: Treatable through one-time curative treatments.
2. Group 2: Requiring long-term treatment at a relatively lower cost.
3. Group 3: Having definitive treatment options that are prohibitively expensive and require lifelong therapy.
The policy includes 63 rare diseases, such as Lysosomal Storage Disorders, Fanconi Anemia, and Gaucher Disease. Under the NPRD, the government has promised up to ₹50 lakhs per patient for treatment at designated Centers of Excellence (CoEs) for rare diseases. There are 12 such CoEs across India, including AIIMS New Delhi, PGIMER Chandigarh, and IPGMER Kolkata.
Despite these measures, gaps remain. According to a study in the Jindal Journal of Public Policy, authored by Dhruvnil Jakasaniya and Rutu Tekwani, the NPRD falls short in making treatments accessible and affordable. Issues include inadequate data, poor communication frameworks among stakeholders, and insufficient incentives for domestic drug manufacturers to invest in research and development.
Moreover, the government’s promise of digital pooled funds is not yet fully operational. Experts suggest utilizing corporate sector CSR funds and public-private partnerships to enhance funding and strengthen CoEs.
Asmika’s case is a grim reminder of these gaps. If the Zolgensma injection is not administered before she turns 15 months old, her chances of survival will diminish significantly. In May 2023, Delhi High Court Justice Prathiba M. Singh emphasized the need for urgent coordination between the medical community, therapy providers, and government agencies.
A public health expert, speaking anonymously, stated: “The government needs to move from sympathy to empathy for rare disease patients. They must reach out to potential donors, such as PSUs with large CSR funds, and implement a more strategic approach to support these families.”
Unfortunately, many healthcare professionals, activists, and foundations remain reluctant to comment publicly on this issue. Despite its critical importance, rare diseases continue to be a largely neglected area in India’s healthcare system.
In November 2024, India Today reported that one baby boy Din Muhammed managed to get Rs 16 crore through crowdfunding and was successfully administered the life-saving drug.
