Archive · 1 Jun 2025
Rare Disease Patients Struggle Despite Government Policies
Imagine kids with rare diseases needing special medicine to stay healthy.
The government made a plan to help, but not everyone is getting the help they need.
Some families can't afford the medicine, and some kids are getting very sick.
The High Court told the government to give more money, but the government is fighting this decision.
Doctors are trying to help, but they also need money to give treatment.
The Lysosomal Storage Disorders Support Society is asking the government to act fast.
While some patients are getting treated, many still need help.
The lack of funds causes significant pain for the patients and families.
Over 300 patients with rare diseases, including LSDs, lack essential medical care despite government policies.
The Delhi High Court directed the Ministry of Health to extend funding, but the Ministry challenged this in the Supreme Court.
The Lysosomal Storage Disorders Support Society (LSDSS) reports that many patients have died while awaiting treatment.
A national fund for rare diseases was mandated but remains pending.
Officials at MAMC report that treatment allocation follows eligibility standards and considers clinical effectiveness of therapies.
- Who
- Patients with rare diseases, especially those with Lysosomal Storage Disorders (LSDs), government officials, and medical professionals.
- What
- Patients with rare diseases are struggling to access life-saving treatment due to funding limitations and bureaucratic hurdles.
- When
- Since 2021 when the NPRD was established and ongoing through October 2024.
- Where
- India, with a focus on Delhi
- Why
- Limited funding, bureaucratic challenges, and delays in treatment implementation undermine the goal of the NPRD and are caused by the ministry's challenges to providing funds and other factors like prioritizing cases to determine who benefits the most from treatments.
LSDSS/Patient Families
Government/MAMC
Adequacy of Funding and Treatment Access
LSDSS/Patient Families
The current funding and treatment access are insufficient and fail to meet the needs of rare disease patients.
Government/MAMC
Treatment allocation follows eligibility standards, prioritizing those with a higher likelihood of benefit, and the government has provided substantial support.
- Untreated Patients
- Over 300
- Deaths Since 2022
- Approximately 50
- One-time Financial Support Limit
- Rs 50 lakh
- National Fund Mandated
- Rs 974 crore
- MAMC Patients Currently Receiving ERT
- 10 out of 28