Rare disease patients in crisis due to delay in implementation of national policy

Why in the NEWS?

• Delay in implementation of India's rare disease policy has left many people, including children, in a life-or-death crisis.

Key Points:

• Rare disease patients in India face delay in implementation of NPRD 2021.
• Advocacy groups urge PM Modi for immediate intervention.
• Children's lives at risk due to lack of treatment.

What will you read next in this topic?

1. National Rare Diseases Policy (NPRD) 2021: Delay in implementation leads to crisis for patients
2. Key demands of advocacy groups:
3. National Rare Diseases Policy (NPRD) 2021
4. Key features of NPRD 2021:
5. Centers of Excellence (COE):
6. Issues and controversies related to NPRD 2021:

National Rare Diseases Policy (NPRD) 2021: Delay in implementation leads to crisis for patients

Critical condition of patients:

• Hundreds of patients, especially children, are in a life-and-death situation due to the incomplete implementation of the Rare Diseases Policy (NPRD) 2021.
• Without financial support, treatment of many patients has been disrupted.

Appeal from advocacy groups and families:

• Patient groups wrote open letters to Prime Minister Narendra Modi and Health Minister Jagat Prakash Nadda.
• They expressed concern over the stalled funding and bureaucratic hurdles of the Rs 974 crore National Rare Diseases Programme.

Severity of rare diseases:

• 30% of children suffering from rare genetic disorders, if left without treatment, do not survive till the age of five.
• The NPRD 2021 policy was made to provide relief to these patients, but the situation worsened due to the delay.

Status of treatment in big hospitals:

• The condition of patients undergoing treatment in hospitals like AIIMS Delhi, IGICH Bangalore and IPGMER Kolkata is deteriorating.
• The one-time financial assistance of Rs 50 lakh under NPRD 2021 has proved inadequate for rare diseases.

Ignoring court orders:

• On October 4, 2024, the Delhi High Court ordered the Ministry of Health to immediately release funds for rare diseases.
• A national fund of Rs 974 crore was directed to be created for the financial years 2024-25 and 2025-26.
• Despite this, no concrete action has been taken by the government yet.

Key demands of advocacy groups:

Sustainable funding for life:

• Implement a long-term financial assistance system by removing the limit of Rs 50 lakh.

Immediate access to treatment:

• Centers of Excellence (COE) should be allocated funds as soon as possible.
• Seamless care should be ensured to patients by simplifying administrative processes.

Effective implementation of policy:

• NPRD 2021 should be implemented as soon as possible so that all rare disease patients can get timely treatment.

National Rare Diseases Policy (NPRD) 2021

Objective of the Policy:

• The National Policy for Rare Diseases (NPRD) 2021 was created to provide affordable and accessible medical facilities to patients suffering from rare diseases. 
• The aim of this policy is to promote patient support, awareness and research.

What are rare diseases?

• Rare diseases are diseases that affect very few people but the cost of their treatment is very high.
• These mostly include genetic disorders, such as Gaucher, Pompe, Fabry, MPS, thalassemia, haemophilia etc.
• There is no fixed definition of rare diseases globally, but in India, diseases affecting less than 1 person in 10,000 are considered rare diseases.

Key features of NPRD 2021:

Rare diseases were divided into three categories:

• Group 1: Cure is possible and once cured, the patient can lead a normal life.
• Group 2: Long-term treatment is required, but it can be managed.
• Group 3: Treatment is very expensive and requires long-term medical care.

Financial assistance:

• The central government provides one-time financial assistance of up to Rs 50 lakh, but this assistance is limited to only a few diseases.
• This assistance is disbursed through the Prime Minister's National Relief Fund (PM CARES Fund) and state governments.

Centers of Excellence (COE):

• Government of India has identified 12 institutions which are actively managing patients suffering from rare diseases. These institutes have been designated as Centres of Excellence (CoE).
  • All India Institute of Medical Sciences, New Delhi
  • Maulana Azad Medical College, New Delhi
  • Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow
  • Post Graduate Institute of Medical Education and Research, Chandigarh 
  • CDFD with Nizam Institute of Medical Sciences, Secunderabad 
  • King Edward Memorial Hospital, Mumbai 
  • Institute of Post- Graduate Medical Education and Research. Kolkata
  • Center For Human Genetics (CHG) with Indira Gandhi Hospital, Bangalore
  • All India Institute of Medical Sciences, Jodhpur
  • Institute Of Child Health and Hospital for Children, Chennai 
  • Government Medical College, Thiruvananthapuram
  • All India Institute of Medical Sciences, Bhopal
• Diagnosis, research and treatment facilities for rare diseases were to be provided at these COE centers.

Drug research and development:

• Indigenous research and development (R&D) was encouraged to make expensive medicines for rare diseases affordable.
• The government encouraged Indian companies to develop affordable medicines for the treatment of rare diseases under the Make in India initiative.

Issues and controversies related to NPRD 2021:

Inadequate financial support:

• The limit of Rs 50 lakh proved to be inadequate for chronic (long-term) and expensive diseases.
• The annual cost of treatment of diseases like Gaucher, Pompe, Fabry and MPS can range from Rs 1 to Rs 3 crore.

Delay in implementation:

• Many patients are not getting timely treatment due to bureaucratic hurdles and lack of funds in implementing the policy.
• A shortage of essential medicines was also observed at COE centers.