• UGI USER
  • Mar 01, 2024
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INDIA’S BATTLE AGAINST RARE DISEASES

Syllabus:

  • GS 2: Issues Relating to Development and Management of Social Sector/Services relating to Health.

Why in the News?

  • The recent tragic demise of 19-year-old actress Suhani Bhatnagar due to dermatomyositis, a rare inflammatory muscle disorder, underscores the challenges faced by individuals with rare diseases.
  • This poignant incident coincided with Rare Disease Day, observed on the last day of February annually, dedicated to raising awareness about the struggles of those affected by uncommon medical conditions.
Source: ToI

Rare Disease Landscape in India

  • According to the World Health Organization, rare diseases impact 1 or less per 1,000 population.
  • India bears a significant burden, constituting one-third of global rare disease cases, encompassing over 450 identified conditions.
  • Despite the prevalence, rare diseases, affecting approximately 8-10 crore Indians, remain largely neglected, especially among children.
Understanding Rare Disease:

·   Definition: 

Broadly, a ‘rare disease’ is defined as a health condition of low prevalence that affects a small number of people when compared with other prevalent diseases in the general population. Many cases of rare diseases may be serious, chronic and life-threatening.

·   These diseases have differing definitions in various countries and range from those that are prevalent in 1 in 10,000 of the population to 6 per 10,000.

·   There are 6,000-8,000 classified rare diseases, but less than 5% have therapies available to treat them.

·   Example: Lysosomal Storage Disorders (LSD), Pompe disease, cystic fibrosis, muscular dystrophy, spina bifida, haemophilia etc.

·   About 95% rare diseases have no approved treatment and less than 1 in 10 patients receive disease-specific treatment.

·   80% of these diseases have genetic origins.

Policy Initiatives

  • The Ministry of Health and Family Welfare formulated a national policy for rare diseases in 2017.
  • However, implementation challenges led to its withdrawal in 2018.
  • The National Policy for Rare Diseases (NPRD) was introduced in 2021, addressing some concerns but facing persistent issues.

National Rare Disease Policy 2021:

  • Objective:
  • Enhance focus on indigenous research and local production of medications.
  • Reduce the treatment expenses associated with rare diseases.
  • Implement early screening and detection measures for the prevention of rare diseases.
  • Key Provisions:

Categorization:

  • Group 1: Conditions treatable with one-time curative interventions.
  • Group 2: Disorders necessitating long-term or lifelong therapies.
  • Group 3: Diseases with available treatments but challenges such as high costs and lifelong therapy requirements.

Financial Assistance:

  • Financial aid of up to Rs. 50 lakhs for patients suffering from any category of rare diseases.
  • Additional support of up to Rs. 20 lakhs under Rashtriya Arogya Nidhi for Group 1 rare diseases.
  • Rashtriya Arogya Nidhi extends assistance to patients irrespective of their financial status.
  • Introduction of a voluntary crowdfunding platform for individual and corporate contributions towards treatment costs.

Centres of Excellence:

  • Designation of eight healthcare facilities as ‘Centres of Excellence’ for rare diseases.
  • Allocation of one-time financial aid of up to Rs. 5 crore for enhancing diagnostic infrastructure.

National Registry:

  • Establishment of a comprehensive hospital-based registry to document rare diseases.
  • Facilitation of comprehensive data collection and standardization for research and developmental purposes.

Challenges:

  • Timely diagnosis is crucial, yet rare disease patients often endure an average of seven years before receiving a diagnosis.
  • Lack of awareness among physicians necessitates enhanced training to improve diagnostic accuracy.
  • Expectant mothers with a family history of rare diseases require mandatory pre-natal screening.
  • Less than 50% of the identified rare diseases in India have available treatments.
  • Inadequate sustainable funding for patients with Group 3 disorders.
  • High costs associated with medications for rare diseases.
  • Limited availability of both global and domestic manufacturers producing drugs for rare diseases.
  • Only 20 rare diseases are covered by approved treatments from the Drugs Controller General of India.
  • Treatment centers, known as Centers of Excellence (CoEs), are few, unevenly distributed, and inadequately coordinated.
  • Budgetary allocations for rare diseases in 2023-2024 stand at ₹93 crore, raising concerns about the adequacy of funds for lifelong management.
  • Financial assistance to CoEs remains underutilized, with significant unspent allocations.
  • The NPRD has suggested crowdfunding for rare disease patients, exemplifying an abdication of governmental responsibility.
  • The crowdfunding portal, with over 1,400 registered patients, has garnered less than ₹3 lakh in three years, questioning its sustainability.

Way Forward

  • Establish a standard definition of rare diseases to guide policy and awareness efforts.
  • Increase budgetary allocations for drug development, therapy, and CoEs.
  • Enhance the number of CoEs and ensure better coordination and responsible fund utilization.
  • Encourage state governments to introduce social assistance programs and develop satellite centers under CoEs.
  • Engage public and private companies in funding rare disease initiatives.
  • Leverage CSR initiatives and partnerships to bridge funding gaps.
  • Addressing Drug Prices and Availability
  • Address the issue of exorbitant drug prices and limited availability.
  • Waive GST on life-saving drugs and incentivize domestic manufacturers under the Production-Linked Incentive Scheme.
  • Explore options like repurposed drugs and bulk imports to make treatments more accessible.

Conclusion

While the government has taken steps to acknowledge rare diseases, a comprehensive and sustained effort is required. Framing a clear definition, augmenting funding, and fostering collaboration between stakeholders can pave the way for a more inclusive approach. Additionally, addressing drug pricing and availability is critical to ensuring that no one is left behind in the battle against rare diseases. The lessons from Rare Disease Day remind us that collective efforts can find solutions even to the most challenging problems.

Source:

https://www.thehindu.com/opinion/op-ed/indias-fight-against-rare-diseases/article67895581.ece/amp/

Mains Practice Question:

Rare diseases continue to pose significant challenges to India’s healthcare system, with millions of individuals grappling with limited treatment options and inadequate support. In light of this, critically analyze the National Rare Disease Policy 2021 and its effectiveness in addressing the multifaceted issues surrounding rare diseases in India.