Issues in the treatment of ‘rare diseases’, and what the govt can do

  • October 14, 2024
  • Posted by: OptimizeIAS Team
  • Category: DPN Topics
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Issues in the treatment of ‘rare diseases’, and what the govt can do

Sub : Sci

Sec :Health

Context:

  • Recently, the Delhi High Court provided directives to enhance the availability of orphan drugs, which are medications designed for treating rare diseases.

About Rare Diseases:

  • According to WHO, rare diseases are debilitating, lifelong conditions affecting 1 or fewer people per 1,000.
  • Around 55 medical conditions including Gaucher’s disease, Lysosomal Storage Disorders (LSDs), and certain forms of muscular dystrophy are classified as rare diseases in India.
  • The National Registry for Rare and Other Inherited Disorders (NRROID) started by the Indian Council of Medical Research (ICMR) has the records of 14,472 rare disease patients in the country.
  • Therapies are available for less than 5% of rare diseases, leading to less than 1 in 10 patients receiving disease-specific care.

Categories of Rare Diseases:

In India, rare diseases are categorised into three groups based on the nature and complexity of available treatment options.

  • Group 1: Curable with one-time procedures.
  • Group 2: Require long-term, less costly treatment; documented benefits.
  • Group 3: Effective but expensive treatments, often lifelong.

Current Funding Policy:

  • National Policy for Rare Diseases (NPRD): Launched in 2021.Provides financial assistance up to ₹50 lakh for treatment at identified Centres of Excellence (CoEs).
  • Centres of Excellence: Include AIIMS (Delhi), PGIMER (Chandigarh), and SSKM Hospital (Kolkata).
  • Crowdfunding Portal: Launched for patient treatment support and donations.

Challenges in Treatment:

  • Lack of Awareness: Limited knowledge among healthcare providers and the general public.
  • High Treatment Costs: Many treatments are expensive, leading to financial burden on families.
  • Limited Research: Insufficient investment in research and development for rare diseases due to small patient populations.
  • Regulatory Hurdles: Complex approval processes for new drugs and therapies.

Cost of orphan drugs:

  • Many medicines for rare diseases are patented, making them costly due to a small market and high development expenses, which discourages pharmaceutical companies from producing them.
  • To lower prices, India could develop these drugs, but the government needs to offer incentives like tax breaks.
  • While patients importing rare disease medicines are exempt from customs duties, companies face 11% customs duty and 12% GST.

High Court ruling:

  • The Delhi High Court has set a 30-day deadline to process necessary exemptions under customs, GST, and Income Tax laws.
  • In 2019, the Department of Pharmaceuticals removed price controls on orphan drugs, but the High Court expressed concerns, saying this can’t go on.
    • If treatments or medicines for rare diseases are not available, the government can, under the Patents Act of 1970, allow a third party to manufacture them against payment of a royalty to the patent holder.
    • The government can also acquire patents to ensure the availability of medicines if the patent holder does not provide them.
  • The court highlighted the need for more research on rare diseases and stressed that working with pharmaceutical companies and boosting local production could help lower treatment costs.
Issues in the treatment of ‘rare diseases’ Science and tech