Local Clinical Trial Waiver for Selected Drugs: A Double-Edged Sword

  • December 27, 2024
  • Posted by: OptimizeIAS Team
  • Category: DPN Topics
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Local Clinical Trial Waiver for Selected Drugs: A Double-Edged Sword

Sub: Sci

Sec: Health

Why in News

  • The Central Licensing Authority (CLA) recently allowed a local clinical trial waiver for five categories of new drugs sourced from six countries under Rule 101 of the New Drugs and Clinical Trials Rules (NDCTR), 2019. This move aims to expedite drug approvals in India, particularly for rare diseases and life-threatening conditions.

What is the Local Clinical Trial Waiver?

  • The waiver allows new drugs from six specified regions — the US, United Kingdom, Japan, Australia, Canada, and the European Union — to bypass local clinical trials during the approval process.
  • It applies to five categories of drugs, including:
  1. Orphan drugs for rare diseases.
  2. Gene and cellular therapy products.
  3. Drugs used during pandemics.
  4. Drugs used for special defence purposes.
  5. Drugs offering significant therapeutic advantages over existing treatments.

Benefits of the Local Clinical Trial Waiver:

  • Ensures timely access to medications, especially for rare diseases and pandemic-related drugs.
  • Reduces long waiting periods for treatment approvals.
  • Reduces bureaucratic hurdles for launching new drugs in the Indian market.
  • Encourages the introduction of innovative therapies in India.

New Drugs and Clinical Trials Rules (NDCTR), 2019:

  • The New Drugs and Clinical Trials Rules, 2019, were introduced by the Ministry of Health and Family Welfare to streamline the drug approval process and ensure the ethical conduct of clinical trials in India.
  • Reduced approval time for drugs in national interest, e.g., those required during pandemics.
  • Defines new drugs to include:
    • Drugs not marketed in India.
    • Drugs with new indications or dosage forms.
    • Drugs undergoing clinical trials for a new use or formulation.
  • Mandatory registration of ethics committees with the Central Drugs Standard Control Organization (CDSCO).
  • Ethics Committees are responsible for the approval and monitoring of clinical trials.
  • Clear provisions for compensation to trial participants in case of:
    • Adverse effects or injuries.
    • Death due to the trial.
  • Clinical Trials: Approvals must be granted within 30 days.
  • New Drugs: Approvals must be granted within 90 days.
  • Orphan drugs (for rare diseases) are exempt from clinical trials in India under certain conditions.
Rare disease in news:

Acid Sphingomyelinase Deficiency (ASMD):

  • A rare disease treatment approved by the Subject Expert Committee (CDSCO) in 2023 is yet to be included under the National Policy for Rare Diseases, 2021.
  • Acid Sphingomyelinase Deficiency (ASMD) is a rare lysosomal storage disorder caused by mutations in the SMPD1 gene.
  • The condition results in the deficiency of acid sphingomyelinase (ASM), an enzyme responsible for breaking down sphingomyelin, leading to its accumulation in cells.
  • ASMD is also referred to as Niemann-Pick Disease Types A and B.

Types of ASMD

  • Type A (Neuropathic):
  • Severe form; affects the nervous system and organs.
  • Early onset in infancy, leading to rapid neurological decline.
  • Poor prognosis; life expectancy is usually less than 3 years.
  • Type B (Non-Neuropathic):
  • Primarily affects organs like the liver, spleen, and lungs.
  • Milder form; patients may survive into adulthood.
  • Intermediate Type (A/B):
  • Symptoms of both Type A and B.

Symptoms

  • Enlarged liver and spleen (hepatosplenomegaly).
  • Breathing difficulties due to lung involvement.
  • Neurological deficits in severe forms.
  • Growth retardation and fatigue.

Diagnosis

  • Enzyme Activity Test: Measures acid sphingomyelinase activity in leukocytes or fibroblasts.
  • Genetic Testing: Confirms mutations in the SMPD1 gene.
  • Imaging techniques (e.g., MRI) to detect organ enlargement.

Treatment

  • Supportive Care: Managing symptoms like organ enlargement and respiratory issues.
  • Olipudase Alfa is under development and offers potential for Type B ASMD.
Local Clinical Trial Waiver for Selected Drugs: A Double-Edged Sword Science and tech