Health Sector – UHC, National Health Policy, Family Planning, Health Insurance, etc.

National guidelines for clinical trials under Gene Therapy

Note4Students

From UPSC perspective, the following things are important :

Prelims level : Not Much

Mains level : Prevention of genetic diseases

In a bid to attract the pharmaceutical industry for pooling investments in drugs for treating rare diseases, the Indian Council of Medical Research (ICMR) has come up with national guidelines for gene therapy-related clinical trials.

About the guidelines

  • The document is titled as the “National Guidelines for Gene Therapy Product Development and Clinical Trials”.
  • Treatments for rare genetic diseases have long been neglected by the traditional pharmaceutical industry because of the notion that it will have uncertain or poor commercial outcomes given the smaller affected population size.
  • It aims to ensure that the gene therapies can be introduced in India and their clinical trials can be performed in an ethical, scientific and safe manner. Also, spur innovation and accelerate research for rare diseases.

What are Genetic diseases?

  • Inherited genetic diseases or rare diseases refer to medical conditions that affect a small percentage of the population but has vast, debilitating and often life threatening effects of the patients, many of whom are in the paediatric age group.
  • According to Health ministry approximately 70 million Indians suffer from some form of rare disease.
  • These include haemophilia, thalassemia, sickle-cell anaemia certain forms of muscular dystrophies, retinal dystrophies such as retinitis pigmentosa, corneal dystrophies, primary immunodeficiency (PID) in children, lysosomal storage disorders such as Pompe disease, Gaucher’s disease, haemangioma, cystic fibrosis etc.

Why need such guidelines?

  • Recognizing huge burden of genetic diseases in India there was a need to accelerate the development of advanced therapeutic options.
  • The guidelines will also serve as an important resource and roadmap for those in the field trying to develop gene and cell therapies.
  • There remain many hurdles that the rare disease fields have yet to overcome. These include:
  1. the appropriate and timely diagnosis including genetic testing and genetic counselling
  2. prohibitive costs of such gene therapies
  3. adequate insurance coverage and
  4. management practices among treating physicians

Way Forward

  • While prospects are bleak for many individuals with conditions classified as rare diseases, policies such as that proposed by the ICMR may offer hope.
  • Gene therapy may be a key avenue of research into many disorders where standard medication has shown little effect as the genetic alterations may directly impact the cause of the disorder.
  • However, for progress to be made, research into such conditions must be incentivized through funding and stakeholder support.
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