As part of the procedure, clinicians treat a disorder by inserting a gene into the cells of patients instead of using drugs or performing surgery.

ICMR Rules To Ensure Uniform Method Across India

The apex health research body ICMR has released national guidelines regarding the procedures to be followed for developing and performing gene therapies to tackle inherited genetic or rare diseases in India.

Gene therapy is a technique that uses genetic modification to treat or prevent ailments. As a part of the procedure, clinicians treat a disorder by inserting a gene into the cells of patients instead of using drugs or performing surgery.

National Guidelines for gene therapy product development and clinical trials document has been released to enable the treatment of diseases by gene therapy, the Indian council of medical research(ICMR) said in a statement.

The aim of the document is to ensure that gene therapies can be introduced in India and clinical trials for gene therapies can be performed in an ethical, scientific and safe manner, the ICMR said. 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 pediatric age group, it said.

Treatments for such diseases have long been neglected by the traditional pharma industry because of the notion that it will have uncertain or poor commercial outcomes given the smaller affected population size.

Recognizing the huge burden of genetic diseases in India and therefore, the need to accelerate the development of advanced therapeutic options for such conditions, this seminal document will also serve as an important resource and roadmap for those in the field trying to develop gene and cell therapies.

"We hope this will spur innovation and accelerate research into RDs in our nation. There remain many hurdles that the scientific and clinical community working in the RD fields are yet to overcome, primarily the appropriate and timely diagnosis including genetic testing and genetic counseling, prohibitive costs of coverage and management practices among treating physicians.

" Towards the goal of crossing these hurdles, the guidelines have been released to the nation, " the ICMR said.

Cumulatively, approximately 70 million Indians suffer from a rare disease. These include hemophilia, thalassemia,sickle-cell anemia 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, and many more.

For a variety of debilitating diseases caused by genetic mutations, gene therapy offers a treatment modality that cannot be provided by conventional including small molecule drugs or alternative medicines.

The government agencies recognize this problem and have been working with many groups around the country to establish rare disease policies as has been done in many western nations, to encourage the development of products intended to diagnose, manage or treat RDs as well as spread awareness and education to prevent inherited conditions, it said.

The Central Drugs Standard Control Organisation(CDSCO) in the new Drugs and Clinical Trials Rules, 2019 (NDCTR), has defined a rare disease drug or "orphan drug" as"a drug intended to treat a condition which affects not more than five lakh persons in India".

These national guidelines provide the general principles for developing gene therapy products (GTPs)for any human ailment and provides the framework for human clinical trials which must follow the established general principles of biomedical research for any human applications.

The guidelines cover all areas of GTP production, pre-clinical testing, and clinical administration, as well as long term, follow up.

 

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