ICMR issues guidelines for gene therapy in India

Gene therapy is a technique that uses genetic modifications 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 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" (RDs) 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, 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 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, the statement said.

"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 counselling, prohibitive costs of such gene therapies, adequate insurance 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 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.

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

The government agencies recognise 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 Organization (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 statement said.

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

Gene Therapy Products (GTP) are defined as any entity which includes a nucleic acid component being delivered by various means for therapeutic benefit to patients, it said.

This term GTP is applicable to entities that are used for, but not limited to, gene augmentation, gene editing, gene silencing, synthetic or chimeric gene augmentation, etc.

All GTP research pertaining to human applications must be conducted within the principles of these guidelines in a scientific and ethical manner following all regulatory requirements as laid down for all forms of GTP.

These national guidelines apply to all stakeholders in the field of gene therapy including researchers, clinicians, oversight/regulatory committees, industry, patient support groups and any others involved in GTP development or their application in humans and their derivatives, the statement said.

In the past three years, several gene and cell therapy products have received approval for patient use from the US and EU regulators, paving the way for development of therapies for a variety of previously untreatable disorders, the statement said.

Worldwide, the market for such treatments for rare diseases is predicted to grow at a compound annual growth rate (CAGR) of 11.3 per cent from 2018 to 2024 and predicted to reach revenues of more than USD 250 billion.

In most countries, such policies for development of gene and cell therapy products is also accompanied by Research and Development support avenues, clinical trial pathways and education and awareness amongst the clinical community.