ICMR comes up with guidelines for gene therapy product development, clinical trials

NEW DELHI: With an aim to ensure better treatment options for 'rare diseases' or inherited genetic diseases such as Gaucher’s disease, haemophilia, thalassemia, sickle-cell anaemia and certain forms of muscular dystrophies, the Indian Council of Medical Reasearch (ICMR) has come up with the national guidelines for gene therapy product development and clinical trials.

The idea is to promote gene therapies in India and ensure clinical trials for gene therapies can be performed in an ethical, scientific and safe manner.

“These are modern and robust guidelines, and are pragmatic for the country. These guidelines will provide the general principles for developing gene therapy products for any human ailment along with the framework for human clinical trials,” ICMR Director General Balram Bhargava said.

Around 70 million Indians are estimated to suffer from rare diseases in India. However, experts say the data is inadequate to give away the actual burden.

Bhargava said both the Prime Minister’s Office (PMO) and NITI Aayog were keen on having these guidelines in place and pushed to fast track the process.

Though the affected population is small, such diseases are often life threatening, many of whom are in the paediatric age group. However, treatments for such diseases has long been neglected by the traditional pharma industry because of commercial unviabilty of orphan drugs. Medicines to treat these rare diseases are often also very expensive. Earlier this year, the health ministry had also written to the Department of Pharmaceuticals to cap prices of these medicines as they continue to be out of access of most patients in the country.

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. 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.

The guidelines are expected to serve as an important resource and roadmap for development of gene and cell therapies. “We hope this will spur innovation and accelerate research into rare diseases in our nation,” ICMR said.

There remain many hurdles that the scientific and clinical community working in the rare disease fields are yet to overcome. This includes 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.

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 such diseases, the apex health research body said.