Release Rs 5 crore to AIIMS for treating children with rare diseases: Delhi High Court to Centre

A genetic disorder, DMD affects muscles and occurs in one in 3,500 to 5,000 newborns. Another genetic disorder, Gaucher, takes place due to the buildup of certain fatty substances in certain organs like the spleen and liver, which causes them to enlarge and can affect their function. As the treatment of the disease is expensive, the pleas sought directions to provide free treatment to children with rare diseases.

Justice Singh further said that each of these children may approach AIIMS Delhi or the respective Centres of Excellence closer to their locations, for the commencement of their treatment in terms of the order of December 14, 2021, wherein the court had directed AIIMS to start the treatment of these children and the Centre to bear the cost of the medicines.

During the last hearing, Justice Singh had directed Alka Sharma, Managing Director Biotechnology Industry Research Assistance Council (BIRAC) and Rajesh Gokhale or any senior official from the Department of Biotechnology (DBT), to hold a meeting with the representatives of Hanugen Therapeutics — a biopharma company which provides therapies for rare diseases, to explore the framework under which funding for clinical study for therapeutic evaluation of indigenous treatment in respect of DMD patients can be done.

However, the HC was informed that in the meeting held on February 2, there had been no progress. BIRAC’s stand was that it is mandatory for Hanugen to contribute 50% of the total project cost. Considering this, Justice Singh referred the matter to the Secretary, the Ministry of Health Family and Welfare to take a decision on clinical trials for testing the therapies for rare diseases including DMD. The HC directed Central Government Standing Counsel Kirtiman Singh to communicate previous orders of the court to the Secretary asking him to file an affidavit on this aspect. The HC also directed the ministry to depute a senior official, at least of the level of Joint Secretary, to join the Court proceedings on March 1, the next hearing.

When the court was informed that a company, M/s Sarepta, had registered for clinical trials in India for the same medicines being procured by AIIMS to treat these children and that AIIMS Delhi is one of the centres to carry out the clinical trial, the HC directed the Drugs Controller General of India (DGCI) to file an affidavit on Sarepta’s clinical trial or any other trial approved by them for DMD, Gaucher, or Hunter’s Syndrome therapies.