Delhi High Court says rare diseases consortium to recommend clinical trials funding

The Delhi High Court on Monday directed the National Consortium for Research, Development, and Therapeutics for Rare Diseases to meet and recommend funding of clinical trials for the rare disease Duchenne muscular dystrophy (DMD).

The consortium, which must hold the meeting by March 26, is free to invite any other organization or individual that it thinks necessary to be required for making the suggestions, which must be comprehensive, a single-judge bench of Justice Pratibha M. Singh said.

The court further instructed the consortium to take into account the age of youngsters whose lives would be drastically shortened if treatment is not started, as well as any potential costs associated with providing the necessary medications.

The consortium can discuss any negotiations or agreements to be made with firms that already have approved medications for rare diseases in India, as well as the likelihood and viability of pursuing indigenous therapies in the trials that have already received approval, it added.

Justice Singh said the consortium will next present its suggestions to the court regarding funding for the clinical trials as well as how a previous court order is to be carried out.

The court was informed at the hearing that four individuals have been determined to be qualified for inclusion in the clinical trials and that on March 9, they would undergo physical examinations and other tests.

According to a doctor from AIIMS, the parents of the children would also be informed about the specifics of the studies, which might begin if they consent.

The doctor informed the court that "M/s Sarepta" is conducting a clinical trial here with a small number of patients, one of whom is the petitioner and the company has now requested permission from the authorities to enroll additional patients.

The judge requested that AIIMS submit a status report on the treatment of these children and stated that the other Centres for Excellence (CoE) could also be consulted at the consortium meeting to determine the estimated number of candidates who will need medications for the treatment of rare diseases across the nation.

Children with several uncommon disorders, including DMD and mucopolysaccharidosis II (MPS II), are the petitioners before the court and since the treatment for these ailments is quite expensive, they have requested directions to the Centre to receive continuous and free care.

On March 1, the HC pulled up the Centre, the AIIMS, and the petitioners' counsel for failing to notify the court of the start of clinical trials.

Expressing its displeasure, the court stated: "The court was 'kept in dark' by all the parties, including the petitioners, about an ongoing trial for DMD by Sarepta Therapeutics." (KB/IANS)