Don’t delay treatment due to lack of long-term clinical trials: Delhi HC

The AIIMS committee also said that there is no evidence to suggest that the drug would stop the disease from progressing, and that long-term outcomes are not known.

The court has been hearing a matter related to the issue of children suffering from rare diseases.

A genetic disorder, DMD affects the muscles, leading to muscle wasting that gets worse over time. DMD occurs primarily among males, though in rare cases may affect females, according to National Institutes of Health, the US’s medical research agency.

In the order, Justice Yashwant Varma said there is admittedly a dearth of authoritative research material in respect of such illnesses. “That, however, should not detract from the right of patients to undergo the treatment options which are currently available and are being administered to similar patients across the globe even if they be viewed as experimental therapies,” the court said.

After the court was told that financial support for patients suffering from rare diseases has been increased from Rs 20 lakh to Rs 50 lakh, the court told AIIMS to forward its proposal to the Union Health Ministry for commencement of treatment to children suffering from rare diseases.

The court also directed the Department of Biotechnology to explore and file a response disclosing “other generic norms of treatment that may be under development and may warrant consideration in respect of children suffering from rare diseases in this batch of writ petitions”.