Govt to waive trials for orphan drugs

HYDERABAD:  In a significant move, the Central Drugs Standard Control Organisation (CDSCO) has decided to relax rules including a possible waiver of clinical trials for orphan drugs used in the treatment of rare diseases. Proposed as part of the New Drugs and Clinical Trials Rules, 2018, expected to be notified in March, the CDSCO intends to provide a pathway so that even with limited data, orphan drugs can be approved, with or without a trial.

In other words, a partial or full waiver of clinical trials is in the offing for specific drugs provided such tests are conducted elsewhere, particularly in advanced nations like the US or Europe. As per Indian norms, any drug, old or new, needs to go through the clinical trials phase before reaching the commercial launch stage.

“The reason is the number of patients (with rare diseases) may be 500-1000 (in India) and with such low numbers it’ll be difficult for them (clinical trials) to be economically viable. The new rules give regulatory freedom to launch orphan drugs,”  Chandrasekhar Ranga, dy director general, CDSCO, said. Orphan drugs are medicines used in the treatment of rare diseases with less than five lakh patients, but are believed to be expensive.

Speaking to media on the sidelines of BioAsia 2019 here on Tuesday, he said, the matter of approval will now be given in few weeks. Currently, as per the notification, there are  no regulatory pathways for approval of orphan drugs, and the upcoming rules will offer a constant pathway for approvals and reduce the cost and time to market the drug at affordable prices. For any new drugs, the incoming rules on clinical trials are expected to reduce the time taken for approval of clinical trials to 45 days for domestic companies and 90 days for foreign companies from the current 120 days. 

However, companies complain that seldom is the deadline adhered to, leading to delays in getting approvals. From next month, companies can proceed with the trials after 45 and 90 days even without a regulatory nod. Meanwhile, dismissing reports about falling number of clinical trials in India, he said trials have been rising over last two to three years.

‘Science needs more clinical trials’
Badhri Srinivasan from the Global drug-maker Novartis Tuesday sought participation of individuals in clinical trials to facilitate translation of science into medicines. He said a piffling 2% of patients participate in clinical research while 98% partake in trials.