New set of rules soon on orphan drugs, trials

The Asian Age.  | Archak Sengupta

Business, In Other News

In the United States, any disease that has less than 2.5 lakh patients is classified as orphan.

Orphan drugs are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases that are rare.

Hyderabad: In a bid to quicken the approval process and accessibility of drugs in the country, the Union government plans to introduce a new set of rules in the coming few weeks, according to an official of the Central Drugs Standard Control Organisation (CDSCO).

India, which first issued a set of guidelines for biosimilars back in 2012, will revise the guidelines again to include orphan drugs and reduce the time needed for approval of clinical trials.

“The new rules, which will substitute the existing rules, have several amendments and are expected in a few weeks,” Ranga Chandrashekar, Deputy Drugs Controller (India), CDSCO, told reporters at the sidelines of the BioAsia expo here in Hyderabad.

Mr Ranga added, “One of the amendments is in regard to the orphan drugs. Previously, there was no definition of orphan drugs. The new rules will define orphan drugs and create a clear, transparent regulatory pathway for approval of orphan drugs. It will also allow approval of drugs with limited data.”

Orphan drugs are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases that are rare.

These drugs are called “orphan” because under normal market conditions the pharmaceutical industry has little interest in developing and marketing such products as these are intended for a small number of patients.

In the United States, any disease that has less than 2.5 lakh patients is classified as orphan. Thanks to India's population, the new rules may set the bar as high as 5 lakh.

Despite orphan drugs being a good business opportunity for Indian companies, they tend to avoid manufacturing them, as it does not make sense for them to run the costly clinical trials for such a small number of patients.

Mr Chandrashekar said companies developing orphan drugs could get partial and full waiver of clinical trials, depending on the drug.

“There will be abridged pathway to approve orphan drugs that reduces the cost and time for the drug to reach the market at affordable cost," he said.

The new rules, to cut costs, will allow drugs that are already approved in well-regulated regions such as in the US or Europe to improve accessibility. However, Mr Chandrashekar was quick to remind that Indian companies must adhere to the intellectual property rights.

The rules will also cut down on the time period for approval of clinical trials. Mr Chandrashekar said, “The timeline will be fixed in the new rules. If the regulator fails to provide the necessary approval within the fixed period, it will be deemed approved for clinical trials. Currently, it takes about four months for the process. According to the new rules, if the drug is discovered in India, it will be given approval in 45 days. For others, it'll be 90 days.” At present, such approval takes 120 days.

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