Terminally ill patients in China might find new hope as the country’s food and drug administration has drafted a regulation that would expand access to unapproved drugs outside of clinical trials.
Published on Dec. 20 to solicit public feedback, the draft from the China Food and Drug Administration proposes that new medication which has yet to be approved should be available to patients in desperate need.
Also known as “compassionate use,” expanded access could make a world of difference to people with critical conditions.
“It takes a minimum of five years for a new drug to get approved and enter the market in China,” Fang Xin, the marketing director of Shanghai Hi-Tech Bioengineering Company, told Sixth Tone. “Many terminally ill patients cannot wait that long. Early intervention could help many control their diseases and ensure that their health conditions don’t deteriorate quickly.”
Expanded access aims to help patients whose lives are at risk — or whose quality of life has been severely compromised — when the approved treatment options have not been effective.
The regulation defines specific conditions under which patients could qualify for expanded access, such as when the patient does not qualify for the clinical trial of the drug in question or does not live in a participating location. Another suggested provision allows access to investigational drugs when clinical trials have concluded but market approval has not yet been granted, though existing data suggests the drug is safe and effective for eligible patients.
The United States in 1987 became the first country to institute measures for expanded access. According to 2016 data from the U.S. Food and Drug Administration, the country’s Center for Drug Evaluation and Research received 999 applications from individual patients for the compassionate use of investigational drugs. Of these, 994 were granted permission to proceed.
Following in the footsteps of the U.S., the European Union and Japan have also developed systems for expanded access.
However, unlike overseas practices, the Chinese draft regulation suggests that applicants for compassionate use should not be individual patients, but rather pharmaceutical companies, which would apply to the China Food and Drug Administration for permission to carry out expanded access. Under the Chinese system, patients would be evaluated by doctors tasked with assessing whether the benefits outweighed the risks in each case.
The public has until Jan. 15, 2018, to submit feedback on the draft regulation.
Editor: Qian Jinghua.
(Header image: Corbis-RM/VCG)