Nature reported that two children died after receiving gene-editing treatments in separate clinical trials in China, putting a spotlight on the country's fast-track trial pathway. Investigator-initiated trials are a popular way for researchers and biotech companies to test new therapies far faster than standard drug-approval systems.
The National Medical Products Administration supervises China's standard drug-approval system, which is roughly equivalent to the US Food and Drug Administration. Investigator-initiated trials drove China's rising biomedical competitiveness, growing the number of annually registered trials in cell and gene therapies 11-fold to 207 between 2015 and 2023. Similar trials exist in the United States but require approval from the FDA.
New regulations
The government introduced stricter rules for trials of new biomedical technologies months before the news of the deaths emerged. Researchers and industry insiders stated that Order 818, which went into effect on May 1, aims to spur innovation while increasing government oversight. Dai Jialing, president of PharmaDJ, said that these fast-track trials often provide safety and efficacy evidence to show whether larger studies are worth pursuing.
Researchers noted that previous rules governing these trials were not strictly enforced or overseen. Iris Zhang, a lawyer at Han Kun Law Offices, stated that cell-therapy activity took place in non-sanjia institutions, private clinics, and hospital-affiliated ventures due to inconsistent supervision. This situation created a grey market where entities charged large sums for unauthorized or fraudulent therapies, leading to medical accidents and deaths.
Order 818 restrictions
Order 818 governs trials using new biomedical technologies such as cell therapies, brain implants, and gene-editing treatments. Hao Bin, an economist at Xi’an Jiaotong-Liverpool University, stated that the purpose of the order is to increase regulatory oversight. Only sanjia hospitals are permitted to conduct trials for all new biomedical technologies under the new rules. Ruby Wang, a physician and China health-policy consultant, stated that these hospitals possess the best clinicians, researchers, and patient access.
The National Health Commission can organize a second round of assessment for registered trials under Order 818, gaining the power to suspend, alter, or cancel trials over technical or ethical concerns. Aaron Gu, a lawyer at Han Kun Law Offices, stated that the new filing system operates somewhat like a de-facto approval process. Penalties under the regulations include millions of yuan in fines, a three-year ban on conducting clinical trials for new biomedical technologies, and the revocation of medical licences for leading physicians.
Lizzi Lee of the Asia Society Policy Institute stated that the real impact of the rules will depend on implementation. Gu stated that the policy allows companies in China to initiate investigator-initiated trials for the first time, making industry collaboration more practical.
