Lawmakers Urge FDA to Up Scrutiny of Clinical Data From China

House Select Committee on China chairman John Moolenaar (R-MI) and Rep. Ben Cline (R-VA) are urging FDA intensify safeguards on clinical trial data generated in China, citing concerns about transparency and potential underreporting of serious adverse events and patient deaths.

In an 8/20 letter to acting FDA commissioner Kyle Diamantas, the lawmakers said the agency should take action to address what they described as “serious risks” associated with clinical trial data generated in China. The lawmakers said FDA has long served as a global standard for drug safety, research ethics and informed consent and argued that those standards should not be weakened when U.S. drug developers rely on data generated overseas.

Their concerns are based on recent reports about three patient deaths in experimental gene-editing investigator-initiated trials in China, including the deaths of two children. According to the lawmakers, one sponsor company halted public updates about a treatment for more than a year and acknowledged the patient's death only after repeated inquiries. In another case, researchers allegedly failed to disclose the death of a child and subsequently published scientific findings without reporting the fatal outcome.

The lawmakers also cited a third case in which a patient reportedly died in March, while the therapy was cleared by FDA in early August to begin clinical trials in the U.S. They said the company had not publicly disclosed the death.

“These events are heartbreaking,” Moolenaar and Cline wrote, arguing that the cases raise broader questions about the transparency of China's clinical research system.

The letter comes as U.S. pharmaceutical and biotechnology companies increasingly incorporate China-generated clinical data into U.S. drug development programs or acquire and license therapies from Chinese companies based on studies conducted in China.

Moolenaar and Cline argued that the FDA should not accept clinical data generated under conditions that would not meet U.S. standards for patient protection.

Moolenaar and Cline asked FDA to take two specific actions: (1) decline China-generated clinical trial data submitted in support of U.S. INDs, NDAs or BLAs unless FDA has conducted an in-person audit of the trial site within the 12 months preceding submission of the application; (2) conduct a comprehensive risk assessment of the extent to which U.S. drug development and marketing approvals rely on data from Chinese investigator-initiated trials and clinical trial applications.