Does FDA Have a Double-Standard for Review for China-Only Data?
(Thursday, April 7, 2022)
In February, FDA pushed hard against a cancer drug arguing that data from the China-only clinical trials was not acceptable for US approval. But does it imply an FDA policy or a one-time decision? Another cancer drug, this time for a rare cancer common in Asian population, with a similar China-only clinical trial may answer the question or muddy the waters further. In recent years, several Chinese pharmaceutical companies have developed novel drugs for various indications using clinical trials done either exclusively or predominantly in China. The strategy is to first get these drugs approved in China and then use the same data for approval in the US and other countries. Additionally, the companies strategize to pursue selected indications based on their rarity in the US to take advantage of the regulatory flexibility available for such indications, unlike their China approvals which were for wider applications. These drugs are mostly designed to compete with drugs that the expensive in the US with a promise to lower drug prices upon market approval. The business goal seems to be to get FDA approval for the rare indication first and then pursue wider approval as supplemental applications. Meanwhile the company could bank on their drug being used off-label for other indications for which data publicly exists, albeit from China, till wider formal FDA approval is obtained. But FDA did not buy it last time. FDA raised concerns about the extrapolation of China-only clinical data to the diverse US population without bridging studies. FDA also raised questions about the integrity of the clinical data from China. Lastly, FDA ignored the company’s promise of lower drug price since such promises are impossible to enforce by the FDA, notwithstanding the fact that FDA’s decisions cannot be legally based on the market price of a drug. Now, another Chinese pharma company is facing a similar situation with their cancer drug with China-only data with a twist. The first indication is nasopharyngeal cancer (also called head and neck cancer) which affects Asian populations more than others justifying use of data from Chinese patients, it is a first in class treatment for this cancer, the drug received priority review designation from the FDA, and so far, FDA has not publicly come out against it. But all the concerns FDA expressed in February exist for this drug as well and the company’s strategy to go for a smaller indication first while touting other bigger indications fits the abovementioned overall business strategy. If FDA were to approve this drug, it would raise serious questions about FDA’s double-standard, and if FDA were to reject the drug based on the previously expressed concerns for a similar situation, it would solidify FDA’s policy to not accept single foreign country clinical data for market approval which in turn should create a clear pathway for other companies. While making promising drugs to patients should take priority, it is equally important that FDA shows consistency and transparency of its processes. Not having that hurts the patients and developers alike, while damaging the perceived integrity of the FDA.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC