The U.S. Food and Drug Administration (FDA) has adopted a more stringent approach to reviewing drug applications based on China-only clinical trials, marking a significant shift in the regulatory landscape for Chinese pharmaceutical companies seeking U.S. market entry.
Recent FDA Rejections Signal Policy Shift
The FDA has recently rejected two cancer therapies developed by Chinese pharmaceutical companies: Hutchmed's surufatinib and the collaborative effort between Coherus BioSciences and Shanghai Junshi Biosciences' toripalimab. This follows the earlier rejection of sintilimab, developed by Eli Lilly and Innovent Biologics, establishing a clear pattern in regulatory decisions.
Dr. Richard Pazdur, director of the FDA's oncology center of excellence, highlighted that approximately 25 applications from China based on single-country data are under review in oncology alone. The agency's primary concern centers on data generalizability to the U.S. population, considering both genetic differences and variations in medical practices between countries.
Impact on Global Drug Development
The FDA's stance has significant implications for China's rapidly expanding pharmaceutical sector. Despite China ranking third among countries exporting drugs and biologics to the U.S., new applications will face heightened scrutiny. The regulatory body now strongly advocates for multi-regional clinical trials, particularly encouraging inclusion of underrepresented regions such as Africa and South America.
China's Growing Pharmaceutical Presence
China's emergence as an R&D powerhouse is evident in its expanding global partnerships. Notable examples include:
- Merck's potential $1.4 billion partnership with Sichuan Kelun Pharmaceutical for an immuno-oncology asset
- Bavarian Nordic's collaboration with Nuance Pharma for RSV vaccine development
- WuXi Biologics' rapid rise to become a top-five biologics manufacturing company since 2015
Regulatory Requirements and Future Implications
The FDA's complete response letters consistently emphasize the need for multi-regional clinical trials. Hutchmed's attempt to bridge this gap with U.S. studies proved insufficient, as the agency maintained its position on requiring broader geographic representation in clinical data.
"The degree of regulatory flexibility in establishing the acceptability of data from a single country and its generalisability to a new population should be balanced against the drug's innovation," stated Dr. Pazdur, suggesting that truly innovative treatments might receive more flexible consideration.
Industry Response and Adaptation
Companies must now weigh the additional time and expense of conducting multi-regional trials against their U.S. market entry strategies. Eli Lilly's attempt to position sintilimab as a cost-competitive alternative in the PD-1 inhibitor space failed to sway the FDA's advisory committee, with only one out of 15 members voting in favor of approval.
The pharmaceutical industry faces a critical decision point: either adapt to these new requirements by implementing multi-regional trials or risk significant delays and potential rejections in the U.S. market. This regulatory evolution may reshape the landscape of global drug development and international pharmaceutical collaboration.
