The FDA’s Approval of Drugs Without U.S. Trial Participants: A Changing Landscape
The FDA’s recent approval of four drugs last year without any U.S. trial participants marks a significant shift in drug approval processes. Among the approved drugs were treatments for a blood disorder, UTIs, cancer, and an ultra-rare disease. This move comes in contrast to previous years, where the FDA rejected drugs for lacking testing on U.S. patients. For example, Eli Lilly and Innovent Biologics received a complete response letter in 2022 for their lung cancer drug sintilimab due to a lack of U.S. trial data.
However, the FDA has been more open to approving drugs with limited data over time, especially since the passage of the 21st Century Cures Act in 2016. This has led to faster reviews for novel drugs in critical disease areas like cancer, with approvals based on fewer supporting studies. FDA Commissioner Dr. Marty Makary has also proposed new strategies to further expedite drug reviews.
Despite these changes, the U.S. is facing competition from China in the field of clinical trials. China has seen a rapid increase in clinical trial activity and has surpassed the U.S. as the top location for drug research. Running trials in China is more cost-effective, and the country has made significant strides in becoming a biotech leader. China has become a major contributor to cancer drug R&D and obesity medicine development, with numerous weight loss drugs in late-stage trials.
While the FDA has approved drugs without U.S. participants, it has maintained a tough stance on China and its reliance on trials conducted solely within the country. The U.S. Justice Department has implemented rules to prevent sensitive information from being accessed by countries like China. Additionally, budget cuts to the NIH have impacted clinical funding in the U.S., leading to concerns from foreign health executives about the future of R&D in the country.
In response to these challenges, China is aiming to expedite clinical trial reviews, proposing to halve the waiting period for approvals. This move signifies China’s commitment to becoming a leader in drug development and clinical research. As the landscape of drug approvals continues to evolve, it is essential for regulatory bodies and industry stakeholders to adapt to these changes and ensure patient safety and access to innovative treatments.
