To understand the innovation points of drugs marketed after the implementation of the U.S. breakthrough therapy designation (BTD) program, and provide a reference basis for the development of innovative drugs and the review of breakthrough therapy drugs in China.

As of 2021, all BTD drugs approved by the U.S. FDA for marketing were collected, and the characteristics of these drugs in terms of innovative therapeutic modalities, treatment mechanisms, and technological breakthroughs were categorized and statistically analyzed.

The US FDA received 1 192 BTD drug applications and approved 242 for marketing. Among the marketed drugs, 42 (17%) were new drugs to fill clinical treatment gaps, 99 (41%) were drugs for expanded indications and new applicable populations, 27 (21%) were drug combinations for combination therapy, 16 (7%) were new drugs with more clinical treatment advantages, 31 (12.8%) were drugs with new mechanisms of action, and 12 (5%) were drugs prepared in new dosage forms or processes.

The substantial improvement of the clinical efficacy of BTD drugs is reflected in the innovation of drugs and advancement of treatment.