Indication Expansion Dynamics and Trial Characteristics of Novel Multi-Indication Anti-Cancer Drugs in China...

To perform a cross-sectional analysis of the indication expansion trends and pivotal trial features of novel multi-indication anti-cancer drugs approved in China from 2000 to 2024.

We conducted a cross‑sectional review of 74 novel multi-indication anti-cancer drugs approved by the National Medical Products Administration (NMPA) in China from 2000 to 2024, encompassing 265 total indications. Data were collected from multiple sources, including the NMPA, the Center for Drug Evaluation (CDE) of the NMPA, the National Healthcare Security Administration (NHSA), ClinicalTrials.gov, and drug package inserts. Descriptive statistics and Fisher's exact test (two-tailed) or the χ² -test with Monte Carlo simulation, were used to analyze indication and clinical trial characteristics across indication approval sequence.

Of the 74 drugs, 32 (43%) held two indications, 23 (31%) held three, and 19 (26%) held four or more. Solid tumors accounted for 80% of all indications, most commonly non-small cell lung cancer. Immune checkpoint inhibitors (ICIs) exhibited the greatest expansion, with the most extensively approved drug reaching 14 indications. However, the inclusion of these drugs in the National Reimbursement Drug List (NRDL) did not keep pace with their market approval. Sequential approvals across indications showed significant differences in treatment type (P<0.001), NMPA approval type (P=0.008), trial arms (P=0.009), trial design (P=0.030), and single-arm endpoints (P<0.001).

Regulatory reforms facilitated the approval of novel multi-indication anti-cancer drugs, although NRDL inclusion has not kept pace. Targeted therapies dominate, while ICIs, though fewer, show the broadest indication expansion. Most drugs are approved for two to three indications, mainly in solid tumors, and later approvals were associated with different regulatory pathways than earlier ones.