CHARACTERISTICS AND REPORTING OF CLINICAL TRIAL EVIDENCE SUPPORTING THE APPROVAL OF CHINESE INNOVATIVE DRUGS (2018-2024): RETROSPECTIVE STUDY OF PUBLICATIONSAND NMPA REVIEW DOCUMENTS
Author(s)
Jing Zhou, PhD, Jay Pan, PhD;
Sichuan University, West China School of Public Health and West China Fourth Hospital, chengdu, China
Sichuan University, West China School of Public Health and West China Fourth Hospital, chengdu, China
OBJECTIVES: Chinese innovative drugs are on the rise and are expected to pose challenges for global regulators and impact patient access worldwide. This study aimed to characterize the clinical trial evidence supporting the approval of Chinese innovative drugs based on publications and to compare the reporting between publications and NMPA regulatory documents.
METHODS: We identified all pivotal clinical trials for Chinese innovative drugs approved between January 2018 and December 2024 using NMPA-issued review reports or drug labels. Corresponding peer-reviewed publications were retrieved from ClinicalTrials.gov, PubMed, and CNKI. We analyzed trial characteristics including trial designs and primary endpoints, and compared them across therapeutic areas and approval programs. We further assessed the discrepancies in reporting between publications and NMPA review documents.
RESULTS: Among 126 Chinese innovative drugs approved during the study period, 109 drugs (114 indications) supported by 126 pivotal trials had both regulatory documents and publications available. Most trials (116 [92%]) were conducted within China, and two-thirds (83 [66%]) were phase III. A total of 80 (63%) were randomized controlled trials using either active (51 [40%]) or placebo (29 [23%]) controls, while 46 (37%) were single-arm trials employing external (40 [32%]) or unspecified controls (6 [5%]). Three-quarters (96 [76%]) of trials evaluated only surrogate primary endpoints. Pivotal trials for cancer treatments were more frequently phase II, open-label, and single-arm trials, using external controls and surrogate primary endpoints. A similar pattern was observed among trials supporting drugs approved through expedited programs. Primary endpoint outcomes were identical in 70 (56%) trials, similar in 41 (33%), and different in 15 (12%) when comparing publications with drug labels.
CONCLUSIONS: The clinical trial evidence supporting the approval of Chinese innovative drugs varied across therapeutic areas and approval programs. The reporting of trial characteristics in publications was sometimes inconsistent with that in drug labels.
METHODS: We identified all pivotal clinical trials for Chinese innovative drugs approved between January 2018 and December 2024 using NMPA-issued review reports or drug labels. Corresponding peer-reviewed publications were retrieved from ClinicalTrials.gov, PubMed, and CNKI. We analyzed trial characteristics including trial designs and primary endpoints, and compared them across therapeutic areas and approval programs. We further assessed the discrepancies in reporting between publications and NMPA review documents.
RESULTS: Among 126 Chinese innovative drugs approved during the study period, 109 drugs (114 indications) supported by 126 pivotal trials had both regulatory documents and publications available. Most trials (116 [92%]) were conducted within China, and two-thirds (83 [66%]) were phase III. A total of 80 (63%) were randomized controlled trials using either active (51 [40%]) or placebo (29 [23%]) controls, while 46 (37%) were single-arm trials employing external (40 [32%]) or unspecified controls (6 [5%]). Three-quarters (96 [76%]) of trials evaluated only surrogate primary endpoints. Pivotal trials for cancer treatments were more frequently phase II, open-label, and single-arm trials, using external controls and surrogate primary endpoints. A similar pattern was observed among trials supporting drugs approved through expedited programs. Primary endpoint outcomes were identical in 70 (56%) trials, similar in 41 (33%), and different in 15 (12%) when comparing publications with drug labels.
CONCLUSIONS: The clinical trial evidence supporting the approval of Chinese innovative drugs varied across therapeutic areas and approval programs. The reporting of trial characteristics in publications was sometimes inconsistent with that in drug labels.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR79
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
SDC: Infectious Disease (non-vaccine), SDC: Oncology