CLINICAL TRIAL EVIDENCE, EXPEDITED REGULATORY PATHWAYS, AND REVIEW EFFICIENCY OF RARE DISEASE DRUGS APPROVED IN CHINA, 2016-2025
Author(s)
Huxinigeer Maimaiti, master1, Chenyan Wang, master2, XIANG FEI, undergraduate2, SU XUAN, undergraduate2, LI SHURONG, undergraduate2, XU ZHUOYAN, undergraduate2, MA TINGTING, undergraduate2, JI WENJIN, Professor2.
1School of Pharmacy, Xi'an Jiaotong University, Xi'an China, xi'an, China, 2School of Pharmacy, Xi'an Jiaotong University, Xi'an China, Xi'an, China.
1School of Pharmacy, Xi'an Jiaotong University, Xi'an China, xi'an, China, 2School of Pharmacy, Xi'an Jiaotong University, Xi'an China, Xi'an, China.
OBJECTIVES: OBJECTIVES: To evaluate the clinical evidence characteristics of rare disease drugs approved in China from 2016 to 2025 and analyze the independent impact of trial designs, drug attributes, and special approval pathways on review efficiency.
METHODS: METHODS: Data of 210 approved rare disease drugs and 252 clinical trials were extracted from NMPA, CDE, and ClinicalTrials.gov. Descriptive statistics, non-parametric tests, and multivariate linear regression (on log-transformed review duration) were used to characterize clinical evidence and identify independent predictors of review time.
RESULTS: RESULTS: Chemical drugs (61.4%), originators (70.5%), and imports (63.8%) dominated the pipeline, with oncology representing the largest therapeutic area (41.0%). Over half (58.6%) of approved drugs fell within the First National Rare Disease Catalogue. While randomized controlled trials (72.6%) and placebo controls (51.7%) remained mainstream, surrogate endpoints (46.7%) and single-arm trials (23.8%) were widely accepted. Furthermore, priority-reviewed trials had significantly smaller median sample sizes compared to non-priority ones (110 vs. 211, P<0.001). Multivariate regression confirmed that after controlling for covariates, the number of utilized special approval pathways was the sole independent driver of shortened review time ( P<0.001); each additional pathway significantly reduced review duration by 17.0% (P=0.019). Although univariate analysis showed no significant delay for single-arm trials (P=0.066) or surrogate endpoints (P=0.640), a higher clinical evidence quality level (0 to 3) was monotonically associated with shorter review duration (P=0.008).
CONCLUSIONS: CONCLUSIONS: China's regulatory framework maintains randomized controlled trials as its evidence baseline while exhibiting high flexibility for expedited pathways. Special approval pathways demonstrate synergistic acceleration effects. These pathways independently accelerate market access, though they entail greater tolerance for lower-phase, open-label, or single-arm trials, highlighting the critical necessity for robust post-marketing confirmatory evidence and real-world evidence to establish a complete regulatory closed loop.
METHODS: METHODS: Data of 210 approved rare disease drugs and 252 clinical trials were extracted from NMPA, CDE, and ClinicalTrials.gov. Descriptive statistics, non-parametric tests, and multivariate linear regression (on log-transformed review duration) were used to characterize clinical evidence and identify independent predictors of review time.
RESULTS: RESULTS: Chemical drugs (61.4%), originators (70.5%), and imports (63.8%) dominated the pipeline, with oncology representing the largest therapeutic area (41.0%). Over half (58.6%) of approved drugs fell within the First National Rare Disease Catalogue. While randomized controlled trials (72.6%) and placebo controls (51.7%) remained mainstream, surrogate endpoints (46.7%) and single-arm trials (23.8%) were widely accepted. Furthermore, priority-reviewed trials had significantly smaller median sample sizes compared to non-priority ones (110 vs. 211, P<0.001). Multivariate regression confirmed that after controlling for covariates, the number of utilized special approval pathways was the sole independent driver of shortened review time ( P<0.001); each additional pathway significantly reduced review duration by 17.0% (P=0.019). Although univariate analysis showed no significant delay for single-arm trials (P=0.066) or surrogate endpoints (P=0.640), a higher clinical evidence quality level (0 to 3) was monotonically associated with shorter review duration (P=0.008).
CONCLUSIONS: CONCLUSIONS: China's regulatory framework maintains randomized controlled trials as its evidence baseline while exhibiting high flexibility for expedited pathways. Special approval pathways demonstrate synergistic acceleration effects. These pathways independently accelerate market access, though they entail greater tolerance for lower-phase, open-label, or single-arm trials, highlighting the critical necessity for robust post-marketing confirmatory evidence and real-world evidence to establish a complete regulatory closed loop.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HPR137
Topic
Health Policy & Regulatory, Methodological & Statistical Research
Topic Subcategory
Approval & Labeling
Disease
Rare & Orphan Diseases