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Published on: September 27, 2024
Is there an efficacy-effectiveness gap between randomized controlled trials and real-world studies in colorectal
Xiao Zhang1, Shihui Fu1, Rui Meng1
1School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, China.
Background:
To investigate whether patients with colorectal cancer (CRC) enrolled in randomized controlled trials (RCTs) and real-world studies (RWS) differ in terms of baseline characteristics, leading to an efficacy-effectiveness gap.
Methods:
A systematic literature reviews was conducted to identify RCTs and RWS with CRC, treated with bevacizumab (BEV), cetuximab (CET) or oxaliplatin combined with capecitabine (XELOX). Using random-effects meta-analyses compared the baseline characteristics and treatment effects of RCTs and RWS, overall and by drug. Correlation between treatment effects and baseline characteristics and study types were estimated using meta-regression analyses.
Results:
Two hundred and fifty-three studies were included. Compared with patients enrolled in RWS, the proportion of male patients in RCTs was 0.032 higher (P=0.004), the proportion of patients with Eastern Cooperative Oncology Group (ECOG) performance ≥2 was 0.085 less (P<0.001). No significant differences in treatment effects [progression-free survival (PFS), overall survival (OS), objective response rate (ORR), disease control rate (DCR)] were found by overall analysis. But the OS of patients in RCTs was 4.184 higher (P=0.023) in the CET group. Meta-regression results showed that OS difference in the CET group was related to the difference in treatment lines, not related to other baseline characteristics and study types.
Conclusions:
No efficacy-effectiveness gap was found in CRC between RCTs and RWS. CRC treatment effects Between RCTs and RWS had high consistency.
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