J Comp Eff Res. 2026 Apr 29:e260006. doi: 10.57264/cer-2026-0006. Online ahead of print.
ABSTRACT
Aim: Single-arm trial data is frequently used during the reimbursement of new oncology interventions. Evaluating treatment effects relative to multiple relevant comparators via network meta-analysis (NMA) using data from single-arm trials; however, remains a challenge. This work introduces a two-stage approach to incorporate single-arm trial data into an NMA and applies this to the LIBRETTO-001 (NCT03157128) trial where selpercatinib (a selective rearranged during transfection [RET] inhibitor) was trialed as a treatment for RET-fusion positive, nonsquamous non-small-cell lung cancer in the first line setting. Materials & methods: Using data from KEYNOTE-189 (NCT02578680) and a real-world database, a pseudo comparator arm was constructed by propensity score matching and adjusted via an acceleration factor to account for the prognostic effect of RET status. NMAs were conducted using a Bayesian random-effects model. Results: The hazard ratios of selpercatinib relative to pemetrexed + platinum-based chemotherapy (the most common comparator in the network used) were found to be 0.304 (95% credible interval [CrI] 0.165, 0.553) and 0.368 (95% CrI 0.178, 0.757) for progression-free survival and overall survival, respectively. The validation of the NMA results could be assessed for progression-free survival of selpercatinib versus pembrolizumab + pemetrexed + platinum-based chemotherapy. A good agreement with published results from the Phase III LIBRETTO-431 trial (NCT04194944) was found (0.586 [95% CrI 0.302,1.123] from the NMA vs 0.46 [95% CI 0.31, 0.70] from LIBRETTO-431 the intention to treat [pembrolizumab] population). Conclusion: The two-stage approach to incorporate single-arm trial data within NMAs is readily applicable within health technology assessment. Enabling the earlier assessment of single-arm trials, via pseudo comparator arms, will provide payers with greater confidence in anticipated treatment effects. In light of the joint clinical assessment, incorporation of single-arm trials within NMA facilitates the reporting of predicted treatment effects relative to multiple relevant comparators, which is important when considering the use of interventions for the global market.
PMID:42052748 | DOI:10.57264/cer-2026-0006
