Augmenting external control arms using Bayesian borrowing: a case study in first-line non-small  cell lung cancer
Conclusion: In this study, the RWD ECA was unable to successfully replicate the OS estimates from the matched population of the selected RCT. The inability to replicate could be due to unmeasured confounding and variations in time-periods, follow-up and subsequent therapy. Despite these findings, we demonstrate how BB can improve precision of comparative effectiveness estimates, potentially aid as a bias assessment tool and mitigate challenges of traditional methods when appropriate external data sources are available.PMID:38573331 | DOI:10.57264/cer-2023-0175 (Source: Journal of Comparative Effectiveness Research)
Source: Journal of Comparative Effectiveness Research - April 4, 2024 Category: General Medicine Authors: Alessandria Struebing Chelsea McKibbon Haoyao Ruan Emma Mackay Natalie Dennis Russanthy Velummailum Philip He Yoko Tanaka Yan Xiong Aaron Springford Mats Rosenlund Source Type: research

The impact of willingness-to-pay threshold on price reduction recommendations for oncology drugs: a review of assessments conducted by the Canadian Agency for Drugs and Technologies in Health
This study showed that there is a divergence between drug sponsor's incremental cost-effectiveness ratio (ICER) and CADTH revised ICER leading to a price reduction to meet the $50,000/QALY threshold. For the submissions with clear clinical benefit the median length of engagement (2.5 vs 3.3 months) and median length of negotiation (3.1 vs 3.6 months) were slightly shorter compared with the submissions where uncertainties were noted in the clinical benefit according to CADTH. This study shows that using a $50,000 per QALY threshold for oncology products potentially impacts timely access to life saving medications.PMID:38567...
Source: Journal of Comparative Effectiveness Research - April 3, 2024 Category: General Medicine Authors: Chakrapani Balijepalli Lakshmi Gullapalli Juhi Joshy Nigel Sb Rawson Source Type: research

Comparing the performance of two-stage residual inclusion methods when using physician's prescribing preference as an instrumental variable: unmeasured confounding and noncollapsibility
Conclusion: 2SRI tends to be less biased than GLM and 2SPS in terms of estimating treatment effect. It can be robust to noncollapsibility in the case of the mild unmeasured confounding effect.PMID:38567965 | DOI:10.57264/cer-2023-0085 (Source: Journal of Comparative Effectiveness Research)
Source: Journal of Comparative Effectiveness Research - April 3, 2024 Category: General Medicine Authors: Lisong Zhang Jim Lewsey Source Type: research

Assessing the performance of physician's prescribing preference as an instrumental variable in comparative effectiveness research with moderate and small sample sizes: a simulation study
Conclusion: Irrespective of sample size, the PPP IV approach leads to less biased estimates of treatment effectiveness than OLS adjusting for known confounding only. Particularly for smaller sample sizes, we recommend constructing PPP from long prescribing histories to improve statistical power.PMID:38567966 | DOI:10.57264/cer-2023-0044 (Source: Journal of Comparative Effectiveness Research)
Source: Journal of Comparative Effectiveness Research - April 3, 2024 Category: General Medicine Authors: Lisong Zhang Jim Lewsey David A McAllister Source Type: research

The impact of willingness-to-pay threshold on price reduction recommendations for oncology drugs: a review of assessments conducted by the Canadian Agency for Drugs and Technologies in Health
This study showed that there is a divergence between drug sponsor's incremental cost-effectiveness ratio (ICER) and CADTH revised ICER leading to a price reduction to meet the $50,000/QALY threshold. For the submissions with clear clinical benefit the median length of engagement (2.5 vs 3.3 months) and median length of negotiation (3.1 vs 3.6 months) were slightly shorter compared with the submissions where uncertainties were noted in the clinical benefit according to CADTH. This study shows that using a $50,000 per QALY threshold for oncology products potentially impacts timely access to life saving medications.PMID:38567...
Source: Journal of Comparative Effectiveness Research - April 3, 2024 Category: General Medicine Authors: Chakrapani Balijepalli Lakshmi Gullapalli Juhi Joshy Nigel Sb Rawson Source Type: research

Comparing the performance of two-stage residual inclusion methods when using physician's prescribing preference as an instrumental variable: unmeasured confounding and noncollapsibility
Conclusion: 2SRI tends to be less biased than GLM and 2SPS in terms of estimating treatment effect. It can be robust to noncollapsibility in the case of the mild unmeasured confounding effect.PMID:38567965 | DOI:10.57264/cer-2023-0085 (Source: Journal of Comparative Effectiveness Research)
Source: Journal of Comparative Effectiveness Research - April 3, 2024 Category: General Medicine Authors: Lisong Zhang Jim Lewsey Source Type: research

Assessing the performance of physician's prescribing preference as an instrumental variable in comparative effectiveness research with moderate and small sample sizes: a simulation study
Conclusion: Irrespective of sample size, the PPP IV approach leads to less biased estimates of treatment effectiveness than OLS adjusting for known confounding only. Particularly for smaller sample sizes, we recommend constructing PPP from long prescribing histories to improve statistical power.PMID:38567966 | DOI:10.57264/cer-2023-0044 (Source: Journal of Comparative Effectiveness Research)
Source: Journal of Comparative Effectiveness Research - April 3, 2024 Category: General Medicine Authors: Lisong Zhang Jim Lewsey David A McAllister Source Type: research

Letter to the editor: network meta-analysis for indirect comparison of lanadelumab and for the treatment of hereditary angioedema
J Comp Eff Res. 2024 Mar 28:e230165. doi: 10.57264/cer-2023-0165. Online ahead of print.NO ABSTRACTPMID:38545965 | DOI:10.57264/cer-2023-0165 (Source: Journal of Comparative Effectiveness Research)
Source: Journal of Comparative Effectiveness Research - March 28, 2024 Category: General Medicine Authors: Max Schlueter Sandra Nestler-Parr Source Type: research

R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 15
J Comp Eff Res. 2024 Mar 28:e240033. doi: 10.57264/cer-2024-0033. Online ahead of print.ABSTRACTIn this latest update we discuss real-world evidence (RWE) guidance from the leading oncology professional societies, the American Society of Clinical Oncology and the European Society for Medical Oncology, and the PRINCIPLED practical guide on the design and analysis of causal RWE studies.PMID:38546012 | DOI:10.57264/cer-2024-0033 (Source: Journal of Comparative Effectiveness Research)
Source: Journal of Comparative Effectiveness Research - March 28, 2024 Category: General Medicine Authors: Alejandra Castanon Benjamin D Bray Sreeram V Ramagopalan Source Type: research

Letter to the editor: network meta-analysis for indirect comparison of lanadelumab and for the treatment of hereditary angioedema
J Comp Eff Res. 2024 Mar 28:e230165. doi: 10.57264/cer-2023-0165. Online ahead of print.NO ABSTRACTPMID:38545965 | DOI:10.57264/cer-2023-0165 (Source: Journal of Comparative Effectiveness Research)
Source: Journal of Comparative Effectiveness Research - March 28, 2024 Category: General Medicine Authors: Max Schlueter Sandra Nestler-Parr Source Type: research