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Pembrolizumab, as monotherapy in first-line recurrent or metastatic (R/M) head and neck squamous cell carcinoma (HNSCC) with combined positive score (CPS) ≥1 and in combination with platinum and 5-fluorouracil (5-FU) in the overall R/M HNSCC population, received US FDA approval based on the KEYNOTE-048 trial. Using public drug prices, from a US payer perspective, we evaluated the cost-effectiveness of each pembrolizumab regimen versus cetuximab + platinum +5-FU (EXTREME regimen, trial comparator), cisplatin + docetaxel + cetuximab (TPEx regimen), cisplatin + paclitaxel, and platinum +5-FU. A three-state partitioned-survival model was used to project costs and outcomes over 20 years with 3% annual discounting. Progression-free and overall survival were modelled using long-term extrapolation of KEYNOTE-048 data and, for alternative comparators, data from a network meta-analysis was used. Time-on-treatment was derived from KEYNOTE-048 or approximated using network meta-analysis progression-free survival estimates. Costs included first-line and subsequent treatments, disease management, adverse events, and terminal care costs. Utilities were derived from the KEYNOTE-048 Euro-QoL five-dimension data and using a US algorithm. In the CPS ≥1 R/M HNSCC population, pembrolizumab monotherapy was dominant versus EXTREME and TPEx regimens, and cost-effective (at $100,000/QALY threshold) versus platinum +5-FU ($86,827/QALY) and cisplatin + paclitaxel ($81,473/QALY). Pembrolizumab combination therapy in the overall R/M HNSCC population, was dominant versus TPEx regimen, and cost-effective versus EXTREME regimen ($1,769/QALY), platinum +5-FU ($81,989/QALY), and cisplatin + paclitaxel ($89,505/QALY). Sensitivity analyses showed a high cost-effectiveness probability for pembrolizumab at the $100,000/QALY threshold. First-line pembrolizumab monotherapy in patients with CPS ≥1, and pembrolizumab combination therapy in the overall R/M HNSCC population is cost-effective from the perspective of US payers.

Pembrolizumab, as monotherapy in first-line recurrent or metastatic (R/M) head and neck squamous cell carcinoma (HNSCC) with a combined positive score (CPS) ≥1 and in combination with platinum and 5-fluorouracil (5-FU) in the overall R/M HNSCC population, received US FDA approval based on the KEYNOTE-048 trial. Using public drug prices, from a US payer perspective, we evaluated the cost-effectiveness of each pembrolizumab regimen vs. cetuximab + platinum+5-FU (EXTREME regimen, trial comparator), cisplatin + docetaxel + cetuximab (TPEx regimen), cisplatin + paclitaxel, and platinum+5-FU. A three-state partitioned-survival model was used to project costs and outcomes over 20 years with 3% annual discounting. Progression-free and overall survival were modeled using long-term extrapolation of KEYNOTE-048 data and, for alternative comparators, data from a network meta-analysis was used. Time-on-treatment was derived from KEYNOTE-048 or approximated using network meta-analysis progression-free survival estimates. Costs included first-line and subsequent treatments, disease management, adverse events, and terminal care costs. Utilities were derived from the KEYNOTE-048 Euro-QoL five-dimension data and using a US algorithm. In the CPS ≥1 R/M HNSCC population, pembrolizumab monotherapy was dominant vs. EXTREME and TPEx regimens, and cost-effective (at $100,000/QALY threshold) vs. platinum+5-FU ($86,827/QALY) and cisplatin + paclitaxel ($81,473/QALY). Pembrolizumab combination therapy in the overall R/M HNSCC population was dominant vs. TPEx regimen, and cost-effective vs. EXTREME regimen ($1769/QALY), platinum+5-FU ($81,989/QALY), and cisplatin + paclitaxel ($89,505/QALY). Sensitivity analyses showed a high cost-effectiveness probability for pembrolizumab at the $100,000/QALY threshold. First-line pembrolizumab monotherapy in patients with CPS ≥1, and pembrolizumab combination therapy in the overall R/M HNSCC population is cost-effective from the perspective of the US payers.

Treatment approaches for hepatocellular carcinoma (HCC) in China are distinct in the use of resection with more advanced HCC, and continued research is needed to re-evaluate the appropriateness of guideline recommendations. Surveillance is recommended among individuals with chronic hepatitis B virus (HBV) infection in East Asia to improve early disease detection; however, only limited evidence exists to quantify its survival impact, and the ideal screening interval is not known. The overall objectives of this dissertation were to (1) compare the effectiveness of HCC treatment approaches in China, (2) evaluate the impact of 6-month and 12-month screening intervals compared with no surveillance among individuals with chronic HBV in China, and (3) examine the survival Impact of surveillance among HCC patients in Taiwan. This dissertation used clinical data extracted from medical records for HCC patients in China and Taiwan, as part of the global BRIDGE to Better Outcomes in HCC (HCC BRIDGE) Study. Multivariate Cox proportional hazard models compared survival with treatment in China, stratified by disease stage, and propensity score (PS) analysis was conducted to address selection bias. An individual-based simulation model combined well-established data on chronic HBV progression and tumor growth in HCC with clinical data from the HCC BRIDGE study in China to project the survival impact of different surveillance strategies. Using the HCC BRIDGE study in Taiwan, survival according to surveillance status was assessed using the Kaplan-Meier method, controlling for selection bias through PS analysis and lead time bias using a range of tumor volume doubling time (DT) estimated across tumor growth studies. The results revealed that patients with intermediate to advanced disease tolerate resection, and have better outcomes than with other HCC therapies. Surveillance improves survival with HCC, after applying lead time adjustments using a plausible range in DT. Results of model simulation suggest that surveillance performed at 12-month versus 6-month intervals can be more easily implemented with little impact on survival with HCC. These findings help inform efforts to ensure that individuals with chronic HBV are properly monitored for HCC given limited resources, and HCC patients receive appropriate treatment to improve survival outcomes in East Asia.

Background: The availability of the tyrosine-kinase inhibitor (TKI), imatinib, and later introduction of second generation TKIs, dasatinib and nilotinib, have not only improved clinical outcomes of patients with chronic myeloid leukemia (CML), but also provide multiple therapeutic options for CML patients. Despite the widespread use of these oral therapies, little is known about the impact of different treatment regimens on patient-reported outcomes (PROs) among CML patients. The objective of this study was to assess the impact of patient-reported treatment restrictions and negative medication experiences (NMEs) on satisfaction and other health outcomes among patients with CML treated with oral TKIs. Methods: Participants recruited from survey panels and patient networks in the United States (US) and Europe completed an online questionnaire. Respondents included adults (≥18 years) with chronic-phase CML currently on TKI treatment. Study variables included treatment difficulty (i.e., difficulty in following treatment regimens), CML dietary/dosing requirements, NMEs, and validated PROs assessing treatment satisfaction, health-related quality of life (HRQoL), activity impairment, and non-adherence. Structural equation models assessed associations among variables, controlling for covariates. Results: 303 patients with CML (US n=152; Europe n=151; mean age 51.5 years; 46.2% male) completed the questionnaire. Approximately 30% of patients reported treatment difficulties; treatment difficulty was higher among nilotinib (63.3%) than among dasatinib (2.6%) or imatinib (19.2%) treated patients (p