ObjectivesOur objectives were to evaluate the cost effectiveness of pembrolizumab compared with standard-of-care (SoC) platinum-based chemotherapy as first-line treatment in patients with metastatic non-small-cell lung cancer (NSCLC) that expresses high levels of programmed death ligand-1 (PD-L1) [tumour proportion score (TPS) ≥50%], from a US third-party public healthcare payer perspective.MethodsWe conducted a partitioned-survival model with a cycle length of 1 week and a base-case time horizon of 20 years. Parametric models were fitted to Kaplan–Meier estimates of time on treatment, progression-free survival and overall survival from the KEYNOTE-024 randomized clinical trial (patients aged ≥18 years with stage IV NSCLC, TPS ≥50%, without epidermal growth factor receptor (EGFR)-activating mutations or anaplastic lymphoma kinase (ALK) translocations who received no prior systemic chemotherapy) and validated with long-term registry data. Quality-adjusted life-years (QALYs) were calculated based on EuroQoL-5 Dimensions (EQ-5D) utility data collected in the trial. Costs ($US, year 2016 values) for drug acquisition/administration, adverse events and clinical management were included. Costs and outcomes were discounted at 3% per year. A series of deterministic and probabilistic sensitivity analyses were performed to test the robustness of the results.ResultsIn the base-case scenario, pembrolizumab resulted in an expected gain of 1.31 life-years (LYs) and 1.05 QALYs and an incremental cost of $US102,439 compared with SoC. The incremental cost per QALY gain was $US97,621/QALY and the incremental cost per LY gain was $US78,344/LY.ConclusionsPembrolizumab is projected to be a cost-effective option compared with SoC platinum-based chemotherapy as first-line treatment in adults with metastatic NSCLC expressing high levels of PD-L1.Electronic supplementary materialThe online version of this article (doi:10.1007/s40273-017-0527-z) contains supplementary material, which is available to authorized users.

Given the ever-increasing burden of end-stage renal disease (ESRD) in a global milieu of limited financial and health resources, interested parties continue to search for ways to optimize dialysis access. Government and payer initiatives to increase access to renal replacement therapies (RRTs), particularly peritoneal dialysis (PD) and hemodialysis (HD), may have meaningful impacts from clinical and health-economic perspectives; and despite similar clinical and humanistic outcomes between the two dialysis modalities, PD may be the more convenient and resource-conscious option. This review assessed country-specific PD-First/Favored policies and their associated background, implementation, and outcomes. It was found that barriers to policy-implementation are broadly associated with government policy, economics, provider or healthcare professional education, modality-related factors, and patient-related factors. Notably, the success of a given country's PD-Favored policy was inversely associated with the extent of HD infrastructure. It is hoped that this review will provide a foundation across countries to share lessons learned during the development and implementation of PD-First/Favored policies.

BackgroundPatient registries have great potential for providing data that describe disease burden, treatments, and outcomes; which can be used to improve patient care. Many renal registries exist, but a central repository of their scope, quality, and accessibility is lacking. The objective of this study was to identify and assess worldwide renal registries reporting on renal replacement therapy and compile a list of those most suitable for use by a broad range of researchers.MethodsRenal registries were identified through a systematic literature review and internet research. Inclusion criteria included information on dialysis use (yes/no), patient counts ≥300, and evidence of activity between June 2007 and June 2012. Public availability of information on dialysis modality, outcomes, and patient characteristics as well as accessibility of patient-level data for external research were evaluated.ResultsOf 144 identified renal registries, 48 met inclusion criteria, 23 of which were from Europe. Public accessibility to annual reports, publications, or basic data was good for 17 registries and moderate for 22. Patient-level data were available to external researchers either directly or through application and review (which may include usage fees) for 13 of the 48 registries, and were inaccessible or accessibility was unknown for 25.ConclusionsThe lack of available data, particularly in emerging economies, leaves information gaps about health care and outcomes for patients with renal disease. Effective multistakeholder collaborations could help to develop renal registries where they are absent, or enhance data collection and dissemination for currently existing registries to improve patient care.Electronic supplementary materialThe online version of this article (doi:10.1186/s12882-015-0028-2) contains supplementary material, which is available to authorized users.

Both the observed rate of BSI and adjusted probability of developing a BSI remained significantly lower for the MCB than the compounded PN group.

Objectives. To quantify the impact of Medicare Part D eligibility on medication utilization, emergency department use, hospitalization, and preference-based health utility among civilian noninstitutionalized Medicare beneficiaries. Study Design. Difference-in-differences analyses were used to estimate the effects of Part D eligibility on health outcomes by comparing a 12-month period before and after Part D implementation using the Medical Expenditure Panel Survey. Models adjusted for sociodemographic characteristics and health status and compared Medicare beneficiaries aged 65 and older with near elderly aged 55-63 years old. Principal Findings. Five hundred and fifty-six elderly and 549 near elderly were included. After adjustment, Part D was associated with a U.S.$179.86 (p 5 .034) reduction in out-of-pocket costs and an increase of 2.05 prescriptions (p 5 .081) per patient year. The associations between Part D and emergency department use, hospitalizations, and preference-based health utility did not suggest cost offsets and were not statistically significant. Conclusions. Although there was a substantial reduction in out-of-pocket costs and a moderate increase in medication utilization among Medicare beneficiaries during the first year after Part D, there was no evidence of improvement in emergency department use, hospitalizations, or preference-based health utility for those eligible for Part D during its first year of implementation.

Pembrolizumab improves survival, increases QALYs, and can be considered as a cost-effective option compared to docetaxel in PD-L1 positive (TPS ≥50%) pre-treated advanced NSCLC patients in the US.

This study was supported by funding from Merck & Co., which reviewed and approved the manuscript before journal submission. Wang, Pellissier, Xu, Stevinson, and Liu are employees of, and own stock in, Merck & Co. Chmielowski has served as a paid consultant for Merck & Co. and received a consultant fee for clinical input in connection with this study. Chmielowski also reports receiving advisory board and speaker bureau fees from multiple major pharmaceutical companies. Wang led the modeling and writing of the manuscript. Chmielowski, Xu, Stevinson, and Pellissier contributed substantially to the modeling design and methodology. Liu led the data collection work and contributed substantially to writing the manuscript. In conducting the analysis and writing the manuscript, the authors followed Merck publication polices and the "cost-effectiveness analysis alongside clinical trials-good research practices and the CHEERS reporting format as recommended by the International Society for Pharmacoeconomics and Outcomes Research.

Aim: To describe recent evolution in treatment patterns and outcomes for advanced melanoma (AMel). Methods: This retrospective observational study analyzed de-identified electronic health record data from the Flatiron Health database for 1140 adult patients who initiated first-line therapy for AMel from 1 January 2014 to 30 June 2016 with follow-up through 28 February 2017. Results: The most common first-line regimens were ipilimumab-based therapies (34%), anti-PD-1 monotherapy (26%) and BRAF/MEK inhibitor(s) (20%). First-line ipilimumab-based and BRAF inhibitor regimens decreased after the third quarter of 2014 (3Q2014), and by 2Q2016, 55 and 91% of BRAF-mutant and BRAF wild-type cohorts, respectively, received a first-line anti-PD-1 regimen. Median overall survival from first-line initiation for all patients was 18.8 months (95% CI: 16.3–23.3). Conclusion: Results illustrate changing paradigms of therapy and real-world patient outcomes for AMel.