Background  A wide range of methods have been used for estimating influenza‐associated deaths in temperate countries. Direct comparisons of estimates produced by using different models with US mortality data have not been published. Objective  Compare estimates of US influenza‐associated deaths made by using four models and summarize strengths and weaknesses of each model. Methods  US mortality data from the 1972–1973 through 2002–2003 respiratory seasons and World Health Organization influenza surveillance data were used to estimate influenza‐associated respiratory and circulatory deaths. Four models were used: (i) rate‐difference (using peri‐season or summer‐season baselines), (ii) Serfling least squares cyclical regression, (iii) Serfling–Poisson regression, (iv) and autoregressive integrated moving average models. Results  Annual estimates of influenza‐associated deaths made using each model were similar and positively correlated, except for estimates from the summer‐season rate‐difference model, which were consistently higher. From the 1976/1977 through the 2002/2003 seasons the, the Poisson regression models estimated that an annual average of 25 470 [95% confidence interval (CI) 19 781–31 159] influenza‐associated respiratory and circulatory deaths [9·9 deaths per 100 000 (95% CI 7·9–11·9)], while peri‐season rate‐difference models using a 15% threshold estimated an annual average of 22 454 (95% CI 16 189–28 719) deaths [8·6 deaths per 100 000 (95% CI 6·4–10·9)]. Conclusions  Estimates of influenza‐associated mortality were of similar magnitude. Poisson regression models permit the estimation of deaths associated with influenza A and B, but require robust viral surveillance data. By contrast, simple peri‐season rate‐difference models may prove useful for estimating mortality in countries with sparse viral surveillance data or complex influenza seasonality.

BackgroundLipodystrophy (LD; non-human immunodeficiency virus [HIV]-associated) syndromes are a rare body of disorders for which true prevalence is unknown. Prevalence estimates of rare diseases are important to increase awareness and financial resources. Current qualitative and quantitative estimates of LD prevalence range from ~0.1 to 90 cases/million. We demonstrate an approach to quantitatively estimate LD prevalence (all, generalized, and partial) through a search of 5 electronic medical record (EMR) databases and 4 literature searches.MethodsEMR and literature searches were conducted from 2012 to 2014. For the EMR database searches (Quintiles, IMS LifeLink, General Electric Healthcare, and Humedica EMR), LD cases were identified by the International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM) code 272.6 (United Kingdom General Practice Research Database used other diagnostic codes to identify LD) plus additional LD-associated clinical characteristics (patients with HIV or documented HIV treatment were excluded). Expert adjudication of cases was used for the Quintiles database only. Literature searches (PubMed and EMBASE) were conducted for each of the 4 major LD subtypes. Prevalence estimates were determined by extrapolating the total number of cases identified for each search to the database population (EMR search) and European population (literature search).ResultsThe prevalence range of all LD across all EMR databases was 1.3–4.7 cases/million. For the adjudicated Quintiles search, the estimated prevalence of diagnosed LD was 3.07 cases/million (95% confidence interval [CI], 2.30–4.02), 0.23 cases/million (95% CI, 0.06–0.59) and 2.84 cases/million (95% CI, 2.10–3.75) for generalized lipodystrophy (GL) and partial lipodystrophy (PL), respectively. For all literature searches, the prevalence of all LD in Europe was 2.63 cases/million (0.96 and 1.67 cases/million for GL and PL, respectively).ConclusionLD prevalence estimates are at the lower range of previously established numbers, confirming that LD is an ultra-rare disease. The establishment of diagnostic criteria and coding specific to the 4 major LD subtypes and future studies/patient registries are needed to further refine our estimates.

Background Pembrolizumab, a monoclonal antibody against programmed death ligand 1 (PD-L1), is approved by several regulatory agencies for first-line treatment of metastatic non-small-cell lung cancer (NSCLC) with a PD-L1 tumor proportion score (TPS) ≥ 50% and no epidermal growth factor receptor (EGFR) or anaplastic lymphoma kinase genomic tumor aberrations. This study was conducted from the perspective of the Hospital Authority in Hong Kong and aimed to evaluate the cost effectiveness of a biomarker (PD-L1) test-and-treat strategy (BTS), in which patients with a TPS ≥ 50% received pembrolizumab and other patients received platinum doublet chemotherapy versus all patients receiving platinum doublet chemotherapy. Methods The model used a partitioned survival approach to estimate the incremental cost-effectiveness ratio (ICER) expressed as the cost per quality-adjusted life-year (QALY) gained. The clinical efficacy, utility and safety data were derived from the KN024 trial. Costs and health outcomes were projected over a 10-year time horizon and discounted at 3% per year. Costs for drug acquisition, PD-L1 testing, drug administration and disease management were used. Sensitivity analyses were conducted to evaluate the robustness of results. Results The BTS approach led to an increase of 0.29 QALYs at an additional cost of Hong Kong dollars (HK$) 249,077 (US$31,933) compared with platinum doublet chemotherapy, resulting in an ICER of HK$865,189 (US$110,922) per QALY gained. This is lower than the World Health Organization cost-effectiveness threshold of three times the 2016 gross domestic product (GDP) per capita for Hong Kong of HK$1017,819 (US$130,490). Probabilistic sensitivity analyses showed a 59.4% chance that the ICER would be below this threshold. Conclusion First-line treatment with pembrolizumab in a BTS to identify patients with NSCLC with PD-L1 TPS ≥ 50% can be considered cost effective in Hong Kong compared with platinum doublet chemotherapy based on a three-times GDP per capita threshold. However, local data on clinical efficacy and safety were not available to estimate overall survival (OS) and progression-free survival (PFS) specific to patients with NSCLC in Hong Kong. Further, uncertainty is inherent in the survival projections/extrapolation of PFS and OS beyond the trial period, and future research may help to further inform these parameters.A programmed death ligand 1 (PD-L1) test-and-treat strategy for the use of pembrolizumab in patients undergoing first-line treatment for non-small-cell lung cancer (NSCLC) with PD-L1 expression ≥ 50% is associated with a gain of 0.29 quality-adjusted life-years (QALYs), at an additional total cost of Hong Kong dollars (HK$) 249,077 per patient compared with platinum doublet chemotherapy (incremental cost-effectiveness ratio [ICER] of HK$865,189 per QALY gained) (year 2016 values).Key cost-effectiveness drivers were the drug acquisition costs and projections of improved survival.The ICER was below the threshold of three times gross domestic product per capita fo...

Our model predicted that universal childhood hepatitis A vaccination led to significant reductions in hepatitis A mortality and morbidity. Consequently, universal vaccination was cost saving compared with a regional vaccination policy. Herd protection effects of hepatitis A vaccination programs had a significant impact on hepatitis A mortality, morbidity, and cost-effectiveness ratios.

This analysis showed that DAPA was cost-saving compared with GLP-1RA and DPP-4i, and cost-effective compared with SU and TZD in the US setting over 1 year. Furthermore, the results suggest that, among the four composite clinical end-points, change in weight and SBP had an impact on cost-effectiveness results.