A reference set of utility values for T2DM and its complications in line with NICE requirements was identified. This research illustrates the challenges associated with systematically selecting utility data for economic evaluations.

This study was funded by InterMune International AG, a wholly owned Roche subsidiary since 2014. Fisher was previously employed by InterMune UK, a wholly owned Roche subsidiary, until July 2015. He is currently employed by FIECON, which has received funding from F. Hoffmann-La Roche for consulting services. Nathan has received consulting fees from Roche-Genentech and Boehringer Ingelheim. He is also on the speakers' bureau for Roche-Genentech and Boehringer Ingelheim and has received research funding from both companies. Hill was previously employed by InterMune UK until October 2014. Hill and Marshall are employees of MAP BioPharma, which has received funding from F. Hoffmann-La Roche for consulting services. Dejonckheere and Thuresson are employees of F. Hoffmann-La Roche. Maher has received grants, consulting fees, and speaker fees from GlaxoSmithKline and UCB, and grants from Novartis. He has also received consulting fees and speaker fees from AstraZeneca, Bayer, Biogen Idec, Boehringer Ingelheim, Cipla, Lanthio, InterMune International AG, F. Hoffmann-La Roche, Sanofi-Aventis, and Takeda. Maher is supported by a National Institute for Health Research Clinician Scientist Fellowship (NIHR Ref: CS: -2013-13-017). Study concept and design were contributed by Fisher, Hill, Marshall, and Dejonckheere. Fisher, Nathan, and Thuresson collected the data, along with Hill and Marshall. Data interpretation was performed by Fisher, Maher, Nathan, and Dejonckheere. The manuscript was written primarily by Fisher, along with Maher and Dejonckheere, and revised by Fisher and Maher, along with the other authors.

For patients with late-stage (metastatic) breast cancer, the impact of treatment on health-related quality of life is a key factor in decision-making. A systematic review was conducted to identify health state utility values (HSUVs) for late-stage breast cancer, derived using methods preferred by health technology assessment (HTA) agencies, by treatment line. The aim was to generate a list of HSUVs, that could help to justify the values used to populate cost-utility models. Areas covered: Ten electronic databases, international congress websites and online HSUV databases were searched (January 1995-May 2014) for HSUVs for adults with late-stage breast cancer that had been derived from methods favoured by HTA agencies. Publications were included only if they reported studies that originated HSUVs. Expert commentary: Large numbers of HSUVs are available for late-stage breast cancer in the published literature. Contrary to expectations, the HSUVs reported in the literature vary greatly for some health states. As a result, the choice of HSUV can have considerable implications for the outcomes of economic evaluations. Standardization of HSUV methodology is expected to reduce variability; however, further research is recommended for assessing the sensitivity of generic preference-based measures in late-stage (metastatic) breast cancer.

Given the improvement in clinical efficacy and a favorable cost per QALY, the addition of pertuzumab in the neoadjuvant setting represents an attractive treatment option for HER2-positive eBC patients.

The results indicate that atazanavir/r is cost-saving and more effective compared to lopinavir/r for patients who have previously not been exposed to antiretroviral drugs in Sweden.

Results: Between 3/2009 and 12/2015, 48 pts were recruited; 46 were assessable (median age 72 ys; range 34-86); 36 pts had de novo DLBCL; most pts had unfavourable features, with an IPI ≥2 in 38 (83%) pts. Salvage therapy before LENA contained high doses of cytarabine or ifosfamide in two-thirds of cases; response at trial registration was complete in 26 pts and partial in 20.LENA was well tolerated after an average of 16 courses/pt (range 3-82); 16 pts received LENA for ≥2 years. 3 pts died of toxicity during maintenance (intestinal infarction, meningitis and sudden death) and 2 died due to myelodysplastic syndrome at 31 and 62 months.LENA was interrupted due to toxicity in other 6 pts, and 25 required dose reduction (transient in 21), mostly due to neutropenia and rash. With the exception of neutropenia, g4 toxicities occurred in <1% of courses. Infections were rare and well controlled with oral antibiotics.After 1 year from registration, 31 pts were progression free, which was significantly higher than the pre-determined efficacy threshold (n≥19).At a median follow-up of 5 years and a median observation period from LENA completion of 35 (8-101) months, 22 pts remain relapsefree, with a 1-and 5-yr PFS of 67 ± 7% and 48 ± 7%, respectively. The duration of response to LENA was longer than response to prior treatment in 30 (65%) pts, and benefit was observed both in de novo and transformed DLBCL, and in GCB and nonGCB subtypes. 27 (59%) pts are alive, with a 1-and 5-yr OS of 80 ± 6% and 58 ± 7%, respectively.Conclusions: These long-term results soundly promote the use of LENA maintenance in pts with chemosensitive relapse of DLBCL not eligible for or failed after ASCT. LENA was well tolerated in this elderly population, even among pts treated for ≥2 ys. Further investigations on immunomodulatory drugs as maintenance in these highrisk pts are warranted. Background: To inform payers/HTA organisations regarding estimated survival benefits when all events have not occurred, statistical methods are used to model projected clinical benefits/costs of new interventions. We estimated overall survival (OS) benefit associated with polatuzumab vedotin (pola) + bendamustine (B) + rituximab (R) vs BR alone in patients (pts) with R/R DLBCL. Methods: Data from a Ph2 randomised controlled trial (RCT; GO29365; NCT02257567; Sehn et al, ASH 2018) of pola-BR vs BR in pts with R/R DLBCL were used (N = 80; follow up 27.6 months [mo]).Most disease progression events occurred ≤12 mo from treatment initiation, after which fewer events were observed, causing Kaplan-Meier estimates of progression-free survival (PFS) and OS to plateau (Fig. A). A mixture model was therefore explored alongside standard parametric survival models (Fig. B). Mixture models combine two processes: (1) OS for pts who died from the disease; (2) OS for long-term survivors (LTS; pts who had not died from their disease, informed by life tables given pts' age/gender/nationality). The mixture model was fitted to the RCT data; from this, the proportion of LTS is estimated....

BackgroundGlucagon-like peptide-1 (GLP-1) receptor agonists are indicated for improvement of glycemic control in adults with type 2 diabetes. Cost is one aspect of treatment to be considered, in addition to clinical benefits, when selecting optimal therapy for a patient. The objective of this study was to estimate the average dose usage and real world daily cost of the GLP-1 receptor agonists, exenatide twice daily and liraglutide once daily, in Germany, the Netherlands, and the UK.MethodsAdministrative databases were used to source the data from longitudinal records of dispensed prescriptions. Data were extracted from the IMS Longitudinal Prescription database which captures details of prescriptions dispensed in pharmacies. Information on the dispensed quantity of each product was used to estimate average daily usage per patient. Daily dose usage was multiplied by the public price per unit to estimate daily cost.ResultsThe dispensed volume in Germany corresponded to a mean dispensed daily dose of 16.81 μg for exenatide twice daily and 1.37 mg for liraglutide (mean daily cost €4.02 and €4.54, respectively). In the Netherlands, average dispensed daily doses of 17.07 μg and 1.49 mg were observed for exenatide twice daily and liraglutide (mean daily cost €3.05 and €3.97, respectively). In the UK, the mean dispensed volume corresponded to a daily usage of 20.49 μg for exenatide twice daily and 1.50 mg for liraglutide (mean daily cost £2.53 and £3.28, respectively).ConclusionEstimates of average daily dispensed doses of GLP-1 receptor agonists derived from pharmacy data in real world settings corresponded to the dosing recommendation of the summaries of product characteristics. Nevertheless, the mean daily cost of exenatide twice daily was lower than that of liraglutide in Germany, the Netherlands, and the UK. Such estimates can be used to inform health care decision-makers on the real world usage and cost of medications effective in achieving glycemic control in patients with type 2 diabetes.

The DES model predicts that increases in compliance may lead to considerable cost savings and health improvements. Therefore, when determining the cost effectiveness of a new antipsychotic, efficacy rates from clinical trials should not be taken at face value, but should be interpreted in tandem with expectations concerning compliance, in light of product characteristics such as adverse effects. These results further suggest that efforts to improve compliance among patients with schizophrenia are expected to prove cost effective if compliance gains and the resulting health improvements and cost savings are in balance with the additional costs.