Search

Background: Inhaled drug delivery is the cornerstone treatment for asthma and chronic obstructive pulmonary disease (COPD). However, use of inhaler devices can be challenging, potentially leading to critical errors in handling that can significantly reduce drug delivery to the lungs and effectiveness of treatment.Methods: A systematic review was conducted to define 'critical' errors and their impact on health outcomes and resource use between 2004 and 2016, using key search terms for inhaler errors in asthma and COPD (Search-1) and associated health-economic and patient burden (Search-2).Results: Search-1 identified 62 manuscripts, 47 abstracts, and 5 conference proceedings (n = 114 total). Search-2 identified 9 studies. We observed 299 descriptions of critical error. Age, education status, previous inhaler instruction, comorbidities and socioeconomic status were associated with worse handling error frequency. A significant association was found between inhaler errors and poor disease outcomes (exacerbations), and greater health-economic burden.Conclusions: We have shown wide variations in how critical errors are defined, and the evidence shows an important association between inhaler errors and worsened health outcomes. Given the negative impact diminished disease outcomes impose on resource use, our findings highlight the importance of achieving optimal inhaler technique, and a need for a consensus on defining critical and non-critical errors. [ABSTRACT FROM AUTHOR]

The primary objective of this review is to develop a conceptual model for Crohn's disease (CD) outlining the disease burden for patients, healthcare systems and wider society, as reported in the scientific literature. A search was conducted using MEDLINE, PsycINFO, EconLit, Health Economic Evaluation Database and Centre for Reviews and Dissemination databases. Patient-reported outcome (PRO) measures widely used in CD were reviewed according to the US FDA PRO Guidance for Industry. The resulting conceptual model highlights the characterization of CD by gastrointestinal disturbances, extra-intestinal and systemic symptoms. These symptoms impact physical functioning, ability to complete daily activities, emotional wellbeing, social functioning, sexual functioning and ability to work. Gaps in conceptual coverage and evidence of reliability and validity for some PRO measures were noted. Review findings also highlight the substantial direct and indirect costs associated with CD. Evidence from the literature confirms the substantial burden of CD to patients and wider society; however, future research is still needed to further understand burden from the perspective of patients and to accurately understand the economic burden of disease. Challenges with existing PRO measures also suggest the need for future research to refine or develop new measures. [ABSTRACT FROM AUTHOR]

Background Despite overall progress in treatment of autoimmune diseases, patients with systemic lupus erythematosus (SLE) experience many inflammatory symptoms representing an unmet medical need. This study aimed to create a conceptual model of the humanistic and economic burden of SLE, and review the patient-reported outcomes (PROs) used to measure such concepts in SLE clinical trials. Methods A conceptual model for SLE was developed from structured review of published articles from 2007 to August 2013 identified from literature databases (MEDLINE, PsycINFO, EconLit) plus other sources (PROLabels, FDA/EMA websites, Clinicaltrials.gov). PROs targeting key symptoms/impacts were identified from the literature. They were reviewed in the context of available guidance and assessed for face and content validity and psychometric properties to determine appropriateness for use in SLE trials. Results The conceptual model identified fatigue, pain, cognition, daily activities, emotional wellbeing, physical/social functioning and work productivity as key SLE concepts. Of the 68 articles reviewed, 38 reported PRO data. From these and the other sources, 15 PROs were selected for review, including SLE-specific health-related quality of life (HRQoL) measures (n = 5), work productivity (n = 1), and generic measures of fatigue (n = 3), pain (n = 2), depression (n = 2) and HRQoL (n = 2). The Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-Fatigue), Brief Pain Inventory (BPI-SF) and LupusQoL demonstrated the strongest face validity, conceptual coverage and psychometric properties measuring key concepts in the conceptual model. All PROs reviewed, except for three Lupusspecific measures, lacked qualitative SLE patient involvement during development. The Hospital Anxiety and Depression Scale (HADS), Short Form [36 item] Health Survey version 2 (SF-36v2),) EuroQoL 5-dimensions (EQ-5D-3L and EQ-5D-5L) and Work Productivity and Activity Impairment Questionnaire:Lupus (WPAI:Lupus) showed suitability for SLE economic models. Conclusions Based on the identification of key symptoms and impacts of SLE using a scientifically sound conceptual model, we conclude that SLE is a condition associated with high unmet need and considerable burden to patients. This review highlights the availability and need for diseasespecific and generic patient-reported measures of relevant domains of disease signs and symptoms, HRQoL and work productivity, providing useful insight for SLE clinical trial design. [ABSTRACT FROM AUTHOR]

Although many patients with type 2 diabetes are initially managed through lifestyle modification, most eventually require insulin therapy. However, insulin initiation is often delayed because of factors such as patients' resistance to insulin therapy and worries about injections. Such delays affect glycemic control, have a direct effect on patient encounters, and may affect medication adherence. Insulin pen delivery systems may address some of these concerns. This study had two phases. First, semi-structured qualitative interviews were conducted to identify the most important features of insulin delivery devices for prandial use from the perspective of patients (n = 8) and health care professionals (HCPs; n = 10). From phase 1, a 26-item questionnaire was developed. In phase 2, patients (n = 33 insulin naive, n = 78 pen users) and HCPs (n = 151) were asked to indicate the most important features to them in insulin delivery devices. Patients then simulated injection using three different pens (SoloSTAR, KwikPen, and FlexPen) and ranked them based on the same features. The most important features were knowing that the entire dose has been injected, ease of reading the dose correctly, and ease of correcting if the dose is over-dialed. In the simulation study, KwikPen and SoloSTAR scored significantly higher (paired t test, P < 0.05) than FlexPen on 'knowing if you have injected the entire dose' (mean score out of 10: KwikPen, 8.9; SoloSTAR, 8.6; and FlexPen, 8.4). No other significant differences among the pens were noted in usability or design, and the mean ranking (from 1 to 3) of the pens was similar (KwikPen, 2.0; FlexPen, 2.1; and SoloSTAR, 1.9). By identifying which insulin delivery pens offer these features, HCPs can choose the most appropriate delivery device for patients, which may lead to earlier insulin initiation, greater patient adherence, and better clinical outcomes. [ABSTRACT FROM AUTHOR]

Introduction: Nearly every individual with schizophrenia is affected by cognitive decline. The aim of this literature review was to: (a) describe the humanistic burden of cognitive impairment associated with schizophrenia (CIAS); (b) develop a conceptual model that depicts the signs and symptoms of CIAS along with key concepts important to patients; and (c) consider the adequacy of potential patient-reported outcome (PRO) instruments for assessing future treatments. Methods: The following electronic databases were searched for articles published between January 1999 and November 2009 related to CIAS and PROs, or cost of illness: Medline; Embase; PsycINFO; the Health Economic Evaluation Database; and the National Health Service Economic Evaluation Database and Health Technology Assessment databases at the Centre for Reviews and Dissemination, University of York. Results: The literature search revealed 3950 abstracts, of which 101 articles were reviewed in detail. Cognitive functions affected include memory, attention/concentration, problem solving, learning, executive function, processing speed, and social cognition. Cognitive impairment impacts the ability of individuals to carry out activities of daily living, work productively, function socially, and adhere to treatment. These effects have economic ramifications through increased direct and indirect costs associated with the treatment of schizophrenia. The literature revealed 39 PRO instruments that have been used to assess functioning. However, no single instrument captures all key concepts of importance to patients with schizophrenia. Conclusion: The significant burden from CIAS for patients and society has implications for designing future treatments and health strategies to improve functional outcomes. [ABSTRACT FROM AUTHOR]

Aims and background With the recent emergence of immunotherapies and novel targeted treatments for advanced and metastatic melanoma such as selective B-Raf inhibitors and checkpoint inhibitors, the treatment landscape in Europe has changed considerably. The aim of this review was to provide an overview of current treatment pathways in Europe for the treatment of advanced melanoma, unresectable stage III–IV. Methods A literature search of four databases was conducted to identify publications reporting on the treatment patterns of advanced and metastatic melanoma (stage III–IV) in European populations. Results Seven full-text publications and two conference abstracts reported on observational studies of melanoma treatment practices in France, Italy and the United Kingdom. Treatment patterns were identified for two time periods: 2005–2009 and 2011–2012. Common treatments reported for both periods included chemotherapy with dacarbazine, fotemustine or temozolomide. The main differences between the two periods were the introduction and prescription of immunotherapy ipilimumab and targeted therapy vemurafenib between 2011 and 2012. Across the three countries studied, the types of treatments prescribed between 2005 and 2009 were relatively similar, however, with noticeable differences in the frequency and priority of administration. Conclusion Treatment practices for advanced melanoma vary markedly across different European countries and continue to evolve with the introduction of new therapies. The results of this review highlight a considerable evidence gap with regards to recent treatment patterns for advanced melanoma in Europe, especially post-2011 after the introduction of novel therapeutic agents, and more recently with the introduction of programmed cell death 1 inhibitors. [ABSTRACT FROM AUTHOR]