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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]

Objective: To provide a framework for addressing payers' criteria during the development of pharmaceuticals.Methods: A conceptual framework was presented to an international health economic expert panel for discussion. A structured literature search (from 2010 to May 2015), using the following databases in Ovid: Medline(®) and Medline(®) In-Process (PubMed), Embase (Ovid), EconLit (EBSCOhost) and the National Health Service Economic Evaluation Database (NHS EED), and a 'grey literature' search, were conducted to identify existing criteria from the payer perspective. The criteria assessed by existing frameworks and guidelines were collated; the most commonly reported criteria were considered for inclusion in the framework. A mnemonic was conceived as a memory aide to summarise these criteria.Results: Overall, 41 publications were identified as potentially relevant to the objective. Following further screening, 26 were excluded upon full-text review on the basis of no framework presented (n = 13), redundancy (n = 11) or abstract only (n = 2). Frameworks that captured criteria developed for or utilised by the pharmaceutical industry (n = 5) and reimbursement guidance (n = 10) were reviewed. The most commonly identified criteria-unmet need/patient burden, safety, efficacy, quality-of-life outcomes, environment, evidence quality, budget impact and comparator-were incorporated into the summary framework. For ease of communication, the following mnemonic was developed: BEACON (Burden/target population, Environment, Affordability/value, Comparator, Outcomes, Number of studies/quality of evidence).Conclusions: The BEACON framework aims to capture the 'essence' of payer requirements by addressing the most commonly described criteria requested by payers regarding the introduction of a new pharmaceutical. [ABSTRACT FROM AUTHOR]

Introduction: Pressurized metered-dose inhalers (pMDI) such as fluticasone propionate and salmeterol (FP/SAL) are commonly used for the treatment of asthma in the UK. Previously, a budget impact analysis demonstrated that use of FP and formoterol fumarate (FP/FORM) pMDI as an alternative to FP/SAL pMDI, would be a cost-saving option for the UK National Health Service (NHS). This budget impact analysis aimed to update the existing analysis with prescription volume data and real-world evidence since the introduction of FP/FORM to the UK market.Methods: Patient Data (IMS Information Solutions UK Ltd) moving annual total (MAT) August 2015 were used to ascertain the number of units of pMDI prescribed. Annual costs to the NHS in terms of drug, administration, monitoring and adverse event costs, were used to estimate the potential budget impact for FP/FORM and FP/SAL. Costs were calculated for current prescription volumes (12% FP/FORM, 88% FP/SAL), and for different prescription volume scenarios (FP/FORM at 0%, 25%, 50% and 100%). Real-world evidence and budget impact at a clinical commissioning group (CCG) level were also considered.Results: Total annual costs per person year were less with FP/FORM (£625) than with FP/SAL (£734). Annual costs to the NHS based on the current prescription volumes and clinical trial data were estimated at £210.0M, however, based on real-world evidence, costs were estimated at £179.8M. For all scenarios with increased FP/FORM prescription volumes, the annual total costs to the NHS decreased. This was reflected at a CCG level.Conclusion: The use of FP/FORM as an alternative to FP/SAL can result in cost savings for the NHS when assessing drug, administration, monitoring and adverse events costs. The inclusion of data released since the launch of FP/FORM within the budget impact analysis demonstrates that the potential cost savings to the NHS that were previously published are being translated to clinical practice.Funding: Mundipharma, UK. [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]

Introduction: Asthma affects 30 million people in Western Europe, leading to substantial burden on healthcare systems and economies. REcognise Asthma and LInk to Symptoms and Experience (REALISE™) was a large European survey across 11 countries assessing patient attitudes and behaviors towards their asthma. The present study utilizes REALISE™ data to understand resource use and absenteeism in asthma. Methods: Data were collected on absenteeism and healthcare resource use from 8000 asthma patients (aged 18-50 years) across the 11 countries. All data were patient reported. Odds ratios (ORs) were calculated against the country with the lowest proportion of respondents for hospitalization (as a proxy for lowest resource use). Results: Patient characteristics were broadly similar across countries. However, self-reported asthma control status varied. More than 50% of respondents in most countries considered primary healthcare professionals (HCPs), i.e., general practitioners and nurses, the main HCP they see about their asthma. However, in some countries, specialists or nurses were considered the main HCP. Hospitalization was lowest amongst patients in the Netherlands. Resource use and productivity loss varied widely across the countries; ORs for hospitalization ranged from 1 in Sweden to 4 in Norway and for productivity loss from 0.6 in Sweden to 2.6 in Italy, compared with the Netherlands. Conclusion: This study quantified utilization of healthcare resources in asthma (number of visits of HCPs, hospitalization, and accident and emergency visits) as well as absenteeism and showed that differences exist across countries. The differences in primary care and specialist use suggest a possible difference in healthcare delivery across countries. Funding: Mundipharma International Limited, Cambridge, UK. [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]

Introduction: The economic burden of asthma on the UK National Health Service (NHS) is the largest among allergic diseases. Current asthma guidelines recommend adding a long acting β 2-agonist (LABA) to a low-dose inhaled corticosteroid (ICS) in patients who are on ICS monotherapy and have uncontrolled asthma. The fixed-dose combination of fluticasone propionate and salmeterol xinafoate (FP/SAL), available in a pressurized metered-dose inhaler (pMDI) device, is the most commonly prescribed ICS/LABA combination. An additional fixed-dose combination of fluticasone propionate and formoterol fumarate (FP/FORM) in pMDI is now available. In a 12-week non-inferiority study, FP/FORM demonstrated comparable efficacy to FP/SAL. The present analysis estimates the annual budget impact for the UK NHS using FP/FORM as an alternative to FP/SAL. Methods: Current pMDI prescribing data were from a real-world UK patient database (Cegedim Strategic Data). Annual costs to the NHS for drug acquisition, administration, and monitoring were estimated for FP/FORM and FP/SAL and used to assess the potential budget impact for the NHS for the use of FP/FORM instead of FP/SAL. Varying rates of uptake, adherence, adverse event-related costs, and resource use associated with switching treatment were assessed in scenario analyses. Results: Assuming similar levels of ICS use with both regimens, annual drug acquisition costs per person were lower with FP/FORM (£412) than with FP/SAL (£509). The difference in acquisition costs and otherwise comparable input costs between the treatments, results in potential annual savings of £15,110,279 to the NHS, assuming uptake of FP/FORM over FP/SAL in 50% of existing patients. The introduction of FP/FORM results in cost savings for the NHS in all of the assessed scenario analyses. Conclusions: The comparable efficacy and lower acquisition costs of FP/FORM compared with FP/SAL make it a cost-saving option for the UK NHS for the treatment of asthma patients requiring combination maintenance therapy using a pMDI. [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]

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]

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]

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, EMBASE, 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 well-being, 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 Lupus-specific 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 disease-specific 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]