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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]

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]

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]

Post-COVID-19 conditions (PCC) is an umbrella term that encompasses a range of signs, symptoms and conditions present weeks after the acute phase of a SARS-CoV-2 infection. This systematic literature review summarises the heterogeneous methodology used to measure PCC across real-world studies and highlights trends by region, age group, PCC follow-up period and data source., Methods: Medline, EMBASE and the Cochrane Library were searched and supplemented with conference and grey literature searches. Eligible studies included individuals with (1) PCC or (2) a positive SARS-CoV-2 test or COVID-19 diagnosis who were followed over time. Included studies were published in English between 1 January 2020 and 14 November 2022., Findings: Of 291 publications included, 175 (60%) followed individuals with confirmed COVID-19 over time for PCC and 116 (40%) used a prespecified PCC definition. There was substantial heterogeneity in study design, geography, age group, PCC conditions/symptoms assessed and their classification and duration of follow-up. Among studies using a prespecified PCC definition, author-defined criteria (51%) were more common than criteria recommended by major public health organisations (19%). Measurement periods for PCC outcomes from date of acute COVID-19 test were primarily 3 to <6 months (39.2%), followed by 6 to <12 months (27.5%) and <3 months (22.9%). When classified by organ/system, constitutional-related PCC were the most frequently assessed in adult (86%) and paediatric (87%) populations. Within constitutional symptoms, fatigue was most frequently assessed in adult (91.6%) and paediatric (95.0%) populations, followed by fever/chills (37.9% and 55%, respectively)., Conclusions: PCC definitions are heterogenous across real-world studies, which limits reliable comparisons between studies. However, some similarities were observed in terms of the most frequently measured PCC-associated symptoms/conditions, which may aid clinical management of patients with PCC.CRD42022376111., Competing Interests: Competing interests: JY, KMA, LJMcG, JLN, MHK, AER and JRS are employees of Pfizer and may hold stock or stock options. LH, KM, IW and VB are employees of Adelphi Values PROVE., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Background: The breadth of protection of National Immunisation Programmes (NIPs) across Europe varies, however, this has not been assessed within published literature. Therefore, a framework was developed to assess the comprehensiveness of pediatric NIPs in Europe. This study aimed to validate and further develop criteria used to cluster countries into three tiers., Research Design and Methods: Independent Europe-based experts ( n  = 23) in the field of pediatric vaccination were invited to participate in a double-blinded modified Delphi panel, with two online survey rounds and a virtual consensus meeting. Consensus was defined as ≥ 80% of experts rating their agreement/disagreement on a 9-point Likert scale., Results: The number of preventable diseases covered by an NIP, simplification of the vaccination calendar, strengthened protection by increasing serotype, degree of funding and epidemiological factors were considered key concepts for consideration of the comprehensiveness of pediatric NIPs in Europe. Experts highlighted that the framework should be extended to include adolescent vaccines and populations up to 18 years of age. Consensus regarding further amendments to the framework was also reached., Conclusions: This Delphi panel validated a framework to assess the comprehensiveness of European NIPs. The framework can be used to facilitate discussions to help countries improve and expand the breadth of protection provided by their NIP.

Background: Assessing the cost-effectiveness of treatments in rheumatoid arthritis (RA) is of growing importance due to the chronic nature of the disease, rising treatment costs, and budget-constrained health care systems. This analysis assesses the cost-effectiveness of modified-release (MR) prednisone compared with immediate-release (IR) prednisone for the treatment of morning stiffness due to RA., Methods: A health state transition model was used to categorize RA patients into four health states, defined by duration of morning stiffness. The model applied a 1-year time horizon and adopted a UK National Health Service (NHS) perspective. Health benefits were measured in quality-adjusted life years (QALYs) and the final output was the incremental cost-effectiveness ratio (ICER). Efficacy data were derived from the CAPRA-1 (Circadian Administration of Prednisone in Rheumatoid Arthritis) study, drug costs from the British National Formulary (BNF), and utility data from a direct elicitation time-trade-off (TTO) study in the general population. Sensitivity analyses were conducted., Results: Mean treatment costs per patient were higher for MR-prednisone (£649.70) than for IR-prednisone (£46.54) for the duration of the model. However, the model generated an incremental QALY of 0.044 in favor of MR-prednisone which resulted in an ICER of £13,577. Deterministic sensitivity analyses did not lead to significant changes in the ICER. Probabilistic sensitivity analysis reported that MR-prednisone had an 84% probability of being cost-effective at a willingness-to-pay threshold of £30,000 per QALY. The model only considers drug costs and there was a lack of comparative long-term data for IR-prednisone. Furthermore, utility benefits were not captured in the clinical setting., Conclusion: This analysis demonstrates that, based on the CAPRA-1 trial and directly elicited public preference values, MR-prednisone is a cost-effective treatment option when compared with IR-prednisone for RA patients with morning stiffness over one year, according to commonly applied UK thresholds (£20,000-£30,000 per QALY). Further research into the costs of morning stiffness in RA is required.