Purpose: To emulate a hypothetical target trial assessing the effect of initiating 5-fluorouracil, folinic acid, irinotecan, and oxaliplatin (FOLFIRINOX) versus gemcitabine plus nab-paclitaxel (GN) within 8 weeks of diagnosis on overall survival.
Methods: An observational cohort study was conducted using population-level data from Alberta, Canada. Individuals diagnosed with advanced pancreatic cancer between April 2015 and December 2019 were identified through the provincial cancer registry and followed until March 2021.
Importance: Quantitative assessment of bias from unmeasured confounding and missing data can help evaluate uncertainty in findings from indirect comparisons using real-world data (RWD).
Objective: To compare the effectiveness of alectinib vs ceritinib in terms of overall survival (OS) in patients with ALK-positive, crizotinib-refractory, non-small cell lung cancer (NSCLC) and to assess the sensitivity of these findings to unmeasured confounding and missing data assumptions.
Design, Setting, And Participants: This comparative effectiveness research study compared patients from 2 phase 2 alectinib trials and real-world patients.
Data other than that from clinical trials are important for healthcare decision making. However, unlike the vocal calls seen for more open access to trial data, there are limited efforts being made to ensure that agencies that collect real-world data (RWD) share this, despite its importance. There are many RWD sources across the world that could be readily exploited for research once shared.
View Article and Find Full Text PDFIn order to understand the current state of data sharing in observational research studies, we reviewed data sharing statements of observational studies published in a general medical journal, the British Medical Journal. We found that the majority (63%) of observational studies published between 2015 and 2017 included a statement that implied that data used in the study could not be shared. If the findings of our exploratory study are confirmed, room for improvement in the sharing of real-world or observational research data exists.
View Article and Find Full Text PDFReal-world data is that collected outside the constraints of controlled clinical trials and is increasingly informing decision-making in healthcare. The landscape of real-world data in the United Kingdom is set to evolve over the coming months as the government plans to build on databases currently in place by collecting patient data from all family practices and linking this information with hospital records. This initiative, called care.
View Article and Find Full Text PDFIntroduction: Although occupational injuries are among the leading causes of death and disability around the world, the burden due to occupational injuries has historically been under-recognized, obscuring the need to address a major public health problem.
Methods: We established the Liberty Mutual Workplace Safety Index (LMWSI) to provide a reliable annual metric of the leading causes of the most serious workplace injuries in the United States based on direct workers compensation (WC) costs.
Results: More than $600 billion in direct WC costs were spent on the most disabling compensable non-fatal injuries and illnesses in the United States from 1998 to 2010.
Aims: This population-based, retrospective study quantified the rates of all-cause and pneumococcal pneumonia, meningitis and septicemia in Norway from 2008 to 2009 and determined the proportions of cases caused by pneumococcal vaccine serotypes.
Methods: Data on patients with all-cause and pneumococcal pneumonia, meningitis and septicemia were obtained from the Norwegian Patient Registry, which collects hospitalization data from all Norwegian public hospitals based on International Classification of Diseases codes. Norwegian Patient Registry case records linked to the Norwegian Surveillance System for Communicable Diseases provided serotype data for invasive pneumococcal disease in patients with microbiological cultures.
Purpose: Cervical dystonia (CD) is a hypertonic condition caused by damage to the central nervous system. Very few studies have assessed the overall economic burden of the disease. The objective of this study was to describe the utilization of health care resources of patients with CD in the UK primary care setting, using a large population-based database.
View Article and Find Full Text PDFBackground: Multiple sclerosis (MS) is a common complex disorder, with new treatment options emerging each year. Social media is being increasingly used to investigate opinions about drugs, diseases and procedures. In this descriptive exploratory study, we sought to investigate opinions about currently available MS treatments.
View Article and Find Full Text PDFPurpose: This study aimed to explore the burden of illness associated with cervical dystonia (CD), including possible demographic and humanistic correlates of baseline disease severity.
Methods: The analysis involved the five multinational randomized, placebo-controlled clinical trials that had evaluated the efficacy and safety of Dysport® in patients with CD, including assessment using the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS). Patient-level TWSTRS scores from the individual studies were meta-analysed to estimate disease severity at baseline.
Background And Purpose: To investigate resource use and burden associated with spina bifida (SB) in Germany.
Methods: A questionnaire was used to obtain information on SB-related healthcare resource use and assistive technologies used for the last 1 and 10 years. Individuals with SB were recruited at a tertiary specialist clinic.
Background: With anticonvulsant, anxiolytic, and analgesic properties, pregabalin has been evaluated for neuropathic pain and fibromyalgia (FM). These chronic conditions diminish patients' quality of life and increase healthcare utilization and costs.
Objective: To assess the current understanding of economic outcomes associated with pregabalin in neuropathic pain and FM.
Objective: Failure of closure of the neural tube often leads to serious malformations, including spina bifida, anencephaly and encephalocoele. Despite improvements in medical and surgical treatment, the burden associated with spina bifida is substantial but country-specific data are lacking outside North America. This study aims to improve understanding of the economic implications and burden associated with the morbidity of children and adults with neural tube defects (NTDs) in Germany.
View Article and Find Full Text PDFStudy Design: Retrospective cohort.
Objective: To explore the ability to capture low back pain (LBP) recurrence using wage-replacement (WR) data.
Summary Of Background Data: LBP can be a recurrent, fluctuating, and disabling condition.
Objectives: Endometriosis presents with significant pain as the most common symptom. Generic health measures can allow comparisons across diseases or populations. However, the Medical Outcomes Study Short Form 36 (SF-36) has not been validated for this disease.
View Article and Find Full Text PDFBackground: A variety of pharmacological and surgical treatments have been developed for heavy menstrual bleeding (HMB), which can have negative physical, social, psychological, and economic consequences. We conducted a systematic literature review and mixed-treatment-comparison (MTC) meta-analysis of available data from randomized controlled trials (RCTs) to derive estimates of efficacy for 8 classes of treatments for HMB, to inform health-economic analysis and future studies.
Methods: A systematic review identified RCTs that reported data on menstrual blood loss (MBL) at baseline and one or more follow-up times.
Objectives: To quantify the change in work productivity and activities of daily living in North American women with heavy menstrual bleeding (HMB) treated with estradiol valerate/dienogest (E2V/DNG; Qlaira(®)/Natazia(®)) compared to placebo.
Methods: Women in the United States and Canada, aged 20-53 years with an objective diagnosis of HMB and no recognizable anatomical pathology, were treated with E2V/DNG or placebo for seven cycles (196 days). Main outcome measures included work productivity (i.
Background: The purpose of this study was to quantify the impact of estradiol-valerate/dienogest (E(2)V/DNG; Qlaira(®)/Natazia(®)) on work productivity and activities of daily living in European and Australian women with heavy menstrual bleeding.
Methods: Women aged 18-54 years with a confirmed diagnosis of heavy menstrual bleeding and no recognizable pathology were recruited across nine European countries (the Czech Republic, Finland, Germany, Hungary, The Netherlands, Poland, Sweden, UK, and Ukraine) and Australia. The women were randomized to receive either E(2)V/DNG (n = 149) or placebo (n = 82) for seven treatment cycles (196 days).
Objective: Allergic diseases are the most common childhood illness in Thailand. Their prevalence has been rising over time, with several studies having revealed substantial economic burden. However, no such study had yet been conducted for Thailand.
View Article and Find Full Text PDFInt J Technol Assess Health Care
January 2012
Objectives: We compared Polish and Scottish Health Technology Assessment (HTA) process in order to elicit recommendations for future development of HTA methodological guidelines in Poland.
Methods: We studied the differences between Polish and Scottish HTA methodological guidelines. HTA recommendations issued by Polish HTA agency (AHTAPol) in the period January 1 through December 31, 2008, were benchmarked to HTA guidance published by Scottish Medical Consortium (SMC) for the same drug technology.
Objective: To examine the association between physical therapy (PT) amount and type (eg, active exercise and passive modalities) received postmeniscectomy with subsequent days of work disability.
Design: Historical prospective study.
Setting: Workers' compensation administrative claims data source.