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The use of patient-reported outcome instruments (PRO) in clinical trials in order to capture the impacts of treatment on patients is widespread. However, regulatory agencies have over the past decade highlighted the need for PROs that are fit for purpose and target relevant aspects of the patient's condition. Many legacy PROs were developed with little patient input, are lengthy, and may lack relevance having not been modified or adapted as medical treatments have advanced. Computer-adaptive test (CAT) systems provide the possibility of targeted approaches to capturing patient-centric data, while minimising patient burden. Coupled with greater patient input in the development of PROs, CAT offers the opportunity of overcoming the shortcomings of the previous generation of PROs. This paper describes the some of the issues facing legacy PROs, current regulatory guidance, and initiatives, such as the Patient-Reported Outcome Measurement Information System (PROMIS), as well as the early signs of use of CAT to capture PRO data in clinical trials.
Arguably, the most common structure currently adopted for oncology modelling is the three-state partitioned survival model with the following states: stable disease, post-progression and dead. This design can, therefore, be adopted to capture the progressive nature of cancer. This chapter outlines the three-state model approach as well as introducing several other key aspects of economic modelling in oncology.
PURPOSE: The Weight-specific Adolescent Instrument for Economic evaluation (WAItE) is a 7-item condition-specific tool assessing the impact of weight status on seven dimensions of quality of life. The content of the WAItE was developed with both treatment-seeking and non-treatment-seeking adolescents aged 11-18 years. The aim of this study was to assess the psychometric properties of the WAItE in adolescent and adult populations.
METHODS: Treatment-seeking adolescents with obesity (females n = 155; males n = 123; mean age = 13.3; 13.1 years, respectively) completed the WAItE twice. An adult general population sample completed the WAItE via an online survey (females n = 236; males n = 231; mean age = 41.2; 44.3 years, respectively). The Partial Credit Model was applied to the data and item fit evaluated against published criteria.
RESULTS: The WAItE had a unidimensional structure both for adolescents and adults. There was no item misfit observed for either participant samples and no differential item functioning (DIF) was present by age or gender for the adolescents. Some DIF was observed across age groups for the adult sample. For the adolescent sample, stable item locations were observed over time.
CONCLUSIONS: The aim of the WAItE is to assess the impact of weight status on the lives of adolescents in cost-effectiveness evaluation of weight management programmes. The results of this study demonstrated that the WAItE has reliable psychometric properties. The instrument may therefore be used to aid informed decision around the identification of cost-effective weight management programmes in both adolescent and adult populations.
OBJECTIVES: This study investigated which databases and which combinations of databases should be used to identify economic evaluations (EEs) to inform systematic reviews. It also investigated the characteristics of studies not identified in database searches and evaluated the success of MEDLINE search strategies used within typical reviews in retrieving EEs in MEDLINE.
METHODS: A quasi-gold standard (QGS) set of EEs was collected from reviews of EEs. The number of QGS records found in nine databases was calculated and the most efficient combination of databases was determined. The number and characteristics of QGS records not retrieved from the databases were collected. Reproducible MEDLINE strategies from the reviews were rerun to calculate the sensitivity and precision for each strategy in finding QGS records.
RESULTS: The QGS comprised 351 records. Across all databases, 337/351 (96 percent) QGS records were identified. Embase yielded the most records (314; 89 percent). Four databases were needed to retrieve all 337 references: Embase + Health Technology Assessment database + (MEDLINE or PubMed) + Scopus. Four percent (14/351) of records could not be found in any database. Twenty-nine of forty-one (71 percent) reviews reported a reproducible MEDLINE strategy. Ten of twenty-nine (34.5 percent) of the strategies missed at least one QGS record in MEDLINE. Across all twenty-nine MEDLINE searches, 25/143 records were missed (17.5 percent). Mean sensitivity was 89 percent and mean precision was 1.6 percent.
CONCLUSIONS: Searching beyond key databases for published EEs may be inefficient, providing the search strategies in those key databases are adequately sensitive. Additional search approaches should be used to identify unpublished evidence (grey literature).
Health economics allows decision-makers to identify the most efficient allocation of limited resources. It involves measuring the benefits of different health care interventions in such a way that they can be compared across the whole health system. Quality-adjusted life years (QALYS) allow us to measure the benefires that acrue from both increased length of life (i.e. life expectancy) and improved quality of life - or even a combination of both. Once we have estimated the QALYS gains from a particular intervention, these can be compared against the QALYS that must be lost elsewhere in the health system in order to release funds to pay for a new intervention. If the QALY gains from a new intervention outweigh the opportunity costs of moving that money form where it is currently spent, then the health care system as a whole will benefit from the new intervention.
OBJECTIVES: To determine the effectiveness and cost implications of using antiseptic passive disinfecting caps (disinfecting alcohol-containing cap that twists onto needle free connector intravenous access devices) as an adjunct to standard care in patients with central or peripheral lines in a NHS hospital setting compared with standard care alone.
METHODS: A systematic review identified studies reporting on the use of antiseptic barrier caps for the reduction of central and peripheral line associated infections. Studies reporting on central line associated bloodstream infections (CLABSI) in general hospital patients and patients in an intensive care setting were pooled using meta-analysis to calculate incidence rate ratios (IRR). A decision-analytic model estimated the costs and consequences associated with the use of an antiseptic barrier cap in these groups of patients from a UK NHS perspective, using the IRRs. Other parameter inputs were obtained from published literature using UK specific sources where possible. Deterministic sensitivity analyses (DSA) and probabilistic sensitivity analyses (PSA) were conducted to assess the robustness of results.
RESULTS: The meta-analyses produced IRRs in favour of the passive disinfecting cap of 0.43 (95% CI 0.22, 0.82) in general hospital patients and 0.29 (95% CI 0.09, 0.97) in intensive care patients. The adjunctive use of the passive disinfecting cap dominated standard care alone in both settings in the economic model, as it was both cost saving and reduced the number of CLABSIs. DSA indicated these results were robust to changes in individual input parameters, and PSA estimated the results to be cost saving in 96.4% of iterations in the general hospital setting and 86.3% in the intensive care setting.
CONCLUSIONS: Based on currently available evidence, the analyses suggest that using passive disinfecting caps to protect needle free connector intravenous access devices is a cost-effective use of resources for patients with central or peripheral line catheters in the UK NHS.
OBJECTIVES: To estimate the relative effectiveness of enzalutamide in chemotherapy-naive metastatic castration-resistant prostate cancer by conducting a systematic literature review and a network meta-analysis (NMA).
METHODS: A systematic literature review identified randomized controlled trials comparing enzalutamide, abiraterone/prednisone, radium-223, sipuleucel-T, or docetaxel with each other or placebo in chemotherapy-naive or mixed populations (with and without prior chemotherapy) with asymptomatic/mildly symptomatic metastatic castration-resistant prostate cancer. Feasibility assessment evaluated the trials' suitability for NMA inclusion. The main outcomes were hazard ratios (HRs) for overall survival (OS) and radiographic progression-free survival (rPFS).
RESULTS: Searches of relevant bibliographic databases, trial registers, Web sites, and conference abstracts conducted in October 2014 identified 25,712 records. Ten randomized controlled trials were eligible for the NMA. Enzalutamide was superior to placebo for OS and rPFS (fixed-effects model). NMA results (fixed-effects model) showed no evidence of a difference between enzalutamide and abiraterone/prednisone (HR 0.95 [95% CrI 0.77–1.16]), sipuleucel-T (HR 1.07 [95% CrI 0.84–1.37]), or radium-223 (HR 1.10 [95% CrI 0.87–1.37]) for OS. HRs were similar for the random-effects model. Nevertheless, results (fixed-effects model) suggested that enzalutamide was superior to abiraterone/prednisone (HR 0.59 [95% CrI 0.48–0.72]) and sipuleucel-T (HR 0.32 [95% CrI 0.25–0.42]) for rPFS. Results also suggested superiority of enzalutamide versus placebo, abiraterone/prednisone, or sipuleucel-T for time to chemotherapy.
CONCLUSIONS: For rPFS, the NMA suggests that enzalutamide is superior to abiraterone/prednisone and sipuleucel-T. There is no evidence of a statistically significant difference in OS between enzalutamide and abiraterone/prednisone, sipuleucel-T, or radium-223. Given the limitations in network construction and underlying assumptions made to complete these analyses, results should be interpreted with caution.
