Interpretation of the Expected Value of Perfect Information and Research Recommendations:A Systematic Review and Empirical Investigation

calculations are increasingly performed to guide and underpin research recommendations. An EVPI value that exceeds the estimated cost of research forms a necessary (although not sufficient) condition for further research to be considered worthwhile. However, it is unclear what factors affect researchers ’ recommendations and whether there is a notional threshold of positive returns below which research is not recommended. The objectives of this study were to explore whether EVPI and other factors have a bearing on research recommendations and to assess whether there exists a threshold EVPI below which research is typically not recommended. Methods. A systematic lit-erature review was undertaken to identify applied EVPI calculations in the health care field. Study characteris-tics were extracted, including funder, location, diseas

Intrathecal drug delivery systems for the management of chronic non-cancer pain : a systematic review of economic evaluations

Background: Intrathecal drug delivery (ITDD) systems are one of a limited number of management options for chronic non-cancer pain, cancer pain and spasticity. Concerns over their effectiveness and high initial costs led NHS England to decommission ITDD for patients with chronic non-cancer pain. However, the extent to which this decision is in line with existing economic evidence is unclear. The aim of this systematic review is to identify and review the existing evidence on the cost-effectiveness of ITDD for chronic non-cancer pain. Methods: Full and partial economic evaluations on ITDD were identified through systematic searches in MEDLINE, EMBASE, Web of Science and the National Health Service Centre for Reviews and Dissemination databases. Database searches were complemented by hand searching of reference lists of relevant studies and searches of grey literature. Study selection was carried out by two assessors, independently. Study quality assessment was performed to inform critical appraisal of health economics studies. Data were extracted using a data extraction form developed for this study. Results: 4464 unique studies were identified, of which seven met the inclusion criteria. With the exception of one study, the studies found ITDD to be either cost-saving or cost-effective compared to conventional medical management. ITDD becomes cost-ineffective in one further study following price year adjustment to 2016. Conclusions: Study findings show ITDD as not cost-effective only in extremely conservative scenarios. There is limited evidence on the effectiveness of ITDD in non-cancer pain; however, the available economic evidence controverts arguments to refute the treatment on economic grounds

Adjusting estimates of the expected value of information for implementation: theoretical framework and practical application

Background: Value of information (VoI) calculations give the expected benefits of decision making under perfect information (EVPI) or sample information (EVSI), typically on the premise that any treatment recommendations made in light of this information will be implemented instantly and fully. This assumption is unlikely to hold in health care; evidence shows that obtaining further information typically leads to “improved” rather than “perfect” implementation. Objectives: To present a method of calculating the expected value of further research that accounts for the reality of improved implementation. Methods: This work extends an existing conceptual framework by introducing additional states of the world regarding information (sample information, in addition to current and perfect information) and implementation (improved implementation, in addition to current and optimal implementation). The extension allows calculating the “implementation-adjusted” EVSI (IA-EVSI), a measure that accounts for different degrees of implementation. Calculations of implementation-adjusted estimates are illustrated under different scenarios through a stylized case study in non–small cell lung cancer. Results: In the particular case study, the population values for EVSI and IA-EVSI were £25 million and £8 million, respectively; thus, a decision assuming perfect implementation would have overestimated the expected value of research by about £17 million. IA-EVSI was driven by the assumed time horizon and, importantly, the specified rate of change in implementation: the higher the rate, the greater the IA-EVSI and the lower the difference between IA-EVSI and EVSI. Conclusions: Traditionally calculated measures of population VoI rely on unrealistic assumptions about implementation. This article provides a simple framework that accounts for improved, rather than perfect, implementation and offers more realistic estimates of the expected value of research. </jats:p

Considerations around the inclusion of children and young people’s time in economic evaluation: findings from an international Delphi study

Background: People’s time is a finite resource and a valuable input that ought to be considered in economic evaluations taking a broad, societal perspective. Yet, evaluations of interventions focusing on children and young people (CYP) rarely account for the opportunity cost of time in this population. As a key reason for this, health economists have pointed to uncertainty around when it is appropriate to include CYP time-related costs in an economic evaluation and highlighted the lack of clear guidance on the topic. Methods: With this in mind, we carried out a Delphi study to establish a list of relevant considerations for researchers to utilise whilst making decisions about whether and when to include CYP time in their economic evaluations. Delphi panellists were asked to propose and rate a set of possible considerations and provide additional thoughts on their ratings. Ratings were summarised using descriptive statistics, and text comments were interrogated through thematic analysis. Findings: A total of 73 panellists across 16 countries completed both rounds of a two-round Delphi study. Panellists’ ratings showed that, when thinking about whether to include displaced CYP time in an economic evaluation, it is very important to consider whether: (1) inclusion would be in line with specified perspective(s) (median score: 9), (2) CYP’s time may already be accounted for in other parts of the evaluation (median score: 8), (3) the amount of forgone time is substantial, either in absolute or relative terms (median score: 7) and (4) inclusion of CYP’s time costs would be of interest to decision-makers (median score: 7). Respondents thought that considerations such as (1) whether inclusion would be of interest to the research community (median score: 6), (2) whether CYP’s time displaced by receiving treatment is ‘school’ or ‘play’ time (median score: 5), and (3) whether CYP’s are old enough for their time to be considered valuable (median score: 5) are moderately important. A range of views was offered to support beliefs and ratings, many of which were underpinned by compelling normative questions.</p

Effectiveness and cost effectiveness of cardiovascular disease prevention in whole populations: modelling study

Objective To estimate the potential cost effectiveness of a population-wide risk factor reduction programme aimed at preventing cardiovascular disease

Economic considerations in designs and modifications of multi-arm, multi-stage adaptive, and adaptive-platform randomised control trials:A systematic literature review

Objective There is uncertainty around whether, and under what circumstances, there is value in embedding economic considerations into multi-arm, multi-stage (MAMS), adaptive, and adaptive-platform trial designs. This systematic review was conducted to assess the analytical methods and factors that are considered when incorporating health economic analyses when designing and modifying MAMS adaptive, and adaptive-platform trials. Methods The review searched for health economic analyses, including planned analyses, of interventions assessed through MAMS adaptive, and adaptive-platform trials. The search for articles was conducted in EMBASE, MEDLINE, Web of Science, Scopus, and ClinicalTrials.gov electronic databases from their inception to 7th August 2023. The screening for articles was conducted by two blinded reviewers who followed a predetermined screening process. A narrative synthesis was conducted on the methods used in the analysis and how the results informed the trial designs and modifications. Results The review included 17 articles of which four were results of economic evaluations while 13 were economic evaluation protocols. No trial reported using pre-trial economic evaluations to inform the trial designs. In 14 articles it was possible to estimate the costs and benefits of the interventions at the interim analysis stages. However, there were only five interim cost-effectiveness analyses and three of these informed decisions to drop or maintain trial arms. Conclusions Health economics is being embedded in some MAMS adaptive, and platform-adaptive trials to inform trial modifications. However, the use of economic evidence is limited, both by design and circumstance, despite its potential important to adoption decisions

Validation of the Bluebelle Wound Healing questionnaire (WHQ) for assessment of surgical site infection in primary surgical wounds after hospital discharge

Background Accurate assessment of surgical‐site infection (SSI) is crucial for surveillance and research. Self‐reporting patient measures are needed because current SSI tools are limited for assessing patients after leaving hospital. The Bluebelle Wound Healing Questionnaire (WHQ) was developed for patient or observer completion; this study tested its acceptability, scale structure, reliability and validity in patients with closed primary wounds after abdominal surgery. Methods Patients completed the WHQ (self‐assessment) within 30 days after leaving hospital and returned it by post. Healthcare professionals completed the WHQ (observer assessment) by telephone or face‐to‐face. Questionnaire response rates and patient acceptability were assessed. Factor analysis and Cronbach's α examined scale structure and internal consistency. Test–retest and self‐ versus observer reliability assessments were performed. Sensitivity and specificity for SSI discrimination against a face‐to‐face reference diagnosis (using Centers for Disease Control and Prevention criteria) were examined. Results Some 561 of 792 self‐assessments (70·8 per cent) and 597 of 791 observer assessments (75·5 per cent) were completed, with few missing data or problems reported. Data supported a single‐scale structure with strong internal consistency (α greater than 0·8). Reliability between test–retest and self‐ versus observer assessments was good (κ 0·6 or above for the majority of items). Sensitivity and specificity for SSI discrimination was high (area under the receiver operating characteristic (ROC) curve 0·91). Conclusion The Bluebelle WHQ is acceptable, reliable and valid with a single‐scale structure for postdischarge patient or observer assessment of SSI in closed primary wounds

Cost-Effectiveness of Treatments for the Management of Bone Metastases: A Systematic Literature Review

Background: Metastatic cancers occur when cancer cells break away from the primary tumour. One of the most common sites of metastasis is the bone. Several therapeutic options are currently available for managing bone metastases. In a resource constrained environment, policy makers and practitioners need to know which options are cost-effective. Objective: To review and appraise published economic evaluations on treatments for the management of bone metastases. Methods: We searched eight bibliographic databases (MEDLINE, MEDLINE in Process, EMBASE, CSDR, DARE, HTA, EED and CPCI) for relevant economic evaluations published from each database’s inception date until March 2017. Study selection, quality assessment and data extraction were carried out according to published guidelines. Results: Twenty-four relevant economic analyses were identified. Seventeen of these studies focus on bone metastases resulting from a particular type of cancer (prostate (n=8), breast (n=7), lung (n=1) or renal (n=1)) while seven report results for various primary tumours. Across types of cancer, evidence suggests that bisphosphonates result in lower morbidity and improved quality of life, for an additional cost which is typically below conventional cost-effectiveness thresholds. While denosumab leads to health gains compared to zoledronic acid, it also results in substantial additional costs and it is unlikely to represent value for money. The limited literature on the radiopharmaceutical strontium-89 (Sr89) and external beam radiotherapy (EBR) suggest that these treatments are cost-effective compared to no treatment. Conclusions: The reviewed evidence suggests that bisphosphonate treatments are cost-effective options for bone metastases, while denosumab is unlikely to represent value for money. Evidence on EBR and Sr89 is limited and less conclusive