Rosanna Orlando

Prucalopride for the treatment of women with chronic constipation in whom standard laxative regimens have failed to provide adequate relief.

This paper presents a summary of the evidence review group (ERG) report into the clinical effectiveness and cost-effectiveness of prucalopride for the treatment of women with chronic constipation in whom standard laxative regimens have failed to provide adequate relief. The ERG report is based on the manufacturers submission (MS) to the National Institute for Health and Clinical Excellence as part of the single technology appraisal process. In the submission, quality-of-life data [Patient Assessment of Constipation Quality of Life (PAC-QOL) and Patient Assessment of Constipation Symptoms (PAC-SYM) questionnaires] from trials of prucalopride were extrapolated to EQ-5D (European Quality of Life-5 Dimensions) data and used to inform effectiveness in an economic model. Response rates to prucalopride were derived from observed response rates in trials, defined as the proportion of patients achieving an average of three or more spontaneous complete bowel movements over the 4- or 12-week trial periods. Adult (18-64 years) and elderly (≥ 65 years) patients were considered separately in the model. Cost-effectiveness was determined from estimated improvements in EQ-5D and anticipated response rates, adjusted for baseline severity of chronic constipation. The ERG considered that the patients participating in these trials were not representative of those in the licensed indication. They were not all refractory to laxatives, and baseline EQ-5D scores showed a large spread in quality of life, with many patients experiencing little baseline dissatisfaction. The mapping of quality-of-life data from trials (PAC-QOL and PAC-SYM data) to EQ-5D was unclear and invalidated. The assumption of the long-term effectiveness and safety of prucalopride to 1 year was considered unjustified. There was no justification or sources given for coefficients used to predict effectiveness in the economic model, and no costs other than the cost of prucalopride were incorporated into the model. Owing to the many areas of uncertainty, particularly the effectiveness of prucalopride in the licensed patient group and its long-term effectiveness and safety, it was considered that the MS provided no evidence for whether prucalopride is effective or not in women with laxative-refractory chronic constipation. Further subgroup analysis of the actual patient group of interest may have better guided decision-making. However, long-term efficacy data, with validated estimates of quality of life incorporated in a well-founded model, would be important for an evidence-based judgement to be made.

Social Network Type and Long-Term Condition Management Support: A Cross-Sectional Study in Six European Countries.

Background Network types and characteristics have been linked to the capacity of inter-personal environments to mobilise and share resources. The aim of this paper is to examine personal network types in relation to long-term condition management in order to identify the properties of network types most likely to provide support for those with a long-term condition. Method A cross-sectional observational survey of people with type 2 diabetes using interviews and questionnaires was conducted between April and October 2013 in six European countries: Greece, Spain, Bulgaria, Norway, United Kingdom, and Netherlands. 1862 people with predominantly lower socio-economic status were recruited from each country. We used k-means clustering analysis to derive the network types, and one-way analysis of variance and multivariate logistic regression analysis to explore the relationship between network type socio-economic characteristics, self-management monitoring and skills, well-being, and network member work. Results Five network types of people with long-term conditions were identified: restricted, minimal family, family, weak ties, and diverse. Restricted network types represented those with the poorest self-management skills and were associated with limited support from social network members. Restricted networks were associated with poor indicators across self-management capacity, network support, and well-being. Diverse networks were associated with more enhanced self-management skills amongst those with a long-term condition and high level of emotional support. It was the three network types which had a large number of network members (diverse, weak ties, and family) where healthcare utilisation was most likely to correspond to existing health needs. Discussion Our findings suggest that type of increased social involvement is linked to greater self-management capacity and potentially lower formal health care costs indicating that diverse networks constitute the optimal network type as a policy in terms of the design of LTCM interventions and building support for people with LTCs.

ECONOMIC EVALUATIONS AND DIAGNOSTIC TESTING: AN ILLUSTRATIVE CASE STUDY APPROACH

OBJECTIVE The aim of this study was to present a clear process of synthesizing test accuracy data when conducting economic evaluations of diagnostic tests for health technology assessment (HTA) assessors and health economists. METHODS We appraised the methods advocated for using diagnostic test accuracy data in economic evaluations. We used a case study of fetal anemia in which data from a screening test are used in combination with a confirmatory test. RESULTS We developed a step-by-step guide and consider two scenarios: when data on test accuracy from several studies are based on (i) the same test threshold for positivity and (ii) different test thresholds. CONCLUSIONS We conclude that each approach has its strengths and limitations. We show that the optimal operating point of the test should be identified to determine the true cost-effectiveness of the test. We advocate that these issues require a multidisciplinary team of health economists, decision modelers and statisticians.

A team approach to recruitment in hospice research: engaging patients, close people and health professionals

Research is vital to the future development of hospice care. However, research in hospice settings is very challenging. This paper describes a case study of a successful multidisciplinary research team approach (MDRT) to the recruitment of participants (hospice patients, family members and health professionals) for a study in a hospice setting on the economic evaluation of end-of-life care. A successful recruitment plan includes three key strategies: identifying key members of the MDRT early in the research process; having a clear and constant communication stream; and creating an environment where all team members have a shared commitment to the research, all voices are heard and valued, and everyone contributes to the research aims. An MDRT approach will be helpful to guide the development of successful recruitment plans for academic-community research partnerships in the hospice setting.

‘The ICECAP-SCM tells you more about what I’m going through’: A think-aloud study measuring quality of life among patients receiving supportive and palliative care

Background: The ICECAP-Supportive Care Measure is a self-complete questionnaire developed to aid economic evaluation of supportive care interventions. Aim: To determine the feasibility of completing ICECAP–Supportive Care Measure alongside EQ-5D-5L and ICECAP-A (generic measures used in economic evaluation) among patients receiving hospice care, close persons and healthcare professionals. Design: Participants were asked to ‘think aloud’ while completing ICECAP-Supportive Care Measure and two other generic measures used in economic evaluation, EQ-5D-5L and ICECAP-A, and then participate in a semi-structured interview. From verbatim transcripts, five raters identified the frequency of errors in comprehension, retrieval, judgement and response. Qualitative data were analysed using constant comparison. Setting/participants: Eligible patients were identified from one UK hospice by a research nurse. Close persons and healthcare professionals were identified by the patient. In all, 72 semi-structured interviews were conducted with patients (n = 33), close persons (n = 22) and healthcare professionals (n = 17). Results: Patients and close persons reported that the ICECAP-Supportive Care Measure was most appropriate for measuring their quality of life. It appeared more meaningful, easier to complete and had fewest errors (3.9% among patients, 4.5% among close persons) compared to EQ-5D-5L (9.7% among patients, 5.5% among close persons). Healthcare professionals acknowledged the value of the ICECAP-Supportive Care Measure but had fewer errors in completing the EQ-5D-5L (3.5% versus 6.7%). They found it easier to complete because it focuses on observable health states. Conclusions: The ICECAP-Supportive Care Measure is feasible to use and perceived as appropriate for evaluating palliative care interventions. Healthcare professionals with limited knowledge of the patient who act as proxy completers may find the measure difficult to complete.

“THE ICECAP-SCM TELLS YOU MORE ABOUT WHAT I'M GOING THROUGH”—MEASURING THE QUALITY OF LIFE AMONGST PATIENTS RECEIVING SUPPORTIVE AND PALLIATIVE CARE

Introduction The ICECAP Supportive Care Measure (ICECAP-SCM) is a self-complete questionnaire developed to rate quality towards the end of life, particularly for economic evaluation. It measures a persons capability to experience a good life and death. Aim(s) and method(s) The study aimed to determine the feasibility of completing ICECAP-SCM alongside EQ-5D-5L and ICECAP-A (measures commonly used for economic evaluation). Each measure was completed by hospice patients (n=33), close persons (n=22) and healthcare professionals (HCPs n=17) in a ‘think aloud’ semi-qualitative interview. Five raters identified the frequency of error (e.g. misunderstanding, poor memory) from interview transcripts. Qualitative data were analysed using constant comparison, focusing on issues affecting response. Results Amongst patients, fewest errors were identified for ICECAP-SCM (3.9%) and the most for EQ5D-5L (9.7%). Amongst close persons there were also fewest errors in the ICECAP-SCM (4.5%) compared to ICECAP-A (5.5%) and EQ-5D-5L (5.5%). HCPs had most errors for ICECAP-SCM (6.7%) and fewest for EQ-5D-5L (3.5%). Qualitative data suggested that HCPs found EQ-5D-5L easiest to answer because it focuses on health states which could be directly observed. The ICECAP-SCM, appeared more meaningful to patients near the end of life and close persons; it was also easier to complete. Conclusion(s) This paper provides insight into the meaning of quality of life for those approaching death, those close to them and those involved in their care. Complexities in disease trajectories and adaptation to poor health make quality of end of life difficult to measure. The ICECAP-SCM captures the subtleties required and may be useful in evaluating future palliative care interventions.

Adaptation, Acceptance and Adaptive Preferences in Health and Capability Well-Being Measurement Amongst Those Approaching End of Life

Background and ObjectivesAdaptive preferences occur when people subconsciously alter their views to account for the possibilities available to them. Adaptive preferences may be problematic where these views are used in resource allocation decisions because they may lead to underestimation of the true benefits of providing services. This research explored the nature and extent of both adaptation (changing to better suit the context) and adaptive preferences (altering preferences in response to restricted options) in individuals approaching the end of life (EoL).MethodsQualitative data from ‘thinkaloud’ interviews with 33 hospice patients, 22 close persons and 17 health professionals were used alongside their responses to three health/well-being measures for use in resource allocation decisions: EQ-5D-5L (health status); ICECAP-A (adult capability); and ICECAP-SCM (Supportive Care Measure; EoL capability). Constant comparative analysis combined a focus on both verbalised perceptions across the three groups and responses to the measures.ResultsData collection took place between October 2012 and February 2014. Informants spoke clearly about how patients had adapted their lives in response to symptoms associated with their terminal condition. It was often seen as a positive choice to accept their state and adapt in this way but, at the same time, most patients were fully aware of the health and capability losses that they had faced. Self-assessments of health and capability generally appeared to reflect the pre-adaptation state, although there were exceptions.ConclusionDespite adapting to their conditions, the reference group for individuals approaching EoL largely remained a healthy, capable population, and most did not show evidence of adaptive preferences.

The ICECAP-SCM tells you more about what I'm going through

Background: The ICECAP-Supportive Care Measure is a self-complete questionnaire developed to aid economic evaluation of supportive care interventions. Aim: To determine the feasibility of completing ICECAP–Supportive Care Measure alongside EQ-5D-5L and ICECAP-A (generic measures used in economic evaluation) among patients receiving hospice care, close persons and healthcare professionals. Design: Participants were asked to ‘think aloud’ while completing ICECAP-Supportive Care Measure and two other generic measures used in economic evaluation, EQ-5D-5L and ICECAP-A, and then participate in a semi-structured interview. From verbatim transcripts, five raters identified the frequency of errors in comprehension, retrieval, judgement and response. Qualitative data were analysed using constant comparison. Setting/participants: Eligible patients were identified from one UK hospice by a research nurse. Close persons and healthcare professionals were identified by the patient. In all, 72 semi-structured interviews were conducted with patients (n = 33), close persons (n = 22) and healthcare professionals (n = 17). Results: Patients and close persons reported that the ICECAP-Supportive Care Measure was most appropriate for measuring their quality of life. It appeared more meaningful, easier to complete and had fewest errors (3.9% among patients, 4.5% among close persons) compared to EQ-5D-5L (9.7% among patients, 5.5% among close persons). Healthcare professionals acknowledged the value of the ICECAP-Supportive Care Measure but had fewer errors in completing the EQ-5D-5L (3.5% versus 6.7%). They found it easier to complete because it focuses on observable health states. Conclusions: The ICECAP-Supportive Care Measure is feasible to use and perceived as appropriate for evaluating palliative care interventions. Healthcare professionals with limited knowledge of the patient who act as proxy completers may find the measure difficult to complete.

The ICECAP-SCM tells you more about what I'm going through: A think aloud study measuring quality of life amongst patients receiving supportive and palliative care

Background: The ICECAP-Supportive Care Measure is a self-complete questionnaire developed to aid economic evaluation of supportive care interventions. Aim: To determine the feasibility of completing ICECAP–Supportive Care Measure alongside EQ-5D-5L and ICECAP-A (generic measures used in economic evaluation) among patients receiving hospice care, close persons and healthcare professionals. Design: Participants were asked to ‘think aloud’ while completing ICECAP-Supportive Care Measure and two other generic measures used in economic evaluation, EQ-5D-5L and ICECAP-A, and then participate in a semi-structured interview. From verbatim transcripts, five raters identified the frequency of errors in comprehension, retrieval, judgement and response. Qualitative data were analysed using constant comparison. Setting/participants: Eligible patients were identified from one UK hospice by a research nurse. Close persons and healthcare professionals were identified by the patient. In all, 72 semi-structured interviews were conducted with patients (n = 33), close persons (n = 22) and healthcare professionals (n = 17). Results: Patients and close persons reported that the ICECAP-Supportive Care Measure was most appropriate for measuring their quality of life. It appeared more meaningful, easier to complete and had fewest errors (3.9% among patients, 4.5% among close persons) compared to EQ-5D-5L (9.7% among patients, 5.5% among close persons). Healthcare professionals acknowledged the value of the ICECAP-Supportive Care Measure but had fewer errors in completing the EQ-5D-5L (3.5% versus 6.7%). They found it easier to complete because it focuses on observable health states. Conclusions: The ICECAP-Supportive Care Measure is feasible to use and perceived as appropriate for evaluating palliative care interventions. Healthcare professionals with limited knowledge of the patient who act as proxy completers may find the measure difficult to complete.