General population normative values for the EORTC QLQ-C30 by age, sex, and health condition for the French general population.

BACKGROUND: General population normative values for the widely used health-related quality of life (HRQoL) measure EORTC QLQ-C30 support the interpretation of trial results and HRQoL of patients in clinical practice. Here, we provide sex-, age- and health condition-specific normative values for the EORTC QLQ-C30 in the French general population. METHODS: French general population data was collected in an international EORTC project. Online panels with quota samples were used to recruit sex and age groups. Number and type of comorbidities were assessed. Descriptive statistics were used to calculate general population values for each QLQ-C30 scale, separately for sex, age, and presence of one- and more chronic health conditions. A multivariate linear regression model has been developed to allow estimating the effect of sex, age, and the presence for one- and more chronic health conditions on EORTC QLQ-C30 scores. Data was weighted according to United Nation statistics adjusting for the proportion of sex and age groups. RESULTS: In total, 1001 French respondents were included in our analyses. The weighted mean age was 47.9 years, 514 (51.3%) participants were women, and 497 (52.2%) participants reported at least one health condition. Men reported statistically significant better scores for Emotional Functioning (+9.6 points, p = 0.006) and Fatigue (-7.8 point; p = 0.04); women reported better profiles for Role Functioning (+8.7 points; p = 0.008) and Financial Difficulty (-7.8 points, p = 0.011). According to the regression model, the sex effect was statistically significant in eight scales; the effect of increasing age had a statistically significant effect on seven of the 15 EORTC QLQ-C30 scales. The sex- and age effect varied in its direction across the various scales. The presence of health conditions showed a strong negative effect on all scales. CONCLUSION: This is the first publication of detailed French normative values for the EORTC QLQ-C30. It aims to support the interpretation of HRQoL profiles in French cancer populations. The strong impact of health conditions on QLQ-C30 scores highlights the importance of considering the impact of comorbidities in cancer patients when interpreting HRQoL data.

Cancer-Specific Health Utilities: Evaluation of Core Measurement Properties of the EORTC QLU-C10D in Lung Cancer Patients-Data from Four Multicentre LUX-Lung Trials, Applying Six Country Tariffs.

BACKGROUND: Cost-utility analysis generally requires valid preference-based measures (PBMs) to assess the utility of patient health. While generic PBMs are widely used, disease-specific PBMs may capture additional aspects of health relevant for certain patient populations. This study investigates the construct and concurrent criterion validity of the cancer-specific European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Utility-Core 10 dimensions (QLU-C10D) in non-small-cell lung cancer patients. METHODS: We retrospectively analysed data from four multicentre LUX-Lung trials, all of which had administered the EORTC Quality of Life Questionnaire (QLQ-C30) and the EQ-5D-3L. We applied six country-specific value sets (Australia, Canada, Italy, the Netherlands, Poland, and the United Kingdom) to both instruments. Criterion validity was assessed via correlations between the instruments' utility scores. Correlations of divergent and convergent domains and Bland-Altman plots investigated construct validity. Floor and ceiling effects were assessed. RESULTS: The comparison of the EORTC QLU-C10D and EQ-5D-3L produced homogenous results for five of the six country tariffs. High correlations of utilities (r > 0.7) were found for all country tariffs except for the Netherlands. Moderate to high correlations of converging domain pairs (r from 0.472 to 0.718) were found with few exceptions, such as the Social Functioning-Usual Activities domain pair (max. r = 0.376). For all but the Dutch tariff, the EORTC QLU-C10D produced consistently lower utility values compared to the EQ-5D-3L (x̄ difference from - 0.082 to 0.033). Floor and ceiling effects were consistently lower for the EORTC QLU-C10D (max. 4.67% for utilities). CONCLUSIONS: The six country tariffs showed good psychometric properties for the EORTC QLU-C10D in lung cancer patients. Criterion and construct validity was established. The QLU-C10D showed superior measurement precision towards the upper and lower end of the scale compared to the EQ-5D-3L, which is important when cost-utility analysis seeks to measure health change across the severity spectrum.

The EORTC QLU-C10D is a valid cancer-specific preference-based measure for cost-utility and health technology assessment in the Netherlands.

BACKGROUND: Cost-utility analysis typically relies on preference-based measures (PBMs). While generic PBMs are widely used, disease-specific PBMs can capture aspects relevant for certain patient populations. Here the EORTC QLU-C10D, a cancer-specific PBM based on the QLQ-C30, is validated using Dutch trial data with the EQ-5D-3L as a generic comparator measure. METHODS: We retrospectively analysed data from four Dutch randomised controlled trials (RCTs) comprising the EORTC QLQ-C30 and the EQ-5D-3L. Respective Dutch value sets were applied. Correlations between the instruments were calculated for domains and index scores. Bland-Altman plots and intra-class correlations (ICC) displayed agreement between the measures. Independent and paired t-tests, effect sizes and relative validity indices were used to determine the instruments' performance in detecting clinically known-group differences and health changes over time. RESULTS: We analysed data from 602 cancer patients from four different trials. In overall, the EORTC QLU-C10D showed good relative validity with the EQ-5D-3L as a comparator (correlations of index scores r = 0.53-0.75, ICCs 0.686-0.808, conceptually similar domains showed higher correlations than dissimilar domains). Most importantly, it detected 63% of expected clinical group differences and 50% of changes over time in patients undergoing treatment. Both instruments showed poor performance in survivors. Detection rate and measurement efficiency were clearly higher for the QLU-C10D than for the EQ-5D-3L. CONCLUSIONS: The Dutch EORTC QLU-C10D showed good comparative validity in patients undergoing treatment. Our results underline the benefit that can be achieved by using a cancer-specific PBM for generating health utilities for cancer patients from a measurement perspective.

The impact of electronic versus paper-based data capture on data collection logistics and on missing scores in thyroid cancer patients.

PURPOSE: The purpose of this study was to investigate the impact of the type of data capture on the time and help needed for collecting patient-reported outcomes as well as on the proportion of missing scores. METHODS: In a multinational prospective study, thyroid cancer patients from 17 countries completed a validated questionnaire measuring quality of life. Electronic data capture was compared to the paper-based approach using multivariate logistic regression. RESULTS: A total of 437 patients were included, of whom 13% used electronic data capture. The relation between data capture and time needed was modified by the emotional functioning of the patients. Those with clinical impairments in that respect needed more time to complete the questionnaire when they used electronic data capture compared to paper and pencil (ORadj 24.0; p = 0.006). This was not the case when patients had sub-threshold emotional problems (ORadj 1.9; p = 0.48). The odds of having the researcher reading the questions out (instead of the patient doing this themselves) (ORadj 0.1; p = 0.01) and of needing any help (ORadj 0.1; p = 0.01) were lower when electronic data capture was used. The proportion of missing scores was equivalent in both groups (ORadj 0.4, p = 0.42). CONCLUSIONS: The advantages of electronic data capture, such as real-time assessment and fewer data entry errors, may come at the price of more time required for data collection when the patients have mental health problems. As this is not uncommon in thyroid cancer, researchers need to choose the type of data capture wisely for their particular research question.

The European Organisation for Research and Treatment of Cancer Quality of Life Utility-Core 10 Dimensions: Development and Investigation of General Population Utility Norms for Canada, France, Germany, Italy, Poland, and the United Kingdom.

OBJECTIVES: The European Organisation for Research and Treatment of Cancer Quality of Life Utility-Core 10 Dimensions (EORTC QLU-C10D) is a cancer-specific preference-based measure, providing health utilities for use in economic evaluations derived from the widely used health-related quality of life measure, EORTC QLQ-C30. Several EORTC QLU-C10D country-specific value sets are available. This article aimed to provide EORTC QLU-C10D general population utility norms for Canada, France, Germany, Italy, Poland, and the United Kingdom, to aid interpretability of obtained utilities in these countries. METHODS: Data were collected in aforementioned countries via a quota-sampled, cross-sectional online survey (n = 100/age-sex group; N = approximately 1000/country). Participants were asked to complete the EORTC QLQ-C30 and provide sociodemographic data. Country-specific utility norms were calculated using the respective country tariff on the country's EORTC QLQ-C30 data after weighting to achieve population representativeness for age and sex. Norm values are provided as means (SDs) by country, age, and sex groups. Tukey's multiple comparison test investigated mean differences among countries. The impact of country, age, and sex on utility values was investigated with a multiple linear regression model. RESULTS: Country-specific mean utilities range from 0.724 (United Kingdom) to 0.843 (Italy). Country-, sex-, and age-specific mean utilities range from 0.664 for 30- to 39-year-old male Canadians to 0.899 for > 70-year-old male Italians. Utilities were lower in females in 4 of 6 countries, and the impact of age differed among countries. Independent of the impact of age and sex, between-country differences were found (P ≤ .05). CONCLUSION: Results showed a varying impact of age and sex on EORTC QLU-C10D utilities and significant between-country differences. Using national utility norms and utility decrements is recommended.

The EORTC QLU-C10D discrete choice experiment for cancer patients: a first step towards patient utility weights.

BACKGROUND: The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Utility-Core 10 Dimensions (QLU-C10D) is a novel cancer-specific preference-based measure (PBM) for which value sets are being developed for an increasing number of countries. This is done by obtaining health preferences from the respective general population. There is an ongoing discussion if instead patients suffering from the disease in question should be asked for their preferences. We used the QLU-C10D valuation survey, originally designed for use in the general population, in a sample of cancer patients in Austria to assess the methodology's acceptability and applicability in this target group before obtaining QLU-C10D patient preferences. METHODS: The core of the QLU-C10D valuation survey is a discrete choice experiment in which respondents are asked to give preferences for certain health states (described by a relatively large number of 10 quality of life domains) and an associated survival time. They therewith are asked to trade off quality of life against life time. As this might be a very burdensome task for cancer patients undergoing treatment, a cognitive interview was conducted in a pilot sample to assess burden and potential additional needs for explanation in order to be able to use the DCE for the development of QLU-C10D patient preferences. In addition, responses to general feedback questions on the survey were compared against responses from a matched control group from the already completed Austrian general population valuation survey. RESULTS: We included 48 patients (mean age 59.9 years; 46% female). In the cognitive interview, the majority indicated that their experience with the survey was positive (85%) and overall clarity as good (90%). In response to the general feedback questions, patients rated the presentation of the health states less clear than matched controls (p = 0.008). There was no difference between patients and the general population concerning the difficulty in choosing between the health states (p = 0.344). CONCLUSION: Despite the relatively large number of DCE domains the survey was manageable for patients and allows going on with the QLU-C10D patient valuation study.

EORTC QLQ-C30 general population normative data for Italy by sex, age and health condition: an analysis of 1,036 individuals.

BACKGROUND: General population normative values for the widely used health-related quality of life (HRQoL) measure, European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire - Core 30 (EORTC QLQ-C30), are available for a range of countries. These are mostly countries in northern Europe. However, there is still a lack of such normative values for southern Europe. Therefore, this study aims to provide sex-, age- and health condition-specific normative values for the general Italian population for the EORTC QLQ-C30. MATERIAL AND METHODS: This study is based on Italian EORTC QLQ-C30 general population data previously collected in an international EORTC project comprising over 15,000 respondents across 15 countries. Recruitment and assessment were carried out via online panels. Quota sampling was used for sex and age groups (18|-|39, 40-49, 50-59, 60-69 and ≥ 70 years), separately for each country. We applied weights to match the age and sex distribution in our sample with UN statistics for Italy. Along with descriptive statistics, linear regression models were estimated to describe the associations of sex, age and health condition with the EORTC QLQ-C30 scores. RESULTS: A total of 1,036 respondents from Italy were included in our analyses. The weighted mean age was 49.3 years, and 536 (51.7%) participants were female. Having at least one health condition was reported by 60.7% of the participants. Men reported better scores than women on all EORTC QLQ-C30 scales but diarrhoea. While the impact of age differed across scales, older age was overall associated with better HRQoL as shown by the summary score. For all scales, differences were in favour of participants who did not report any health condition, compared to those who reported at least one. CONCLUSION: The Italian normative values for the EORTC QLQ-C30 scales support the interpretation of HRQoL profiles in Italian cancer populations. The strong impact of health conditions on EORTC QLQ-C30 scores highlights the importance of adjusting for the impact of comorbidities in cancer patients when interpreting HRQoL data.

Impact of Blinding on Patient-Reported Outcome Differences Between Treatment Arms in Cancer Randomized Controlled Trials.

Some concerns have been raised about potential bias in patient-reported outcome (PRO) results from open-label cancer randomized controlled trials (RCTs). We investigated if open-label trials favor the experimental treatment over the standard treatment more frequently than blinded trials. We also examined if the effect of blinding differs for distal vs more proximal PROs. We assessed 538 RCTs with a PRO endpoint conducted in the most prevalent cancers, of which 366 (68.0%) were open-label, 148 (27.5%) were blinded, and 24 (4.5%) were categorized as unclear. In our multivariable logistic regression model, we did not observe a statistically significant association of the independent variable treatment concealment (blinded vs open-label) on the dependent variable measuring the proportion of trials favoring the experimental treatment (adjusted odds ratio = 1.19, 95% confidence interval = 0.79 to 1.79; 2-sided P = .40). This was also the case when comparing distal and proximal PROs. Our findings provide novel evidence-based data that support the validity of PRO results from open-label cancer RCTs.

Past and Current Practice of Patient-Reported Outcome Measurement in Randomized Cancer Clinical Trials: A Systematic Review.

OBJECTIVES: In our systematic review, we assessed past and current practice of patient-reported outcome (PRO) measurement in cancer randomized, controlled trials (RCTs). METHODS: We included RCTs with PRO endpoints evaluating conventional medical treatments, conducted in patients with the most prevalent solid tumor types (breast, lung, colorectal, prostate, bladder, and gynecological cancers) and either published in 2004 to 2018 or registered on clinicaltrials.gov and initiated in 2014 to 2019. Frequency of use of individual PRO measures was assessed overall, over time, and by cancer site. RESULTS: Screening of 42 095 database records and 3425 registered trials identified 480 published and 537 registered trials meeting inclusion criteria. Among published trials, the European Organisation for Research and Treatment of Cancer (EORTC) measures were used most often (54.8% of trials), followed by the Functional Assessment of Chronic Illness Therapy (FACIT) measures (35.8%), the EQ-5D (10.2%), the SF-36 (7.3%), and the MD Anderson Symptom Inventory (MDASI; 2.5%). Among registered trials, the EORTC measures were used in 66.1% of the trials, followed by the FACIT measures (25.9%), the EQ-5D (23.1%), the SF-36 (4.8%), the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE; 2.2%), the Patient-Reported Outcomes Measurement Information System (PROMIS) measures (1.7%), and the MDASI measures (1.1%). CONCLUSION: The PRO measures most frequently used in RCTs identified in our review differ substantially in terms of content and domains, reflecting the ongoing debate among the scientific community, healthcare providers, and regulators on the type of PRO to be measured. Current findings may contribute to better informing the development of an internationally agreed core outcome set for future cancer trials.

French Value-Set of the QLU-C10D, a Cancer-Specific Utility Measure Derived from the QLQ-C30.

BACKGROUND AND OBJECTIVE: The EORTC Quality of Life Utility Measure-Core 10 Dimensions (QLU-C10D) is a new multi-attribute utility instrument derived from the EORTC Quality of Life Questionnaire-Core 30 (QLQ-C30), a widely used cancer-specific quality-of-life questionnaire. It covers ten dimensions: physical, role functioning, social, emotional functioning, pain, fatigue, sleep, appetite, nausea and bowel problems. To allow national health preferences to be reflected, country-specific valuations are being performed through collaboration between the Multi-Attribute Utility Cancer (MAUCa) Consortium and the EORTC. The aim of this study was to determine the utility weights for health states in the French version of the QLU-C10D. METHODS: Valuations were run in a web-based setting in a general population sample of 1033 adults. Utilities were elicited using a discrete-choice experiment (DCE). Data were analyzed by conditional logistic regression and mixed logits. RESULTS: The sample was representative of the general French population in terms of gender and age. Dimensions with the largest impact on utility weights were, in this order: physical functioning, pain and emotional functioning. The impact on utilities was lower for role functioning, nausea, bowel problems and social functioning. The dimensions of sleep, fatigue and lacking appetite were associated with the smallest utility decrement. CONCLUSION: The results of the present study provide utility weights for the QLU-C10D and offer interesting prospects, as some cancer-specific dimensions also received sizeable utility weights (nausea and bowel problems). In fact, the EQ-5D and the HUI 3 are recommended in France and commonly used for cancer-related CUA; however, both these instruments are generic. The availability of a new cancer-specific utility instrument, such as the QLU-C10D, could improve the quality and the pertinence of future CUA in oncology.

Health-related quality of life in neuroendocrine neoplasia: a critical review.

Incidence of neuroendocrine neoplasia (NEN) is increasing, as is use of health-related quality of life (HRQoL) measurement in clinical trials. Following development of validated questionnaires, HRQoL is widely used to assess outcomes. This review is intended for healthcare professionals and is based on a selection of data published in the last decade. HRQoL is on par with other clinical endpoints such as performance status. Assessments in clinical trials have been particularly useful for monitoring the symptom burden of NEN, for the effects of treatments on patients' lives, and have provided new data allied to the usual clinical endpoints. QoL expressed as quality-adjusted life years (QALYs) have become the most important primary outcome to establish cost-effectiveness in health economic evaluation. From looking at clinical trials over the last 10 years, we see that the quality of HRQoL evidence reported in published studies has improved and, in general, recent studies are likely to be more methodologically robust. Assessment of HRQoL in clinical trials is likely to become a standard part of clinical practice in NEN, as in other cancers. However, clear methods for calculating the clinical meaningfulness of changes in scores are needed. Other limitations of HRQoL measurement include lack of specificity to certain symptom sets and ease of completion and administration. An international group taking a lead on developing HRQoL research specifically in NEN patients is needed to address limitations of the evidence base. In order for greater weight to be placed on HRQoL data, agreement on optimal, validated scoring systems is needed.

Correction to: do patients consider computer-adaptive measures more appropriate than static questionnaires?

Following publication of the original article [1], the authors reported three of their given name have been erroneously tagged as their family names. The correct names are: give name Caroline family name Martini, give name Irene family name Virgolini, give name Bernhard family name Holzner.

Do patients consider computer-adaptive measures more appropriate than static questionnaires?

OBJECTIVE: Computer-adaptive tests (CAT) use individualised sets of questions to assess patient-reported health states, whereas static (conventional) questionnaires present the same questions to all patients. CAT has been shown to increase measurement precision and reduce assessment length. In our study, we investigated if patients perceive CAT questions as more appropriate than static questionnaires, a claim that is frequently associated with CAT measures. METHODS: We compared the static European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) against its corresponding CAT measures focusing on two domains: Physical Functioning (PF) and Emotional Functioning (EF). Cancer patients completed the questionnaires and participated in a cognitive interview to assess how appropriate they perceive the QLQ-C30 and the CAT questions for their current health state. RESULTS: Forty-four cancer patients (mean age = 54.6; 56.8% female) were assessed. For the PF domain, patients considered the CAT items more appropriate (p = 0.002) than the QLQ-C30 items (56.8% vs. 15.9%; 27.2% indifferent). For the EF domain, patients were in favour of the QLQ-C30 items (p < 0.001), with 54.5% considering the QLQ-C30, and 4.5% considering the CAT items to be more appropriate; 40.9% were indifferent. Most patients (N = 36) commented on the preference for the CAT (PF), mentioning better matching of the questions and the health state (38.6%) and better item wording (15.9%). CONCLUSION: For the PF domain the CAT measure better matched the score distribution in the patient sample than the QLQ-C30 PF scale and was consequently considered more appropriate by patients. For the EF domain, the CAT measure did not show better fit than the QLQ-C30 and hence no such preference in terms of appropriateness was observed.

Test-Retest Reliability of Discrete Choice Experiment for Valuations of QLU-C10D Health States.

BACKGROUND: Recently, a newly developed cancer-specific multiattribute utility instrument based on the widely used health-related quality of life instrument, the European Organisation for Research and Treatment of Cancer QLQ-C30, was introduced: the QLU-C10D. For the elicitation of utility weights, a discrete choice experiment (DCE) was designed. Our aim was to investigate the DCE in terms of individual choice consistency and utility estimate consistency by applying a test-retest design. METHODS: We conducted the study in general population samples in Germany and France. The DCE was administered via a web-based self-complete survey using online panels. Respondents were presented 16 choice sets comprising 11 attributes with 4 levels each. Retest was conducted 4 to 6 weeks after first assessment. We used kappa and percentage agreement as measures of choice consistency and both intraclass correlations and mean utility differences as measures of utility estimate consistency. RESULTS: A total of 300 German respondents (31% female, mean age 48 years [SD 14]) and 305 French respondents (46% female, mean age 47 years [SD 16]) completed test and retest assessments. Individual choice consistency was moderate to high (Germany: κ = 0.605, percentage agreement = 80.2%; France: κ = 0.411, percentage agreement = 70.6%). Utility estimate consistency was high when considering intraclass correlations (all >0.79). Mean utility differences were 0.08 in the German sample and 0.05 in the French sample. CONCLUSIONS: Results indicate that the designed DCE elicits stable health state preferences rather than guesses or mood-specific or condition-specific judgments. Nevertheless, the identified mean utility differences between test and retest need to be taken into account when determining minimal important differences for the QLU-C10D in future research.

Quality of Life in Patients with Metastatic Gastroenteropancreatic Neuroendocrine Tumors Receiving Peptide Receptor Radionuclide Therapy: Information from a Monitoring Program in Clinical Routine.

In patients with metastatic gastroenteropancreatic neuroendocrine tumors (NETs), we evaluated health-related quality of life (HRQoL) from the first peptide receptor radionuclide therapy (PRRT) to the first restaging and compared the scores with general-population (GP) norms. Methods: The data were from routine HRQoL monitoring using the core quality-of-life questionnaire of the European Organization for Research and Treatment of Cancer (EORTC QLQ-C30). Patients received 4-6 cycles of 177Lu-DOTATATE or 90Y-DOTATOC. To be eligible for analysis, patients had to have at least one HRQoL assessment before PRRT and at least one HRQoL assessment at the end of or after treatment completion. Linear mixed models were used to consider HRQoL changes over time. Results: In total, 61 gastroenteropancreatic NET patients (small-intestine NETs, n = 37; pancreatic NETs, n = 24) were eligible for analysis. Clear improvements from baseline to the first restaging were found for diarrhea in small-intestine NET patients, showing a decrease of 16 points, which represents a moderately large change. We observed a clinically relevant decrease in appetite loss (17 points), but for female small-intestine NET patients only. Other HRQoL changes were also restricted to sociodemographic or clinical subgroups and mainly reflected improvements, except for physical and social functioning, which showed decreasing scores in older small-intestine NET patients. Compared with HRQoL GP norms, patients had impairments consisting of diarrhea; fatigue; appetite loss; reduced physical, social, and role functioning; and reduced global HRQoL. Except for diarrhea and appetite loss, patient scores at the first restaging did not reach GP levels. Conclusion: Our analyses support previous findings of overall stable HRQoL under PRRT. Yet, significant HRQoL impairments compared with the GP and potentially specific subgroup patterns need to be considered.

The EORTC CAT Core-The computer adaptive version of the EORTC QLQ-C30 questionnaire.

BACKGROUND: To optimise measurement precision, relevance to patients and flexibility, patient-reported outcome measures (PROMs) should ideally be adapted to the individual patient/study while retaining direct comparability of scores across patients/studies. This is achievable using item banks and computerised adaptive tests (CATs). The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 (QLQ-C30) is one of the most widely used PROMs in cancer research and clinical practice. Here we provide an overview of the research program to develop CAT versions of the QLQ-C30's 14 functional and symptom domains. METHODS: The EORTC Quality of Life Group's strategy for developing CAT item banks consists of: literature search to identify potential candidate items; formulation of new items compatible with the QLQ-C30 item style; expert evaluations and patient interviews; field-testing and psychometric analyses, including factor analysis, item response theory calibration and simulation of measurement properties. In addition, software for setting up, running and scoring CAT has been developed. RESULTS: Across eight rounds of data collections, 9782 patients were recruited from 12 countries for the field-testing. The four phases of development resulted in a total of 260 unique items across the 14 domains. Each item bank consists of 7-34 items. Psychometric evaluations indicated higher measurement precision and increased statistical power of the CAT measures compared to the QLQ-C30 scales. Using CAT, sample size requirements may be reduced by approximately 20-35% on average without loss of power. CONCLUSIONS: The EORTC CAT Core represents a more precise, powerful and flexible measurement system than the QLQ-C30. It is currently being validated in a large independent, international sample of cancer patients.

Critical appraisal of health-state utility values used in breast cancer-related cost-utility analyses.

PURPOSE: To review the data sources of health-state utility values (HSUVs), as well as their elicitation and use, in 140 breast cancer-related cost-utility analyses (CUAs), and to provide a critical appraisal of these. METHODS: A checklist was developed to guide the process of the critical appraisal. It is divided into three parts: the data source (three questions), elicitation method (four questions), and use (ten questions) of HSUVs in CUAs. Two independent reviewers performed the data extraction. A consensus was reached in case of disagreements. Data sources were categorized as "original study," "derived from the literature," or "other." RESULTS: The data source of HSUVs was always specified. When HSUVs were derived from the literature (90% of cases), the authors referred to a median number of two references as data sources. The critical appraisal of the elicitation of HSUVs in CUAs revealed considerable variability in terms of the quality of the reporting of the data source selection of HSUV. More details were provided by authors when HSUVs were elicited from an original study rather than derived from the literature. The use of HSUVs elicited from an original study was generally better described in terms of the checklist than were those derived from the literature. CONCLUSIONS: Based on the developed checklist, we were able to highlight the challenges that authors are facing when trying to adequately report HSUV used in CUAs. Our proposed checklist offers a good starting point for encouraging more explicit and comprehensive reporting of HSUVs in CUAs.

Evaluation of Noncompletion Bias and Long-Term Adherence in a 10-Year Patient-Reported Outcome Monitoring Program in Clinical Routine.

BACKGROUND: Currently there is little knowledge on real-life sustainability of routine patient-reported outcome (PRO) measurement and the representativeness of collected data. OBJECTIVES: The investigation of routine PRO with regard to noncompletion bias and long-term adher- ence, considering the potential impact of mode of assessment (MOA) (paper-pencil vs. electronic PRO [ePRO]) and patient characteristics. METHODS: At our department, routine PRO measurement in oncological patients is being done since 2005 using different MOA (paper-pencil assessment until 2011 and ePRO assessment from 2011 onward). We analyzed two different patient groups: patients eligible in both periods (both-MOA group) and patients eligible in only one period (one-MOA group). The primary outcome was PRO noncompletion (100% missing questionnaires). The secondary outcome was poor PRO adherence (>20% missing questionnaires). Multivariate logistic regression models were developed, testing the impact of MOA and patient characteristics on the outcomes in the different patient groups. RESULTS: Data from 1484 eligible patients were included in the analyses. Most of the patients could be included in PRO assessment at least once. PRO noncompletion rates were clearly higher during paper-pencil assessment (odds ratios between 2.72 and 4.31), as were poor PRO adherence rates (odd ratio 2.23). Analyses of potential bias by patient characteristics showed that male patients had a higher risk of poor adherence. Other factors with significant impact were age, country, and cancer diagnosis, but results were indecisive. CONCLUSIONS: ePRO increased the feasibility of our clinical routine PRO data for retrospective analyses by increasing completion rates. In general, potential completion bias regarding certain patient characteristics requires attention before generalizing results to the respective populations.

Systematic review reveals lack of quality in reporting health-related quality of life in patients with gastroenteropancreatic neuroendocrine tumours.

BACKGROUND: Gastroenteropancreatic neuroendocrine tumours (GEP-NET) are often slow-growing and patients may live for years with metastasised disease. Hence, along with increasing overall and progression-free survival, treatments aim at preserving patients' well-being and health-related quality of life (HRQoL). However, studies on systematic HRQoL assessment in patients with GEP-NET are scarce. Therefore, the purpose of the current review is to systematically evaluate the methodological quality of the identified studies. METHODS: A targeted database search was performed in PubMed, EMBASE, and CENTRAL. Data extraction was conducted by two independent researchers according to predefined criteria. For study evaluation, the Minimum Standard Checklist for Evaluating HRQoL Outcomes in Cancer Clinical Trials and the CONSORT Patient-Reported Outcome extension were adapted. RESULTS: The database search yielded 48 eligible studies. We found the awareness for the need of HRQoL measurement to be growing and application of cancer-specific instruments gaining acceptance. Overall, studies were too heterogeneous in terms of patient characteristics and treatment interventions to draw clear conclusions for clinical practice. More importantly, a range of methodological shortcomings has been identified which were mainly related to the assessment and statistical analysis, as well as the reporting and interpretation of HRQoL data. CONCLUSION: Despite an increasing interest in HRQoL in GEP-NET patients, there is still a lack of knowledge on this issue. A transfer of HRQoL results into clinical practice is hindered not only by the scarceness of studies, but also by the often limited quality of HRQoL processing and reporting.

Cost-utility analyses of drug therapies in breast cancer: a systematic review.

The economic evaluation (EE) of health care products has become a necessity. Their quality must be high in order to trust the results and make informed decisions. While cost-utility analyses (CUAs) should be preferred to cost-effectiveness analyses in the oncology area, the quality of breast cancer (BC)-related CUA has been given little attention so far. Thus, firstly, a systematic review of published CUA related to drug therapies for BC, gene expression profiling, and HER2 status testing was performed. Secondly, the quality of selected CUA was assessed and the factors associated with a high-quality CUA identified. The systematic literature search was conducted in PubMed, MEDLINE/EMBASE, and Cochrane to identify published CUA between 2000 and 2014. After screening and data extraction, the quality of each selected CUA was assessed by two independent reviewers, using the checklist proposed by Drummond et al. The analysis of factors associated with a high-quality CUA (defined as a Drummond score ≥7) was performed using a two-step approach. Our systematic review was based on 140 CUAs and showed a wide variety of methodological approaches, including differences in the perspective adopted, the time horizon, measurement of cost and effectiveness, and more specially health-state utility values (HSUVs). The median Drummond score was 7 [range 3-10]. Only one in two of the CUA (n = 74) had a Drummond score ≥7, synonymous of "high quality." The statistically significant predictors of a high-quality CUA were article with "gene expression profiling" topic (p = 0.001), consulting or pharmaceutical company as main location of first author (p = 0.004), and articles with both incremental cost-utility ratio and incremental cost-effectiveness ratio as outcomes of EE (p = 0.02). Our systematic review identified only 140 CUAs published over the past 15 years with one in two of high quality. It showed a wide variety of methodological approaches, especially focused on HSUVs. A critical appraisal of utility values is necessary to better understand one of the main difficulties encountered by authors and propose areas for improvement to increase the quality of CUA. Since the last 5 years, there is a tendency toward an improvement in the quality of these studies, probably coupled with economic context, a better and widely spreading of recommendations and thus appropriation by medical practitioners. That being said, there is an urgent need for mandatory use of European and international recommendations to ensure quality of such approaches and to allow easy comparison.