The Valuation of Health Outcomes: A Contribution to the QALY Approach

The aim of any medical intervention is to improve or sustain health. Therefore, the assessment of health outcome is an important aspect in the scientific evaluation of medical effectiveness. Nowadays this is an important activity. Three major aspects of health outcome can be distinguished. The most prominent is survival, which defines the prevention of premature death as a major goal of care. Disease-specific clinical characteristics of the serio liS ness of the pathology are a second aspect of health outcome. More recently, a third aspect has been developed to express an entity that was previously more or less implicit: health status. Especially in the more prosperous part of the world where life expectancy has shown an impressive increase, a shifr of emphasis from the attention of quantity of life towards the quality of health, i.e., to health status can be noticed

Patient-reported health outcomes in long-term lung transplantation survivors

During the last three decades lung transplantation (LTx) has become a proven modality for increasing both survival and health-related quality of life (HRQoL) in patients with various end-stage lung diseases. Most previous studies have reported improved HRQoL shortly after LTx. With regard to long-term effects on HRQoL, however, the evidence is less solid. This prospective cohort study was started with 828 patients who were on the waiting list for LTx. Then, in a longitudinal follow-up, 370 post-LTx patients were evaluated annually for up to 15 years. For all wait-listed and follow-up patients, the following four HRQoL instruments were administered: State-Trait Anxiety Inventory, Zung Self-rating Depression Scale, Nottingham Health Profile, and a visual analogue scale. Cross-sectional and generalized estimating equation (GEE) analysis for repeated measures were performed to assess changes in HRQoL during follow-up. After LTx, patients showed improvement in all HRQoL domains except pain, which remained steady throughout the long-term follow-up. The level of anxiety and depressive symptoms decreased significantly and remained constant. In conclusion, this study showed that HRQoL improves after LTx and tends to remain relatively constant for the entire life span

Measurement properties of the EQ-5D across four major geriatric conditions: Findings from TOPICS-MDS

Background: As populations age, chronic geriatric conditions linked to progressive organ failure jeopardize health-related quality of life (HRQoL). Thus, this research assessed the validity and applicability of the EQ-5D (a common HRQoL instrument) across four major chronic geriatric conditions: hearing issues, joint damage, urinary incontinence, or dizziness with falls. Methods: The study sample comprised 25,637 community-dwelling persons aged 65 years and older residing in the Netherlands (Data source: TOPICS-MDS, www.topics-mds.eu ). Floor and ceiling effects were examined. To assess convergent validity, random effects meta-correlations (Spearman's rho) were derived between individual EQ-5D domains and related survey items. To further examine construct validity, the association between sociodemographic characteristics and EQ-5D summary scores were assessed using linear mixed models. Outcomes were compared to the overall study population as well as a 'healthy' subgroup reporting no major chronic conditions. Results: Whereas ceiling effects were observed in the overall study population and the 'healthy' subgroup, such was not the case in the geriatric condition subgroups. The majority of hypotheses regarding correlations between survey items and sociodemographic associations were supported. EQ-5D summary scores were lower in respondents who were older, female, widowed/single, lower educated, and living alone. Increasing co-morbidity had a clear negative effect on EQ-5D scores. Conclusion: This study supported the construct validity of the EQ-5D across four major geriatric conditions. For older persons who are generally healthy, i.e. reporting few to no chronic conditions, the EQ-5D confers poor discriminative ability due to ceiling effects. Although the overall dataset initially suggested poor discriminative ability for the EQ-5D, such was not the case within subgroups presenting with major geriatric conditions

The bilirubin albumin ratio in the management of hyperbilirubinemia in preterm infants to improve neurodevelopmental outcome: A randomized controlled trial - BARTrial

Background and Objective: High bilirubin/albumin (B/A) ratios increase the risk of bilirubin neurotoxicity. The B/A ratio may be a valuable measure, in addition to the total serum bilirubin (TSB), in the management of hyperbilirubinemia. We aimed to assess whether the additional use of B/A ratios in the management of hyperbilirubinemia in preterm infants improved neurodevelopmental outcome. Methods: In a prospective, randomized controlled trial, 615 preterm infants of 32 weeks' gestation or less were randomly assigned to treatment based on either B/A ratio and TSB thresholds (consensus-based), whichever threshold was crossed first, or on the TSB thresholds only. The primary outcome was neurodevelopment at 18 to 24 months' corrected age as assessed with the Bayley Scales of Infant Development III by investigators unaware of treatment allocation. Secondary outcomes included complications of preterm birth and death. Results: Composite motor (100±13 vs. 101±12) and cognitive (101±12 vs. 101±11) scores did not differ between the B/A ratio and TSB groups. Demographic characteristics, maximal TSB levels, B/A ratios, and other secondary outcomes were similar. The rates of death and/or severe neurodevelopmental impairment for th

Examining the construct and known-group validity of a composite endpoint for the Older Persons and Informal Caregivers Survey Minimum Data Set (TOPICS-MDS); A largescale data sharing initiative

Background Preference-weighted multi-faceted endpoints have the potential to facilitate comparative effectiveness research that incorporates patient preferences. The Older Persons and Informal Caregivers Survey Composite endpoint (TOPICS-CEP) is potentially a valuable outcome measure for evaluating interventions in geriatric care as it combines multiple outcomes relevant to older persons in a single metric. The objective of this study was to validate TOPICS-CEP across different study settings (general population, primary care and hospital). Methods Data were extracted from TOPICS Minimum Dataset (MDS), a pooled public-access national database with information on older persons throughout the Netherlands. Data of

Understanding drug preferences, different perspectives

Aims To compare the values regulators attach to different drug effects of oral antidiabetic drugs with those of doctors and patients. Methods We administered a 'discrete choice' survey to regulators, doctors and patients with type 2 diabetes in The Netherlands. Eighteen choice sets comparing two hypothetical oral antidiabetic drugs were constructed with varying drug effects on glycated haemoglobin, cardiovascular risk, bodyweight, duration of gastrointestinal complaints, frequency of hypoglycaemia and risk of bladder cancer. Responders were asked each time which drug they preferred. Results Fifty-two regulators, 175 doctors and 226 patients returned the survey. Multinomial conditional logit analyses showed that cardiovascular risk reduction was valued by regulators positively (odds ratio 1.98, 95% confidence interval 1.11-3.53), whereas drug choices were negatively affected by persistent gastrointestinal problems (odds ratio 0.24, 95% confidence interval 0.14-0.41) and cardiovascular risk increase (odds ratio 0.49, 95% confidence interval 0.27-0.87). Doctors and patients valued these effects in a similar manner to regulators. The values that doctors attached to large changes in glycated haemoglobin and that both doctors and patients attached to hypoglycaemia and weight gain also reached statistical significance. No group's drug choice was affected by a small absolute change in risk of bladder cancer when presented in the context of other drug effects. When comparing the groups, the value attached by regulators to less frequent hypoglycaemic episodes was significantly smaller than by patients (P = 0.044). Conclusions Regulators may value major benefits and risks of drugs for an individual diabetes patient mostly in the same way as doctors and patients, but differences may exist regarding the value of minor or short-term drug effects