Public stated preferences and predicted uptake for genome-based colorectal cancer screening

Background Emerging developments in nanomedicine allow the development of genome-based technologies for non-invasive and individualised screening for diseases such as colorectal cancer. The main objective of this study was to measure user preferences for colorectal cancer screening using a nanopill. Methods A discrete choice experiment was used to estimate the preferences for five competing diagnostic techniques including the nanopill and iFOBT. Alternative screening scenarios were described using five attributes namely: preparation involved, sensitivity, specificity, complication rate and testing frequency. Fourteen random and two fixed choice tasks, each consisting of three alternatives, were offered to 2225 individuals. Data were analysed using the McFadden conditional logit model. Results Thirteen hundred and fifty-six respondents completed the questionnaire. The most important attributes (and preferred levels) were the screening technique (nanopill), sensitivity (100%) and preparation (no preparation). Stated screening uptake for the nanopill was 79%, compared to 76% for iFOBT. In the case of screening with the nanopill, the percentage of people preferring not to be screened would be reduced from 19.2% (iFOBT) to 16.7%. Conclusions Although the expected benefits of nanotechnology based colorectal cancer screening are improved screening uptake, assuming more accurate test results and less preparation involved, the relative preference of the nanopill is only slightly higher than the iFOBT. Estimating user preferences during the development of diagnostic technologies could be used to identify relative performance, including perceived benefits and harms compared to competitors allowing for significant changes to be made throughout the process of developmen

Patients’ Priorities for Oral Anticoagulation Therapy in Non-valvular Atrial Fibrillation:a Multi-criteria Decision Analysis

Introduction: Effectiveness of oral anticoagulants (OACs) is critically dependent on patients’ adherence to intake regimens. We studied the relative impact of attributes related to effectiveness, safety, convenience, and costs on the value of OAC therapy from the perspective of patients with non-valvular atrial fibrillation. Methods: Four attributes were identified by literature review and expert interviews: effectiveness (risk of ischemic stroke), safety (risk of major bleeding, minor bleeding, gastrointestinal complaints), convenience (intake frequency, diet restrictions, international normalized ratio [INR] blood monitoring, pill type/intake instructions), and out-of-pocket costs. Focus groups were held in Spain, Germany, France, Italy and the United Kingdom (N = 48) to elicit patients’ preferences through the use of the analytical hierarchy process method. Results: Effectiveness (60%) and side effects (27%) have a higher impact on the perceived value of OACs than drug convenience (7%) and out-of-pocket costs (6%). As for convenience, eliminating monthly INR monitoring was given the highest priority (40%), followed by reducing diet restrictions (27%), reducing intake frequency (17%) and improving the pill type/intake instructions (15%). The most important side effect was major bleeding (75%), followed by minor bleeding (15%) and gastrointestinal complaints (10%). Furthermore, 71% of patients preferred once-daily intake to twice-daily intake. Discussion: Although the relative impact of convenience on therapy value is small, patients have different preferences for options within convenience criteria. Besides considerations on safety and effectiveness, physicians should also discuss attributes of convenience with patients, as it can be assumed that alignment to patient preferences in drug prescription and better patient education could result in higher adherence

Postpartum depression and anxiety:a community-based study on risk factors before, during and after pregnancy

Background: Depression and anxiety occur frequently postpartum, calling for early detection and treatment. Evidence on risk factors may support early detection, but is inconclusive. Our aim was to identify risk factors for postpartum depression and anxiety, before, during and after pregnancy. Methods: We used data from 1406 mothers of the intervention arm of the Post-Up study. Risk factors were collected at 3 weeks and 12 months postpartum. Depression and anxiety symptoms were measured in the first month postpartum by the Edinburgh Postnatal Depression Scale (EPDS) and 6-item State-Trait Anxiety Inventory (STAI-6), respectively. We used stepwise logistic regression to identify relevant risk factors. Results: Of the mothers, 8.0% had EPDS-scores ≥9 and 14.7% STAI-6-scores ≥42. Factors associated with higher risk of depression were: foreign language spoken at home, history of depression, low maternal self-efficacy and poor current health of the mother. No initiation of breastfeeding was associated with lower risk of depression, no breastfeeding at 3 weeks postpartum increased the risk. Factors associated with higher risk of anxiety were: higher educational level, history of depression, preterm birth, negative experience of delivery and first week postpartum, excessive infant crying, low maternal self-efficacy, low partner support and poor current maternal health. Limitations: Use of a self-report instrument, potential bias by postpartum mood status, and no inclusion of emerging depression cases after one month postpartum. Conclusions: The shared and separate risk factors for postpartum depression and anxiety may help professionals in identifying mothers at increased risk and provide opportunities for preventive interventions and treatment

Helmet therapy in infants with positional skull deformation: randomised controlled trial

Objective To determine the effectiveness of helmet therapy for positional skull deformation compared with the natural course of the condition in infants aged 5-6 months. Design Pragmatic, single blinded, randomised controlled trial (HEADS, HElmet therapy Assessment in Deformed Skulls) nested in a prospective cohort study. Setting 29 paediatric physiotherapy practices; helmet therapy was administered at four specialised centres. Participants 84 infants aged 5 to 6 months with moderate to severe skull deformation, who were born after 36 weeks of gestation and had no muscular torticollis, craniosynostosis, or dysmorphic features. Participants were randomly assigned to helmet therapy (n=42) or to natural course of the condition (n=42) according to a randomisation plan with blocks of eight. Interventions Six months of helmet therapy compared with the natural course of skull deformation. In both trial arms parents were asked to avoid any (additional) treatment for the skull deformation. Main outcome measures The primary outcome was change in skull shape from baseline to 24 months of age assessed using plagiocephalometry (anthropometric measurement instrument). Change scores for plagiocephaly (oblique diameter difference index) and brachycephaly (cranioproportional index) were each included in an analysis of covariance, using baseline values as the covariate. Secondary outcomes were ear deviation, facial asymmetry, occipital lift, and motor development in the infant, quality of life (infant and parent measures), and parental satisfaction and anxiety. Baseline measurements were performed in infants aged between 5 and 6 months, with follow-up measurements at 8, 12, and 24 months. Primary outcome assessment at 24 months was blinded. Results The change score for both plagiocephaly and brachycephaly was equal between the helmet therapy and natural course groups, with a mean difference of −0.2 (95% confidence interval −1.6 to 1.2, P=0.80) and 0.2 (−1.7 to 2.2, P=0.81), respectively. Full recovery was achieved in 10 of 39 (26%) participants in the helmet therapy group and 9 of 40 (23%) participants in the natural course group (odds ratio 1.2, 95% confidence interval 0.4 to 3.3, P=0.74). All parents reported one or more side effects. Conclusions Based on the equal effectiveness of helmet therapy and skull deformation following its natural course, high prevalence of side effects, and high costs associated with helmet therapy, we discourage the use of a helmet as a standard treatment for healthy infants with moderate to severe skull deformatio

Response to Pediatric Physical Therapy in Infants With Positional Preference and Skull Deformation

Background Pediatric physical therapy (PPT) seems to reduce skull deformation in infants with positional preference. However, not all infants show improvement. \ud \ud Objective The purpose of this study was to determine which infant and parent characteristics were related to response to PPT in 2-4 month-old infants with positional preference and/or skull deformation. \ud \ud Design A prospective cohort study. \ud \ud Methods Infants 2–4 months old with positional preference and/or skull deformation were recruited by pediatric physical therapists at the start of PPT. Primary outcome was good or poor response (moderate/severe skull deformation) at 4.5 to 6.5 months of age. Potential predictors for response to PPT were assessed at baseline using questionnaires, plagiocephalometry, and the Alberta Infant Motor Scale. Univariate and multiple logistic regression analyses using a stepwise backward elimination method were performed. \ud \ud Results 657 infants participated in the study. At follow-up 364 infants (55.4%) showed poor response and 293 infants (44.6%) good response to therapy. Multiple logistic regression analysis resulted in the identification of four significant predictors at baseline for poor response to PPT: starting therapy after 3 months of age (adjusted odds ratio [aOR]: 1.50, 95% CI 1.04 to 2.17), skull deformation (plagiocephaly (aOR: 2.64, 1.67 to 4.17), brachycephaly (aOR: 3.07, 2.09 to 4.52)) and a low parental satisfaction score (aOR: 2.64, 1.67 to 4.17). \ud \ud Limitations Information about PPT was collected retrospectively and concerned general therapy characteristics. Subsequently no adjustment for therapy for the individual participants could be made. \ud \ud Conclusions Four predictors for response to PPT in infants of 2-4 months of age with positional preference and/or skull deformation were identified. Health professionals can use these predictors in daily practice to provide infants with more individualized therapy, resulting in better chances of a good outcom

Research Priorities to Increase Confidence in and Acceptance of Health Preference Research:What Questions Should be Prioritized Now?

Background and Objective: There has been an increase in the study and use of stated-preference methods to inform medicine development decisions. The objective of this study was to identify prioritized topics and questions relating to health preferences based on the perspective of members of the preference research community. Methods: Preference research stakeholders from industry, academia, consultancy, health technology assessment/regulatory, and patient organizations were recruited using professional networks and preference-targeted e-mail listservs and surveyed about their perspectives on 19 topics and questions for future studies that would increase acceptance of preference methods and their results by decision makers. The online survey consisted of an initial importance prioritization task, a best-worst scaling case 1 instrument, and open-ended questions. Rating counts were used for analysis. The best-worst scaling used a balanced incomplete block design. Results: One hundred and one participants responded to the survey invitation with 66 completing the best-worst scaling. The most important research topics related to the synthesis of preferences across studies, transferability across populations or related diseases, and method topics including comparison of methods and non-discrete choice experiment methods. Prioritization differences were found between respondents whose primary affiliation was academia versus other stakeholders. Academic researchers prioritized methodological/less studied topics; other stakeholders prioritized applied research topics relating to consistency of practice. Conclusions: As the field of health preference research grows, there is a need to revisit and communicate previous work on preference selection and study design to ensure that new stakeholders are aware of this work and to update these works where necessary. These findings might encourage discussion and alignment among different stakeholders who might hold different research priorities. Research on the application of previous preference research to new contexts will also help increase the acceptance of health preference information by decision makers.</p

Exploring the importance of controlling heteroskedasticity and heterogeneity in health valuation: a case study on Dutch EQ-5D-5L

Background: Respondents in a health valuation study may have different sources of error (i.e., heteroskedasticity), tastes (differences in the relative effects of each attribute level), and scales (differences in the absolute effects of all attributes). Although prior studies have compared values by preference-elicitation tasks (e.g., paired comparison [PC] and best–worst scaling case 2 [BWS]), no study has yet controlled for heteroskedasticity and heterogeneity (taste and scale) simultaneously in health valuation. Methods: Preferences on EQ-5D-5L profiles were elicited from a random sample of 380 adults from the general population of the Netherlands, using 24 PC and 25 BWS case 2 tasks. To control for heteroskedasticity and heterogeneity (taste and scale) simultaneously, we estimated Dutch EQ-5D-5L values using conditional, heteroskedastic, and scale-adjusted latent class (SALC) logit models by maximum likelihood. Results: After controlling for heteroskedasticity, the PC and BWS values were highly correlated (Pearson's correlation: 0.9167, CI: 0.9109–0.9222) and largely agreed (Lin's concordance: 0.7658, CI: 0.7542–0.7769) on a pits scale. In terms of preference heterogeneity, some respondents (mostly young men) failed to account for any of the EQ-5D-5L attributes (i.e., garbage class), and others had a lower scale (59%; p-value: 0.123). Overall, the SALC model produced a consistent Dutch EQ-5D-5L value set on a pits scale, like the original study (Pearson's correlation:0.7295; Lin's concordance: 0.6904). Conclusions: This paper shows the merits of simultaneously controlling for heteroskedasticity and heterogeneity in health valuation. In this case, the SALC model dispensed with a garbage class automatically and adjusted the scale for those who failed the PC dominant task. Future analysis may include more behavioral variables to better control heteroskedasticity and heterogeneity in health valuation. Highlights: The Dutch EQ-5D-5L values based on paired comparison [PC] and best-worst scaling [BWS] responses were highly correlated and largely agreed after controlling for heteroskedasticity.Controlling for taste and scale heterogeneity simultaneously enhanced the Dutch EQ-5D-5Lvalues by automatically dispensing with a garbage class and adjusting the scale for those who failed the dominant task.After controlling for heteroskedasticity and heterogeneity, this study produced Dutch EQ-5D-5L values on a pits scale moderately concordant with the original values

Valuing Treatments for Parkinson Disease Incorporating Process Utility: Performance of Best-Worst Scaling, Time Trade-Off, and Visual Analogue Scales

Objective The objective of this study was to compare treatment profiles including both health outcomes and process characteristics in Parkinson disease using best-worst scaling (BWS), time trade-off (TTO), and visual analogue scales (VAS). Methods From the model comprising of seven attributes with three levels, six unique profiles were selected representing process-related factors and health outcomes in Parkinson disease. A Web-based survey (N = 613) was conducted in a general population to estimate process-related utilities using profile-based BWS (case 2), multiprofile-based BWS (case 3), TTO, and VAS. The rank order of the six profiles was compared, convergent validity among methods was assessed, and individual analysis focused on the differentiation between pairs of profiles with methods used. Results The aggregated health-state utilities for the six treatment profiles were highly comparable for all methods and no rank reversals were identified. On the individual level, the convergent validity between all methods was strong; however, respondents differentiated less in the utility of closely related treatment profiles with a VAS or TTO than with BWS. For TTO and VAS, this resulted in nonsignificant differences in mean utilities for closely related treatment profiles. Conclusions This study suggests that all methods are equally able to measure process-related utility when the aim is to estimate the overall value of treatments. On an individual level, such as in shared decision making, BWS allows for better prioritization of treatment alternatives, especially if they are closely related. The decision-making problem and the need for explicit trade-off between attributes should determine the choice for a metho