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Introduction: Loss of cognitive function is a common feature in schizophrenia. However, generic measures of health-related quality of life favored by decision-makers, such as the EQ-5D, are not designed to detect changes in cognitive function. We report the valuation of the Schizophrenia Cognition Rating Scale (SCoRS), a schizophrenia-specific measure of cognitive impairment.Methods: Expert opinion and psychometric analysis of the SCoRS from clinical trial data was undertaken to select 5 key items from the measure. These items were combined orthogonally to develop health-state vignettes. Vignettes were valued using composite time trade-off (cTTO) in one-on-one video calls. Several econometric models were fitted to the data to estimate disutilities. Performance of EQ-5D- and SCoRS-based utilities were compared in the trial data. Results: The SCoRS items selected for the valuation study represented attention, learning, processing speed, social cognition and memory. Four hundred respondents participated in the valuation study. The best observed health state was valued at 0.855 [standard deviation (SD) = 0.179] and the worst at 0.152 (SD = 0.575). At the most severe levels, ‘social cognition’ received the largest disutility followed by ‘learning’ and ‘memory’. The final model to estimate utilities had 15 parameters. SCoRS-based utilities were sensitive to change in cognition, but the EQ-5D was not. Conclusion: It is feasible to value different dimensions of cognition separately using a validated instrument for proxy assessment. The resulting utilities indicate loss of quality of life due to reduced cognitive functioning.

Objectives: Evidence comparing utilities for adults and children consistently report higher utility values for child health states. This study investigates the reasons why child health states are valued differently. Methods: A total of 80 respondents (United Kingdom, Belgium, The Netherlands) participated in 1.5-hour face-to-face interviews. Respondents valued 4 health states from 2 perspectives (8-year-old child, 40-year-old adult) using visual analog scale and time trade-off. A total of 32 respondents participated in think-aloud interviews. Audio recordings were analyzed by 2 independent coders using NVIVO software. Statements, nodes, and themes were reviewed cyclically until consensus was reached. Results: Qualitative results: a total of 5 themes were identified in the data regarding child and adult valuation—intergenerational responsibility and dependency (childhood is crucial for forming life skills based on new experiences; adulthood is an important time to take care of the family), staying alive is important (life is worth living even with impaired health-related quality of life (HRQoL), for children and adults), awareness of poor HRQoL and ability to make decisions (children have difficulties comprehending poor HRQoL and their parents make their healthcare decision; adults can assess their own HRQoL and decide for themselves), coping ability (children are flexible and resilient; adults have experience with dealing with difficulties), and practical organization of care (children are cared for by their parents; adults are able to organize and pay for care). Mixed methods: comparing qualitative statements with respondents’ higher utilities for child health states confirmed concordance between results. Conclusions: Quality-adjusted life-years are interpreted differently for children and adults. Child-specific value sets are needed to reflect society's preferences and to adequately conduct health technology assessment of pediatric treatments.

Andrew J Lloyd,1 Robin Thompson,2 Katy Gallop,1 Megan Teynor3 1Acaster Lloyd Consulting Ltd., London, UK; 2Value and Access, Biogen, Baar, Switzerland; 3Value and Access, Biogen, Cambridge, MA, USACorrespondence: Andrew J LloydAcaster Lloyd Consulting Ltd., 16 Upper Woburn Place, London WC1H0BS, UKTel +44 20 3978 1681Email andrew.lloyd@acasterlloyd.comBackground: Spinal muscular atrophy (SMA) is a rare, genetic, progressive neuromuscular disorder characterized by severe muscle atrophy and weakness and is a leading genetic cause of death in infants and children. Nusinersen was the first treatment targeting the underlying cause of disease approved by the FDA, EMA and other countries for patients with SMA. There are currently very limited data available on the health-related quality of life (HRQoL) burden of SMA suitable for use in a cost-effectiveness analysis.Objective: This study was designed to estimate quality of life weights or utilities for different SMA states.Methods: SMA case studies were developed describing Type I (infantile onset) and Type II (later-onset) patients and different outcomes from treatment. These were developed so that quality of life weights or utilities (where the value of health ranges from 1 – full health to 0 – dead) could be estimated for cost-effectiveness analysis. Clinical experts (n=5) rated each of the case studies using standardized HRQoL instruments – the EQ-5D-Y and PedsQL-NMM (baseline states only).Results: The SMA Type I utilities ranged from −0.33 (requires ventilation) to 0.71 (Type I patient reclassified as Type III following treatment), with quite substantial differences between some states. Most Type I states had a utility score below zero indicating the severity of the states. The SMA Type II utilities ranged from −0.13 (worsened) to 0.72 (stands/walks unaided). In general, the results showed HRQoL improved in line with better health states.Conclusion: The utility scores o

Lina Eliasson,1 Anthony P Bewley,2 Farhan Mughal,3 Karissa M Johnston,4 Andreas Kuznik,5 Chloe Patel,1 Andrew J Lloyd1 1Clinical Outcomes Assessment, ICON Clinical Research UK Ltd, 2Department of Dermatology, Whipps Cross University Hospital, Barts Health National Health Service Trust, London, 3Health Economics Outcomes Research, Celgene Ltd, Uxbridge, UK; 4Epidemiology, ICON Commercialisation and Outcomes, Vancouver, BC, Canada; 5Global Health Economics and Outcomes Research, Celgene Corporation, Summit, NJ, USA Objective: Treatment options for psoriasis offer trade-offs in terms of efficacy, convenience, and risk of adverse events. We evaluated patients’ preferences with respect to benefit–risk in the treatment of psoriasis. Methods: A discrete choice experiment was conducted in adults from the UK with moderate-to-severe psoriasis using an orthogonal design with 32 hypothetical choice sets. Participants were randomly assigned to one of two surveys with 16 choice sets. Patients’ preferences were investigated with respect to the following attributes: reduction in body surface area affected by psoriasis, treatment administration (frequency and mode of delivery), short-term diarrhea or nausea risk, and 10-year risk of developing melanoma or nonmelanoma skin cancer, tuberculosis, or serious infections. A mixed effects logistic regression model generated relative preferences between treatment profiles. Results: Participants (N=292) had a strong preference to avoid increased risk of melanoma or nonmelanoma skin cancer (odds ratio [OR]: 0.44 per 5% increased 10-year risk) and increased risks of tuberculosis and serious infections (both ORs: 0.73 per 5% increased 10-year risk) and preferred once-weekly to twice-daily tablets (OR: 0.76) and weekly (OR: 0.56) or fortnightly (OR: 0.65) injections. Participants preferred avoiding treatments that may cause diarrhea or nausea in the first 2 weeks (OR: 0.87 per 5% increase) and preferred treatments t

Beenish Nafees,1 Andrew J Lloyd,2 Rachel S Ballinger,2 Anton Emmanuel3 1Health Outcomes Research, Nafees Consulting Limited, London, 2Patient-Reported Outcomes Research, ICON plc, Oxford, 3Department of Gastroenterology and Nutrition, University College Hospital, London, UK Background: Most patients with bowel dysfunction secondary to neurological illness are managed by a range of nonsurgical methods, including dietary changes, laxatives, and suppository use to transanal irrigation (TAI). The aim of the present study was to explore individuals’ preferences regarding TAI devices and furthermore investigate willingness to pay (WTP) for attributes in devices in the UK. Methods: A discrete choice experiment survey was conducted to evaluate the patients’ perceived value of TAI devices. Attributes were selected based upon a literature review and input from clinicians. Interviews were conducted with three clinicians and the survey was developed and finalized with the input from both patients and professionals. The final attributes were “risk of urinary tract infections” (UTIs), “risk of fecal incontinence” (FI), “frequency of use”, “time spent on toilet”, “ease of use”, “level of control/independence”, and “cost”. Participants were recruited by a patient panel of TAI device users in the UK. Data were analyzed using the conditional logit model whereby the coefficients obtained from the model provided an estimate of the (log) odds ratios (ORs) of preference for attributes. WTP was also estimated for each attribute. Results: A total of 129 participants were included in the final analyses. Sixty two percent of the participants had suffered from three UTIs in the preceding year and 58% of patients reported currently experiencing FI using their current device. All attributes were significant predictors of choice.

PurposeThe aim of this study was to elicit utilities for radioactive iodine-refractory differentiated thyroid cancer (RR-DTC) and evaluate the impact of treatment response and toxicities on quality of life.Patients and methodsRR-DTC health states were developed based on data from a previous qualitative study and iterative review by clinical experts. Following piloting, health states underwent valuation by 100 members of the UK public during time trade-off interviews. Mean utilities and descriptive distribution statistics were calculated, and a logistic regression analysis was conducted.ResultsThe demographic characteristics of the study sample were generally reflective of the UK population. Clear differentiation in valuation between health states was observed. No response/stable disease had an adjusted utility value of 0.87, with a corresponding gain of +0.04 following a treatment response and a decline of -0.35 for disease progression. Adverse events were associated with utility decrements between -0.47 (grade III diarrhea) and -0.05 (grade I/II alopecia).ConclusionThe trade-off interviews derived utility weights show clear differentiation between RR-DTC health states in response to treatment. The values reported in this study are suitable for cost-effectiveness evaluations for new treatments in RR-DTC.

PurposeThe aim of this study was to elicit utilities for radioactive iodine-refractory differentiated thyroid cancer (RR-DTC) and evaluate the impact of treatment response and toxicities on quality of life.Patients and methodsRR-DTC health states were developed based on data from a previous qualitative study and iterative review by clinical experts. Following piloting, health states underwent valuation by 100 members of the UK public during time trade-off interviews. Mean utilities and descriptive distribution statistics were calculated, and a logistic regression analysis was conducted.ResultsThe demographic characteristics of the study sample were generally reflective of the UK population. Clear differentiation in valuation between health states was observed. No response/stable disease had an adjusted utility value of 0.87, with a corresponding gain of +0.04 following a treatment response and a decline of -0.35 for disease progression. Adverse events were associated with utility decrements between -0.47 (grade III diarrhea) and -0.05 (grade I/II alopecia).ConclusionThe trade-off interviews derived utility weights show clear differentiation between RR-DTC health states in response to treatment. The values reported in this study are suitable for cost-effectiveness evaluations for new treatments in RR-DTC.

Beth A Fordham,1 Cicely Kerr,1 Hayley M de Freitas,1 Andrew J Lloyd,1 Karissa Johnston,2 Corey L Pelletier,3 Gabriel Tremblay,3 Anna Forsythe,3 Bryan McIver,4 Ezra EW Cohen5 1ICON Patient Reported Outcomes, Oxford, UK; 2ICON Epidemiology, Toronto, ON, Canada; 3Eisai Inc., NJ, 4Moffitt Cancer Center, FL, 5University of California San Diego Moores Cancer Center, La Jolla, CA, USA Purpose: The aim of this study was to elicit utilities for radioactive iodine-refractory differentiated thyroid cancer (RR-DTC) and evaluate the impact of treatment response and toxicities on quality of life.Patients and methods: RR-DTC health states were developed based on data from a previous qualitative study and iterative review by clinical experts. Following piloting, health states underwent valuation by 100 members of the UK public during time trade-off interviews. Mean utilities and descriptive distribution statistics were calculated, and a logistic regression analysis was conducted.Results: The demographic characteristics of the study sample were generally reflective of the UK population. Clear differentiation in valuation between health states was observed. No response/stable disease had an adjusted utility value of 0.87, with a corresponding gain of +0.04 following a treatment response and a decline of –0.35 for disease progression. Adverse events were associated with utility decrements between –0.47 (grade III diarrhea) and –0.05 (grade I/II alopecia).Conclusion: The trade-off interviews derived utility weights show clear differentiation between RR-DTC health states in response to treatment. The values reported in this study are suitable for cost-effectiveness evaluations for new treatments in RR-DTC. Keywords: differentiated thyroid cancer, radioactive iodine-refractory, health-related quality of life, health utility, vignette

Andrew J Lloyd,1 Beenish Nafees,1 Eddy Ziani,2 Laurence Nicolas,2 Beth Alice Fordham,1 Benoit Soubeyrand,2 Christoph Bornhöft3 1Patient Reported Outcomes, ICON Plc, Oxford, UK; 2Medical Affairs, Sanofi Pasteur MSD, Lyon, France; 3Practice for Pediatric and Adolescent Medicine, Bensheim, Germany Abstract: Diphtheria, tetanus and acellular pertussis (DTaP) based combined vaccines have led to significant reduction in incidence of several serious pediatric infectious diseases. A new, fully liquid combined hexavalent vaccine has been introduced and has been shown to reduce administration time. This fully liquid vaccine may also be simpler to administer and could reduce handling errors. The present study was designed to understand the value that health care providers (HCPs) place on aspects of injection devices for combined hexavalent vaccine programs in Germany. A discrete choice experiment (DCE) was designed to elicit the views of HCPs regarding hexavalent vaccines. The key attributes of injection devices were identified through a focused literature search and interviews with HCPs. Five key attributes, each with two or three levels were described which included: type of device, experience of this hexavalent vaccine on the German market, preparation time, probability of handling errors, and dosage errors. Physicians (n=150) and nurses (n=150) who administered hexavalent vaccines in Germany completed the survey. Choice data were analyzed using the conditional logit procedure. All attributes were significant and important independent influences on physicians’ and nurses’ choices. Reducing any “probability of dosage errors” was the most important attribute. Both physicians and nurses had a strong preference to reduce preparation time. All other things equal both groups also significantly preferred a fully liquid hexavalent vaccine. They also preferred vaccines that had been on the market for a few years compared to ones that