Recommendations and guidelines for the collection, generation, source and usage of utility data for health technology assessment (HTA) vary across different countries, with no international consensus. Many international agencies generate their own guidelines providing details on their preferred methods for HTA submissions, and there is variability in both what they recommend and the clarity and amount of detail provided in their guidelines. This article provides an overview of international regulations and recommendations for utility data in HTA for a selection of key HTA countries: Australia, Canada, France, Germany, the Netherlands, Spain (Catalonia), Sweden and the UK (England/Wales and Scotland). International guidelines are typically clear and detailed for the selection of countries assessed regarding the source description of health states (e.g. generic preference-based measure) and who should provide preference weights for these health states (e.g. general population for own country). Many guidelines specify the use of off-the-shelf generic preference-based measures, and some further specify a measure, such as EQ-5D. However, international guidelines are either unclear or lack detailed guidance regarding the collection (e.g. patients report own health), source (e.g. clinical trial) and usage (e.g. adjusting for comorbidities) of utility values. It is argued that there is a need for transparent and detailed international guidelines on utility data recommendations to provide decision makers with the best possible evidence. Where this is not possible it is recommended that best practice should be used to inform the collection, source and usage of utility values in HTA.

A condition-specific preference-based measure (CSPBM) is a measure of health-related quality of life (HRQOL) that is specific to a certain condition or disease and that can be used to obtain the quality adjustment weight of the quality-adjusted life-year (QALY) for use in economic models. This article provides an overview of the role and the development of CSPBMs, and presents a description of existing CSPBMs in the literature. The article also provides an overview of the psychometric properties of CSPBMs in comparison with generic preference-based measures (generic PBMs), and considers the advantages and disadvantages of CSPBMs in comparison with generic PBMs. CSPBMs typically include dimensions that are important for that condition but may not be important across all patient groups. There are a large number of CSPBMs across a wide range of conditions, and these vary from covering a wide range of dimensions to more symptomatic or uni-dimensional measures. Psychometric evidence is limited but suggests that CSPBMs offer an advantage in more accurate measurement of milder health states. The mean change and standard deviation can differ for CSPBMs and generic PBMs, and this may impact on incremental cost-effectiveness ratios. CSPBMs have a useful role in HTA where a generic PBM is not appropriate, sensitive or responsive. However, due to issues of comparability across different patient groups and interventions, their usage in health technology assessment is often limited to conditions where it is inappropriate to use a generic PBM or sensitivity analyses.

Methodological issues of how to use health state utility values (HSUVs) in decision models arise frequently, including the most appropriate evidence to use as the baseline (e.g. the baseline HSUVs associated with avoiding a particular health condition or event), how to capture changes due to adverse events and how to appropriately capture uncertainty in progressive conditions where the expected change in quality of life is likely to be monotonically decreasing over time. As preference-based measures provide different values when collected from the same patient, it is important to ensure that all HSUVs used within a single model are obtained from the same instrument where ever possible. When people enter the model without the condition of interest (e.g. primary prevention of cardiovascular disease, screening or vaccination programmes), appropriate age- and gender-adjusted HSUVs from people without the particular condition should be used as the baseline. General population norms may be used as a proxy if the exact condition-specific evidence is not available. Individual discrete health states should be used for serious adverse reactions to treatment and the corresponding HSUVs sourced as normal. Care should be taken to avoid double counting when capturing the effects for both less severe adverse reactions (e.g. itchy skin rash or dry cough) and more severe adverse events (e.g. fatigue in oncology). Transparency in reporting standards for both the justification of the evidence used and any 'adjustments' is important to increase readers' confidence that the evidence used is the most appropriate available.

ObjectiveTo examine the clinical and economic impact of vedolizumab compared with conventional therapy in the treatment of moderately-to-severely active ulcerative colitis (UC) in the UK based on results of the GEMINI I trial.MethodsA decision-analytic model in Microsoft Excel was used to compare vedolizumab with conventional therapy (aminosalicylates, corticosteroids, immunomodulators) for the treatment of patients with UC in the UK. We considered the following three populations: the overall intent-to-treat population from the GEMINI I trial, patients naïve to anti-TNF therapy, and those who had failed anti-TNF-therapy. Population characteristics and efficacy data were obtained from the GEMINI I trial. Other inputs (eg, unit costs, probability of surgery, mortality) were obtained from published literature. Time horizon was a lifetime horizon, with costs and outcomes discounted by 3.5% per year. One-way and probabilistic sensitivity analyses were conducted to measure the impact of parameter uncertainty.ResultsVedolizumab had incremental cost-effectiveness ratios of £4,095/quality-adjusted life-year (QALY), £4,423/QALY, and £5,972/QALY compared with conventional therapy in the intent-to-treat, anti-TNF-naïve, and anti-TNF-failure populations, respectively. Patients on vedolizumab accrued more QALYs while incurring more costs than patients on conventional therapy. The sensitivity analyses showed that the results were most sensitive to induction response and transition probabilities for each treatment.ConclusionThe results suggest that vedolizumab results in more QALYs and may be a cost-effective treatment option compared with conventional therapy for both anti-TNF-naïve and anti-TNF-failure patients with moderately-to-severely active UC.

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come directly from patients whereas NICE in the UK, prefer values from the general population. This is essentially a normative judgement, but do these values differ? The aim of this study is to compare utility values elicited from the two samples of UK respondents: a general population sample and a sample of patients with Crohn's disease (CD). METHODS: Eight health state vignettes associated with complex perianal fistulae in CD were developed and refined from interviews with patients and these were validated by clinicians. An on-line time trade off (TTO) was used to elicit preferences. In addition to valuing the hypothetical vignettes, patients with CD were asked to value their own current health state. RESULTS: In total 835 respondents were included in the general population study of the and were reflective of the UK population in age and gender. In the patient study, 162 patients with CD were included. Non-remission states were valued much lower than the remission state by both samples: ranging from 0.20 for proctectomy with negative outcome to 0.66 for chronic symptomatic fistulae with mild symptoms. Patients reported slightly higher values than the general population for most health states. The relative difference in mean values was greatest for the most severe health states. This is consistent with our finding from the subgroup analysis of the general population sample in which respondents with experience (self, family or friend has CD) provided higher estimates than those without any experience of CD. CONCLUSIONS: Patients reported slightly higher values than the general population for most health states; the difference was greatest for the most severe health states.

OBJECTIVES: Under current economic and financial framework, some important revisions were made to the National Pharmaceutical Policy in Portugal, aimed at decreasing prices and contributing to lower public expenditure in the Health Sector. The Decree-Law 112/2011 introduced a new margin system both for pharmacies and wholesalers. A linear margin scheme, expressed as a percentage mark-up on the consumer price before VAT was thus replaced by a regressive system combining: i) a fixed fee that increases with the ex-factory price of drugs; ii) a regressive margin expressed as a percentage of the ex-factory price. This paper aims to assess: costs associated to the pharmacy dispensing and other pharmacy services provided; the proportion of purchased medicines on total prescribed; the perception of pharmacists and patients regarding changes in access to medicines, namely possible shortages of medicines in Portuguese pharmacies, and other issues. METHODS: In order to access the effects of this policy measure, surveys were carried out to consumers and pharmacies across the country. Four surveys were administered in week 25 to 29 June: survey to Pharmacy Owner; survey following each prescription sale (one day census); survey about pharmacy services not associated to the dispensing of medicines (5-day census). Pharmacists were also requested to administer a survey to patients presenting a prescription. RESULTS: To be completed after analysis of the surveys (after 29 June) CONCLUSIONS: We will elaborate on a more efficient distribution of medicines in view of both the economic sustainability of pharmacies and the impact on patients. OBJECTIVES:To explore opinions among professionals in health economics and related fields on global trends in the use of health economic (HE) data in various market access decision-making processes. METHODS: An on-line survey was administered to professionals who work with HE data. The survey captured professional background characteristics and respondents' opinions on trends in the role of HE data in various decision-making areas and specifically in applying HE analysis to individualized medicine and orphan medications. RESULTS: Seventy three professionals completed the survey; 53% from Europe, 30% United States, and 16% from other countries. 25% were from the pharmaceutical/medical technology industry, while 75% were from academia/government and other institution types. The area where most respondents expected an increasing role for HE data was in reimbursement decisions (89%), followed by manufacturers' internal pricing (78%), clinical guideline development (70%), and clinical practice (59%). Opinions on whether cost-effectiveness analysis of individualized medicine will become a dominant approach in the next three years varied widely with 49% of respondents in agreement and 19% disagreeing. Equally, 37% of the respondents agree and 37% disagree that orphan drugs should be subjected to the same value-based assessments as other products, with 26% being neutral. CONCLUSIONS: While there is strong...