Incorporating MCDA into HTA: challenges and potential solutions, with a focus on lower income settings

Background: Multicriteria decision analysis (MCDA) has the potential to bring more structure and transparency to health technology assessment (HTA). The objective of this paper is to highlight key methodological and practical challenges facing the use of MCDA for HTA, with a particular focus on lower and middle-income countries (LMICs), and to highlight potential solutions to these challenges. Methodological challenges: Key lessons from existing applications of MCDA to HTA are summarized, including: that the socio-technical design of the MCDA reflect the local decision problem; the criteria set properties of additive models are understood and applied; and the alternative approaches for estimating opportunity cost, and the challenges with these approaches are understood. Practical challenges: Existing efforts to implement HTA in LMICs suggest a number of lessons that can help overcome the practical challenges facing the implementation of MCDA in LMICs, including: adapting inputs from other settings and from expert opinion; investing in technical capacity; embedding the MCDA in the decision-making process; and ensuring that the MCDA design reflects local cultural and social factors. Conclusion: MCDA has the potential to improve decision making in LMICs. For this potential to be achieved, it is important that the lessons from existing applications of MCDA are learned

Emerging Use of Early Health Technology Assessment in Medical Product Development: A Scoping Review of the Literature

Early health technology assessment is increasingly being used to support health economic evidence development during early stages of clinical research. Such early models can be used to inform research and development about the design and management of new medical technologies to mitigate the risks, perceived by industry and the public sector, associated with market access and reimbursement. Over the past 25 years it has been suggested that health economic evaluation in the early stages may benefit the development and diffusion of medical products. Early health technology assessment has been suggested in the context of iterative economic evaluation alongside phase I and II clinical research to inform clinical trial design, market access, and pricing. In addition, performing early health technology assessment was also proposed at an even earlier stage for managing technology portfolios. This scoping review suggests a generally accepted definition of early health technology assessment to be “all methods used to inform industry and other stakeholders about the potential value of new medical products in development, including methods to quantify and manage uncertainty”. The present review also aimed to identify recent published empirical studies employing an early-stage assessment of a medical product. With most included studies carried out to support a market launch, the dominant methodology was early health economic modeling. Further methodological development is required, in particular, by combining systems engineering and health economics to manage uncertainty in medical product portfolios

Patient and Public Preferences for Treatment Attributes in Parkinson's Disease

BACKGROUND: Patient and public preferences for therapeutic outcomes or medical technologies are often elicited, and discordance between the two is frequently reported. OBJECTIVE: Our main objective was to compare patient and public preferences for treatment attributes in Parkinson's disease (PD). METHODS: A representative sample from Dutch PD patients and the general public were invited to complete a best-worst scaling case 2 experiment consisting of six health-related outcomes and one attribute describing the specific treatment (brain surgery, pump, oral medication). Data were analyzed using mixed logit models, and attribute impact was estimated and compared between populations (and population subgroups). RESULTS: Both the public (N = 276) and patient (N = 198) populations considered treatment modality the most important attribute, although patients assigned higher relative importance. Both groups assigned high disutility to pump infusion and brain surgery and preferred drug treatment. Most health outcomes were valued equally by patients and the public, with the exception of reducing dizziness (more important to the public) and improving slow movement (more important to patients). DISCUSSION: Although these data do not support definite conclusions on whether patients are less likely to undergo invasive treatments, the (predicted) choice probability of undergoing brain surgery or having pump infusion technology would be low based on the (un)desirability of the attribute levels. Patients with PD might have adapted to their condition and are not willing to undergo advanced treatments in order to receive health improvements. Both public and patient preferences entail information that is potentially relevant for decision makers, and patient preferences can inform decision makers about the likelihood of adaptation to a specific condition

Tech-assisted language learning tasks in an EFL setting: use of hand phone recording feature

Technology with its speedy great leaps forward has undeniable impact on every aspect of our life in the new millennium. It has supplied us with different affordances almost daily or more precisely in a matter of hours. Technology and Computer seems to be a break through as for their roles in the Twenty-First century educational system. Examples are numerous, among which CALL, CMC, and Virtual learning spaces come to mind instantly. Amongst the newly developed gadgets of today are the sophisticated smart Hand phones which are far more ahead of a communication tool once designed for. Development of Hand phone as a wide-spread multi-tasking gadget has urged researchers to investigate its effect on every aspect of learning process including language learning. This study attempts to explore the effects of using cell phone audio recording feature, by Iranian EFL learners, on the development of their speaking skills. Thirty-five sophomore students were enrolled in a pre-posttest designed study. Data on their English speaking experience using audio–recording features of their Hand phones were collected. At the end of the semester, the performance of both groups, treatment and control, were observed, evaluated, and analyzed; thereafter procured qualitatively at the next phase. The quantitative outcome lent support to integrating Hand phones as part of the language learning curriculum

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

Involving Patients in Weighting Benefits and Harms of Treatment in Parkinson's Disease

INTRODUCTION: Little is known about how patients weigh benefits and harms of available treatments for Parkinson's Disease (oral medication, deep brain stimulation, infusion therapy). In this study we have (1) elicited patient preferences for benefits, side effects and process characteristics of treatments and (2) measured patients' preferred and perceived involvement in decision-making about treatment. METHODS: Preferences were elicited using a best-worst scaling case 2 experiment. Attributes were selected based on 18 patient-interviews: treatment modality, tremor, slowness of movement, posture and balance problems, drowsiness, dizziness, and dyskinesia. Subsequently, a questionnaire was distributed in which patients were asked to indicate the most and least desirable attribute in nine possible treatment scenarios. Conditional logistic analysis and latent class analysis were used to estimate preference weights and identify subgroups. Patients also indicated their preferred and perceived degree of involvement in treatment decision-making (ranging from active to collaborative to passive). RESULTS: Two preference patterns were found in the patient sample (N = 192). One class of patients focused largely on optimising the process of care, while the other class focused more on controlling motor-symptoms. Patients who had experienced advanced treatments, had a shorter disease duration, or were still employed were more likely to belong to the latter class. For both classes, the benefits of treatment were more influential than the described side effects. Furthermore, many patients (45%) preferred to take the lead in treatment decisions, however 10.8% perceived a more passive or collaborative role instead. DISCUSSION: Patients weighted the benefits and side effects of treatment differently, indicating there is no "one-size-fits-all" approach to choosing treatments. Moreover, many patients preferred an active role in decision-making about treatment. Both results stress the need for physicians to know what is important to patients and to share treatment decisions to ensure that patients receive the treatment that aligns with their preferences

Women’s preferences, willingness-to-pay, and predicted uptake for single-nucleotide polymorphism gene testing to guide personalized breast cancer screening strategies: a discrete choice experiment

Xin Yi Wong,1 Catharina GM Groothuis-Oudshoorn,2 Chuen Seng Tan,3 Janine A van Til,2 Mikael Hartman,3,4 Kok Joon Chong,5 Maarten J IJzerman,2,6,7 Hwee-Lin Wee1,3 1Department of Pharmacy, Faculty of Science, National University of Singapore, Singapore, Republic of Singapore; 2Department of Health Technology and Services Research, Faculty of Behavioural, Management and Social Sciences, Technical Medical Centre, University of Twente, Enschede, The Netherlands; 3Saw Swee Hock School of Public Health, National University of Singapore and National University Health System, Singapore, Republic of Singapore; 4Department of Surgery, Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Republic of Singapore; 5Department of Medicine, Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Republic&nbsp;of Singapore; 6Faculty of Medicine, Dentistry and Health Sciences, University of Melbourne, Melbourne, Australia; 7Victorian Comprehensive Cancer Centre, Melbourne, Australia Background: Single-nucleotide polymorphism (SNP) gene test is a potential tool for improving the accuracy of breast cancer risk prediction. We seek to measure women&rsquo;s preferences and marginal willingness-to-pay (mWTP) for this new technology. Materials and methods: We administered a discrete choice experiment (DCE) to English-speaking Singaporean women aged 40&ndash;69 years without any history of breast cancer, enrolled via door-to-door recruitment with quota sampling by age and ethnicity. DCE attributes comprise: 1) sample type (buccal swab and dried blood spot), 2) person conducting pretest discussion (specialist doctor, non-specialist doctor, and nurse educator), 3) test location (private family clinic, public primary-care clinic, and hospital), and 4) out-of-pocket cost (S$50, S$175, and S300). Mixed logit model was used to estimate the effect of attribute levels on women’s preferences and mWTP. Interactions between significant attributes and respondent characteristics were investigated. Predicted uptake rates for various gene testing scenarios were studied. Results: A total of 300 women aged 52.6±7.6 years completed the survey (100 Chinese, Malay, and Indian women, respectively). Sample type (P=0.046), person conducting pretest discussion, and out-of-pocket cost (P<0.001) are significantly associated with going for SNP gene testing. Women with higher income and education levels are more willing to pay higher prices for the test. Preferences in terms of mWTP across ethnic groups appear similar, but Chinese women have greater preference heterogeneity for the attributes. Predicted uptake for a feasible scenario consisting of buccal swab, pretest discussion with nurse educator at the hospital costing S50 is 60.5%. Only 3.3% of women always opted out of the SNP gene test in real life. Reasons include high cost, poor awareness, and indifference toward test results. Conclusion: SNP gene testing may be tailored according to individual preferences to encourage uptake. Future research should focus on outcomes and cost-effectiveness of personalized breast cancer screening using SNP gene testing. Keywords: single-nucleotide polymorphisms, gene testing, personalized breast cancer screening, preci&shy;sion medicine, women&rsquo;s preferences, willingness-to-pay, predicted uptake, discrete choice experimen

How psychological distance of a study sample in discrete choice experiments affects preference measurement: a colorectal cancer screening case study

Jorien Veldwijk,1&ndash;3 Catharina GM Groothuis-Oudshoorn,4 Ulrik Kihlbom,2,5,6 Sophie Langenski&ouml;ld,2,5,6 Evelien Dekker,7 Frank GJ Kallenberg,7 G Ardine de Wit,3,8 Mattijs S Lambooij3 1Erasmus School of Health Policy and Management, Erasmus University, Rotterdam, the Netherlands; 2Centre for Research Ethics &amp; Bioethics, Uppsala University, Uppsala, Sweden; 3Centre for Nutrition, Prevention and Health Services, National Institute for Public Health and the Environment, Bilthoven, the Netherlands; 4Health Technology and Services Research, University of Twente, Enschede, the Netherlands; 5Department of Health Economics, Uppsala University, Uppsala, Sweden; 6Department of Learning, Informatics, Management and Ethics, Karolinska Institute, Stockholm, Sweden; 7Department of Gastroenterology and Hepatology, Academic Medical Center, Amsterdam, the Netherlands; 8Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, the Netherlands Purpose: The purpose of this study was to investigate to what extent the outcomes of a discrete choice experiment (DCE) differ based on respondents&rsquo; psychological distance to the decision at hand.Methods: A DCE questionnaire regarding individuals&rsquo; preferences for genetic screening for colorectal cancer (CRC) within the Dutch national CRC screening program was created. The DCE contained nine D-efficient designed choice tasks and was distributed among two populations that differ in their psychological distance to the decision at hand: 1) a representative sample of the Dutch general population aged 55&ndash;65 years, and 2) a sample of Dutch individuals who attended an information appointment regarding colonoscopies following the detection of blood in their stool sample in the CRC screening program. The DCE consisted of four attributes related to the decision whether to participate in genetic screening for CRC: 1) risk of being genetically predisposed, 2) risk of developing CRC, 3) frequency of follow-up colonoscopies, and 4) survival. Direct attribute ranking, dominant decision-making behavior, and relative importance scores (based on panel MIXL) were compared between the two populations. Attribute level estimates were compared with the Swait and Louviere test.Results: The proportion of respondents who both ranked survival as the most important attribute, and showed dominant decision-making behavior for this attribute, was significantly higher in the screened population compared to the general population. The relative importance scores of the attributes significantly differed between populations. Finally, the Swait and Louviere test also revealed significant differences in attribute level estimates in both the populations.Conclusion: The study outcomes differed between populations depending on their psychological distance to the decision. This study shows the importance of adequate sample selection; therefore, it is advocated to increase attention to study sample selection and reporting in DCE studies. Keywords: discrete choice experiment, preferences, stated preferences, sample, psychological distance, genetic screening&nbsp

Association between poor therapy adherence to inhaled corticosteroids and tiotropium and morbidity and mortality in patients with COPD

Kirsten Koehorst-ter Huurne,1 Catharina GM Groothuis-Oudshoorn,2 Paul DLPM vanderValk,1 Kris LL Movig,3 Job van der Palen,4,5 Marjolein Brusse-Keizer4 1Department of Pulmonary Medicine, Medisch Spectrum Twente, Enschede, the Netherlands; 2Department of Health Technology and Services Research, University of Twente, Enschede, the Netherlands; 3Department of Clinical Pharmacy, Medisch Spectrum Twente, Enschede, the Netherlands; 4Medical School Twente, Medisch Spectrum Twente, Enschede, the Netherlands; 5Department of Research Methodology, Measurement, and Data Analysis, University of Twente, Enschede, the Netherlands Aim: The aim of this study was to analyze the association between therapy adherence to inhaled corticosteroids (ICSs) and tiotropium on the one hand and morbidity and mortality in COPD on the other hand. Methods: Therapy adherence to ICSs and tiotropium over a 3-year period of, respectively, 635 and 505 patients was collected from pharmacy records. It was expressed as percentage and deemed optimal at &ge;75&ndash;&le;125%, suboptimal at &ge;50%&ndash;&lt;75%, and poor at &lt;50% (underuse) or &gt;125% (overuse). The association between adherence and time to first hospital admission for an acute exacerbation of chronic obstructive pulmonary disease (AECOPD), community acquired pneumonia (CAP), and mortality was analyzed, with optimal use as the reference category. Results: Suboptimal use and underuse of ICSs and tiotropium were associated with a substantial increase in mortality risk: hazard ratio (HR) of ICSs was 2.9 (95% CI 1.7&ndash;5.1) and 5.3 (95% CI 3.3&ndash;8.5) and HR of tiotropium was 3.9 (95% CI 2.1&ndash;7.5) and 6.4 (95% CI 3.8&ndash;10.8) for suboptimal use and underuse, respectively. Suboptimal use and overuse of tiotropium were also associated with an increased risk of CAP, HR 2.2 (95% CI 1.2&ndash;4.0) and HR 2.3 (95% CI 1.2&ndash;4.7), respectively. Nonadherence to tiotropium was also associated with an increased risk of severe AECOPD: suboptimal use HR 3.0 (95% CI 2.01&ndash;4.5), underuse HR 1.9 (95% CI 1.2&ndash;3.1), and overuse HR 1.84 (95% CI 1.1&ndash;3.1). Nonadherence to ICSs was not related to time to first AECOPD or first CAP. Conclusion: Poor adherence to ICSs and tiotropium was associated with a higher mortality risk. Furthermore, nonadherence to tiotropium was associated with a higher morbidity. The question remains whether improving adherence can reduce morbidity and mortality. Keywords: COPD, adherence, morbidity, mortality, pneumonia, exacerbation, hospitalizatio