Development of a standard set of PROs and generic PROMs for Dutch medical specialist care : Recommendations from the Outcome-Based Healthcare Program Working Group Generic PROMs.

PURPOSE: The added value of measuring patient-reported outcomes (PROs) for delivering patient-centered care and assessment of healthcare quality is increasingly evident. However, healthcare system wide data collection initiatives are hampered by the proliferation of patient-reported outcome measures (PROMs) and conflicting data collection standards. As part of a national initiative of the Dutch Ministry of Health, Welfare and Sport we developed a consensus-based standard set of generic PROs and PROMs to be implemented across Dutch medical specialist care. METHODS: A working group of mandated representatives of umbrella organizations involved in Dutch medical specialist care, together with PROM experts and patient organizations worked through a structured, consensus-driven co-creation process. This included literature reviews, online expert and working group meetings, and feedback from national patient- and umbrella organizations. The 'PROM-cycle' methodology was used to select feasible, valid, and reliable PROMs to obtain domain scores for each of the PROs included in the set. RESULTS: Eight PROs across different domains of health were ultimately endorsed: symptoms (pain & fatigue), functioning (physical, social/participation, mental [anxiety & depression]), and overarching (quality of life & perceived overall health). A limited number of generic PROMs was endorsed. PROMIS short forms were selected as the preferred instruments for all PROs. Several recommendations were formulated to facilitate healthcare system level adoption and implementation of the standard set. CONCLUSIONS: We developed a consensus-based standard set of Generic PROMs and a set of recommendations to facilitate healthcare system wide implementation across Dutch medical specialist care.

Cocreation with Dutch patients of decision-relevant information to support shared decision-making about adjuvant treatment in breast cancer care.

BACKGROUND: To support patients in shared decision-making about treatment options, patient decision aids (PtDAs) usually provide benefit/harm information and value clarification methods (VCMs). Recently, personalized risk information from prediction models is also being integrated into PtDAs. This study aimed to design decision-relevant information (i.e., personalized survival rates, harm information and VCMs) about adjuvant breast cancer treatment in cocreation with patients, in a way that suits their needs and is easily understandable. METHODS: Three cocreation sessions with breast cancer patients (N = 7-10; of whom N = 5 low health literate) were performed. Participants completed creative assignments and evaluated prototypes of benefit/harm information and VCMs. Prototypes were further explored through user testing with patients (N = 10) and healthcare providers (N = 10). The researchers interpreted the collected data, for example, creative and homework assignments, and participants' presentations, to identify key themes. User tests were transcribed and analysed using ATLAS.ti to assess the understanding of the prototypes. RESULTS: Important information needs were: (a) need for overview/structure of information directly after diagnosis and; (b) need for transparent benefit/harm information for all treatment options, including detailed harm information. Regarding VCMs, patients stressed the importance of a summary/conclusion. A bar graph seemed the most appropriate way of displaying personalized survival rates; the impact of most other formats was perceived as too distressful. The concept of 'personalization' was not understood by multiple patients. CONCLUSIONS: A PtDA about adjuvant breast cancer treatment should provide patients with an overview of the steps and treatment options, with layers for detailed information. Transparent information about the likelihood of benefits and harm should be provided. Given the current lack of information on the likelihood of side effects/late effects, efforts should be made to collect and share these data with patients. Further quantitative studies are needed to validate the results and to investigate how the concept of 'personalization' can be communicated. PATIENT OR PUBLIC CONTRIBUTION: Ten breast cancer patients participated in three cocreation sessions to develop decision-relevant information. Subsequent user testing included 10 patients. The Dutch Breast Cancer Association (BVN) was involved as an advisor in the general study design.

Case-studies of displacement effects in Dutch hospital care.

BACKGROUND: Under a constrained health care budget, cost-increasing technologies may displace funds from existing health services. However, it is unknown what services are displaced and how such displacement takes place in practice. The aim of our study was to investigate how the Dutch hospital sector has dealt with the introduction of cost-increasing health technologies, and to present evidence of the relative importance of three main options to deal with cost-increases in health care: increased spending, increased efficiency, or displacement of other services. METHODS: We conducted six case-studies and interviewed 84 professionals with various roles and responsibilities (practitioners, heads of clinical department, board of directors, insurers, and others) to investigate how they experienced decision making in response to the cost pressure of cost-increasing health technologies. Transcripts were analyzed thematically in Atlas.ti on the basis of an item list. RESULTS: Direct displacement of high-value care due to the introduction of new technologies was not observed; respondents primarily pointed to increased spending and efficiency measures to accommodate the introduction of the cost-increasing technologies. Respondents found it difficult to identify the opportunity costs; partly due to limited transparency in the internal allocation of funds within a hospital. Furthermore, respondents experienced the entry of new technologies and cost-containment as two parallel processes that are generally not causally linked: cost containment was experienced as a permanent issue to level costs and revenues, independent from entry of new technologies. Furthermore, the way of financing was found important in displacement in the Netherlands, especially as there is a separate budget for expensive drugs. This budget pressure was found to be reallocated horizontally across departments, whereas the budget pressure of other services is primarily reallocated vertically within departments or divisions. Respondents noted that hospitals have reacted to budget pressures primarily through a narrowing in the portfolio of their services, and a range of (other) efficiency measures. The board of directors is central in these processes, insurers are involved only to a limited extent. CONCLUSIONS: Our findings indicate that new technologies were generally accommodated by greater efficiency and increased spending, and that hospitals sought savings or efficiency measures in response to cumulative cost pressures rather than in response to single cost-increasing technologies.

Towards Integrated Health Technology Assessment for Improving Decision Making in Selected Countries.

OBJECTIVES: To assess the level of comprehensiveness of health technology assessment (HTA) practices around the globe and to formulate recommendations for enhancing legitimacy and fairness of related decision-making processes. METHODS: To identify best practices, we developed an evaluation framework consisting of 13 criteria on the basis of the INTEGRATE-HTA model (integrative perspective on assessing health technologies) and the Accountability for Reasonableness framework (deliberative appraisal process). We examined different HTA systems in middle-income countries (Argentina, Brazil, and Thailand) and high-income countries (Australia, Canada, England, France, Germany, Scotland, and South Korea). For this purpose, desk research and structured interviews with relevant key stakeholders (N = 32) in the selected countries were conducted. RESULTS: HTA systems in Canada, England, and Scotland appear relatively well aligned with our framework, followed by Australia, Germany, and France. Argentina and South Korea are at an early stage, whereas Brazil and Thailand are at an intermediate level. Both desk research and interviews revealed that scoping is often not part of the HTA process. In contrast, providing evidence reports for assessment is well established. Indirect and unintended outcomes are increasingly considered, but there is room for improvement. Monitoring and evaluation of the HTA process is not well established across countries. Finally, adopting transparent and robust processes, including stakeholder consultation, takes time. CONCLUSIONS: This study presents a framework for assessing the level of comprehensiveness of the HTA process in a country. On the basis of applying the framework, we formulate recommendations on how the HTA community can move toward a more integrated decision-making process using HTA.

Impact of Survey Administration Mode on the Results of a Health-Related Discrete Choice Experiment: Online and Paper Comparison.

BACKGROUND: Electronic data collection is increasingly being used for discrete choice experiments (DCEs). OBJECTIVES: To study whether paper or electronic administration results in measurement effects. METHODS: Respondents were drawn from the same sample frame (an Internet panel) and completed a nearly identical DCE survey either online or on paper during the same period. A DCE on preferences for basic health insurance served as a case study. We used panel mixed logit models for the analysis. RESULTS: In total, 898 respondents completed the survey: 533 respondents completed the survey online, whereas 365 respondents returned the paper survey. There were no significant differences with respect to sociodemographic characteristics between the respondents in both samples. The median response time was shorter for the online sample than for the paper sample, and a smaller proportion of respondents from the online sample were satisfied with the number of choice sets. Although some willingness- to-pay estimates were higher for the online sample, the elicited preferences for basic health insurance characteristics were similar between both modes of administration. CONCLUSIONS: We find no indication that online surveys yield inferior results compared with paper-based surveys, whereas the price per respondent is lower for online surveys. Researchers might want to include fewer choice sets per respondent when collecting DCE data online. Because our findings are based on a nonrandomized DCE that covers one health domain only, research in other domains is needed to support our findings.

Exploring how individuals complete the choice tasks in a discrete choice experiment: an interview study.

BACKGROUND: To be able to make valid inferences on stated preference data from a Discrete Choice Experiment (DCE) it is essential that researchers know if participants were actively involved, understood and interpreted the provided information correctly and whether they used complex decision strategies to make their choices and thereby acted in accordance with the continuity axiom. METHODS: During structured interviews, we explored how 70 participants evaluated and completed four discrete choice tasks aloud. Hereafter, additional questions were asked to further explore if participants understood the information that was provided to them and whether they used complex decision strategies (continuity axiom) when making their choices. Two existing DCE questionnaires on rotavirus vaccination and prostate cancer-screening served as case studies. RESULTS: A large proportion of the participants was not able to repeat the exact definition of the risk attributes as explained to them in the introduction of the questionnaire. The majority of the participants preferred more optimal over less optimal risk attribute levels. Most participants (66%) mentioned three or more attributes when motivating their decisions, thereby acting in accordance with the continuity axiom. However, 16 out of 70 participants continuously mentioned less than three attributes when motivating their decision. Lower educated and less literate participants tended to mention less than three attributes when motivating their decision and used trading off between attributes less often as a decision-making strategy. CONCLUSION: The majority of the participants seemed to have understood the provided information about the choice tasks, the attributes, and the levels. They used complex decision strategies (continuity axiom) and are therefore capable to adequately complete a DCE. However, based on the participants' age, educational level and health literacy additional, actions should be undertaken to ensure that participants understand the choice tasks and complete the DCE as presumed.

Public preferences for vaccination programmes during pandemics caused by pathogens transmitted through respiratory droplets - a discrete choice experiment in four European countries, 2013.

This study aims to quantify and compare preferences of citizens from different European countries for vaccination programme characteristics during pandemics, caused by pathogens which are transmitted through respiratory droplets. Internet panel members, nationally representative based on age, sex, educational level and region, of four European Union Member States (Netherlands, Poland, Spain, and Sweden, n = 2,068) completed an online discrete choice experiment. These countries, from different geographical areas of Europe, were chosen because of the availability of high-quality Internet panels and because of the cooperation between members of the project entitled Effective Communication in Outbreak Management: development of an evidence-based tool for Europe (ECOM). Data were analysed using panel latent class regression models. In the case of a severe pandemic scenario, vaccine effectiveness was the most important characteristic determining vaccination preference in all countries, followed by the body that advises on vaccination. In Sweden, the advice of family and/or friends and the advice of physicians strongly affected vaccine preferences, in contrast to Poland and Spain, where the advice of (international) health authorities was more decisive. Irrespective of pandemic scenario or vaccination programme characteristics, the predicted vaccination uptakes were lowest in Sweden, and highest in Poland. To increase vaccination uptake during future pandemics, the responsible authorities should align with other important stakeholders in the country and communicate in a coordinated manner.

Taking shared decision making for prostate cancer to the next level: Requirements for a Dutch treatment decision aid with personalized risks on side effects.

Background: Different curative treatment modalities need to be considered in case of localized prostate cancer, all comparable in terms of survival and recurrence though different in side effects. To better inform patients and support shared decision making, the development of a web-based patient decision aid including personalized risk information was proposed. This paper reports on requirements in terms of content of information, visualization of risk profiles, and use in practice. Methods: Based on a Dutch 10-step guide about the setup of a decision aid next to a practice guideline, an iterative and co-creative design process was followed. In collaboration with various groups of experts (health professionals, usability and linguistic experts, patients and the general public), research and development activities were continuously alternated. Results: Content requirements focused on presenting information only about conventional treatments and main side effects; based on risk group; and including clear explanations about personalized risks. Visual requirements involved presenting general and personalized risks separately; through bar charts or icon arrays; and along with numbers or words, and legends. Organizational requirements included integration into local clinical pathways; agreement about information input and output; and focus on patients' numeracy and graph literacy skills. Conclusions: The iterative and co-creative development process was challenging, though extremely valuable. The translation of requirements resulted in a decision aid about four conventional treatment options, including general or personalized risks for erection, urinary and intestinal problems that are communicated with icon arrays and numbers. Future implementation and validation studies need to inform about use and value in practice.

Development and testing of a tailored online fertility preservation decision aid for female cancer patients.

BACKGROUND: Decision making regarding future fertility can be very difficult for female cancer patients. To support patients in decision making, fertility preservation decision aids (DAs) are being developed. However, to make a well-informed decision, patients need personalized information tailored to their cancer type and treatment. Tailored cancer-specific DAs are not available yet. METHODS: Our DA was systematically developed by a multidisciplinary steering group (n = 21) in an iterative process of draft development, three rounds of alpha testing, and revisions. The drafts were based on current guidelines, literature, and patients' and professionals' needs. RESULTS: In total, 24 cancer-specific DAs were developed. In alpha testing, cancer survivors and professionals considered the DA very helpful in decision making, and scored an 8.5 (scale 1-10). In particular, the cancer-specific information and the tool for recognizing personal values were of great value. Revisions were made to increase readability, personalization, usability, and be more careful in giving any false hope. CONCLUSIONS: A fertility preservation DA containing cancer-specific information is important in the daily care of female cancer patients and should be broadly available. Our final Dutch version is highly appraised, valid, and usable in decision making. After evaluating its effectiveness with newly diagnosed patients, the DA can be translated and adjusted according to (inter)national guidelines.

Designing Unforced Choice Experiments to Inform Health Care Decision Making: Implications of Using Opt-Out, Neither, or Status Quo Alternatives in Discrete Choice Experiments.

Background. Discrete choice experiments (DCEs) are increasingly used in the health care context to inform on patient preferences for health care services. In order for such experiments to provide useful and policy-relevant information, it is vital that the design includes those options that the respondent faces in the real-life situation. Whether to include opt-out, neither, or status quo alternatives has, however, received little attention in the DCE literature. We aim to investigate whether the use of different unforced choice formats affects DCE results in different settings: 1) opt-out versus neither in a health care market where there is no status quo and 2) including status quo in addition to opt-out in a health care market with a status quo. Design. A DCE on Dutch citizens' preferences for personal health records served as our case, and 3189 respondents were allocated to the different unforced choice formats. We used mixed logit error component models to estimate preferences. Results. We found that the use of different unforced choice formats affects marginal utilities and welfare estimates and hence the conclusions that will be drawn from the DCE to inform health care decision making. Conclusions. To avoid biased estimates, we recommend that researchers are hesitant to use the neither option and consider including a status quo in addition to opt-out in settings where a status quo exists.

Personal health records in the Netherlands: potential user preferences quantified by a discrete choice experiment.

OBJECTIVE: To identify groups of potential users based on their preferences for characteristics of personal health records (PHRs) and to estimate potential PHR uptake. METHODS: We performed a discrete choice experiment, which consisted of 12 choice scenarios, each comprising 2 hypothetical PHR alternatives and an opt-out. The alternatives differed based on 5 characteristics. The survey was administered to Internet panel members of the Dutch Federation of Patients and Consumer Organizations. We used latent class models to analyze the data. RESULTS: A total of 1,443 potential PHR users completed the discrete choice experiment. We identified 3 latent classes: "refusers" (class probability 43%), "eager adopters" (37%), and "reluctant adopters" (20%). The predicted uptake for the reluctant adopters ranged from 4% in the case of a PHR with the worst attribute levels to 68% in the best case. Those with 1 or more chronic diseases were significantly more likely to belong to the eager adopter class. The data storage provider was the most decisive aspect for the eager and reluctant adopters, while cost was most decisive for the refusers. Across all classes, health care providers and independent organizations were the most preferred data storage providers. CONCLUSION: We identified 3 groups, of which 1 group (more than one-third of potential PHR users) indicated great interest in a PHR irrespective of PHR characteristics. Policymakers who aim to expand the use of PHRs will be most successful when health care providers and health facilities or independent organizations store PHR data while refraining from including market parties.

Future pandemics and vaccination: Public opinion and attitudes across three European countries.

BACKGROUND: Understanding public opinion and attitudes regarding vaccination is crucial for successful outbreak management and effective communication at the European level. METHODS: We explored national differences by conducting focus group discussions in The Netherlands, Poland and Sweden. Discussions were structured using concepts from behavioural models. RESULTS: Thematic analysis revealed that participants would base their vaccination decision on trade-offs between perceived benefits and barriers of the vaccine also taking into account the seriousness of the new outbreak. Except for those having chronic diseases, participants expected a low infection risk, resulting in a low willingness to get vaccinated. Information about the health status of cases was considered important since this might change perceived susceptibility. Participants displayed concerns about vaccine safety due to the limited available time to produce and test vaccines in the acute situation of a new pandemic. Swedish participants mentioned their tendency of doing the right thing and following the rules, as well as to get vaccinated because of solidarity with other citizens and social influences. This appeared much less prominent for the Dutch and Polish participants. However, Swedish participants indicated that their negative experiences during the Influenza A/H1N1 2009 pandemic decreases their acceptance of future vaccinations. Polish participants lacked trust in their national (public) health system and government, and were therefore sceptical about the availability and quality of vaccines in Poland. CONCLUSIONS: Although participants overall expressed similar considerations, important differences between countries stand out, such as previous vaccination experiences, the degree of adherence to social norms, and the degree of trust in health authorities.

What health plans do people prefer? The trade-off between premium and provider choice.

Within a healthcare system with managed competition, health insurers are expected to act as prudent buyers of care on behalf of their customers. To fulfil this role adequately, understanding consumer preferences for health plan characteristics is of vital importance. Little is known, however, about these preferences and how they vary across consumers. Using a discrete choice experiment (DCE) we quantified trade-offs between basic health plan characteristics and analysed whether there are differences in preferences according to age, health status and income. We selected four health plan characteristics to be included in the DCE: (i) the level of provider choice and associated level of reimbursement, (ii) the primary focus of provider contracting (price, quality, social responsibility), (iii) the level of service benefits, and (iv) the monthly premium. This selection was based on a literature study, expert interviews and focus group discussions. The DCE consisted of 17 choice sets, each comprising two hypothetical health plan alternatives. A representative sample (n = 533) of the Dutch adult population, based on age, gender and educational level, completed the online questionnaire during the annual open enrolment period for 2015. The final model with four latent classes showed that being able to choose a care provider freely was by far the most decisive characteristic for respondents aged over 45, those with chronic conditions, and those with a gross income over €3000/month. Monthly premium was the most important choice determinant for young, healthy, and lower income respondents. We conclude that it would be very unlikely for half of the sample to opt for health plans with restricted provider choice. However, a premium discount up to €15/month by restricted health plans might motivate especially younger, healthier, and less wealthy consumers to choose these plans.

Discrete choice experiments in health economics: a review of the literature.

BACKGROUND: Discrete choice experiments (DCEs) are increasingly used in health economics to address a wide range of health policy-related concerns. OBJECTIVE: Broadly adopting the methodology of an earlier systematic review of health-related DCEs, which covered the period 2001-2008, we report whether earlier trends continued during 2009-2012. METHODS: This paper systematically reviews health-related DCEs published between 2009 and 2012, using the same database as the earlier published review (PubMed) to obtain citations, and the same range of search terms. RESULTS: A total of 179 health-related DCEs for 2009-2012 met the inclusion criteria for the review. We found a continuing trend towards conducting DCEs across a broader range of countries. However, the trend towards including fewer attributes was reversed, whilst the trend towards interview-based DCEs reversed because of increased computer administration. The trend towards using more flexible econometric models, including mixed logit and latent class, has also continued. Reporting of monetary values has fallen compared with earlier periods, but the proportion of studies estimating trade-offs between health outcomes and experience factors, or valuing outcomes in terms of utility scores, has increased, although use of odds ratios and probabilities has declined. The reassuring trend towards the use of more flexible and appropriate DCE designs and econometric methods has been reinforced by the increased use of qualitative methods to inform DCE processes and results. However, qualitative research methods are being used less often to inform attribute selection, which may make DCEs more susceptible to omitted variable bias if the decision framework is not known prior to the research project. CONCLUSIONS: The use of DCEs in healthcare continues to grow dramatically, as does the scope of applications across an expanding range of countries. There is increasing evidence that more sophisticated approaches to DCE design and analytical techniques are improving the quality of final outputs. That said, recent evidence that the use of qualitative methods to inform attribute selection has declined is of concern.

Acceptance of vaccinations in pandemic outbreaks: a discrete choice experiment.

BACKGROUND: Preventive measures are essential to limit the spread of new viruses; their uptake is key to their success. However, the vaccination uptake in pandemic outbreaks is often low. We aim to elicit how disease and vaccination characteristics determine preferences of the general public for new pandemic vaccinations. METHODS: In an internet-based discrete choice experiment (DCE) a representative sample of 536 participants (49% participation rate) from the Dutch population was asked for their preference for vaccination programs in hypothetical communicable disease outbreaks. We used scenarios based on two disease characteristics (susceptibility to and severity of the disease) and five vaccination program characteristics (effectiveness, safety, advice regarding vaccination, media attention, and out-of-pocket costs). The DCE design was based on a literature review, expert interviews and focus group discussions. A panel latent class logit model was used to estimate which trade-offs individuals were willing to make. RESULTS: All above mentioned characteristics proved to influence respondents' preferences for vaccination. Preference heterogeneity was substantial. Females who stated that they were never in favor of vaccination made different trade-offs than males who stated that they were (possibly) willing to get vaccinated. As expected, respondents preferred and were willing to pay more for more effective vaccines, especially if the outbreak was more serious (€6-€39 for a 10% more effective vaccine). Changes in effectiveness, out-of-pocket costs and in the body that advises the vaccine all substantially influenced the predicted uptake. CONCLUSIONS: We conclude that various disease and vaccination program characteristics influence respondents' preferences for pandemic vaccination programs. Agencies responsible for preventive measures during pandemics can use the knowledge that out-of-pocket costs and the way advice is given affect vaccination uptake to improve their plans for future pandemic outbreaks. The preference heterogeneity shows that information regarding vaccination needs to be targeted differently depending on gender and willingness to get vaccinated.