A scattered landscape: assessment of the evidence base for 71 patient decision aids developed in a hospital setting.

BACKGROUND: Recent publications reveal shortcomings in evidence review and summarization methods for patient decision aids. In the large-scale "Share to Care (S2C)" Shared Decision Making (SDM) project at the University Hospital Kiel, Germany, one of 4 SDM interventions was to develop up to 80 decision aids for patients. Best available evidence on the treatments' impact on patient-relevant outcomes was systematically appraised to feed this information into the decision aids. Aims of this paper were to (1) describe how PtDAs are developed and how S2C evidence reviews for each PtDA are conducted, (2) appraise the quality of the best available evidence identified and (3) identify challenges associated with identified evidence. METHODS: The quality of the identified evidence was assessed based on GRADE quality criteria and categorized into high-, moderate-, low-, very low-quality evidence. Evidence appraisal was conducted across all outcomes assessed in an evidence review and for specific groups of outcomes, namely mortality, morbidity, quality of life, and treatment harms. Challenges in evidence interpretation and summarization resulting from the characteristics of decision aids and the type and quality of evidence are identified and discussed. RESULTS: Evidence reviews assessed on average 25 systematic reviews/guidelines/studies and took about 3 months to be completed. Despite rigorous review processes, nearly 70% of outcome-specific information derived for decision aids was based on low-quality and mostly on non-directly comparative evidence. Evidence on quality of life and harms was often not provided or not in sufficient form/detail. Challenges in evidence interpretation for use in decision aids resulted from, e.g., a lack of directly comparative evidence or the existence of very heterogeneous evidence for the diverse treatments being compared. CONCLUSIONS: Evidence reviews in this project were carefully conducted and summarized. However, the evidence identified for our decision aids was indeed a "scattered landscape" and often poor quality. Facing a high prevalence of low-quality, non-directly comparative evidence for treatment alternatives doesn't mean it is not necessary to choose an evidence-based approach to inform patients. While there is an urgent need for high quality comparative trials, best available evidence nevertheless has to be appraised and transparently communicated to patients.

Cost-Effectiveness of Targeted Genetic Testing for Breast and Ovarian Cancer: A Systematic Review.

BACKGROUND: Targeted genetic testing is a tool to identify women at increased risk of gynaecological cancer. OBJECTIVE: This systematic review evaluates the results and quality of cost-effectiveness modeling studies that assessed targeted genetic-based screen-and-treat strategies to prevent breast and ovarian cancer. METHODS: Using MEDLINE and databases of the Centre for Reviews and Dissemination, we searched for health economic modeling evaluations of targeted genetic-based screen-and-treat strategies to prevent inheritable breast and ovarian cancer (until August 2020). The incremental cost-effectiveness ratios (ICERs) were compared. Methodological variations were addressed by evaluating the model conceptualizations, the modeling techniques, parameter estimation and uncertainty, and transparency and validation of the models. Additionally, the reporting quality of each study was assessed. RESULTS: Eighteen studies met our inclusion criteria. From a payer perspective, the ICERs of (1) BRCA screening for high-risk women without cancer ranged from dominating the no test strategy to an ICER of $21 700/quality-adjusted life years (QALY). In studies that evaluated (2) BRCA cascade screening (ie, screening of women with cancer plus their unaffected relatives) compared with no test, the ICERs were between $6500/QALY and $50 200/QALY. Compared with BRCA alone, (3) multigene testing in women without cancer had an ICER of $51 800/QALY (one study), while for (4) multigene-cascade screening the ICERs were $15 600/QALY, $56.500/QALY, and $69 600/QALY for women in the United Kingdom, Norway, and the United States, respectively (2 studies). More recently published studies showed a higher methodological and reporting quality. CONCLUSIONS: Targeted BRCA or multiple gene screening is likely to be cost-effective. Methodological variations could be decreased by the development of a reference model, which may serve as a tool for validation of present and future cost-effectiveness models.

Comparing Analytic Hierarchy Process and Discrete-Choice Experiment to Elicit Patient Preferences for Treatment Characteristics in Age-Related Macular Degeneration.

BACKGROUND: In this study, we conducted an analytic hierarchy process (AHP) and a discrete choice experiment (DCE) to elicit the preferences of patients with age-related macular degeneration using identical attributes and levels. OBJECTIVES: To compare preference-based weights for age-related macular degeneration treatment attributes and levels generated by two elicitation methods. The properties of both methods were assessed, including ease of instrument use. METHODS: A DCE and an AHP experiment were designed on the basis of five attributes. Preference-based weights were generated using the matrix multiplication method for attributes and levels in AHP and a mixed multinomial logit model for levels in the DCE. Attribute importance was further compared using coefficient (DCE) and weight (AHP) level ranges. The questionnaire difficulty was rated on a qualitative scale. Patients were asked to think aloud while providing their judgments. RESULTS: AHP and DCE generated similar results regarding levels, stressing a preference for visual improvement, frequent monitoring, on-demand and less frequent injection schemes, approved drugs, and mild side effects. Attribute weights derived on the basis of level ranges led to a ranking that was opposite to the AHP directly calculated attribute weights. For example, visual function ranked first in the AHP and last on the basis of level ranges. CONCLUSIONS: The results across the methods were similar, with one exception: the directly measured AHP attribute weights were different from the level-based interpretation of attribute importance in both DCE and AHP. The dependence/independence of attribute importance on level ranges in DCE and AHP, respectively, should be taken into account when choosing a method to support decision making.

Communicating the parameter uncertainty in the IQWiG efficiency frontier to decision-makers.

The Institute for Quality and Efficiency in Health Care (IQWiG) developed-in a consultation process with an international expert panel-the efficiency frontier (EF) approach to satisfy a range of legal requirements for economic evaluation in Germany's statutory health insurance system. The EF approach is distinctly different from other health economic approaches. Here, we evaluate established tools for assessing and communicating parameter uncertainty in terms of their applicability to the EF approach. Among these are tools that perform the following: (i) graphically display overall uncertainty within the IQWiG EF (scatter plots, confidence bands, and contour plots) and (ii) communicate the uncertainty around the reimbursable price. We found that, within the EF approach, most established plots were not always easy to interpret. Hence, we propose the use of price reimbursement acceptability curves-a modification of the well-known cost-effectiveness acceptability curves. Furthermore, it emerges that the net monetary benefit allows an intuitive interpretation of parameter uncertainty within the EF approach. This research closes a gap for handling uncertainty in the economic evaluation approach of the IQWiG methods when using the EF. However, the precise consequences of uncertainty when determining prices are yet to be defined.

Patient-reported effects of hospital-wide implementation of shared decision-making at a university medical centre in Germany: a pre-post trial.

OBJECTIVES: To evaluate the feasibility and effectiveness of the SHARE TO CARE (S2C) programme, a complex intervention designed for hospital-wide implementation of shared decision-making (SDM). DESIGN: Pre-post study. SETTING: University Hospital Schleswig-Holstein (UKSH), Kiel Campus. PARTICIPANTS: Healthcare professionals as well as inpatients and outpatients from 22 departments of the Kiel Campus of UKSH. INTERVENTIONS: The S2C programme is a comprehensive implementation strategy including four core modules: (1) physician training, (2) SDM support training for and support by nurses as decision coaches, (3) patient activation and (4) evidence-based patient decision aid development and integration into patient pathways. After full implementation, departments received the S2C certificate. MAIN OUTCOME MEASURES: In this paper, we report on the feasibility and effectiveness outcomes of the implementation. Feasibility was judged by the degree of implementation of the four modules of the programme. Outcome measures for effectiveness are patient-reported experience measures (PREMs). The primary outcome measure for effectiveness is the Patient Decision Making subscale of the Perceived Involvement in Care Scale (PICSPDM). Pre-post comparisons were done using t-tests. RESULTS: The implementation of the four components of the S2C programme was able to be completed in 18 of the 22 included departments within the time frame of the study. After completion of implementation, PICSPDM showed a statistically significant difference (p<0.01) between the means compared with baseline. This difference corresponds to a small to medium yet clinically meaningful positive effect (Hedges' g=0.2). Consistent with this, the secondary PREMs (Preparation for Decision Making and collaboRATE) also showed statistically significant, clinically meaningful positive effects. CONCLUSIONS: The hospital-wide implementation of SDM with the S2C-programme proved to be feasible and effective within the time frame of the project. The German Federal Joint Committee has recommended to make the Kiel model of SDM a national standard of care.

Integrating patients' views into health technology assessment: Analytic hierarchy process (AHP) as a method to elicit patient preferences.

BACKGROUND: Patient involvement is widely acknowledged to be a valuable component in health technology assessment (HTA) and healthcare decision making. However, quantitative approaches to ascertain patients' preferences for treatment endpoints are not yet established. The objective of this study is to introduce the analytic hierarchy process (AHP) as a preference elicitation method in HTA. Based on a systematic literature review on the use of AHP in health care in 2009, the German Institute for Quality and Efficiency in Health Care (IQWiG) initiated an AHP study related to its HTA work in 2010. METHODS: The AHP study included two AHP workshops, one with twelve patients and one with seven healthcare professionals. In these workshops, both patients and professionals rated their preferences with respect to the importance of different endpoints of antidepressant treatment by a pairwise comparison of individual endpoints. These comparisons were performed and evaluated by the AHP method and relative weights were generated for each endpoint. RESULTS: The AHP study indicates that AHP is a well-structured technique whose cognitive demands were well handled by patients and professionals. The two groups rated some of the included endpoints of antidepressant treatment differently. For both groups, however, the same six of the eleven endpoints analyzed accounted for more than 80 percent of the total weight. CONCLUSIONS: AHP can be used in HTA to give a quantitative dimension to patients' preferences for treatment endpoints. Preference elicitation could provide important information at various stages of HTA and challenge opinions on the importance of endpoints.

Transcatheter aortic valve implantation versus surgical aortic valve replacement in patients with severe aortic stenosis: a systematic review and meta-analysis.

OBJECTIVES: Patients undergoing surgery for severe aortic stenosis (SAS) can be treated with either transcatheter aortic valve implantation (TAVI) or surgical aortic valve replacement (SAVR). The choice of procedure depends on several factors, including the clinical judgement of the heart team and patient preferences, which are captured by actively informing and involving patients in a process of shared decision making (SDM). We synthesised the most up-to-date and accessible evidence on the benefits and risks that may be associated with TAVI versus SAVR to support SDM in this highly personalised decision-making process. DESIGN: Systematic review and meta-analysis. DATA SOURCES: MEDLINE (Ovid), Embase (Ovid) and the Cochrane Central Register of Controlled Trials (CENTRAL; Wiley) were searched from January 2000 to August 2020 with no language restrictions. Reference lists of included studies were searched to identify additional studies. ELIGIBILITY CRITERIA: Randomised controlled trials (RCTs) that compared TAVI versus SAVR in patients with SAS and reported on all-cause or cardiovascular mortality, length of stay in intensive care unit or hospital, valve durability, rehospitalisation/reintervention, stroke (any stroke or major/disabling stroke), myocardial infarction, major vascular complications, major bleeding, permanent pacemaker (PPM) implantation, new-onset or worsening atrial fibrillation (NOW-AF), endocarditis, acute kidney injury (AKI), recovery time or pain were included. DATA EXTRACTION AND SYNTHESIS: Two independent reviewers were involved in data extraction and risk of bias (ROB) assessment using the Cochrane tool (one reviewer extracted/assessed the data, and the second reviewer checked it). Dichotomous data were pooled using the Mantel-Haenszel method with random-effects to generate a risk ratio (RR) with 95% CI. Continuous data were pooled using the inverse-variance method with random-effects and expressed as a mean difference (MD) with 95% CI. Heterogeneity was assessed using the I2 statistic. RESULTS: 8969 records were retrieved and nine RCTs (61 records) were ultimately included (n=8818 participants). Two RCTs recruited high-risk patients, two RCTs recruited intermediate-risk patients, two RCTs recruited low-risk patients, one RCT recruited high-risk (≥70 years) or any-risk (≥80 years) patients; and two RCTs recruited all-risk or 'operable' patients. While there was no overall change in the risk of dying from any cause (30 day: RR 0.89, 95% CI 0.65 to 1.22; ≤1 year: RR 0.90, 95% CI 0.79 to 1.03; 5 years: RR 1.09, 95% CI 0.98 to 1.22), cardiovascular mortality (30 day: RR 1.03, 95% CI 0.77 to 1.39; ≤1 year: RR 0.90, 95% CI 0.76 to 1.06; 2 years: RR 0.96, 95% CI 0.83 to 1.12), or any type of stroke (30 day: RR 0.83, 95% CI 0.61 to 1.14;≤1 year: RR 0.94, 95% CI 0.72 to 1.23; 5 years: RR 1.07, 95% CI 0.88 to 1.30), the risk of several clinical outcomes was significantly decreased (major bleeding, AKI, NOW-AF) or significantly increased (major vascular complications, PPM implantation) for TAVI vs SAVR. TAVI was associated with a significantly shorter hospital stay vs SAVR (MD -3.08 days, 95% CI -4.86 to -1.29; 4 RCTs, n=2758 participants). Subgroup analysis generally favoured TAVI patients receiving implantation via the transfemoral (TF) route (vs non-TF); receiving a balloon-expandable (vs self-expanding) valve; and those at low-intermediate risk (vs high risk). All RCTs were rated at high ROB, predominantly due to lack of blinding and selective reporting. CONCLUSIONS: No overall change in the risk of death from any cause or cardiovascular mortality was identified but 95% CIs were often wide, indicating uncertainty. TAVI may reduce the risk of certain side effects while SAVR may reduce the risk of others. Most long-term (5-year) results are limited to older patients at high surgical risk (ie, early trials), therefore more data are required for low risk populations. Ultimately, neither surgical technique was considered dominant, and these results suggest that every patient with SAS should be individually engaged in SDM to make evidence-based, personalised decisions around their care based on the various benefits and risks associated with each treatment. PROSPERO REGISTRATION NUMBER: CRD42019138171.

Making shared decision-making (SDM) a reality: protocol of a large-scale long-term SDM implementation programme at a Northern German University Hospital.

INTRODUCTION: Shared decision-making (SDM) is not yet widely used when making decisions in German hospitals. Making SDM a reality is a complex task. It involves training healthcare professionals in SDM communication and enabling patients to actively participate in communication, in addition to providing sound, easy to understand information on treatment alternatives in the form of evidence-based patient decision aids (EbPDAs). This project funded by the German Innovation Fund aims at designing, implementing and evaluating a multicomponent, large-scale and integrative SDM programme-called SHARE TO CARE (S2C)-at all clinical departments of a University Hospital Campus in Northern Germany within a 4-year time period. METHODS AND ANALYSIS: S2C tackles the aforementioned components of SDM: (1) training physicians in SDM communication, (2) activating and empowering patients, (3) developing EbPDAs in the most common/relevant diseases and (4) training other healthcare professionals in SDM coaching. S2C is designed together with patients and providers. The physicians' training programme entails an online and an in situ training module. The decision coach training is based on a similar but less comprehensive approach. The development of online EbPDAs follows the International Patient Decision Aid Standards and includes written, graphical and video-based information. Validated outcomes of SDM implementation are measured in a preintervention and postintervention evaluation design. Process evaluation accompanies programme implementation. Health economic impact of the intervention is investigated using a propensity-score-matched approach based on potentially preference-sensitive hospital decisions. ETHICS AND DISSEMINATION: Ethics committee review approval has been obtained from Medical Ethics Committee of the Medical Faculty of the Christian-Albrechts-University Kiel. Project information and results will be disseminated at conferences, on project-hosted websites at University Hospital Medical Center Schleswig Holstein and by S2C as well as in peer-reviewed and professional journals.

Economic modeling of risk-adapted screen-and-treat strategies in women at high risk for breast or ovarian cancer.

BACKGROUND: The 'German Consortium for Hereditary Breast and Ovarian Cancer' (GC-HBOC) offers women with a family history of breast and ovarian cancer genetic counseling. The aim of this modeling study was to evaluate the cost-effectiveness of genetic testing for BRCA 1/2 in women with a high familial risk followed by different preventive interventions (intensified surveillance, risk-reducing bilateral mastectomy, risk-reducing bilateral salpingo-oophorectomy, or both mastectomy and salpingo-oophorectomy) compared to no genetic test. METHODS: A Markov model with a lifelong time horizon was developed for a cohort of 35-year-old women with a BRCA 1/2 mutation probability of ≥ 10%. The perspective of the German statutory health insurance (SHI) was adopted. The model included the health states 'well' (women with increased risk), 'breast cancer without metastases', 'breast cancer with metastases', 'ovarian cancer', 'death', and two post (non-metastatic) breast or ovarian cancer states. Outcomes were costs, quality of life years gained (QALYs) and life years gained (LYG). Important data used for the model were obtained from 4380 women enrolled in the GC-HBOC. RESULTS: Compared with the no test strategy, genetic testing with subsequent surgical and non-surgical treatment options provided to women with deleterious BRCA 1 or 2 mutations resulted in additional costs of €7256 and additional QALYs of 0,43 (incremental cost-effectiveness ratio of €17,027 per QALY; cost per LYG: €22,318). The results were robust in deterministic and probabilistic sensitivity analyses. CONCLUSION: The provision of genetic testing to high-risk women with a BRCA1 and two mutation probability of ≥ 10% based on the individual family cancer history appears to be a cost-effective option for the SHI.

Assessing the Importance of Treatment Goals in Patients with Psoriasis: Analytic Hierarchy Process vs. Likert Scales.

BACKGROUND: To define treatment benefit, the Patient Benefit Index contains a weighting of patient-relevant treatment goals using the Patient Needs Questionnaire, which includes a 5-point Likert scale ranging from 0 ("not important at all") to 4 ("very important"). These treatment goals have been assigned to five health dimensions. The importance of each dimension can be derived by averaging the importance ratings on the Likert scales of associated treatment goals. OBJECTIVE: As the use of a Likert scale does not allow for a relative assessment of importance, the objective of this study was to estimate relative importance weights for health dimensions and associated treatment goals in patients with psoriasis by using the analytic hierarchy process and to compare these weights with the weights resulting from the Patient Needs Questionnaire. Furthermore, patients' judgments on the difficulty of the methods were investigated. METHODS: Dimensions of the Patient Benefit Index and their treatment goals were mapped into a hierarchy of criteria and sub-criteria to develop the analytic hierarchy process questionnaire. Adult patients with psoriasis starting a new anti-psoriatic therapy in the outpatient clinic of the Institute for Health Services Research in Dermatology and Nursing at the University Medical Center Hamburg (Germany) were recruited and completed both methods (analytic hierarchy process, Patient Needs Questionnaire). Ratings of treatment goals on the Likert scales (Patient Needs Questionnaire) were summarized within each dimension to assess the importance of the respective health dimension/criterion. Following the analytic hierarchy process approach, consistency in judgments was assessed using a standardized measurement (consistency ratio). RESULTS: At the analytic hierarchy process level of criteria, 78 of 140 patients achieved the accepted consistency. Using the analytic hierarchy process, the dimension "improvement of physical functioning" was most important, followed by "improvement of social functioning". Concerning the Patient Needs Questionnaire results, these dimensions were ranked in second and fifth position, whereas "strengthening of confidence in the therapy and in a possible healing" was ranked most important, which was least important in the analytic hierarchy process ranking. In both methods, "improvement of psychological well-being" and "reduction of impairments due to therapy" were equally ranked in positions three and four. In contrast to this, on the level of sub-criteria, predominantly a similar ranking of treatment goals could be observed between the analytic hierarchy process and the Patient Needs Questionnaire. From the patients' point of view, the Likert scales (Patient Needs Questionnaire) were easier to complete than the analytic hierarchy process pairwise comparisons. CONCLUSIONS: Patients with psoriasis assign different importance to health dimensions and associated treatment goals. In choosing a method to assess the importance of health dimensions and/or treatment goals, it needs to be considered that resulting importance weights may differ in dependence on the used method. However, in this study, observed discrepancies in importance weights of the health dimensions were most likely caused by the different methodological approaches focusing on treatment goals to assess the importance of health dimensions on the one hand (Patient Needs Questionnaire) or directly assessing health dimensions on the other hand (analytic hierarchy process).

Measuring the importance of health domains in psoriasis - discrete choice experiment versus rating scales.

BACKGROUND: Psoriasis affects different aspects of health-related quality of life (eg, physical, psychological, and social impairments); these health domains can be of different importance for patients. The importance of domains can be measured with the Patient Benefit Index (PBI). This questionnaire weights the achievement of treatment goals by Likert scales (0, "not important at all" to 4, "very important") using the Patient Needs Questionnaire (PNQ). Treatment goals assessed with the PBI have been assigned to five health domains; the importance of each domain can be calculated as the average importance of the respective treatment goals. In this study, the PBI approach of deriving importance weights is contrasted to a discrete choice experiment (DCE), in order to determine the importance of health domains in psoriasis, and to find if the resulting weights will differ when derived from these two methods. METHODS: Adult patients with psoriasis completed both questionnaires (PNQ, DCE). The PBI domains were used as attributes in the DCE with the levels "did not help at all", "helped moderately", and "helped a lot". RESULTS: Using DCE, "improving physical functioning" was the most important health domain, followed by "improving psychological well-being". Using PNQ, these domains were ranked in position two and three following "strengthening confidence in the therapy and in a possible healing". The latter was least important using DCE. The only agreement of ranking was shown in "reducing impairments due to therapy" (position four). "Improving social functioning" was ranked in position three (DCE) and five (PNQ). CONCLUSION: Health domains have different importance to patients with psoriasis. Using PNQ or DCE to determine the importance of domains results in markedly different rankings; both approaches can thus not be considered equivalent. However, in this study, importance was assessed at the domain level in DCE and at the single item level in PNQ, which may have added to the differences.

How to weight patient-relevant treatment goals for assessing treatment benefit in psoriasis: preference elicitation methods vs. rating scales.

In psoriasis, several patient-relevant treatment goals must be met to be able to consider a treatment beneficial. To assess treatment benefit, the validated questionnaire Patient Benefit Index (PBI) can be used. Its global score summarizes the degree of patient-relevant treatment goals achieved after treatment, weighted by their individual importance on rating scales. These treatment goals have empirically been assigned to five dimensions. While the weighting procedure of the PBI provides information about the importance patients attach to treatment goals on a rating scale from 0 to 4, methods of preference elicitation provide information on how patients would trade off certain treatment goals against each other. However, since the treatment goals defined in the PBI often overlap conceptually, the dimensions of the PBI might be more suitable for exploration in preference elicitation methods. We used an analytic hierarchy process (AHP) and a discrete choice experiment (DCE) to generate preference-based importance weights for the PBI dimensions, and compared these weights to those derived from the rating scales. We were further interested in the effect of importance weights on the calculation of the PBI score. A total of 120 patients with psoriasis completed a questionnaire at baseline, including AHP, DCE and the rating scales, and at follow-up, regarding the attainment of treatment goals, to calculate the PBI score. In contrast to the results derived from the average rating scores, use of AHP and DCE resulted in both similar importance weights and rankings of dimensions. Presumably, patients rated treatment goals differently than the respective dimension they belong to. However, the differently calculated importance weights led to similar values of the PBI score. Our findings nevertheless provide clear evidence that, regardless of the method used, the importance of treatment goals differs between psoriasis patients, and this should be reflected in treatment decisions.

Cost-effectiveness of different strategies to prevent breast and ovarian cancer in German women with a BRCA 1 or 2 mutation.

BACKGROUND: Women with a BRCA1 or BRCA2 mutation are at increased risk of developing breast and/or ovarian cancer. This economic modeling study evaluated different preventive interventions for 30-year-old women with a confirmed BRCA (1 or 2) mutation. METHODS: A Markov model was developed to estimate the costs and benefits [i.e., quality-adjusted life years (QALYs), and life years gained (LYG)] associated with prophylactic bilateral mastectomy (BM), prophylactic bilateral salpingo-oophorectomy (BSO), BM plus BSO, BM plus BSO at age 40, and intensified surveillance. Relevant input data was obtained from a large German database including 5902 women with BRCA 1 or 2, and from the literature. The analysis was performed from the German Statutory Health Insurance (SHI) perspective. In order to assess the robustness of the results, deterministic and probabilistic sensitivity analyses were performed. RESULTS: With costs of €29,434 and a gain in QALYs of 17.7 (LYG 19.9), BM plus BSO at age 30 was less expensive and more effective than the other strategies, followed by BM plus BSO at age 40. Women who were offered the surveillance strategy had the highest costs at the lowest gain in QALYs/LYS. In the probabilistic sensitivity analysis, the probability of cost-saving was 57% for BM plus BSO. At a WTP of 10,000 € per QALY, the probability of the intervention being cost-effective was 80%. CONCLUSIONS: From the SHI perspective, undergoing BM plus immediate BSO should be recommended to BRCA 1 or 2 mutation carriers due to its favorable comparative cost-effectiveness.

Estimation of Input Costs for a Markov Model in a German Health Economic Evaluation of Newer Antidepressants.

Background: Estimating input costs for Markov models in health economic evaluations requires health state-specific costing. This is a challenge in mental illnesses such as depression, as interventions are not clearly related to health states. We present a hybrid approach to health state-specific cost estimation for a German health economic evaluation of antidepressants. Methods: Costs were determined from the perspective of the community of persons insured by statutory health insurance ("SHI insuree perspective") and included costs for outpatient care, inpatient care, drugs, and psychotherapy. In an additional step, costs for rehabilitation and productivity losses were calculated from the societal perspective. We collected resource use data in a stepwise hierarchical approach using SHI claims data, where available, followed by data from clinical guidelines and expert surveys. Bottom-up and top-down costing approaches were combined. Results: Depending on the drug strategy and health state, the average input costs varied per patient per 8-week Markov cycle. The highest costs occurred for agomelatine in the health state first-line treatment (FT) ("FT relapse") with €506 from the SHI insuree perspective and €724 from the societal perspective. From both perspectives, the lowest costs (excluding placebo) were €55 for selective serotonin reuptake inhibitors in the health state "FT remission." Conclusion: To estimate costs in health economic evaluations of treatments for depression, it can be necessary to link different data sources and costing approaches systematically to meet the requirements of the decision-analytic model. As this can increase complexity, the corresponding calculations should be presented transparently. The approach presented could provide useful input for future models.

Patients' preferences in periodontal disease treatment elicited alongside an IQWiG benefit assessment: a feasibility study.

BACKGROUND AND PURPOSE: The German Institute for Quality and Efficiency in Health Care (IQWiG) previously tested two preference elicitation methods in pilot projects and regarded them as generally feasible for prioritizing outcome-specific results of benefit assessment. The present study aimed to investigate the feasibility of completing a discrete choice experiment (DCE) within 3 months and to determine the relative importance of attributes of periodontal disease and its treatment. PATIENTS AND METHODS: This preference elicitation was conducted alongside the IQWiG benefit assessment of systematic treatments of periodontal diseases. Attributes were defined based on the benefit assessment, literature review, and patients' and periodontologists' interviews. The DCE survey was completed by patients with a history of periodontal disease. Preferences were elicited for the attributes "tooth loss within next 10 years", "own costs for treatment, follow-up visits, re-treatment", "complaints and symptoms", and "frequency of follow-up visits". Patients completed a self-administered questionnaire including 12 choice tasks. Data were analyzed using a random parameters logit model. The relative attribute importance was calculated based on level ranges. RESULTS: Within 3 months, survey development, data collection among 267 patients, data analysis, and provision of a study report could be completed. The analysis showed that tooth loss (score 0.73) was the most important attribute in patients' decisions, followed by complaints and symptoms (0.22), frequency of follow-up visits (0.02), and costs (0.03) (relative importance scores summing up to 1). CONCLUSION: A preference analysis performing a DCE can be generally feasible within 3 months; however, a good research infrastructure and access to patients is required. Outcomes used in benefit assessments might need to be adapted to be used in preference analyses.

Patients' Preferences for Outcome, Process and Cost Attributes in Cancer Treatment: A Systematic Review of Discrete Choice Experiments.

INTRODUCTION: As several studies have been conducted to elicit patients' preferences for cancer treatment, it is important to provide an overview and synthesis of these studies. This study aimed to systematically review discrete choice experiments (DCEs) about patients' preferences for cancer treatment and assessed the relative importance of outcome, process and cost attributes. METHODS: A systematic literature review was conducted using PubMed and EMBASE to identify all DCEs investigating patients' preferences for cancer treatment between January 2010 and April 2016. Data were extracted using a predefined extraction sheet, and a reporting quality assessment was applied to all studies. Attributes were classified into outcome, process and cost attributes, and their relative importance was assessed. RESULTS: A total of 28 DCEs were identified. More than half of the studies (56%) received an aggregate score lower than 4 on the PREFS (Purpose, Respondents, Explanation, Findings, Significance) 5-point scale. Most attributes were related to outcome (70%), followed by process (25%) and cost (5%). Outcome attributes were most often significant (81%), followed by process (73%) and cost (67%). The relative importance of outcome attributes was ranked highest in 82% of the cases where it was included, followed by cost (43%) and process (12%). CONCLUSION: This systematic review suggests that attributes related to cancer treatment outcomes are the most important for patients. Process and cost attributes were less often included in studies but were still (but less) important to patients in most studies. Clinicians and decision makers should be aware that attribute importance might be influenced by level selection for that attribute.

How Well Can Analytic Hierarchy Process be Used to Elicit Individual Preferences? Insights from a Survey in Patients Suffering from Age-Related Macular Degeneration.

BACKGROUND: In this study, we tested the feasibility of an interviewer-assisted analytic hierarchy process (AHP) in a special patient population with age-related macular degeneration (AMD). OBJECTIVES: One aim was to generate preference weights regarding AMD treatment characteristics. A secondary aim was to explore the consistency of preference judgments and reasons for inconsistency. METHODS: We generated quantitative importance weights for decision criteria using the matrix multiplication method. A qualitative study component in the form of asking patients to think aloud throughout their judgments was implemented to facilitate understanding of quantitative findings. Consistency ratios were calculated as a measure of logical judgment performance within AHP. If consistency ratios exceeded 0.2, we explored reasons for inconsistency. RESULTS: We interviewed 86 patients and generated preference weights for criteria. Patients rated the injection's effect on visual function the highest (0.44), followed by the frequency of monitoring visits (0.18), approval status (0.13), injection frequency (0.13), and side effects (0.12). Inconsistency in judgments was prevalent at the subcriteria level. Whereas much of the observed inconsistency was due to an excessive use of high/extreme value judgments, these judgments seemed to result from patients reasonably trying to highlight their strong preferences. CONCLUSION: Our study combines quantitative with qualitative data to explore patients' preference weights and decision processes using the AHP. It suggests that the type of inconsistency observed in judgments of AMD patients mostly results from rational decision making, not from error or lack of understanding. Further research should address which type and extent of inconsistency might be acceptable in different AHP settings.

Focus Groups in Elderly Ophthalmologic Patients: Setting the Stage for Quantitative Preference Elicitation.

OBJECTIVES: Patients suffering from age-related macular degeneration (AMD) are rarely actively involved in decision-making, despite facing preference-sensitive treatment decisions. This paper presents a qualitative study to prepare quantitative preference elicitation in AMD patients. The aims of this study were (1) to gain familiarity with and learn about the special requirements of the AMD patient population for quantitative data collection; and (2) to select/refine patient-relevant treatment attributes and levels, and gain insights into preference structures. METHODS: Semi-structured focus group interviews were performed. An interview guide including preselected categories in the form of seven potentially patient-relevant treatment attributes was followed. To identify the most patient-relevant treatment attributes, a ranking exercise was performed. Deductive content analyses were done by two independent reviewers for each attribute to derive subcategories (potential levels of attributes) and depict preference trends. RESULTS: The focus group interviews included 21 patients. The interviews revealed that quantitative preference surveys in this population will have to be interviewer assisted to make the survey feasible for patients. The five most patient-relevant attributes were the effect on visual function [ranking score (RS): 139], injection frequency (RS: 101), approval status (RS: 83), side effects (RS: 79), and monitoring frequency (RS: 76). Attribute and level refinement was based on patients' statements. Preference trends and dependencies between attributes informed the quantitative instrument design. CONCLUSION: This study suggests that qualitative research is a very helpful step to prepare the design and administration of quantitative preference elicitation instruments. It especially facilitated familiarization with the target population and its preferences, and it supported attribute/level refinement.

Using qualitative research to facilitate the interpretation of quantitative results from a discrete choice experiment: insights from a survey in elderly ophthalmologic patients.

BACKGROUND: Age-related macular degeneration (AMD) is the leading cause of visual impairment and blindness in industrialized countries. Currently, mainly three treatment options are available, which are all intravitreal injections, but differ with regard to the frequency of injections needed, their approval status, and cost. This study aims to estimate patients' preferences for characteristics of treatment options for neovascular AMD. METHODS: An interviewer-assisted discrete choice experiment was conducted among patients suffering from AMD treated with intravitreal injections. A Bayesian efficient design was used for the development of 12 choice tasks. In each task patients indicated their preference for one out of two treatment scenarios described by the attributes: side effects, approval status, effect on visual function, injection and monitoring frequency. While answering the choice tasks, patients were asked to think aloud and explain the reasons for choosing or rejecting specific characteristics. Quantitative data were analyzed with a mixed multinomial logit model. RESULTS: Eighty-six patients completed the questionnaire. Patients significantly preferred treatments that improve visual function, are approved, are administered in a pro re nata regimen (as needed), and are accompanied by bimonthly monitoring. Patients significantly disliked less frequent monitoring visits (every 4 months) and explained this was due to fear of deterioration being left unnoticed, and in turn experiencing disease deterioration. Significant preference heterogeneity was found for all levels except for bimonthly monitoring visits and severe, rare eye-related side effects. Patients gave clear explanations of their individual preferences during the interviews. CONCLUSION: Significant preference trends were discernible for the overall sample, despite the preference heterogeneity for most treatment characteristics. Patients like to be monitored and treated regularly, but not too frequently or infrequently. The results of our qualitative research facilitated the interpretation of the quantitative data collected in this study.

Price elasticities in the German Statutory Health Insurance market before and after the health care reform of 2009.

This paper investigates the change in price elasticity of health insurance choice in Germany after a reform of health insurance contributions. Using a comprehensive data set of all sickness funds between 2004 and 2013, price elasticities are calculated both before and after the reform for the entire market. The general price elasticity is found to be increased more than 4-fold from -0.81 prior to the reform to -3.53 after the reform. By introducing a new kind of health insurance contribution the reform seemingly increased the price elasticity of insured individuals to a more appropriate level under the given market parameters. However, further unintended consequences of the new contribution scheme were massive losses of market share for the more expensive sickness funds and therefore an undivided focus on pricing as the primary competitive element to the detriment of quality.