The Patient - Patient-Centered Outcomes Research

Patient Perspectives on the Value of Patient Preference Information in Regulatory Decision Making: A Qualitative Study in Swedish Patients with Rheumatoid Arthritis Abstract Background There is increasing interest in involving patient preferences for benefits and risks in regulatory decision making. Therefore, it is essential to identify patient perspectives regarding the value of patient preference information (PPI). Objectives The aim of this study was to explore how patients with rheumatoid arthritis (RA) value the use of PPI in regulatory decision making regarding medical products. Methods Regulators and patients with RA were interviewed to gather initial insights into opinions on the use of PPI in regulatory decisions regarding medical products. The interviews were used to draft and validate the interview guide for focus groups with patients with RA. Participants were purposively sampled in collaboration with the Swedish Rheumatism Association in Stockholm and Uppsala. Each focus group consisted of three to six patients (18 in total). All interviews were audio-recorded, transcribed verbatim, and analysed using content analysis. Results According to the participants, PPI could lead to regulators considering patients’ needs, lifestyles and well-being when making decisions. PPI was important in all stages of the medical product lifecycle. Participants reported that, when participating in a preference study, it is important to be well-informed about the use of the study and the development, components, administration, and risks related to the medical products. Conclusions Patients thought PPI could be valuable to consider in regulatory decisions. It is essential for patients to be well-informed when asked for their preferences. Research on information materials to inform patients in preference studies is needed to increase the value of PPI in regulatory decision making.

High Proportion of Subjective Component to the Disease Activity Score is Associated with Favorable Response to Abatacept in Rheumatoid Arthritis Abstract Background Response prediction of certain biologic agents for the treatment of rheumatoid arthritis (RA) remains an unmet need in real-world clinical practice. The contribution of patient-reported components to the 28-joint Disease Activity Score (DAS28) was termed DAS28-P and investigated as a predictor of response to biologic agents, mostly tumor necrosis factor inhibitors. We aimed to evaluate DAS28-P as a predictor of the European League Against Rheumatism (EULAR) response to abatacept in patients with RA. Methods The study population was a prospective, observational, multicenter cohort of Korean patients with RA, who were followed up for a nationwide post-marketing surveillance study of abatacept. Correlation of DAS28-P with DAS28, change of DAS28, and EULAR response groups were evaluated. Logistic regression analysis was used to predict good-to-moderate EULAR response to abatacept in the study population. Results A total of 341 patients were involved in the analysis stratified on the EULAR response criteria. Presence of comorbidities, previous exposure to biologic agents, baseline DAS28, three of its components (tender joint counts, global health visual analog scale, erythrocyte sedimentation rate), and baseline DAS28-P were significantly associated with EULAR response to abatacept at 6 months. Stratified upon EULAR response, a group with good-to-moderate response had a higher baseline value and lower interval change for DAS28-P. Logistic regression analysis showed that a DAS28-P cut-off of > 0.44 was more positively associated with good-to-moderate EULAR response with abatacept treatment than naivety to biologic agents. Conclusions The DAS28-P could be predictive of response to abatacept. A higher baseline DAS28-P is associated with a favorable therapeutic response to abatacept. Trial registration Trial name, Korean Post-marketing Surveillance for Orencia®. Trial registration number, NCT01583244. Registered on April 20, 2012.

Healthcare Experience and their Relationship with Demographic, Disease and Healthcare-Related Variables: A Cross-Sectional Survey of Patients with Chronic Diseases Using the IEXPAC Scale Abstract Background Patient experience is acknowledged as a principal aspect of quality healthcare delivery, and it has implications with regard to outcomes. Objectives Our objective was to evaluate the healthcare experience of patients with chronic diseases to identify patient-perceived healthcare gaps and to assess the influence of demographic and healthcare-related variables on patient experiences. Methods A cross-sectional survey was delivered to adult patients with chronic diseases: diabetes mellitus (DM), human immunodeficiency virus (HIV) infection, inflammatory bowel disease (IBD) or rheumatic diseases. Patient experiences were assessed with the Instrument for Evaluation of the Experience of Chronic Patients (IEXPAC) questionnaire, with possible scores ranging from 0 (worst) to 10 (best experience). Results Of the 2474 patients handed the survey, 1618 returned it (response rate 65.4%). Patients identified gaps in healthcare related mainly to access to reliable information and services, interaction with other patients and continuity of healthcare after hospital discharge. The mean ± standard deviation (SD) IEXPAC score was 6.0 ± 1.9 and was higher for patients with HIV (6.6 ± 1.7) than for those with rheumatic disease (5.5 ± 2.0), IBD (5.9 ± 2.0) or DM (5.9 ± 1.9) (p < 0.001). In multivariate models, better overall IEXPAC experience was associated with follow-up by the same physician, follow-up by a nurse, receiving healthcare support from others and treatment with subcutaneous or intravenous drugs. The multivariate model that confirmed patients with HIV or DM had better experience than did those with rheumatic diseases. Conclusions Through IEXPAC, patients identified aspects for healthcare quality improvements and circumstances associated with better experience, which may permit greater redirection of healthcare toward patient-centered goals while facilitating improvements in social care and long-term healthcare quality.

Elicitation of Health-Related Utility in Perianal Fistula in Crohn’s Disease Abstract Background and Objective Perianal fistulae are a common complication of Crohn’s disease (CD) and pose a substantial burden on quality of life. Data capturing health-related utility associated with perianal fistulae in CD are scarce. The current study aims to value health states related to different stages of the disease to quantitatively evaluate the impact of complex perianal fistulae on CD patients’ quality of life. Methods Eight health state descriptions associated with complex perianal fistulae in CD were developed following qualitative research with patients and validation by clinicians. Following pre-testing, a survey was administered online in two samples of UK respondents: the general population and patients with CD. A choice-based valuation technique, the time trade-off (TTO), was used for direct utility measurement. CD patients also valued their current health state using the TTO. Exclusion criteria for respondents displaying logical inconsistencies were applied. Results Usable responses were received from 835 respondents, reflective of the UK population in age and sex, in the general population survey and 162 CD patients in the patient survey. Non-remission states were valued much lower than the remission state by both samples, ranging from 0.20 for proctectomy with a negative outcome to 0.66 for chronic symptomatic fistulae with mild symptoms. Patients currently experiencing fistulae reported lower values for current health than those without fistulae. Conclusion Low utility values were assigned to the non-remission health states for perianal fistulae in CD by the general public and patients with CD. This demonstrates the high humanistic burden of inadequately managed perianal fistula in CD.

Creation of a Decision Support Tool for Expectant Parents Facing Threatened Periviable Delivery: Application of a User-Centered Design Approach Abstract Background Shared decision-making (SDM) is optimal in the context of periviable delivery, where the decision to pursue life-support measures or palliation is both preference sensitive and value laden. We sought to develop a decision support tool (DST) prototype to facilitate SDM by utilizing a user-centered design research approach. Methods We convened four patient and provider advisory boards with women and their partners who had experienced a surviving or non-surviving periviable delivery, pregnant women who had not experienced a prior preterm birth, and obstetric providers. Each 2-h session involved design research activities to generate ideas and facilitate sharing of values, goals, and attitudes. Participant feedback shaped the design of three prototypes (a tablet application, family story videos, and a virtual reality experience) to be tested in a final session. Results Ninety-five individuals (48 mothers/partners; 47 providers) from two hospitals participated. Most participants agreed that the prototypes should include factual, unbiased outcomes and probabilities. Mothers and support partners also desired comprehensive explanations of delivery and care options, while providers wanted a tool to ease communication, help elicit values, and share patient experiences. Participants ultimately favored the tablet application and suggested that it include family testimonial videos. Conclusion Our results suggest that a DST that combines unbiased information and understandable outcomes with family testimonials would be meaningful for periviable SDM. User-centered design was found to be a useful method for creating a DST prototype that may lead to improved effectiveness, usability, uptake, and dissemination in the future, by leveraging the expertise of a wide range of stakeholders.

A Guide to Measuring and Interpreting Attribute Importance Abstract Stated-preference (SP) methods, such as discrete-choice experiments (DCE) and best–worst scaling (BWS), have increasingly been used to measure preferences for attributes of medical interventions. Preference information is commonly characterized using attribute importance. However, attribute importance measures  can vary in value and interpretation depending on the method used to elicit preferences, the specific context of the questions, and the approach used to normalize attribute effects. This variation complicates the interpretation of preference results and the comparability of results across subgroups in a sample. This article highlights the potential consequences of ignoring variations in attribute importance measures, and makes the case for reporting more clearly how these measures are obtained and calculated. Transparency in the calculations can clarify what conclusions are supported by the results, and help make more accurate and meaningful comparisons across subsamples.

Issues in the Design of Discrete Choice Experiments

The Value of Treatment Processes in Germany: A Discrete Choice Experiment on Patient Preferences in Complementary and Conventional Medicine Abstract Background The effects of health interventions are often complex, and it is argued that they comprise more than pure changes in clinical parameters. Aspects of the treatment process, so-called ‘benefits beyond health’, are often overlooked in the evaluation of health interventions but can be of value to the patients. Objectives The aim of this study was to assess patients’ preferences and willingness to pay regarding the treatment process and its attributes in patients using acupuncture, homeopathy or general medicine (GM). Methods A systematic literature search, six semi-structured interviews and a stakeholder involvement were conducted to determine the attributes of the treatment process. Five process attributes and one cost attribute were used to construct the experimental design of the discrete choice experiment (DCE) (6 × 3), a cross sectional survey method. Patients were recruited by outpatient physicians practicing in Berlin and Munich, Germany. Process attributes were effects-coded. Data were analyzed in a conditional logit regression. Results Data from 263 patients were analyzed. DCE results showed that the treatment process attributes ‘active listening’ and ‘time’ were most relevant to all patients. Preferences for the attributes ‘holistic treatment’ (more relevant to the acupuncture and homeopathy groups) and ‘information’ (more relevant to the GM group) seemed to differ slightly between the groups. Willingness-to-pay values were higher in the acupuncture and homeopathy groups. Conclusions The time physicians take and the extent to which they listen attentively are most important and are equally important to all patients. These results may contribute to the debate about more patient-centered healthcare. They support a strengthening of medical consultations in the German healthcare system. We suggest giving physicians the opportunity to spend more time with their patients, which may be achieved by changing the general conditions of remuneration (e.g., improved reimbursement of medical consultations). German Clinical Trial Register DRKS00013160.

Systematic Review of Public Preferences for the Allocation of Donor Organs for Transplantation: Principles of Distributive Justice Abstract Background Solid organ transplantation is the treatment of choice for organ failure, but donor organs are a scarce resource because of a large mismatch between supply and demand. This scarcity leads to an ethical dilemma, forcing priority setting in organ allocation to individual patients. Little is known about public preferences regarding priority setting in organ allocation. A systematic review was performed to review the existing evidence and provide an overview of the criteria and criterion levels in regard to ethical aspects of distributive justice. Methods The PubMed, Web of Science, EBSCO and PsycINFO databases were searched for literature published between January 2000 and December 2018. Only original studies were selected. The criteria were identified, extracted and grouped into a self-developed matrix according to the principles of distributive justice to ascertain public preferences. Results Overall, 9645 references were identified, and 15 studies were included. In total, 27 criteria clustered in seven theory-guided groups could be identified: “equality”, “effectiveness/benefit”, “medical urgency”, “own fault”, “value for society”, “medical background” and “sociodemographic status”. It was shown that not only a single principle but rather a combination of principles are relevant for the allocation. Therefore, a public propensity towards a rational utilitarian ethical model of allocation could be recognised. Conclusions The general public not only wanted to allocate organs mainly to those with a good probability of having a successful transplantation but also wanted to consider those who need an organ most urgently to prevent fatal consequences, resulting in unclear trade-offs between effectiveness/benefit and medical urgency. Public preferences for organ allocation are therefore complex, and data regarding clear trade-offs are still lacking.

Multi-Method Patient-Engagement Approach: A Case Example from a PCORI-Funded Training Project