Patient-Reported Outcome Measures in Atopic Dermatitis and Chronic Hand Eczema in Adults Abstract Patient-reported outcome measures (PROMs) provide an important complement to physician-assessed clinical outcome measures in dermatologic diseases such as atopic dermatitis (AD) and chronic hand eczema (CHE). AD and CHE are chronic and relapsing inflammatory skin conditions that often co-occur. While both diseases result in various signs and symptoms that are burdensome and can negatively affect patients’ lives, there may be distinct differences in the signs, symptoms, burden, and health-related quality of life (HRQOL) impact of these diseases. The objective of this study was to identify and evaluate PROMs used in studies of AD and CHE. The aim was to explore the assessment of key symptoms and impacts, and identify any gaps in the measures in use. A structured review of the PubMed database was conducted to identify PROMs used or developed for use in AD or CHE. The Dermatology Life Quality Index (DLQI), the Pruritus/Itch Numeric Rating Scale (NRS), the Patient-Oriented Eczema Measure (POEM), and the Quality of Life in Hand Eczema Questionnaire (QOLHEQ) were identified and reviewed in detail. With these measures, the AD and CHE symptoms and impacts most commonly evaluated in the literature include dermatology-related HRQOL in the domains of symptoms and feelings, daily activities, leisure, work and school, personal relationships, and adverse effects; pruritus; sleep disturbance; AD-specific symptoms (dryness, itching, flaking, cracking, bleeding, and weeping/oozing); and CHE-specific symptoms (pain, itch, fissuring, redness, bleeding, and dryness). A review of regulatory labels of drugs approved for AD by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) found that, among the four measures reviewed, the Pruritus NRS was included in the FDA and EMA labels for dupilumab, the DLQI was included in the EMA labels for dupilumab and tacrolimus, and the POEM was included in the EMA label for dupilumab. Key symptoms of AD (e.g. itching, flaking, cracking) and CHE (e.g. pain, itching, fissuring) are increasingly being assessed with PROMs; however, primary endpoints in clinical trials are often based on clinician-reported outcome measures. As therapeutic strategies in dermatology are targeted at specific dermatologic symptoms and diseases affecting specific sites (e.g. CHE), future research should explore patients’ experiences with these symptoms and sites and the changes with treatment that are most meaningful to them.

A Patient-Centered Description of Severe Asthma: Patient Understanding Leading to Assessment for a Severe Asthma Referral (PULSAR) Abstract Background Although severe asthma can be life-threatening, many patients are unaware they have this condition. Objectives Patient Understanding Leading to Assessment for a Severe Asthma Referral (PULSAR) is a novel, multidisciplinary working group aiming to develop and disseminate a global, patient-centered description of severe asthma to improve patient understanding of severe asthma and effect a change in patient behavior whereby patients are encouraged to visit their healthcare professional, when appropriate. Methods Current definitions from patient organization websites, asthma guidelines, and medication information for key asthma drugs were assessed and informed a multidisciplinary working group, convened to identify common concepts and terminology used to define severe asthma. A patient-centered description of severe asthma and patient checklist were drafted based on working-group discussions and reviewed by an external behavioral scientist for patient understanding and relevance. These were tested using an online US/Canadian survey. Results The patient-centered description of severe asthma and patient checklist were reviewed and re-drafted by the authors. The text was simplified following the behavioral-scientist review. The survey (n = 153) included 105 patients with severe asthma. Of those with severe asthma, 92.2% of patients reported that the description was consistent with their experiences of severe asthma and 92.6% of patients reported that the PULSAR initiative would encourage them to visit their healthcare provider. Conclusion A patient-centered description of severe asthma has been developed and tested using patients with severe asthma; this description will allow patients to assess whether they might have severe asthma and prompt them to visit their healthcare provider, if appropriate.

Do Non-participants at Screening have a Different Threshold for an Acceptable Benefit–Harm Ratio than Participants? Results of a Discrete Choice Experiment Abstract Objective The objective of the study was to investigate non-participants’ preferences for cardiovascular disease screening programme characteristics and whether non-participation can be rationally explained by differences in preferences, decision-making styles and informational needs between non-participants and participants. Methods We conducted a discrete choice experiment at three screening sites between June and December 2017 among 371 male non-participants and 830 male participants who were asked to trade different levels of five key programme characteristics (chance of health benefit, risk of overtreatment, risk of later regret, screening duration and screening location). Data were analysed using a multinomial mixed-logit model. Health benefit was used as a payment vehicle for estimation of marginal substitution rates. Results Non-participants were willing to accept that 0.127 (95% confidence interval 0.103–0.154) fewer lives would be saved to avoid overtreatment of one individual, whilst participants were willing to accept 0.085 (95% confidence interval 0.077–0.094) fewer lives saved. This translates into non-participants valuing health benefits 7.9 times higher than overtreatment. The corresponding value of participants is 11.8. Similarly, non-participants had higher requirements than participants for advanced technology and a quicker screening duration. With regard to their participation decision, 64% of the non-participants felt certain about their choice compared with 89% among participants. Conclusions This study shows that non-participants have different preferences than participants at screening as they express relatively more concern about overtreatment and have higher requirements for a high-tech screening programme. Non-participants also report to be more uncertain about their participation decision and more often seek additional information to the standard information provided in the invitation letter. Further studies on informational needs and effective communication strategies are warranted to ensure that non-participation is a fully informed choice.

Analysis of Clinical Trial Exit Interview Data in Patients with Treatment-Resistant Depression Abstract Background Clinical outcome assessments may not fully capture patients’ perspectives of treatment benefit or tolerability. Incorporating individual exit interviews might enhance the description of the patient experience of drug effects. Objective The objective of this study was to evaluate the patient treatment experience in a clinical trial of treatment-resistant depression utilizing exit interview methodology. Methods Individual patient interviews were conducted with subjects exiting two phase II clinical trials involving investigational agents for treatment-resistant depression. Interviews included standardized questions about patients’ perceptions of health changes and interest in continued use of the investigational agent. Constant comparative analysis of blinded data was used to identify, code, and categorize the data followed by a subsequent analysis of unblinded data to evaluate any potential treatment differences. Results Ninety subjects completed exit interviews across the two trials. Most subjects (90%, Trial 2001; 74%, Trial 2002) reported at least one health change. Most subjects rated these changes to be at least moderately important, with most being rated “very important” to “extremely important.” After unblinding, participants receiving active therapy alone reported most of the positive health changes (80% of overall positive changes in Trial 2001, 89% in Trial 2002), whereas patients taking placebo alone reported the majority of negative health changes (57% in Trial 2002). Positive changes included not only anticipated changes in mood but also potential cognitive benefits such as mental alertness, improved sleep, and better concentration. Conclusions Standardized interview data provided direct patient insight into the treatment experience from the patient perspective. Data from these interviews assisted in phase III endpoint selection by providing data on relevant concepts in the target treatment-resistant depression population receiving a new treatment, thus enabling the selection of tools to capture noted treatment effects and, by eliminating irrelevant constructs or measures, thereby reducing data “noise.” Trial Registration ClinicalTrials.gov NCT01640080; NCT01627782.

Patient Preferences in the Medical Product Life Cycle: What do Stakeholders Think? Semi-Structured Qualitative Interviews in Europe and the USA Abstract Background Patient preferences (PP), which are investigated in PP studies using qualitative or quantitative methods, are a growing area of interest to the following stakeholders involved in the medical product lifecycle: academics, health technology assessment bodies, payers, industry, patients, physicians, and regulators. However, the use of PP in decisions along the medical product lifecycle remains limited. As the adoption of PP heavily relies on these stakeholders, knowledge of their perceptions of PP is critical. Objective This study aimed to characterize stakeholders’ attitudes, needs, and concerns with respect to PP in decision making along the medical product lifecycle. Methods Semi-structured interviews (n = 143) were conducted with academics (n = 24), health technology assessment/payer representatives (n = 24), industry representatives (n = 24), patients, caregivers and patient representatives (n = 24), physicians (n = 24), and regulators (n = 23) from seven European countries and the USA. Interviews were conducted between April and August 2017. The framework method was used to organize the data and identify themes and key findings in each interviewed stakeholder group. Results Interviewees reported being unfamiliar (43%), moderately familiar (42%), or very familiar (15%) with preference methods and studies. Interviewees across stakeholder groups generally supported the idea of using PP in the medical product lifecycle but expressed mixed opinions about the feasibility and impact of using PP in decision making. Interviewees from all stakeholder groups stressed the importance of increasing stakeholders’ understanding of the concept of PP and preference methods and ensuring patients’ understanding of the questions asked in PP studies. Key concerns and needs in each interviewed stakeholder group were as follows: (1) academics: investigating the validity, reliability, reproducibility, and generalizability of preference methods; (2) health technology assessment/payer representatives: developing quality criteria for evaluating PP studies and gaining insights into how to weigh them in reimbursement/payer decision making; (3) industry representatives: obtaining guidance on PP studies and recognition on the importance of PP from decision makers; (4) patients, caregivers, and patient representatives: providing an incentive and adequate information towards patients when participating in PP studies; (5) physicians: avoiding bias as a result of commercial agendas in PP studies and clarifying how to deal with subjective and emotional elements when measuring PP; and (6) regulators: avoiding the misuse of PP study results to overrule the traditional efficacy and safety criteria used for marketing authorization and obtaining robust PP study results. Conclusions Despite the interest all interviewed stakeholder groups reported in PP, the effective use of PP in decision making across the medical product lifecycle is currently hampered by a lack of standardization and consensus on how to both measure and use PP.

Patient Experience with Congenital (Hereditary) Thrombotic Thrombocytopenic Purpura: A Conceptual Framework of Symptoms and Impacts Abstract Background and Objective Thrombotic thrombocytopenic purpura is a rare, life-threatening disorder characterized by microangiopathic hemolytic anemia and thrombocytopenia, with variable clinical manifestations (e.g., central nervous system, renal, gastrointestinal, and cardiac effects). This study’s objective was to gain an in-depth understanding of patients’ experiences with the congenital form of thrombotic thrombocytopenic purpura, including the most salient symptoms and impacts associated with congenital thrombotic thrombocytopenic purpura and its treatment. Methods An initial conceptual model of thrombotic thrombocytopenic purpura symptoms and impacts was derived from a targeted literature review, refined by interviews with expert hematologists, and further refined by concept elicitation telephone interviews with adults with congenital thrombotic thrombocytopenic purpura in the USA. Patients reported the duration, frequency, and severity experienced for each concept, and rated level of disturbance on a minimum to maximum scale of 0–10. Results Interviews were conducted with 11 patients (mean age, 38.2 years; range 21–52 years) in three waves (n = 4, n = 4, n = 3). The most salient symptoms (reported most frequently and rated by patients as most disturbing) were fatigue, headache, bruising, joint pain, muscular pain, forgetfulness, and difficulty communicating. The most salient impacts included diminished ability to work/study, financial distress, feeling depressed, feeling anxious, and mood swings. Patients’ comments reflected the pervasive nature of congenital thrombotic thrombocytopenic purpura symptoms and impacts, and unmet treatment needs. Conclusions The final conceptual model, which includes salient symptoms and impacts of congenital thrombotic thrombocytopenic purpura and reflects the disease burden, was derived by integrating inputs from the literature review, expert opinion, and patient interviews, and will be used to develop a congenital thrombotic thrombocytopenic purpura–specific, patient-reported outcome instrument.

Support Tools for Preference-Sensitive Decisions in Healthcare: Where Are We? Where Do We Go? How Do We Get There?

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.

Humanization of Care: Key Elements Identified by Patients, Caregivers, and Healthcare Providers. A Systematic Review Abstract Background Given the automatization of care and rationing of time and staff due to economic imperatives, often resulting in dehumanized care, the concept of ‘humanization of care’ has been increasingly discussed in the scientific literature. However, it is still an indistinct concept, lacking well-defined dimensions and to date no literature review has tried to capture it. Objectives The objectives of this systematic review were to identify the key elements of humanization of care by investigating stakeholders’ (patients, patients’ caregivers, healthcare providers) perspectives and to assess barriers and strategies for its implementation. Methods We carried out a systematic search of five electronic databases up to December 2017 as well as examining additional sources (e.g., gray literature). Search terms included “humanization/humanisation of care” and “dehumanization/dehumanisation of care”. We conducted a thematic synthesis of the extracted study findings to identify descriptive themes and produce key elements. Results Of 1327 records retrieved, 14 full-text articles were included in the review. Three main areas (relational, organizational, structural) and 30 key elements (e.g., relationship bonding, holistic approach, adequate working conditions) emerged. Several barriers to implementation of humanization of care exist in all areas. Conclusion Our systematic review and synthesis contributes to a deeper understanding of the concept of humanization of care. The proposed key elements are expected to serve as preliminary guidance for healthcare institutions aiming to overcome challenges in various forms and achieve humanized and efficient care. Future studies need to fully examine specific practices of humanized care and test quantitatively their effectiveness by examining psychosocial and health outcomes.

African Americans Want a Focus on Shared Decision-Making in Asthma Adherence Interventions Abstract Background and Objective Inhaled corticosteroids (ICS) reduce asthma-related morbidity and mortality. However, ICS non-adherence is more common in African American (AA) adults than White adults and explains, in part, the marked asthma disparities that AAs experience. We aimed to understand how ICS non-adherence could be addressed from the perspective of AA adults with asthma, their family, and friends. Methods We held six focus groups at two urban federally qualified health centers separately with adult asthma patients (n = 2), patients’ family/friends (n = 2), and patients and family/friends together (n = 2). Qualitative descriptive methodology guided the design and the conduct of focus groups. Verbatim transcripts were analyzed by three coders working independently using conventional content analysis to capture responses to interview questions and identify emergent categories. Results Forty-six AA adults participated (32 patients, 14 family/friends); 67% were female. Participants stated that ICS adherence could be improved if they were heard, respected, and received patient-centered care, and if providers highlighted the risk of ICS non-adherence at clinic visits. Though not explicitly described by participants as shared decision-making (SDM), what they described included many essential elements of SDM. Conclusions Participants desired SDM and offered reasons for ICS non-adherence that could be used to inform an SDM intervention for clinical application. Strategies informed by the recipients of care and delivered by providers during routine office visits offer a scalable approach to narrowing asthma disparities experienced by AA adults. Trial Registration ClinicalTrials.gov identifier NCT03036267.