Patient and Caregiver Perspectives on Care-Seeking During a Vaso-Occlusive Crisis in Sickle Cell Disease: Results from Qualitative Interviews in Canada.

Purpose: There is little research on care-seeking preferences during active pain crises for sickle cell disease (SCD) patients and their caregivers. The objective of this study was to identify relevant, patient or caregiver narratives of the pain crisis experience, to understand the factors that contribute to care-seeking during a pain crisis, and to identify preferences when making care-seeking decisions during a pain crisis. Patients and Methods: Qualitative semi-structured interviews were conducted with Canadian residents with a self-reported SCD diagnosis, who were either ≥18 years of age or an adolescent between the ages of 12-18. Interviews were hosted virtually, audio-recorded, and transcribed verbatim. Results: A total of 23 individuals participated (74% female; 26% male), including six adolescents with parent dyads and 11 adults. Almost all (N = 21, 91.3%) participants were Black/African American. Participants mentioned many factors that influenced care-seeking decisions, mainly the symptom and pain experience; institutional factors (waiting time, the presence of and adherence to treatment guidelines, and the empathy or racial bias felt from medical staff); and subject-level factors (age and a flexibility in daily responsibilities). Conclusion: This study identified important institutional and subject-level considerations involved in care-seeking decisions. Most importantly, this study highlights the racial stigma faced by many patients when care-seeking in the ER and the lack of care protocol implemented, which hinders care-seeking in a dedicated medical facility. From the patient perspective, these are clear gaps to fill to encourage patients to seek and receive the care they deserve.

The evolving role of patient preference studies in health-care decision-making, from clinical drug development to clinical care management.

Introduction: There is a growing trend of using patient preference studies to help incorporate the patient perspective into clinical drug development, care management, and health-care decision-making. Collecting and interpreting patient preference data is integral to multi-stakeholder engagement, patient-centric drug development, and clinical care management. Operationally, challenges exist in understanding 'when' and 'how' to embark on patient preference studies. This review will provide a brief overview of stated-preference methods, discuss applications throughout the clinical drug development and care management, and highlight how preference studies serve as a powerful tool for quantifying patient experiences for better outcomes. Areas covered: We present case studies to complement the different applications of stated-preference methods in clinical drug development and care management. We discuss the applications of preference data to help inform evidence-based patient advocacy, clinical development strategy, operational feasibility, regulator benefit-risk assessments, health technology assessments, and clinical decision-making. Expert commentary: Patient preference studies can serve as a powerful tool to engage patients and their communities as well as quantify the patient voice across different stages of clinical drug development and care management to support patient-centric health-care decision-making. It is expected that the application of these strategies will quickly advance in the coming years.

AcroVoice: eliciting the patients' perspective on acromegaly disease activity.

PURPOSE: To determine how patients define acromegaly disease activity and treatment success and to quantify from the patients' perspective the relative importance of each disease parameter included in the ACRODAT®. METHODS: One hundred acromegaly patients on medical therapy (mean age = 47.1 years; SD = 11.96) completed an online preference study evaluating hypothetical patient profiles described in terms of insulin-like growth factor-I (IGF-I) levels, tumor size, comorbid conditions, signs/symptoms, and quality of life (QoL). Participants first completed a single-profile task experiment by rating 20 single patient profiles as exhibiting stable, mild, or significant disease activity based on treatment success. Next, participants completed a double-profile discrete choice experiment (DCE) by selecting the patient that was doing "better" from 15 profile pairs. Results were analyzed using logistic and conditional logistic models. RESULTS: When choosing between stable vs. mild or significant disease activity, signs/symptoms, tumor size, and IGF-I levels were weighted equally; IGF-I and signs and symptoms were valued equally when selecting mild vs. significant disease activity. The DCE showed that, statistically, all disease parameters, except comorbid conditions, predicted health status equally. Tumor size and IGF-I levels each accounted for 23% of the decision-making process; QoL, signs/symptoms, and comorbid conditions accounted for 21%, 19%, and 14%, respectively. CONCLUSION: All five ACRODAT® parameters had some influence on disease activity from the patients' perspective. To account for patients' preferences and optimize treatment and outcomes, a holistic disease management approach should be employed.