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.

Haemophilia home care: A qualitative evaluation study of the Be Involved infusion program.

INTRODUCTION: In Canada, patients with haemophilia can receive health care through a limited number of specialized haemophilia treatment centres. Treatment of this X-linked bleeding disorder through self-infusion of factor replacement is common; however, some individuals with haemophilia require additional infusion support from medical professionals. While provincially funded outreach clinics and telehealth are available, pharmaceutical companies that supply factor have developed novel home infusion programs wherein registered nurses assist/manage factor administration in the patients' homes. AIM: The current qualitative evaluation explored the real-world effectiveness and impact of the Be Involved (Pfizer Canada) home infusion service, from provider, program end-user and needs analysis perspectives. METHODS: Qualitative interviews with patients and caregivers who had used Be Involved (n = 7) and Be Involved Staff (n = 8) provided important insights and recommendations for program improvement initiatives. Also included were needs assessments of potential end-users (n = 11) as a means to making program adjustments. RESULTS: The inductively derived findings revealed Be Involved as a highly valued, though key specialized service providing significant assistance to a small number of haemophilia patients. Augmenting traditional services, the program was endorsed as easing the burden of haemophilia care, while the Be Involved nurses were affirmed as highly effective and critical to the program's success. A lack of program promotion and awareness was evident - limiting uptake, amid communication and systems delivery challenges. CONCLUSIONS: The current evaluation findings inform our recommendations for, and brief discussion of, the need for stronger promotional strategies and improved communication lines to increase the reach and efficiencies of the Be Involved program.

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.

Nonvocational outcomes of vocational rehabilitation: reduction in health services utilization.

BACKGROUND: Numerous studies have demonstrated the beneficial effects of vocational rehabilitation on vocational outcomes in individuals with a mental illness, yet effects on secondary outcomes remain largely unexplored. This study investigates the impact of vocational rehabilitation on the utilization of emergency, ambulatory care and inpatient services in individuals with a mental illness. METHODS: Using a repeated measures study design, the utilization of health services by individuals with a mental illness (n= 37) was compared before and during their engagement in training and employment at a social enterprise - a form of vocational rehabilitation. RESULTS: Individuals with a mental illness had significantly less emergency department visits (p=0.01), ambulatory care visits (p=0.01) and hospital admissions (p=0.05), but no difference in hospital length of stay (p=0.39), during training/employment, compared to pre-training/employment at a social enterprise. CONCLUSION: The reduction in health services utilization found in this study may reflect symptom and overall health improvement, highlighting the importance of vocational rehabilitation programs for individuals with a mental illness. In addition, results from this study can inform stakeholders and policymakers about the impact of vocational rehabilitation on the healthcare system to help guide decisions regarding program implementation or continuation, and funding allocation.