Young Adult and Parent Willingness to Pay for Meningococcal Serogroup B Vaccination.

Unlabelled: Serogroup B (MenB) is the leading cause of invasive meningococcal disease among adolescents and young adults in the United States. The US Advisory Committee on Immunization Practices (ACIP) recommends MenB vaccination based on shared clinical decision making between patients and providers. However, suboptimal understanding of these recommendations could contribute to low vaccination awareness and coverage.

Use of Patient Preferences Data Regarding Multiple Risks to Inform Regulatory Decisions.

Unlabelled: Risk-tolerance measures from patient-preference studies typically focus on individual adverse events. We recently introduced an approach that extends maximum acceptable risk (MAR) calculations to simultaneous maximum acceptable risk thresholds (SMART) for multiple treatment-related risks. We extend these methods to include the computation and display of confidence intervals and apply the approach to 3 published discrete-choice experiments to evaluate its utility to inform regulatory decisions.

How Does the Public Evaluate Vaccines for Low-Incidence, Severe-Outcome Diseases? A General-Population Choice Experiment.

Background: Because immunizing large numbers of healthy people could be required to reduce a relatively small number of infections, disease incidence has a large impact on cost effectiveness, even if the infection is associated with very serious health outcomes. In addition to cost effectiveness, the US Advisory Committee on Immunization Practices requires evidence of stakeholders' values and preferences to help inform vaccine recommendations. This study quantified general-population preferences for vaccine trade-offs among disease severity, disease incidence, and other vaccine features.

Method for Calculating the Simultaneous Maximum Acceptable Risk Threshold (SMART) from Discrete-Choice Experiment Benefit-Risk Studies.

Background: Medical decisions require weighing expected benefits of treatment against multiple adverse outcomes under uncertainty (i.e., risks) that must be accepted as a bundle.

Primary Immune Deficiency: Patients' Preferences for Replacement Immunoglobulin Therapy.

Purpose: Immunoglobulin (Ig) replacement therapy is an important life-saving treatment modality for patients with primary antibody immune deficiency disorders (PAD). IVIG and SCIg are suitable alternatives to treat patients with PAD but vary in key ways. Existing evidence on patient preferences for Ig treatments given the complexities associated with IVIG and SCIg treatment is limited and fails to account for variations in preferences across patients.

Women's Willingness to Accept Risks of Medication for Inflammatory Bowel Disease During Pregnancy.

Background: Women with inflammatory bowel disease (IBD) face difficult decisions regarding treatment during pregnancy: while the majority of IBD medications are safe, there is substantial societal pressure to avoid exposures during pregnancy. However, discontinuation of IBD medications risks a disease flare occurring during pregnancy.

Objective: This study quantified women's knowledge about pregnancy and IBD and their willingness to accept the risks of adverse pregnancy outcomes to avoid disease activity or medication use during pregnancy.

Patients' Willingness to Accept Mitral Valve Procedure-Associated Risks Varies Across Severity of Heart Failure Symptoms.

Background: The Food and Drug Administration's Center for Drugs and Radiological Health issued Guidance in 2016 on generating patient preference information to aid evaluation of medical devices. Consistent with this guidance, we aimed to provide quantitative patient preference evidence on benefit-risk tradeoffs relevant to transcatheter mitral valve repair versus medical therapy for patients with heart failure and symptomatic secondary mitral regurgitation.

Methods: A discrete-choice experiment survey was designed to quantify patients' tolerance for 30-day mortality or serious bleeding risks to achieve improvements in physical functioning or reductions in heart failure hospitalizations.

Patient Preferences for Device-Aided Treatments Indicated for Advanced Parkinson Disease.

Background: Effective treatment for advanced Parkinson disease (PD) uncontrolled with oral medication includes device-aided therapies such as deep brain stimulation (DBS) and continuous levodopa-carbidopa infusion to the duodenum via a portable pump.

Objective: Our objective was to quantify patient preferences for attributes of these device-aided treatments.

Methods: We administered a Web-enabled survey to 401 patients in the United States.

What is clearance worth? Patients' stated risk tolerance for psoriasis treatments.

Purpose: The purpose of this study was to provide quantitative evidence of patients' tolerance for therapeutic risks associated with psoriasis treatments that could offer psoriasis improvements beyond the PASI 75 benchmark.

Materials And Methods: We used a discrete-choice experiment in which respondents chose between competing psoriasis treatments characterized by benefits (i.e.

Effective Partnering in Conducting Benefit-Risk Patient Preference Studies: Perspectives From a Patient Advocacy Organization, a Pharmaceutical Company, and Academic Stated-Preference Researchers.

Background: Formal incorporation of patients' perspectives is becoming increasingly important in medical product development and decision making. This article shares practical advice regarding how patient advocacy organizations, the pharmaceutical industry, and academic experts in stated-preference research can effectively partner on benefit-risk patient preference studies.

Methods: The authors partnered on a benefit-risk patient preference study related to the treatment of psoriasis.

Patient Benefit-Risk Tradeoffs for Radioactive Iodine-Refractory Differentiated Thyroid Cancer Treatments.

Background. The aims of this study were to assess patients' preferences to wait or start systemic treatment and understand how patients would make tradeoffs between certain severe adverse events (AEs) and additional months of progression-free survival (PFS). Materials and Methods.

Inflammatory Bowel Disease Patients' Willingness to Accept Medication Risk to Avoid Future Disease Relapse.

Objectives: Biomarkers, endoscopy and imaging tests can identify patients at increased risk for early recurrence of symptomatic inflammatory bowel disease (IBD). However, patients may be unwilling to accept additional medical therapy risks related to therapy escalation to avoid a future disease relapse. We sought to quantify IBD patients' willingness to accept medication risk to avoid future disease relapse.

Patient preferences for surgical versus medical therapy for ulcerative colitis.

Background: Therapy options for mesalamine-refractory ulcerative colitis (UC) include immunosuppressive medications or surgery. Chronic immunosuppressive therapy increases risks of infection and cancer, whereas surgery produces a permanent change in bowel function. We sought to quantify the willingness of patients with UC to accept the risks of chronic immunosuppression to avoid colectomy.

Quantifying benefit-risk preferences for medical interventions: an overview of a growing empirical literature.

Decisions regarding the development, regulation, sale, and utilization of pharmaceutical and medical interventions require an evaluation of the balance between benefits and risks. Such evaluations are subject to two fundamental challenges-measuring the clinical effectiveness and harms associated with the treatment, and determining the relative importance of these different types of outcomes. In some ways, determining the willingness to accept treatment-related risks in exchange for treatment benefits is the greater challenge because it involves the individual subjective judgments of many decision makers, and these decision makers may draw different conclusions about the optimal balance between benefits and risks.