Quantifying Benefit-Risk Preferences for Heart Failure Devices: A Stated-Preference Study
Patient Introduction
This published article summarizes the work of a multi-stakeholder group of experts convened by MDIC to develop and conduct a patient preference study among patients living with heart failure. The goal of this effort was to gain a better understanding of attributes for a potential medical device that patients would value, and how they view potential benefit-risk trade-offs associated with that potential device. This paper describes the unique nature of this project, in which multiple medical device companies worked together with academic experts to tease out important aspects of the patient experience with heart failure and how that experience shapes patients’ views. Patients may be interested in reviewing this article to better understand how patient preference studies are developed, conducted, and applied to clinical development of medical devices.
Summary
Through a private-public collaborative effort, a multi-stakeholder group of experts convened by MDIC applied rigorous stated-preference methods to quantify patients’ trade-off values for benefits and risks for hypothetical heart failure devices. Findings from this study provide a consistent set of benefit-risk valuations that can be used across stakeholder applications to support product development, clinical trial design, and regulatory evaluation. These data also strengthen the call for considering structured patient preferences in shared decision-making.
This article summarizes the process and key steps by which this collaborative study was developed and conducted. For example, the paper reviews how best-practice methods were used to design a discrete-choice experiment (DCE) survey to quantify patients’ willingness to accept adverse risks in exchange for improved effectiveness for hypothetical implantable devices intended to treat heart failure. The paper also discusses the approach used to test the draft survey instrument and determine maximum acceptable risk (MAR) among participants living with heart failure. Additionally, the paper reviews the statistical analysis plan used to predefine data-quality checks, specify models, determine subgroups of interest. In the paper’s discussion section, key results are described, including evidence that most participants living with heart failure were willing to accept single-digit risks of mortality for one-year gains in survival during which their physical functioning would be stable or would improve. For these health gains, the majority would accept in-hospital complication risks occurring as frequently as one in four to one in five patients. However, about one in seven patients generally were unwilling to accept these risks despite improvements in functioning and survival consistent with the device scenarios offered. Finally, the paper describes a variety of ways that results from this type of patient preference study can be used to inform clinical trial design for medical devices.