March 4, 2016: Patient-Focused Benefit-Risk Assessment
Topic
Patient-Focused Benefit-Risk Assessment
Presenter
Bennett Levitan, MD, PhD, Janssen Research and Development
Keywords
Patient-centered research; Patient preferences; Benefit-risk assessment; Clinical decision-making
Key Points
- Benefit-risk assessment is an evaluation of medical products that considers both benefits and harms; it involves more than the union of efficacy and safety data. This growing field combines regulatory, clinical, decision-making, behavioral economics, and risk-communication sciences.
- Patient-focused benefit-risk assessment is an important component of patient engagement and includes the following aspects:
- Characterizing the burden of disease and unmet need
- Developing novel endpoints and patient-reported outcome (PRO) instruments
- Determining which endpoints are most important
- Assessing preference tradeoffs between endpoints
- Identifying variations in preferences - “Preferences” in this context are qualitative or quantitative statements of the relative desirability or acceptability of attributes that differ among alternative health interventions.
- Using patient preference information in a regulatory context requires more than expressions of feelings or opinions. It needs defensible data, and preference studies can obtain these data on the maximum additional risk that patients would accept for an increase in benefit.
Discussion Themes
What challenges exist for using the patient voice in decision-making?
Would it be possible and desirable to assess real-time patient preferences to optimize individual decision-making?
Patient preferences are considered part of personalized medicine and precision treatment. But how do we learn about variations—preferences that vary not only between people but also within people across time and situations?
For More Information
Read more about patient-focused benefit-risk assessment at the following links:
- FDA’s “Voice of the Patient” Report Series
- FDA CDRH’s Patient Preference Initiative and 2016-2017 Strategic Priorities (PDF)
- Medical Device Innovation Consortium’s Patient-Centered Benefit-Risk Assessment
Tags
#PatientPreferences; #ClinicalDecisionMaking; #pctGR
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