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Quantitative Benefit-Risk Assessment: State of the Practice Within Industry
Meredith Y Smith1,2, Janine van Til3, Rachael L DiSantostefano4
1Alexion Pharmaceuticals, Inc., 121 Seaport Boulevard, Boston, MA, 02210, USA. meredith.smith@alexion.com.
Therapeutic Innovation & Regulatory Science
|October 28, 2020
Summary
Life sciences companies widely use quantitative benefit-risk assessment (qBRA) methods, mainly for internal decisions and regulatory submissions. Adoption is growing, but clarity on value and regulatory guidance are key for broader implementation.
Area of Science:
- Pharmacoeconomics and Health Technology Assessment
- Regulatory Science
- Decision Analysis
Background:
- Benefit-risk assessments for medical products have advanced.
- Quantitative benefit-risk assessment (qBRA) methods are increasingly relevant.
Purpose of the Study:
- To assess the utilization of quantitative benefit-risk assessment (qBRA) methods in the life sciences industry.
- To understand the application, impact, and challenges of qBRA.
Main Methods:
- Semi-structured interviews with 20 industry professionals in drug and medical device benefit-risk assessment.
- Thematic analysis of recorded, transcribed interviews.
Main Results:
- Most surveyed companies applied qBRA, but primarily for a limited number of assets, supporting internal decisions and regulatory submissions.
- Positive impacts included enhanced team decision-making and communication.
- Multi-criteria decision analysis and discrete choice experiment were common qBRA methods.
Conclusions:
- qBRA use is widespread but concentrated; its primary applications are internal decision-making and regulatory support.
- Successful adoption hinges on leadership support and regulatory receptivity.
- Future capacity building is planned, but some await further regulatory guidance.
Keywords:
Benefit–risk frameworkDiscrete choice experimentMedical devicesMedicinal productsMulti-criteria decision analysisStructured benefit–risk assessmentMore Related Videos
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