Lecture 7: Pharmacoepidemiology: risk-benefit analysis and meta analysis Flashcards Preview

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Flashcards in Lecture 7: Pharmacoepidemiology: risk-benefit analysis and meta analysis Deck (16):

Introduction to Risk Benefit Assessment (RBA)

Risk and benefit assessment monitoring are significant contributors to promoting safety and quality in the delivery of health care


RBA (What is it?)

Quantitative methods for systematically evaluating the risks and benefits of new or existing medical interventions


RBA (Why?)

These methods evaluate risk-benefit tradeoffs to assist regulatory and clinical decision making in the absence of directly comparable metrics


RBA (Who?)

Regulators, clinicians and patients who routinely make decisions that require trading safety for desired clinical benefits



-any favorable outcome of the research to society or to the individual



-refers both to the probability of a harm resulting from an activity and also to its magnitude (its HR, IRR, AEs)


Challenges of RBA

-Heterogeneity of metrics (few common denominators)
-Multiplicity (multiple benefits and risks)
-Uncertainty (temporality)
-Paucity of data (exposure: patients and time distribution and outcomes)


Some metrics of RBA

-NNT, QALYs (quality of life)


Risk Management

1) Risk identification
2) Risk assessment
3) Risk prioritization and communication (improve collective and individual decision making)


Current approaches to RBA

-there is no single approach to RBA, so regulatory bodies in the US and abroad are looking for ways to standardize it


Problem states in RBA

-development strategies (when does increased benefit outweigh potential increased risk)
-regulatory approval (how do you know if risk outweighs benefit?)
-risk management (when do you require risk/benefit plans)


Quantitative Approches and Techniques for RBA

1) Quantitative framework for risk and benefit assessment (QFRBA)
2) Beneft-less risk analysis (BLRA)
3) Quality adjusted time without symptoms and toxicity (Q-TWIST)
4) Number needed to treat (NNT) vs. Number Needed to Harm (NNH)
5) Relative Value adjusted number needed to treat (RV-NNT)
6) Minimum Clinical Efficacy (MCE)
7) Incremental Net Health Benefit (INHB)
8) Risk Benefit Plane (RBP) and Risk Benefit Acceptability Threshold (RBAT)
9) Probabilistic Simulation Methods (PSM)
10) Multi-Criteria Decision Analysis (MCDA)
11) Risk-Benefit Contour (RBC)


RBA Framework

Measure background benefit, characterize background risk, design study, analyze relative risk benefit, communicate risk benefit to FDA all to optimize risk benefit of drug of interest


Optimizing benefit-risk profile

demonstrate product benefit, characterize risk, quantify the benefit/risk, ensure appropriate use and communicate benefit/risk to stakeholders



-Quantitative RBA and meta-analytic methods supplement, but do not replace the existing paradigms used by regulators and researchers
-New methods should be mathematically sound, involve relatively straight-forward computations and enhance the scientific validity of the current RB assessment
-multiple RBA techniques may be developed to triangulate the risk benefit profile of a product


Points to Consider

-Deploy RBA as a continuous feedback cycle starting early in clinical development
-engage stakeholders to better understand and participate in RBA
-focus on the patient perspective when appraising risk-benefit