Benefit-Risk Assessment Flashcards
7 cards from real PVC practice questions. Tap to flip, then mark Knew It or Still Learning — missed cards come back until you master them.
Read the first 7 Benefit-Risk Assessment flashcards as text
When a drug's serious adverse event rate is 1 in 10,000 but the disease it treats kills 1 in 100 untreated patients, this situation BEST illustrates:
Answer: A favorable benefit-risk profile where the mortality reduction substantially outweighs the rare serious adverse event
When the risk of harm (1/10,000) is orders of magnitude lower than the risk of the disease outcome (1/100), the benefit-risk balance strongly favors the drug.
In pharmacovigilance, 'framing' in benefit-risk assessment refers to:
Answer: How the problem, context, and objectives are defined, which can influence the assessment outcome
Framing defines the scope, perspective, and criteria of the assessment; poor framing can introduce bias by omitting relevant outcomes or comparators.
A benefit-risk assessment for a pediatric oncology drug concludes that severe but manageable toxicities are acceptable. Which principle MOST supports this conclusion?
Answer: The principle of beneficence applied to a life-threatening condition with no alternative
In life-threatening pediatric cancer with no alternatives, the principle of beneficence—acting in the patient's best interest by providing meaningful benefit—justifies accepting significant toxicities.
Which signal type emerging during post-marketing surveillance would MOST urgently trigger a formal benefit-risk reassessment?
Answer: A previously unrecognized fatal adverse reaction in a non-serious indication
A new fatal adverse reaction in a non-serious indication dramatically alters the benefit-risk balance and requires immediate formal reassessment.
Under ICH E2C(R2), the Periodic Benefit-Risk Evaluation Report (PBRER) must include which of the following?
Answer: An integrated benefit-risk evaluation summarizing new findings and their impact on the overall benefit-risk balance
ICH E2C(R2) requires the PBRER to include an integrated benefit-risk evaluation that synthesizes all cumulative data and assesses the current benefit-risk profile.
Patient preference studies are increasingly incorporated into benefit-risk assessments because they:
Answer: Provide quantitative data on how patients weigh benefits versus risks, reflecting real-world values
Patient preference studies use methods like discrete choice experiments to quantify how patients trade off benefits and risks, ensuring their values inform regulatory decisions.
If a drug's benefit-risk balance is considered favorable only when used with a specific diagnostic test to identify appropriate patients, this is an example of:
Answer: A companion diagnostic-based restricted use strategy to optimize benefit-risk
When a diagnostic test identifies the patients who benefit most (or are at lowest risk), restricting use to test-positive patients is a benefit-risk optimization strategy.