Quiz 6

Question 1

Pharmacovigilance

Answer 1

continual monitoring for unwanted side effects and other safety-related aspects of marketed drugs

detection, evaluation, understanding, and prevention of adverse drug reactions

normally involves the collection of spontaneous reports of drug related morbidity and mortality

Question 2

How they conduct Pharmacovigilance?

Answer 2

use of observational studies conducted across different large databases, EMR’s

involves post marketing surveillance, signal detection, surveillance, data mining, social media

Question 3

Why do they conduct Pharmacovigilance?

Answer 3

premarketing studies exclude patients who have complicated comorbidities

premarketing studies only determine the efficacy of specific drug vs placebo

premarketing studies power is too weak to detect infrequent adverse events (low sample size)

Question 4

Examples of Programs

Answer 4

FDA Adverse Event Reporting System (FAERS)
- postmarketing adverse event reports received by FDA
- receives medwatch reports

FDA Sentinel System
- monitors safety of FDA regulated products
- submitted by clinical, academic, claims, etc

FDA VARES (vaccine adverse event reporting system)

Question 5

Comparative Effectiveness Research

Answer 5

conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat, and monitor health conditions in the REAL WORLD

aim to assist consumers, clinicians, purchasers, and policy makers to make informed decisions to improve both individual and population outcomes

Question 6

Types of CER

Answer 6

RCT with active treatment arms
observational studies
patient-centered health research

Question 7

Efficacy vs Effectiveness

Answer 7

Efficacy: whether a drug has the ability to bring about a given intended effect in controlled settings

Effectiveness: whether, in real-world patients and settings, a treatment can in fact achieve its desired effect

Question 8

Goals of CER

Answer 8

inform decisions on interventions or approaches to healthcare in real-world settings with regard to intended and unintended outcomes relevant to patients

put new treatment into perspective in relation to older treatment

identify patients who are more or less likely to respond to a given intervention

overcome external validity associated with RCT

Question 9

Pragmatic Research

Answer 9

studies (using randomization) that tests practical changes that could bring about big impact within health outcomes (randomization in real-world)

hybrid between RCT and routine care

Question 10

Why use pragmatic research?

Answer 10

include real world patients with diverse backgrounds
include providers from diverse settings
comparators are those actually used in practice
outcomes matter to clinicians and patients

Question 11

RCT vs Pragmatic

Answer 11

Efficacy vs effectiveness

highly controlled vs normal practice

highly selected patients excluding subjects with confounding characteristics vs little selection beyond indication of interest

intervention is strictly enforced with adherence vs more flexible real world

placebo vs current standard of care

high internal validity vs high external validity

short term surrogate/composite/intermediate endpoints vs real world relevant outcomes

limited generalization and impact on practice vs direct relevance to practice