Pharmacoepidemiologic Assessment

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Pharmacoepidemiologic Assessment

• Judy A. Staffa, PhD, RPh
• Epidemiology Team Leader
• Division of Surveillance, Research
  Communication Support
• Office of Drug Safety, CDER

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When and why are pharmacoepidemiologic studies
recommended?

• To further characterize potential safety signals
  from controlled clinical trials
• chronic exposures
• patients with co-morbid conditions
• To further evaluate and quantify a safety signal
  identified after approval
• incidence rate vs. reporting rate

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Pharmacoepidemiologic Study Design

• Cohort
• Case-control
• Nested case-control
• Hybrid designs

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Minimum Requirements for a Good
Pharmacoepidemiologic Study Protocol

• Clearly specified study objectives
• Critical review of the literature
• Detailed research methods
• Study population
• Data sources
• Projected study size
• Methods for data collection/management/analysis

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Studies using Automated Data Factors
affecting Choice of Database

• Demographics of patients
• Turnover rate/mobility
• Plan coverage of study medications
• Size of population available for study
• Availability of outcomes of interest
• Coding of outcomes of interest
• Access to medical records

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Registries

• A systematic collection of defined events or
  product exposures in a defined patient population
  for a defined period of time

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Good Registry Protocols

• Objectives
• Literature review
• Detailed research methods
• Patient recruitment and follow up
• Projected sample size
• Methods for data collection, management and
  analysis
• Carefully designed data collection forms
• Validation of findings

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Surveys

• Further evaluate safety signals
• Assess knowledge of labeled events
• Assess product use
• Assess compliance with risk management programs1
• Address proprietary name confusion

1Concept Paper Risk Management Programs
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Good Survey Protocols

• Objectives
• Detailed research methods
• Patient or provider recruitment and follow up
• Projected sample size
• Methods for data collection, management and
  analysis
• Carefully designed survey instruments
• Validation of findings

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Reporting Safety Signals to FDA

• Submit a synthesis of all available safety
  information
• Provide an assessment of the risk/benefit profile
  of the product
• Propose steps to further investigate through
  additional studies
• Propose risk management programs as appropriate1

1Concept Paper Risk Management Programs
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FDAs Assessment of Safety Signals

• Magnitude of the signal
• Precision of the signal
• Consistency of findings across available data
  sources
• Biological plausibility
• Seriousness of the event
• Degree of benefit
• Availability of other therapies
• Mitigate events occurring in population(s)
  through risk management programs1

1Concept Paper Risk Management Programs
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Questions for Public Comment

• Under what circumstances would a registry be
  useful as a surveillance tool and when would it
  cease to be useful?

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Questions for Public Comment

• Under what circumstances would active
  surveillance strategies prove useful to identify
  as yet unreported adverse events?

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Questions for Public Comment

• Under what circumstances would additional
  pharmacoepidemiologic studies be useful?