Transcript stemhagen_2

Developing, Pilot Testing & Evaluating
RiskMAP Interventions
Annette Stemhagen, DrPH, FISPE
Vice President
UBC Epidemiology & Risk Management
The FDA Regulatory and Compliance Symposium
August 25, 2005
Risk Minimization (RiskMAP) Guidance
• Goal: minimize a product’s risks while
preserving its benefits.
• For the majority of products, routine risk
minimization measures are sufficient
• RiskMAP -- strategic safety program
minimizing known risks of a product while
preserving its benefits.
• A RiskMAP uses tools to achieve goals.
Development and Use of Risk Minimization Action Plans (RiskMAPs)
http://www.fda.gov/cder/guidance/6358fnl.htm
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How to Design a Risk Minimization Program
• Complete risk and benefit assessment
• Define goals and objectives for risk
minimization intervention
• Identify appropriate “tools”
• Conduct feasibility
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Risk and Benefit Assessment
• Quantify risks, if possible
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High risk populations?
Specific doses?
Inappropriate use?
Off-label use?
Misuse or abuse?
• Evaluate risk in context of benefit
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Goals and Objectives for Risk Minimization
• Ideal Goals
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No deaths
No abuse
No pregnancies
No patients taking drug X also take Drug Y
• Specify objectives
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Inform all health care professionals
Inform all patients
Increase knowledge of appropriate use
Require pregnancy test before each prescription fill
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RiskMAP “Tool” Selection
• Maintain widest accessibility with least
burden to health care system
• Target key groups most likely to be
able to minimize risk
• Determine the feasibility of proposed
approach
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Categories of RiskMAP Tools
Targeted Education and Outreach
Increase appropriate knowledge and behaviors of
stakeholders who have the capacity to prevent or
mitigate risks
Reminder Systems
Systems that prompt, remind or guide in
prescribing, dispensing, or using a product to
minimize risk
Performance-Linked Access Systems
Link product access to laboratory testing or other
documentation.
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RiskMAP “Tools”
• Targeted education and outreach
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Health care provider letters
Training programs
Continuing education
Patient package inserts
Medication Guides
Promotional techniques
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RiskMAP “Tools”
• Reminder systems, processes or forms
– Patient agreement or acknowledgement forms
– Health care practitioner certification or
attestation programs
– Limited Rx amount or refills
– Specialized packaging to enhance safety
– Prescription stickers
– Special prescription pads
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RiskMAP “Tools”
• “Performance-linked access systems” that
guide prescribing, dispensing and use
– Compulsory reminder systems
– Certification of prescribers/dispensers
– Documentation of safe-use conditions
• blood tests for agranulocytosis
• pregnancy tests
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How Can You Determine if Your Approach Will Succeed?
• Feasibility assessments prior to designing
intervention
• Pilot testing prior to full implementation, if
feasible
• Ongoing evaluation over the life of the
program
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Initial Feasibility Assessments
• Determine likelihood of success with
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Physicians
Pharmacists
Patients and caregivers
Wholesalers/distributors
Others
– Program Design
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Fit busy practice routine?
Represent actual clinical practice?
Need no extraordinary testing?
Requires minimal training?
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Assessing Feasibility Using Existing Data
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Medical chart review
Natural history of disease
Cross sectional surveys, e.g., NCHS
Registries
Large automated databases
Market research data
– Competitive products
– Physician prescribing behavior
• ISS and ISE (particularly placebo groups)
• Literature
• Past experience
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Ad Hoc Data Collection for Assessing Feasibility
• Panel of experts
• Focus groups
• Stakeholder surveys
– Health Care Providers
– Patients
– General population sample
• Time and Motion studies
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Ongoing Feasibility Throughout the Program
• Conduct Failure Mode and Effects
Analysis (FMEA)
• Test your assumptions
– How good were the data?
– How good were the assumptions?
• Projected vs. actual
• Implement mid-course corrections
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Pilot Test “Tools”
• Qualitative and quantitative testing of
educational materials
– Health literacy
– Comprehension
• Randomized trial of educational programs
• User acceptance testing of systems
• Phased roll out of program to small group of
physicians prior to widespread
implementation
– Registry
– Large streamlined study
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RiskMAP Evaluation
• Continually assess & modify
• Use evidence based performance
measures
– At least two different approaches
– Quantitative, representative, minimally biased
• Measure health outcome goal
– Surrogates
– Process measures
– KAB
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Evaluation Strategies
• Root Cause Analysis
• Spontaneous AE report monitoring
(Medication errors)
• Record review
• Stakeholder surveys
– Knowledge, Attitude and Behavior
• Program audits
• System audit trail
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Evaluation Strategies
• Large automated database study
• National surveys & databases,
e.g., DAWN, TESS
• Market research data, e.g., IMS-Health
• Registries
– Product exposure registry
– Pregnancy registry
• Large streamlined study
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What Works in Risk Minimization?
• Really don’t have enough history to know
• Involves changing behaviors and/or
systems—education? marketing?
• Limited/controlled distribution works
– “No blood, no drug”
• Attestation, testing, limiting access in other
ways
– Lotronex®
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What Doesn’t Work in Risk Minimization?
• Severe restriction of use via complex program
– Dofetilide®
– Plenaxis®
• Unintended consequences
• Unrealistic study size or expectations
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