Observational Studies:Strategic, Operational and Budgetary Considerations
11 January 2011
Jeff Trotter – Executive Vice President – Phase IV Development
Post-approval research today – Safety and value► Requirement
► In some countries, ‘real world’ post-approval experience data must be submitted to maintain market approval.
► Increasingly, some form of safety surveillance / risk management program will be mandated and enforced.
► Responsibility
► Corporate accountability for post-approval safety is increasingly expected by various constituencies.
► Documentation of clinical / economic / humanistic value is critical for commercial acceptance and accelerated product uptake.
► Opportunity
► If managed proactively, safety surveillance obligation can be controlled.
► An observational study can be a cost-effective, high ROI mechanism for fulfilling the post-approval obligation for both safety and effectiveness data.
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Real world perspectives
Real world research
“The conditions under which products are examined for regulatory approval are generally not the conditions under which they are actually used…”
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► …RCTs can be too artificial in intent and design, and therefore poorly reflective of actual medical practice
► Tight inclusion criteria
► Experimental protocol
► Tight procedural control
► Randomization, blinding, placebo, etc.
► Short in duration
► Homogeneous sites
► We need to know how a product is used and how it “performs” under real world conditions
► Safety
► Clinical outcomes (CER)
► Economic value
► Humanistic value
Real world (observational) studies are needed, because…
Operational Issues and Challenges
►Site selection
►Site training and start-up
►Site “interaction” (monitoring) and management
► Site motivation
► Protocol “adherence”
> Inclusion
> Procedures
►Data management► Accommodating multiple
measures
► EDC issues
► Data quality> SDV
►Analysis► Biases, etc.
► Findings
► Reporting (communications
Observational Studies are a “different animal”
So, who “owns” it…?
► HEOR
► Epidemiology
► Medical Affairs
► Marketing / Product Management
► Clinical Operations
► Development
► Safety / PV
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CRO
Proposal
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Product Management, HEOR
Clin OpsRFP/Bid Grid
Outsourcing
Highlights from survey on Observational Research ► Motivation: “Schizophrenic” RFPs
► i.e., uncertainty, inconsistency, imprecision, over-engineering, etc.
► Many functional areas have some involvement in observational research studies
► Many different purposes underlie these studies
► “Observational research” goes by many names
► Sponsors have varying levels of “comfort” with observational research
► Most sponsors do not have defined processes for observational studies
► Design, Procurement, Operational, Analytical, etc.
► Sponsors have varying expectations for the “conclusiveness” of findings from observational studies
► Sponsors are concerned that regulatory/health authorities “don’t get it”
► Sponsors plan to become increasingly involved in observational research
Observational Research: Budgetary Implications
► Observational Research is a different animal
► Strategic objectives
► Activities, resources, units
► Process
► Applying “traditional” processes to an Observational Study leads to…
► Over-engineered, overly expensive study
► Study not properly designed
► Project not undertaken
► Need for clarity and flexibility
► Observational Research may be outside traditional comfort zones
► Specialized skills are required
► Observational Studies can be very cost-efficient
► Balance objectives, analytical granularity, data management, etc.