1. Community-Based and Cluster-Randomized Studies –‘Pragmatic’ Approaches for Life Cycle Evidence?
Florian Eichmann, PhD Principal Scientific Affairs and Real World Evidence, Late Stage inVentiv Health

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4. Community-Based and Cluster-Randomized Studies –‘Pragmatic’ Approaches for Life Cycle Evidence?
RCT- Expl. Trial
….a third way….?
Non-Interv. Study
Protocol driven
Protocol AND usual care
Usual Care
Scheduled visits
Observed visits
Case Report Form (CRF)
CRFs
Data Collection Form (DCF)
“Efficacy”
Effectiveness
“Effectiveness”
Blinded unless not feasible
Usually unblinded
Unblinded
Follow-up for proxy endpoints
Real life endpoint follow-up
Follow-up for real life endpoints
Homogeneous ‘ideal’ patients
Heterogenous ‘real’ patients
Heterogeneous 'real' patients
‘Experienced’ investigators
Everyday practicioners
‘Everyday’ practitioners
‘Motivated’ patients
Usual patients
’Usual' patients / subjects
Pharmacological differences (Clinical Research)
‘Pharmacological’ AND ‘Management’ differences
’Management’ differences (Health Care Research)
Can it work…if given?
Does it work...better?
Does it work … if offered?
Internal validity focus
Internal AND external validity
External validity focus
All phases, focus on II, III
Usually Late Stage (disease observation)
CAUSALITY—Compare two or more groups (control heterogeneity)
CAUSALITY—Compare two or more groups
ASSOCIATION—matching, multiple regression, propensity score, instrumental variables ..
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5. Community-Based and Cluster-Randomized Studies –‘Pragmatic’ Approaches for Life Cycle Evidence?
Increasing discussion on pragmatic study types
But do we also see more of these studies?
Medline search
Qualitative review
Results
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6. Annual Medline Publication Title Hits
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7. Medicinal Product Studies among Medline Title Hits
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8. Medicinal Product Studies – Qualitative
Study Design N
Recent Medicinal Product Title Hit Studies
Pragmatic 5
asthma, skin infection, pneumonia, rheumatoid arthritis, osteoarthritis
Community-based 3
pneumonia, myeloma, colorectal cancer
Two studies used subject based randomization,
One study was observational.
Cluster-randomized 1
vaccines
All studies studies analyzed
medicinal products with regard to
clinical and/or patient reported and economic outcomes in
real life situations versus
standard therapy or an active comparator.
No reference to life cycle evidence
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9. Conclusions & Outlook
Growing numbers of cluster-randomized and pragmatic studies
Few studies on medicinal products
Various indications
Many studies on public health or non-medical or complementary medicine
Practical use for life cycle evidence is still limited
Most studies still use non-interventional designs
Strategic, financial, regulatory, and educational reasons
Modifications e.g. “stepped wedge” designs to overcome hurdles
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10. Thanks for your attention!
Poster Nr. 3
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