Beyond Efficacy: Hard Outcomes in Treatment Studies Guy Goodwin, - PowerPoint PPT Presentation

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Beyond Efficacy: Hard Outcomes in Treatment Studies Guy Goodwin, University of Oxford, UK ISCTM ~ ECNP Joint Conference 1 September 2017 Paris France Is there a problem? Efficacy and safety Effectiveness, safety (and established

Beyond Efficacy: Hard Outcomes in Treatment Studies Guy Goodwin, University of Oxford, UK ISCTM ~ ECNP Joint Conference ▪ 1 September 2017 ▪ Paris ▪ France
Is there a problem? Efficacy and safety Effectiveness, safety (and established reimbursemen) Well validated scales Hard outcomes Randomized clinical trial (economic data) Placebo Non-random Short & long term data No placebo Key is long term
Randomized clinical trials Pro Contra Narrow inclusion and exclusion Highest level of evidence criteria limits generalisability Low risk for systematic bias Difficult to demonstrate effect Demonstrate “ efficacy ” of old drugs “Enriched design” benefits the study drug Do not show “ effectiveness ” in the real world Expensive Long-term trials might be unethical
The agenda for this session

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