Announcement BBS/EFSPI seminar Small populations and level of evidence
The seminar takes place on June 27, 2018 from 9:00am to 5:00pm
Venue: Idorsia Pharmaceuticals, Hegenheimermattweg 91, Allschwil, Switzerland
TO REGISTER PLEASE GO TO: www.efspi.org
Introduction, European Statistical Meeting onSmall populations and level of evidence
Daniel O’Connor, Rare diseases and orphan drugs: A Regulator’s (clinical)Perspectives
Stephen Senn, In search of the lost loss function
Henriette Thole, The potential and challenges of registry use when generating evidence in small populations
Anja Schiel, Rare diseases and orphan drugs:The HTA perspective
Adele Morganti, Borrowing external controls for an event-driven pediatric trial in PAH: a case study
Andreas Kaiser, Bayesian analysis for small sample size trials using informative priors derived from historical data
Elina Asikanius, Using a non-interventional study to strengthen the evidence collected in a Phase III program: a Hemophilia A case Study
Hans Hockey & Kristian Brock, Hockey sticks and broken sticks – a design for a single-treatment, placebo-controlled, double-blind, randomized clinical trial suitable for chronic diseases
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