4th EFSPI Workshop on Regulatory Statistics


23rd - 24th September 2019


Oekolampad Church Allschwilerplatz 22 CH – 4055 Basel Switzerland

After three very successful workshops on regulatory statistics in the past three years, EFSPI is pleased to announce the 4th regulatory statistics workshop taking place in Basel on 23rd and 24th September 2019

For the event program please
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Real World Data, Real World Evidence, Big Data
BBS spring seminar on synthetic controls: summary & follow-up
Development of a digital endpoint in Multiple Sclerosis -challenges and opportunities
A look on Best Practices in Pragmatic Trials
One single arm study plus a historic comparator equals two historic regulatory approvals
Development of a smartphone based monitoring tool for people with Multiple Sclerosis Challenges and Opportunities
Using Real-World Data to Extrapolate Evidence from Randomized Controlled Trials
Use of RWD in gene - therapy approvals
Use of historical data to support gene-therapy approval: example from Kymriah
How far can we trust “Real World Data”?
Comparison of statistical methods to analyse safety data
Pooling and harmonizing of safety data for a robust statistical analysis
A shiny app to explore clinical data
Matching up
How statisticians deal with the difference between efficacy and safety reporting
How the estimand framework becomes standard practice in applications, and where we still need to learn
Treatment policy and hypothetical strategies for intercurrent events in chronic pain and Parkinson’s disease
Estimand framework: opportunity to rethink some old (and new) problems in Oncology trials?
Regulatory experience with the Estimand framework
Observational vs. randomized analysesof digoxin-mortality in the DIG trial
Goal Attainment Scaling: Validation & use for rare disease
A regulator’s view on rare cancer drug development: Histology independent indications
Masking of open label studies
To blind or not to blind? FDA guidance on placebos and blinding in oncology clinical trials
Underpowered RCTs and external data
EMA (2018) Q&A on Mahalanobisdistance (MD) to assess drug dissolution profiles