| Number | Session | Link (if available) |
|
|---|---|---|---|
| 1 | Overture: Strategic Priorities in Pharmaceutical Statistics | x | |
| 2 | ICH E20 Guideline on Adaptive Designs for Clinical Trials – A Critical Discussion from Different Perspectives | x | |
| 3 | Anniversary session | x | |
| 4 | From Trials to Target Populations: Extending Evidence for Decision-Making | x | |
| 5 | From Black Box to Pandora’s Box: Navigating AI in Clinical Trials | x | |
| 6 | Characterizing the Effect of Treatment Using Hierarchical Composite Endpoints – Risks and Benefits of Win Statistics and Beyond | x | |
| 7 | Keeping it Real or Losing Control? Adventures in Target Trial Emulation | x | |
| 8 | The Added Value of Bayesian Methods for Pivotal Clinical Trials – Just a Communication Issue? | x | |
| 9 | How to Deal with Assumptions in Drug Development? What Insights Can We Gain from ICH M15? | x | |
| 10 | Short Topics | x |
Slidedecks and recordings will be continuously added as soon as we have them and the presenter’s consent.
Download all materials as .zip file
Recordings
Recordings of those sessions where all presenters have given consent:
Slidedecks and posters
Session 1: Overture: Strategic Priorities in Pharmaceutical Statistics
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Kit Roes (Chair of MWP EMA, Radboud UMC, NL) | Navigating priorities from regulatory perspective | x |
| 2 | Mouna Akacha (Co-chair EFSPI statistical methodology leaders, Novartis, CH) | Strategic priorities in pharmaceutical statistics – A quantitative drug developer’s perspective | x |
Session 2: ICH E20 Guideline on Adaptive Designs for Clinical Trials – A Critical Discussion from Different Perspectives
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Frank Pétavy (EMA, NL), Khadija Rantell (MHRA, UK) | Moving towards harmonisation for (confirmatory) trials with an adaptive design | x |
| 2 | Christopher Jennison (University of Bath, UK) | ICH E20 guideline on adaptive designs for clinical trials: my reflections as a statistician working in Academia | x |
| 3 | Silke Jörgens (J&J/University of Cologne, DE) | ICH E20 guideline on adaptive designs for clinical trials: my reflections as a statistician working in Industry | x |
| 4 | Maria Grünewald (MWP member, MPA, SE) | ICH E20 guideline on adaptive designs for clinical trials: my reflections as a statistician working in Regulatory assessment | x |
| 5 | Seamus Kent (ESHPM, NL) | ICH E20 guideline on adaptive designs for clinical trials: my reflections as a statistician working in HTA assessment | x |
Session 3: Anniversary session
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Emmanuel Zuber (cogitamen, CH), Hans Ulrich Burger (Medical University of Graz, DE), Egbert Biesheuvel (Viatris, NL) | 10th EFSPI regulatory statistics workshop | x |
| 2 | Julie Jones (Novartis), Andreas Brandt (BfARM), Elina Asikanius (fimea), Jenny Devenport (Roche) | Tackling the Hard Topics: Shaping What’s Next in Regulatory Science | x |
Session 4: From Trials to Target Populations: Extending Evidence for Decision-Making
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Seamus Kent (ESHPM, NL) | Intro | x |
| 2 | Antonio Remiro-Azócar (Novo Nordisk, ES) | Evidence synthesis: estimands, transportability and external validity | x |
| 3 | Miguel Hernán (Harvard T.H. Chan School of Public Health, US) | Emulation of target trials using observational data: because randomized trials cannot possibly answer all causal questions | x |
Short topics: Present a problem on 2-3 slides and receive input from a panel of regulators
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Gaelle Klingelschmitt, Jianmei Wang, Audrey Boruvka, Jenny Devenport (Roche, CH / UK / CA) | Surprises matter! Limiting broad disclosure of futility analysis criteria to preserve trial integrity | x |
| 2 | Rob Hemmings (Consilium, UK) | Accessing accumulating data in open-label studies | x |
| 3 | Franco Mendolia, Katja Brandau (Bayer, DE) | The future of per protocol set analyses in non-inferiority trials | x |
| 4 | Thomas Hoefelder, Beate Presser (Boehringer Ingelheim, DE) | How can we strengthen statisticians’ impact in CMC related ICH guidelines? - Special focus on ICH M13b concerning dissolution profiles | x |
| 5 | Bohdana Ratitch, Alfredo Farjat (Bayer, CA/NL) | Acceptability of prognostic covariate adjustment and Targeted Maximum Likelihood Estimation (TMLE) methods for the primary and key analyses of pivotal clinical trials in absence of prior clinical knowledge about strong predictive factors | x |
Session 6: Characterizing the Effect of Treatment Using Hierarchical Composite Endpoints – Risks and Benefits of Win Statistics and Beyond
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Henrik F. Thomsen (Novo Nordisk, DK), Mickaël De Backer (UCB, BE) | Hierarchical composite endpoints: more nuance, more insight and … more confusion? | x |
| 2 | Lukas Aguirre Dávila (alternate SAWP member, PEI, DE) | Hierarchical composite endpoints – time to untie the not’s? | x |
Session 7: Keeping it Real or Losing Control? Adventures in Target Trial Emulation
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Olaf Klungel (MWP member, University of Utrecht, NL) | Bridging the Target Trial Emulation Framework and the Estimand Framework | x |
| 2 | Rima Izem (Novartis, CH) | Target Trial Emulation meets clinical trial design: two case studies | x |
| 3 | Angelika Geroldinger (AGES, AT) | From a regulatory perspective: what can we gain from TTE? | x |
Session 8: The Added Value of Bayesian Methods for Pivotal Clinical Trials – Just a Communication Issue?
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Katharina Hees and Florian Krach (PEI, DE) | Bayesian approaches in clinical trials: a discussion on regulatory expectations | x |
| 2 | Nicky Best (GSK, UK) | Communicating the value of Bayesian approaches in clinical trials: is it just a prior issue? | x |
Session 9: How to Deal with Assumptions in Drug Development? What Insights Can We Gain from ICH M15?
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Flora Musuamba Tshinanu (SAWP & MWP member, University of Namur, BE) | From risk assessment to optimal regulatory decision: first having the question right | x |
| 2 | Oliver Sailer (Boehringer Ingelheim, DE) | Bridging disciplines with ICH M15: a case study on assumption testing for pharmacometric-enhanced Bayesian borrowing | x |
| 3 | Tobias Mielke (J&J, DE) | ICH-M15 to support credibility assessment for Bayesian modelling | x |
Posters
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Sanne Roels, Alex Ocampo, Kelly Van Lancker (EFSPI Working Group Causal Inference SIG) | Causal Inference Special Interest Group | |
| 2 | Beate Presser, Christian Schmid, Jens Lamerz, Kevin Lief, Martin Motava (EFSPI Working Group CMC Statistical Network EU SIG) | Introduction to CMC statistical network Europe and areas for priority | x |
| 3 | Jonas Häggström (EFSPI/EFPIA EIWG with phuse sub-team) | EFSPI Scientific & Training Academy – Past and Present | |
| 4 | Armin Schüler, David Wright, Amel Besseghir, Khadija Rantell, Andreas Sashegyi, Katarina Hedman, George Kordzakhia, Mike Colopy, Liangcai Zhang (EFSPI statistical method leaders) | Estimands in safety analytics | x |
| 5 | Mouna Akacha, Kaspar Rufibach (EFSPI statistical methodology leaders) | Statistical methodology leaders in drug development, an EFSPI group | x |
| 6 | Frank Pétavy, Eftychia Eirini Psarelli, Marie Annie Orre (HMA-EMA Network) | Clinical study data submission in Europe: An EMA-CHMP proof-of-concept pilot | x |
| 7 | Oliver Sailer, Monika Jelizarow (Historical Data SIG) | Historical Data: A PSI/EFSPI Special Interest Group | x |
| 8 | Maximo Carreras, Chrissie Fletcher (HTA SIG) | Adaptive Clinical Trials – what are the challenges and opportunities from an HTA perspective? | x |
| 9 | Birte Geusens, Rachid Massaad, Anders Gorst-Rasmussen, Fred Sorenson, Shahrul Mt-Isa (HTA SIG) | Characterizing the effect of treatment using hierarchical composite endpoints – risks and benefits of win statistics and beyond in the HTA context | x |
| 10 | Katrin Kupas, Min-Hua Jen, Orlando Dohring, Rima Izem (HTA SIG / RWE SIG) | Target trial emulation and incorporation of observational data into clinical trials from an HTA perspective | x |
| 11 | Ash Bullement, Orlando Dohring, Peter Pemberton-Ross, Grammati Sarri, Katrin Kupas (HTA SIG) | From trials to target populations: extending and extrapolating evidence for HTA decision-making | x |
| 12 | Lada Mitchell, Cornelia Dunger-Baldauf, Jenny Devenport, Yulia Dyachkova (Launch & Lifecycle SIG) | Updates from the Launch & Lifecycle SIG | x |
| 13 | Audrey Yeo, Daniel Sabanés Bové, Alessandro Gasparini, Nils Penard (openstatsware / Software Engineering SIG) | openstatsguide: checklist for good statistical software packages | x |
| 14 | Josephine Wolfram, Rima Izem, Elizabeth Merrall, Helen Broadhurst (Real World Data SIG) | Welcome to the Real World Data SIG! | |
| 15 | Christoph Gerlinger (Regulatory SIG) | EFSPI/PSI regulatory European Special Interest Group | x |
| 16 | Giles Partington, Maeva Dupuis, Aysun Cetinyurek Yavuz (Small populations SIG) | The PSI/EFSPI small population Special Interest Group | x |
| 17 | Bjoern Bornkamp, Kostas Sechidis, David Svensson, Ashwini Venkatasubramaniam (Treatment Effect Heterogeneity SIG) | Data-driven evaluation of treatment effect heterogeneity | x |