| Number | Session | Link (if available) |
|
|---|---|---|---|
| 1 | Regulatory and HTA update | x | |
| 2 | Synthetic and other baseline covariates: The promise of smaller and faster clinical trials through prognostic digital twins | x | |
| 3 | Complex innovative trials for regulatory decision-making | x | |
| 4 | How to get most out of Scientific Advice | x | |
| 5 | Use of non-RCT studies in regulatory decision-making | x | |
| 6 | Short Topics | x |
Recordings
Recordings of those sessions where all presenters have given consent (Sessions 5 might be added later, pending speaker consent):
Material
Opening
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Justine Rochon (Boehringer Ingelheim, EFSPI president) | Opening remarks | x |
Session 1: Regulatory and HTA update
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Kristin Karlsson (Swedish Medical Products Agency, Uppsala University, EMA, Vice-chair MWP) | EMA Methodology Working Party update | x |
| 2 | Eftychia-Eirini Psarelli (University of Liverpool, EMA) | EMA update on submission of individual patient data from clinical trials | x |
| 3 | David McConnell (Statistician National Centre for Pharmacoeconomics, Ireland, Methodology Subgroup of HTA CG) | Update on joint HTA work under the EU HTA Regulation | x |
Short topics
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Fredrik Öhrn (Janssen) | Formal statistical requirements for a pivotal trial to support approval of a combination drug | x |
| 2 | Betty Molloy (Novartis) | Use of RWE in drug labelling | x |
| 3 | Patrick Schloemer (Bayer) | Hierarchical Composite Endpoints for Chronic Kidney Disease Trials | x |
| 4 | Simon Wandel (Novartis) | What’s the rule for the pool? | x |
| 5 | Sandro Gsteiger (Roche) and Anders Gorst-Rasmussen (Novo Nordisk on behalf of the PSI/EFSPI HTA Special Interest Group) | Surrogate endpoints - can EMA and EU HTA align on common standards? | x |
| 6 | Susan Robson (Roche) | Data Quality issues in IITs | x |
Session 2: Synthetic and other baseline covariates: The promise of smaller and faster clinical trials through prognostic digital twins
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Andreas Brandt (BfArM, EMA) | Covariate Adjustment: Traditional principles and challenges by new approaches | x |
| 2 | Courtney Schiffman (Genentech Roche) | Strategic considerations and value of covariate adjustment | x |
| 3 | Daniel Rubin (FDA) | Adjusting for covariates in randomized clinical trials for drugs and biological products | x |
Session 3: Complex innovative trials for regulatory decision-making
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Khadija Rantell (MHRA), Frank Bretz (Novartis), Hans Ulrich Burger (Roche) | Some guidance on when applying adaptive designs | x |
| 2 | Wolfgang Jacquet (Vrije Universiteit Brussel, EMA) and Benjamin Hofner (Paul-Ehrlich Institut, EMA) | Platform trials in a confirmatory regulatory setting – generating thoughts and directions | x |
| 3 | Dieter Haering (Novartis) | Experience with and learnings from regulatory interactions around innovative trial designs based on the NEOS study | x |
Session 4: How to get most out of Scientific Advice
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Tommi Nurminen (fimea, EMA) and Antero Kallio (fimea, EMA) | How to efficiently leverage scientific advice - a joint statistical and clinical perspective | x |
| 2 | Gergő Merész (Co-Chair of the JSC Subgroup (HTA R)) | HTA Joint Scientific Consultation | x |
| 3 | David Wright (AstraZeneca) | Tips on a) what questions to ask regulators and HTAs, b) interpreting the answers given and c) what to do when different authorities give conflicting advice | x |
Session 5: Use of non-RCT studies in regulatory decision-making
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | Kit Roes (Radboud Universiteit, EMA) | Single Arm Trials: The EMA Reflection Paper | x |
| 2 | Mark Levenson (FDA) | United States FDA Real World Evidence Program: A Quick Tour | x |
| 3 | Pallavi Mishra-Kalyani (FDA) | Regulatory Issues with the Use of External Controls and the US FDA RWE Program | x |
| 4 | Emmanuel Zuber (Novartis) | External control for approval and labeling: Two case studies trying to figure out when it is worth it | x |
| 4 | Stefan Lange (IQWiG) | HTA view on the EMA draft reflection paper and the FDA guidance | x |
Posters
| Talk | Speaker and affiliation | Talk title | Download (if available) |
|---|---|---|---|
| 1 | EFSPI / PSI & ASA biopharmaceutical section oncology estimand working group (conditional marginal taskforce) | Conditional and Unconditional treatment effects in randomized clinical trials: trials: EstimandsEstimands, Estimation, and Interpretation, Estimation, and Interpretation | x |
| 2 | EFSPI / PSI Regulatory SIG | Regulatory ESIG | x |
| 3 | EFPIA / EFSPI estimand implementation working group | How the “Estimand Implementation Working Group” brings together statisticians and clinicians to support the estimand journeystatisticians and clinicians to support the estimand journey | x |
| 4 | EFSPI / PSI biomarker SIG | The Biomarkers European Special Interest Group: European Special Interest Group: Where are we now?Where are we now? | x |
| 5 | EFSPI / PSI historical data SIG | Beyond the classical type I error: Bayesian metrics for Bayesian designs using informative priors | x |
| 6 | EFSPI / PSI Launch & Lifecycle Special Interest Group | EFSPI / PSI Launch & Lifecycle Special Interest Group | x |
| 7 | EFSPI / PSI HTA SIG | EU HTA – How can statisticians help navigate the problem of multiplicity? | x |
| 8 | EFSPI / PSI HTA SIG | What’s the HTA ESIG doing to prepare for EU HTA? | x |
| 9 | BBS Next Generation | Summary of BBS Next Generation Seminar: “Future of the Next Generation Statisticians and Quantitative Scientists” | x |