Past events: agendas, slidedecks, recordings

Published

November 25, 2022

15.07.2022: On market approval and market access: Breaking the linear thinking or how to innovate in a crowded space?

Agenda

Recording

Speaker Institution Title Download slides
Frank Bretz Novartis Welcome link
Anja Schiel (starts at 00:04:00) NOMA & EMA Start thinking HTA early link
Nigel Yateman (01:01:15) Novartis Challenges in developing next generation CAR-T link
Iain Bennett (01:22:30) Roche No comparators no problem? link
Karen Facey (01:42:50) University of Edinburgh and self-employed Comments to introduce panel discussion link
Speakers + Gorana Capkun, Lilla di Scala, Andrew Thomson (01:58:15) Panel discussion No presentation available

23.06.2022: BBS / EFSPI Seminar Registry studies and HTA

Agenda

Recording

Speaker Institution Title Download slides
Fred Sorenson Xcenda, Switzerland Welcome No presentation available
Eric Faulkner and Omar Dabbous Novartis Gene Therapies, USA An open dialog on the issues faced and lessons learned with respect to Novartis Gene Therapies’ Spinal Muscular Atrophy (SMA) registries No presentation available
Entela Xoxi Università Cattolica del Sacro Cuore, Italy AIFA registries: past and current & upcoming challenges link
Jinma Ren and Friedhelm Leverkus Pfizer, USA and Pfizer, Germany Issues in the design and analysis of registry-based studies for regulatory and HTA purposes link
Mei Yang Happy Life Technology, China & USA Registries in China: Guidelines, trends and new technologies link
Rossella Di Bidino Fondazione Policlinico Universitario Agostino Gemelli, Italy Hospital Perspective on using Registry Data for HTA link
Kirk Geale Quantify Research, Sweden Case studies using Registry Data for HTA in Scandinavia link
Kat Belendiuk Genentech, USA FlywheelMS Case Study - A complete web-based digital registry of 5,000 patients with Multiple Sclerosis in the USA No presentation available

24.05.2022: BBS Spring Seminar Transforming drug development and Assembly

Agenda

Recording

Recording 2

Speaker Institution Title Download slides
Uli Burger Roche, Basel Welcome No presentation available
Pierre Verweij, Guy Braunstein, Colin Terry, Pavi Rallapalli, Dominik Heinzmann, Lisa Hampson, Marc Vandemeulebroecke, Heinz Schmidli, Sebastian Weber, Laurent Essioux Idorsia Pharmaceuticals, Deloitte, Novo Nordisk, Novartis, Roche BBS Spring Seminar Transforming drug development and assembly link

29.03.2022: Graphical approaches to multiple test problems

Agenda

Recording

Recording 2

Speaker Institution Title Download slides
Marc Vandemeulebroecke Novartis, Basel Welcome No presentation available
Ekkehard Glimm, Frank Bretz & Dong Xi Novartis & Gilead Graphical approaches to multiple test problems link

21.02.2022: Machine Learning in clinical drug development

Agenda

Recording

Speaker Institution Title Download slides
Markus Lange, Lorenz Uhlmann Novartis, Basel Machine Learning in clinical drug development link
Markus Lange, Lorenz Uhlmann Novartis, Basel Questions from the chat, with answers link
Markus Lange, Lorenz Uhlmann Novartis, Basel R exercises link
Markus Lange, Lorenz Uhlmann Novartis, Basel Dataset used for exercises link

27.07.2021: Joint BBS/BES Webinar COVID-19 pandemic and how we deal with data

Agenda

Recording

Speaker Institution Title Download slides
Hans Ulrich Burger Roche, Basel Welcome No presentation available
Tim Friede University Medical Center Göttingen Data and Statistics as a basis for decision making: A discussion of the Corona pandemic link
Thorsten Lehr Saarland University Forecasting: What do we learn? link
Thierry Van Effelterre J&J Vaccination trials: What do we know about long-term efficacy measures and other questions link
Fei Chen Janssen Statistical Considerations Underlying a COVID-19 Vaccine phase 3 design link

28.06.2021: Joint EFSPI/BBS Meeting Precision & Innovative Medicine and Health Technology Assessment

Agenda

Recording

Speaker Institution Title Download slides
Fred Sorensen Xcenda Welcome link
Dan O’Connor MHRA, UK The UK’s Innovative Licensing and Access Pathway (ILAP) for medicines – A joint MHRA, NICE & SMC initiative link
Karen Facey University of Edinburgh IMPACT HTA - Recommendations for Developing Rare Disease Treatments link
Paul Cislo, Jinma Ren and Joseph C. Cappelleri Pfizer Adjusting Global Survival to Make Results More Relevant and Generalizable to Local Markets link
Marc Buyse IDDI and University of Hasselt, Belgium Net benefit and correlation between benefit and harms link
Mark Belger and Marie-Ange Paget Eli Lilly UK & France Closing the efficacy to effectiveness gap: Generalizing from RCTs to real world populations link
Kirsten Herrmann Exact Sciences, Germany Assessments and reimbursement of gene expression signature tests in Europe link
Janneke Boersma Roche, Netherlands Bridging the gap between Regulatory & HTA approval for Precision Medicine therapies: a case study from the Netherlands link

16.06.2021: BBS Webinar Impact of the COVID-19 Pandemic on Clinical Trials

Agenda

Recording

Speaker Institution Title Download slides
Anja Schiel Chair Scientific Advice Working Party, EMA Overview on early experience of the impact of Covid-19 pandemic on clinical trials link
Paul Delmar Roche, Basel COVID-19 pandemics : Impact on Clinical Trials in a chronic progressing disease link
Eva Hua Novartis, Basel Hypothetical strategy for a case study affected by COVID-19 pandemic link
Kelly Van Lancker Ghent University The hypothetical estimand and its potential estimators in clinical trials impacted by COVID-19 link

22.03.2021: BBS Webinar Statistical challenges in the clinical development of CAR T-cell therapies

Agenda

Recording

Speaker Institution Title Download slides
Roland Marion-Gallois BMS, Switzerland Welcome and Introduction link
Caroline Aarber Bath CHUV, Switzerland Introduction to Cell Therapies: A Clinical Perspective link
Elina Asikanius FIMEA, Finland Perspective from EMA link
Zhenzhen Xu FDA, USA Perspective from FDA link
Khadija Rantell MHRA, UK Phase 2 / Phase 3, Treatment effect or therapeutic strategy effect? link
Alessandro Previtali BMS, Switzerland Phase 2 / Phase 3, Estimands in the context of cell therapy development link
Nigel Yateman Novartis, Switzerland Challenges for new CAR-T therapies link
Oriana Ciani Università Bocconi, Italy Introduction on Post-Approval challenges, reimbursement and HTA assessments link
Andrea Smith G-BA, Germany HTA-Perspective on the assessment of CAR-T-Cell Therapies link
Marcelo Pasquini and Zhenuan Hu CIBMTR, USA CAR-T cell therapy registries link

08.03.2021: Graphics for decision-making in biomedical research and drug development

Agenda

Recording

Speaker Institution Title Download slides
Dominik Heinzmann Roche, Basel Welcome No presentation available
Hannah Diehl and Tamara Broderick / Andy Stein and Niladri Roy Chowdhury MIT and Novartis The “See”-Value App: Visual Decision Making for Drug Development link
Neil McQuarrie Flatiron Better understanding and reacting on impact of COVID-19 on RWD collection by combining statistics with visualization link
Tadeusz Lewandowski Roche, Basel Interactive clinical study visualisation in enabling the faster decision making link
Markus Lange Novartis, Basel Unraveling a single number – using graphics to explain Probability of Success link
Anne-Marie Meyer Roche, Basel Population Level Analytics for pandemic response: Predicting vaccine uptake and vaccine hesitancy link
Giusi Moffa University of Basel Closure No presentation available

03.11.2020: Joint EFSPI / BBS Webinar: The application of estimands from a Neuroscience perspective

Agenda

Recording

Speaker Institution Title Download slides
Hans Ulrich Burger Roche, Basel Welcome and introduction to the estimand framework link
Nikolaos Sfikas Novartis, Basel Outline of an estimand strategy in MS link
Mette Krog Josiassen and Peter Quarg Lundbeck and Novartis Outline of an estimand proposal in migraine prevention and neuropathic pain link
Paul Delmar Roche, Basel Using the Estimand Framework to address challenges in AD clinical trial with a closer look at the hypothetical strategy link
Carrie Li Roche, Basel Estimands in Huntington’s disease link
Andrew Hartley PPDI Impact of Covid-19 on studies in Neuroscience link
Joel Raffel and Khadija Rantell MHRA, UK Regulatory aspects of the estimand framework: Clinical and statistical perspectives link
Anja Schiel Chair Scientific Advice Working Party, EMA Panel discussion including all speakers link

07.09.2020: BBS Webinar RCTs meeting causal inference: principal stratum strategy and beyond

Agenda

Recording

Speaker Institution Title Download slides
Kaspar Rufibach Roche, Basel Welcome and scene setting link
Vanessa Didelez Keynote speaker, Leibniz Institute for Prevention Research and Epidemiology, BIPS, Bremen Time-Varying Treatments in Observational Studies: Lessons for Clinical Trials link
Jack Bowden University of Exeter Connecting Instrumental Variable methods for causal inference to the Estimand Framework link
Kelly van Lancker Ghent University Efficient, doubly robust estimation of the effect of dose switching for switchers in a randomised clinical trial link
Björn Bornkamp Novartis, Basel Principal Stratum Strategy: Potential Role in Drug Development link
Dominik Heinzmann Roche, Basel Principal stratum strategy to investigate anti-drug antibody impact on cancer immunotherapy outcome link
Aiesha Zia Novartis, Basel Exploring estimation approaches for principal stratum estimands in Phase III randomized trials in CAR-T anti-cancer therapy link
Fabrizia Mealli University of Florence The ICH E9 addendum from an academic causal inference perspective and feedback on the previous talks link
Giusi Moffa University of Basel Next webinars and closure link
All Question and Answers link

30.06.2020: BBS/EFSPI Webinar on HTA

Agenda

Recording

Speaker Institution Title Download slides
Chrissie Fletcher GSK, UK Celebrating 10 Years HTA SIG link
Christoph Schürmann IQWiG, Germany Health-related quality of life endpoints in benefit assessments: Demands and challenges as seen by IQWiG in Germany link
Lara Wolfson Merck, USA Issues related to transparency with HTA dossiers link
Mark Nuijten A2M and Univ. of Maastricht, NL & ITU, Turkey An innovative pricing model to assess the price of expensive drugs with an orphan indication link
Fred Sorenson Xcenda Introduction to moderated panel discussion link

29.06.2020: Joint EFSPI / BBS Webinar: Estimands addendum is final: Anything new for oncology?

Agenda

Recording

Speaker Institution Title Download slides
Kaspar Rufibach Roche, Basel Welcome and scene setting link
Anja Schiel Chair Scientific Advice Working Party, EMA Experience with the estimand framework in oncology link
Renaud Capdeville Novartis, Basel Challenges and open questions in hematology: RATIFY link
Tina Nielsen Roche, Basel Challenges and open questions in hematology: GALLIUM link
Hannes Buchner and Ingolf Griebsch Staburo and Boehringer-Ingelheim Treatment switching: challenges, estimands, and estimators link
Stefan Englert Abbvie Commentary on previous talks taking COVID-19 into account link
All Question and Answers link

03.06.2020: BBS Webinar: Aspects of COVID-19 pandemic

Agenda

No recording available

Speaker Institution Title Download slides
Jean Lang Sanofi Pasteur Overview on SARS-CoV2 and Challenges for COVID-19 Vaccine Development at Pandemic Speed link
Christian Althaus ISPM, University of Bern Early transmission, pandemic spread and severity of COVID-19 link
Karin Meiser Novartis, Basel Specific aspects of a clinical trial targeting Covid-19 link
Jenny Devenport Roche, Basel The influence of investigator initiated studies in the COVID-19 pandemic link

06.05.2020: BBS Virtual Seminar: Impact of COVID-19 on clinical trials

Agenda

No recording available

Speaker Institution Title Download slides
Frank Petavy EMA EMA perspective and guidance on COVID-19 link
Natalie Dimier Roche, Welwyn Industry perspective on COVID-19 (1/3) link
Mouna Akacha Novartis, Basel Industry perspective on COVID-19 (2/3) link
Cristina Sotto J&J Industry perspective on COVID-19 (3/3) link
Marcel Wolbers Roche, Basel Short Overview of Pharmaceutical Industry COVID-19 Biostatistics Working Group link
Evgeny Degtyarev Novartis, Basel Short overview on COVID-19 from the Cross-Industry Oncology Estimands Working Group link

04.02.2020: BBS Seminar: Network meta-analysis: methods and applications

Agenda

No recording available

Speaker Institution Title Download slides
Sylwia Bujkiewicz  Bivariate network meta-analysis for surrogate endpoint evaluation link
Gerta Rücker  Component network meta-analysis compared to a matching method in a disconnected network: a case study link
Georgia Salanti  CINeMA: a framework and web application to evaluate the confidence in network meta-analysis results link
Mark Pletscher  Network meta-analysis of treatments for previously untreated metastatic PD-L1-positive triple-negative breast cancer link
Bartosz Jenner An experience with indirect treatment comparisons using MAIC methods in a rare disease No presentation available
Marius Thomas  A network meta-analysis to compare efficacy of treatment options for relapsing-remitting multiple sclerosis No presentation available

01.11.2019: BBS Seminar: Predictive modelling, machine learning and causality

Agenda

No recording available

Summary of the event

Speaker Institution Title Download slides
Ewout Steyerberg  Clinical prediction models in the age of artificial intelligence and big data link
Willi Sauerbrei  The EQUATOR networkand guidelines for prediction models link
Torsten Hothorn  Score-based transformation learning link
Peter Bühlmann  Causal regularization for distributional robustness and replicability link
Giusi Moffa  Predicting putative intervention effects after causal structure learning from survey data link
Andrew Shattock Using machine learning and disease models to evaluate target product profiles of novel interventions (No slide deck available) No presentation available
Federico Mattiello  Identifying high-risk patients in Non-Hodgkin lymphoma (and trying to get insights into the disease biology) link
Mark Baillie  Novartis benchmarking initiative: making sense of AI link
Chris Harbron  Experiences from running internal prediction challenges within a pharmaceutical company link

21.08.2019: BBS/BES Seminar: Causal inference in drug development: why, when, how?

Agenda

No recording available

Speaker Institution Title Download slides
Miguel Hernan  Beyond intention-to-treat. Causal inference guidelines for causal inference link
Christine Fletcher  The Final ICH E9(R1) E9 addendum link
Frank Pétavy  A regulator’s perspective No presentation available
Valentine Jehl Adverse reactions confounded by prior medication: (how) can causal inference solve the problem? No presentation available
Thibaut Sanglier Treatment sequencing and effectiveness: challenges and considerations No presentation available
Nikolaos Sfikas Principal stratification techniques in the context of regulatory decision making No presentation available
Jack Bowden  Implementing the Principal Stratum estimand strategy using Instrumental Variable methods: An emulation of the CANTOS trial link

04.06.2019: BBS/EFSPI Seminar: Precision medicine in drug development

Agenda

No recording available

Speaker Institution Title Download slides
Niko Beerenwinkel Bioinformatics for precision oncology No presentation available
Stephen Senn  Precision medicine: The honeymoon is over. It’s high time for tough love link
Tomasz Burzykowski  Generalized pairwise comparisons for precision medicine link
Adam Heathfield  Valuation of Regenerative Medicine/Advance Therapeutics (RM/ATs): Challenges and opportunities for creating a better framework link
Jack Kuipers  Mutational interactions define novel cancer subgroups: can they inform precision oncology? link
Mario Ouwens  Difficulties with network meta-analysis when starting to use PDL1 thresholds link
Georgina Bermann  Cardiovascular medicine: approaches to the use of early biomarker response to identify a patient subgroup with enhanced therapeutic benefit link
Laurent Essioux  Examples of personalized Healthcare at Roche: statistical perspectives link

10.05.2019: BBS Spring Seminar: Synthetic controls - what do we need and how far can we go?

Agenda

No recording available

Speaker Institution Title Download slides
Tom Brookland  RWD/RWE Global Regulatory Overview link
Kaspar Rufibach and Hans Ulrich Burger  External controls in drug development link
Somnath Sarkar  Considerations for Developing External Control Arm from Real-World Data link
Laurence Colin and Yue Li  Making better use of early phase safety data link
Cornelia Dunger-Baldauf  For the sake of the patient – reducing placebo exposure by using historical controls link
Gonzalo Duran-Pacheco  Electronic Health Records used to derive Control Arms for Single-Arm oncology trials: Proof of concept using RCT’s in lung cancer link
Chris Harbron  A Decision Making Framework For Utilising External Control Arms link
Meinhard Kieser  Synthetic controls – what do we need and how far can we go? Rejoinder link
Norbert Benda  Synthetic controls – what do we need and how far can we go? Rejoinder link
Kit Roes  Synthetic controls – what do we need and how far can we go? Rejoinder link
Jan Müller-Berghaus  Synthetic controls – what do we need and how far can we go? Rejoinder No presentation available
Anja Schiel  Synthetic controls – what do we need and how far can we go? Rejoinder No presentation available

27.06.2018: BBS/EFSPI Seminar: Small populations and level of evidence

Agenda

No recording available

Speaker Institution Title Download slides
Fred Sorensen European Statistical Meeting on Small populations and level of evidence link
Daniel O’Connor  Rare diseases and orphan drugs: A Regulator’s (clinical)Perspectives link
Stephen Senn  In search of the lost loss function link
Henriette Thole  The potential and challenges of registry use when generating evidence in small populations link
Anja Schiel  Rare diseases and orphan drugs:The HTA perspective link
Adele Morganti  Borrowing external controls for an event-driven pediatric trial in PAH: a case study link
Andreas Kaiser  Bayesian analysis for small sample size trials using informative priors derived from historical data link
Elina Asikanius  Using a non-interventional study to strengthen the evidence collected in a Phase III program: a Hemophilia A case Study link
Hans Hockey and Kristian Brock  Hockey sticks and broken sticks – a design for a single-treatment double-blind randomized clinical trial suitable for chronic diseases link

26.06.2018: BBS Seminar: RCTs, personalized medicine, and surrogacy

Agenda

No recording available

Speaker Institution Title Download slides
Tomasz Burzykowski Evaluation of Time-to-event Surrogate Endpoints Using Accelerated Failure-time Models link
Everardo D. Saad Precision Medicine Needs Randomized Trials link
Marc Buyse A statistical approach for personalized medicine and benefit / risk assessment link

17.04.2018: BBS Spring Seminar: New developments in HTA, adaptive designs and multiplicity – in remembrance of Willi Maurer

Agenda

No recording available

Speaker Institution Title Download slides
Jason Wang Sense and sensibility of estimands for health technology assessment (HTA) link
Christoph Gerlinger EQ-5D-5L Utility Index for different countries link
Susan Edwards Whose perspective? Implications on cost-effectiveness modelling of differences between country value sets (a case study) link
Carsten Schwenke Real World Evidence and HTA – Experiences with IQWiG link
Tim Friede HTA AND SAFETY Some results of the ATF / APF Project Group link

20.03.2018: BBS Seminar: Competing Risks and Multi-State Models

Agenda

No recording available

Speaker Institution Title Download slides
Claudia Schmoor  Competing risks with applications to oncology link
Jan Beyersmann  Analysis of co-time-to-event outcomes in randomized clinical trials link
Ekkehard Glimm and Lillian Yau  A discrete semi-Markov model for the effect of need-based treatments on the disease states link

14.11.2017: BBS Seminar: Future of Biomedical Research: Are we ready?

Agenda

No recording available

Speaker Institution Title Download slides
Damian Roqueiro Machine Learning and Computational Biology Lab ETH Zurich Applications of Machine Learning and Deep Learning No presentation available
Jonas Dorn Novartis, Basel Machine learning when the ground truth isn’t truth and privacy is a problem – case study from the Assess MS project No presentation available
Hans Ulrich Burger Roche, Basel Big clinical data: What should biometrician do with it? No presentation available

11.09.2017: BBS Seminar Emerging topics for statistical methodology in drug development: Estimands and advanced analytics

Agenda

No recording available

Speaker Institution Title Download slides
Stephen Ruberg Eli Lilly The patient perspective – the estimands they want No presentation available
Stef van Buuren, Netherlands Organisation for Applied Scientific Research and University of Utrecht Individual causal effect: What is it? Why we need it? How to estimate it? No presentation available
Baldur Magnusson Novartis, Basel Using principal stratification to disentangle post-randomization outcomes in a randomized controlled phase 3 study No presentation available
Teresa Barata Roche, Basel Estimands in early Parkinson disease No presentation available
Stephen Ruberg Eli Lilly Making what’s advanced today routine tomorrow No presentation available
Jouni Kerman Google Causal Measurement using Geo Experiments in a Time-Based Regression Framework No presentation available
Cornelia Dunger-Baldauf Novartis, Basel A Smartphone-Based Study Capturing Longitudinal Vision Test Data, Movement Scores and User-reported Outcomes No presentation available
Fabio Pellegrini Biogen Individualized medicine based on a treatment response continuous score No presentation available

26.06.2017: BBS Seminar Innovative model-based dose escalation designs: what next?

Agenda

No recording available

Speaker Institution Title Download slides
Daniel Sabanés Bové  Model-based D/E designs: Current status and next steps link
Daniel Lorand  Tailoring dose escalation designs to early clinical development goals link
Andreas Krause  Guiding dose escalation studies in Phase 1 with unblinded modeling link

15.06.2017: BBS / PSI 1-Day Scientific Meeting: Empower the immune system to fight cancer

Agenda

No recording available

Speaker Institution Title Download slides
Jorge Martinalbo  Realising the potential of cancer immunotherapy link
Andrew Stone Statistical issues in the development of cancer immunotherapy link
Daniel Sabanés Bové Bayesian Learning in Early Phase Cancer Immunotherapy: A Case Study link
Matt Whiley An adaptive phase II basket trial design link
Dominik Heinzmann Statistical, clinical and ethical considerations when minimizing link
Claude Berge Statistical Challenges in Immunotherapy: Non Proportional Hazard Model link
Sergio Fracchia Challenges in development and approval: the case of cell based therapeutics link
Fred Sorenson Cancer Immunotherapy from the Health Technology Assessment (HTA) and Payer Perspectives link
Nicholas Latimer Estimating survival benefit for health technology assessment: New challenges presented by immuno-oncology treatments? link

05.05.2017: BBS Spring Seminar The use of external data for decision making

Agenda

No recording available

Speaker Institution Title Download slides
David Evans Reality and Real-World Data link
Ralf Bender Use of registries and observational data in the benefit assessment of medical interventions link
Frank Bretz Threshold-crossing: A Useful Way to Establish the Counterfactual in Clinical Trials? link
David Dejardin Dynamic borrowing of historical data: Performance and comparison of existing methods based on a case study link
Eva-Maria Didden GetReal: 3 Years on! link
Tim Friede The Use of External Data for Decision Making link

13.03.2017: BBS Seminar: Biomarker analyses

Agenda

No recording available

Speaker Institution Title Download slides
Werner Vach Universitätsspital Basel Statistical perspectives on umbrella trials No presentation available
Kaspar Rufibach Roche, Basel Comparison of clinical development plans for a confirmatory trial with subpopulation selection link

29.11.2016: BBS Seminar: Safety monitoring during the life cycle of a drug

Agenda

No recording available

Speaker Institution Title Download slides
Conny Berlin Safety monitoring during the life cycle of a drug link
Yusuf Tanrikulu Signal Detection – Quantitative Analysis of Safety Data link
Pritibha Singh Adverse Drug Reaction (ADR) screening in clinical trials link
Gianmario Candore Screening for adverse reactions in EudraVigilance link
Soheila Aghlmandi Choice of priors in rare events meta-analysis link

14.11.2016: BBS Seminar: Synthesis of Evidence: Observational studies and Meta-analysis

Agenda

No recording available

Speaker Institution Title Download slides
Kay Brodersen Google, Zurich Inferring causal effects in the absence of an experiment using CausalImpact No presentation available
David Rasmussen ETH Zurich Using HIV phylogenetics to quantify how human mobility impacts antiretroviral-based prevention strategies No presentation available
Almut Mecke Roche, Basel Different endpoints for meta-analyses of safety information No presentation available
Friedhelm Leverkus Pfizer Germany Meta-analysis in support of the German Benefit Assessment for Reimbursement (Arzneimittelmarkt-Neuordnungsgesetz – AMNOG No presentation available

17.10.2016: BBS Seminar: Missing Data and Graphical Models

Agenda

No recording available

Speaker Institution Title Download slides
Jane Hutton Uni Warwick Missing data and how to see biased results using Chain Event Graphs No presentation available
Giusi Moffa Novartis, Basel Cancer profiling and subtype discovery with Bayesian inference for acyclic digraphs No presentation available
Markus Elze Roche, Basel Propensity scores methods and covariate adjustment in practice No presentation available

14.09.2016: PSI/BBS One Day Meeting: Time-to-Event and Recurrent Event Endpoints

Agenda

No recording available

Speaker Institution Title Download slides
Lilla Di Scala Actelion, Basel Informative censoring in a rare disease: a regulatory experience in PAH No presentation available
Dominic Magirr AstraZeneca Unblinded sample-size reassessment in time-to-event clinical trials No presentation available
Tobias Bluhmki Universität Ulm Analyzing non-monotonous time-to-event outcome probabilities in randomized clinical trials No presentation available
Jennifer Rogers University of Oxford The analysis of recurrent events: A summary of methodology No presentation available
Mouna Akacha Novartis, Basel Recurrent event data endpoints in chronic heart failure studies: What is the estimand of interest? No presentation available
Ekkehard Glimm Novartis, Basel Sample size and interim analysis considerations for recurrent event data analyses No presentation available

04.05.2016: BBS Seminar: Adaptive Designs

Agenda

No recording available

Speaker Institution Title Download slides
Franz König Regulatory and methodological issues in adaptive designs for confirmatory trials link
Peter Bauer DMC membership experience link
David Lawrence Using a DMC for dose selection in a phase IIb/III adaptive design: the INHANCE study link
Alexander Strasak Adaptive seamless phase II/III study in gastric cancer No presentation available

28.04.2016: BBS Spring Seminar

Agenda

No recording available

Speaker Institution Title Download slides
Kaspar Rufibach Event projection: quantify uncertainty and manage expectations of broader teams link
Beat Neuenschwander Predicting milestone events for time-to-event trials link
Melissa Penny Model-based public health impact and cost-effectiveness estimates informing the WHO recommendation on malaria vaccine RTS No presentation available
Iain Bennett Designing in Treatment Switching (case study review and recommendations) link
Viktoriya Stalbovskaya Practical aspects of handling treatment switching in randomized clinical trials link
Daniel Sabanes Cancer immunotherapies: Which efficacy endpoints and statistical analyses to use? link
Andrew Stone Non-Proportional Hazards – So What? link
Karine Lheritier Complex study design in patients with Hereditary Periodic Fevers No presentation available
David Dejardin Bayesian dual endpoint decision making in combination studies link
Alessandro Matano Bayesian approach for Combination Phase I Trials in Oncology No presentation available

13.01.2016: BBS Seminar: Disease and Product Registries

Agenda

No recording available

Speaker Institution Title Download slides
Tim Friede Clinical registries: Use and Emerging Best Practices link
Eva‐Maria Didden Learning and Predicting Real‐World Treatment Effect based on Randomized Controlled Trials and Registry Data – A Case Study in Rheumatoid Arthritis link
Heiner C. Bucher Making Better Use of Registry Data in Designing Pragmatic Trials link
Yvonne Geissbühler Use of Registries to Collect Pregnancy Data link
Brian Hennessy Registries in Rare Diseases / Orphan Drugs along with 2 Case Studies No presentation available
Helene Karcher Leveraging Real‐World Registry Data to Optimize the Design of Phase 3 Studies – a Case Study in Schizophrenia link

27.10.2015: BBS Seminar: Prediction

No agenda available

No recording available

Speaker Institution Title Download slides
Kaspar Rufibach Bayesian Predictive Power: the bathtub problem link
Evgeny Degtyarev Designing Phase 2 to predict success in Phase 3 study No presentation available

23.06.2015: Joint BBS/EFSPI Seminar: Health Technology Assessment

Agenda

No recording available

Speaker Institution Title Download slides
Chrissie Fletcher & Matthias Egger Amgen & University of Bern IMI GetReal Initiative Update No presentation available
Panelists Expert panel discussion on trends and burning issues, eg. Structured benefit risk and real world evidence; dual EMA-HTA consultation No presentation available
Pascale Brasseur Medtronic Overview of HTA for medical devices and diagnostics No presentation available
Sheryl Warttig NICE NICE’s approach to the development of guidance for medical devices and diagnostics No presentation available
Karsten Berndt Roche Diagnostics EUnetHTA core model applied to Colorectal Cancer screening No presentation available
Valéry Risson Novartis Uses of Social Media for Outcomes Research – results of a real-world pilot No presentation available
Claire Watkins AstraZeneca Adjusting overall survival for treatment switch/crossover No presentation available
Pierre Ducorneau Roche, Basel Using the EUnetHTA HTA core model as a framework for planning, generating and presenting evidence No presentation available
Yovanna Castro Roche, Basel Predicting long term survival using nonparametric Bayesian methods: the melanoma case No presentation available
Nathalie Barbier Novartis Reimbursement challenges with new emerging cancer therapies No presentation available
Won Lee Xcenda The value of Oncology therapies and emerging access hurdles in Canada and the United States No presentation available

13.04.2015: BBS Seminar: Bayesian Methods in Adaptive Dose-Finding Trials

Agenda

No recording available

Speaker Institution Title Download slides
Daniel Sabanes Bove Bayesian Learning in Oncology: A Case Study link
Ying Yuan Bayesian Data Augmentation Continual Reassessment Method (DA-CRM) for Phase I Trials with Delayed Toxicities link
Daniel Lorand Bayesian modelling for combination dose-escalation trial that incorporates pharmacokinetic data link

13.11.2014: Joint BBS-EFSPI Seminar: Data Sharing in Clinical Development

Agenda

No recording available

Speaker Institution Title Download slides
Sabine Atzor  Clinical Trial Data Transparency – Environment & Expectations link
Stefan Driessen  EFSPI position on EMA policy on publication of clinical data link
Franz König  Big thunder link
Rebecca Sudlow  Overview of Data Sharing Initiatives in Industry and Current Experiences link
Janice Branson  Practicalities of accessing and using data – Advice for Researchers link
Sally Hollis  Considerations for analysis plans for data sharing requests link
Katherine Tucker  Principles to maintain patient confidentiality+H211 link
Guilliaume Breton  Patient Confidentiality Implementation link

02.10.2014: BBS Seminar: Meta-Analysis of Clinical Safety Data

Agenda

No recording available

Speaker Institution Title Download slides
Stephen Evans  Do meta-analyses of adverse events have adverse effects? link
Brenda Crowe  Comparison of Bayesian and Frequentist Meta-Analytical Approaches for Analyzing Time to Event Data link
Michael Gaffney  Model Selection in Meta‐Analysis of Clinical Safety Data – Fixed or Random Study Effect link
Amy Xia  Bayesian Meta-Analysis in Drug Safety Evaluation link
Jim Slattery  Meta-analysis in EU regulation link
Tarek A. Hammad  Sources of Bias in Meta-analysis of RCTs link
Mark Levenson  Meta‐Analysis for Safety: Context and Examples at US FDA link

07.05.2014: BBS Seminar: Applications for statisticians working with “Real-World Data”

Agenda

No recording available

Speaker Institution Title Download slides
Pamela Landsman-Blumberg Applications for statisticians working with “Real-World Data” No presentation available
Fred Sorensen No presentation available

04.02.2014: BBS seminar: Adaptive / Flexible Designs in early development

No agenda available

No recording available

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18.06.2013: BBS Seminar: IPD meta-analysis of treatment-covariate interaction with a continuous predictor

No agenda available

No recording available

Speaker Institution Title Download slides
Willi Sauerbrei, Patrick Royston, Benjamin Kasenda and Matthias Briel A method for IPD meta-analysis of treatment-covariate interaction with a continuous predictor in randomised trials link
Willi Sauerbrei and Patrick Royston Towards stratified medicine – instead of dichotomization link
Willi Sauerbrei and Patrick Royston A new strategy for meta-analysis of continuous covariates in observational studies with IPD link

04.06.2013: Joint BBS/EFSPI Seminar: Health Technology Assessment

Agenda

No recording available

Speaker Institution Title Download slides
Mike Branson Welcome link
Fred Sorenson  Health Technology Assessment – Why is it so important? link
Matthias Egger and Mike Chambers  Moving HTA forward: The challenges of incorporating real world evidence into Health Technology Assessment link
Mike Chambers and Matthias Egger  GetReal: Clinical effectiveness in drug development link
Claudia Nicolay  Health Technology Assessment – What’s in for Stats? link
Jens Grüger  HTA and personalized healthcare link
Skip Olson  HTA – Use of observational link
Bruno Falissard  The place of subjectivity in the French system (HAS): a good thing or an archaism? link
Ralf Bender  Biometrical topics of Health Technology Assessment in Germany link
Friedhelm Leverkus  30 Months AMNOG Health Technology Assessment: Outcomes and Issues link
Lou Garrison  HTA in Emerging Markets: A Framework and Examples link
Richard Nixon  Using early health economic models to support drug development decisions link
Chrissie Fletcher  Using indirect treatment comparisons to support an HTA link
All Summary of event link

28.11.2012: BBS Seminar: Optimal Design for Non-linear Models

Agenda

No recording available

Speaker Institution Title Download slides
Anthony Atkinson London School of Economics Experiments for Building Enzyme Kinetic Models No presentation available
Tobias Mielke Aptiv Solutions Optimal Population Designs for Non-linear Mixed Effects Models No presentation available
Barbara Bogacka Queen Mary College, University of London Population Optimum Design for Non-linear Mixed Effects Models in the Presence of Covariates No presentation available

25.09.2012: BBS Seminar: Benefit-Risk & Comparative Effectiveness

Agenda

No recording available

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Andrea Beyer Understanding the risk tolerance of regulatory assessors in Europe: the role of quantitative models in risk communication link
Christoph Dierig and Richard Nixon A case study using the BRAT framework for benefit risk assessment link
Christian Hove Rasmussen Benefit-risk assessment from a clinical point of view: a structured approach with focus on transparency link
Ralf Bender Biometrical requirements in (early) benefit assessments link
Fred Sorenson Benefit-risk assessment and comparative effectiveness research: are they really converging? link

09.07.2012: BBS Seminar: Emerging Topics in Pharmaceutical Statistics

Agenda

No recording available

Speaker Institution Title Download slides
Stephen A. Julious Sample sizes for multiple must-win trials link
Eric Derobert A parameterized strategy of gatekeeping link
Mouna Akacha & Günther Müller-Velten Recurrent event approaches in cardiovascular outcome trials No presentation available
Lisa Hampson Group sequential tests for delayed responses link

22.05.2012: BBS Seminar: Experiences in the Development and Implementation of Flexible Designs

No agenda available

No recording available

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29.11.2011: BBS Seminar

Agenda

No recording available

Speaker Institution Title Download slides
Boris Choy Business School The University of Sydney Nonignorable dropout models for longitudinal binary data with random effects: An application of Monte Carlo approximation through the Gibbs output No presentation available
Mouna Akacha Novartis, Basel Implementing Current Regulatory Guidance on the Treatment of Missing Data: An Industry Perspective No presentation available

16.09.2011: BBS Fall Conference: Current Topics in Pharmaceutical Statistics: Dose Finding and Multiregional Trials

Agenda

No recording available

Speaker Institution Title Download slides
Andy Grieve  Dose Selection in Drug Development: What Can Go Wrong? Can we put it Right? link
Didier Renard  Use of modeling & simulation to support design and analysis of a new dose and regimen finding study link
Bjoern Bornkamp  On the efficiency of two-stage adaptive designs link
Georg Gutjahr Powerful Modification of Step Down Procedures for Dose Finding No presentation available
H.M. James Hung  Planning and Analyzing Multi-regional Clinical Trials: A Regulatory Perspective link
Philip Hougaard  Global drug development strategies link
Jorgen Seldrup  Designing clinical trials in a multiregional regulatory environment No presentation available
Kevin J. Carroll  Consistency of treatment effect across regions in a multi-regional trial: reasonable goal or unrealistic requirement? link

21.07.2011: BBS Seminar Quantitative Benefit-Risk: What Matters to Whom and How? – Getting the Values and Weights

Agenda

No recording available

Speaker Institution Title Download slides
John Ferguson Structured Benefit-Risk Optimization (BRO): A Framework Quantitative Decision Support Tools link
Gordon Francis Clinical Perspective on Benefit-Risk Assessments link
Lawrence Phillips Quantitative Benefit-Risk: Determining Values & Assessing Weights link
Andrea Beyer Beyond the Probability of Risk: Measuring Perception link

10.05.2011: BBS Spring Conference: Comparative Quantitative Assessments: Benefit-Risk & Effectiveness

Agenda

No recording available

Speaker Institution Title Download slides
Chrissie Fletcher Industry Perspective on Comparative Effectiveness Research (CER) and the impact of Health Technology Assessment (HTA) in Europe link
Melvin (Skip) Olson Some Issues with Indirect Comparisons link
Friedhelm Leverkus Health Care Reform in Germany and Update on IQWiG Requirements link
John Doyle Effect of Comparative Effectiveness Research on Drug Development Innovation: a 360° Appraisal and Summary Discussion link
Deborah Ashby Current Methodological Approaches to Benefit-Risk Decision-Making link
John Ferguson Structured Benefit-Risk Optimization (BRO): State-of-the-art and Role of Fully Quantitative Decision Support Tools link
Michael Forstner Considerations for Implementing a Quantitative Benefit-Risk Assessment link
Richard Nixon Benefit-Risk Assessment of Multiple Sclerosis Treatments: Lessons Learnt in the use of Multi-Criteria Decision Analysis link
John Doyle Convergence of CER and BRA and Concluding Summary Discussion link

07.05.2011: BBS Spring Seminar: Multiplicity in Clinical Trials

Agenda

No recording available

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Huque Mohammad Some key multiplicity questions on primary and secondary endpoints of RCCTs and possible answers link
Ferber Georg Confirmatory statistics in PK/PD studies link
Kornelius Rohmeyer gMCP: A GUI for graphical multiple test procedures No presentation available
Wolf Michael Control of the false discovery rate under dependence using the bootstrap and sub sampling link
Maurer Willi / Glimm Ekkehard Multiple and repeated testing of primary link

13.01.2011: BBS Seminar: Statistical Challenges in Biomedical Research

Agenda

No recording available

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Kalisch Markus  Can one extract causal information from high-dimensional observational data? link
Sauerbrei Willi  Regression model-building with continuous variables – multivariable fractional polynomials link

30.11.2010: BBS Seminar

Agenda

No recording available

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Harald Binder Fitting and evaluating risk prediction models with high-dimensional molecular data No presentation available
Martin Wolkewitz Healthcare epidemiology hospital-acquired infections statistical modeling of outbreaks No presentation available

04.10.2010: BBS Fall Conference

Agenda

No recording available

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Helmut Schäfer University of Marburg Optimized and flexible designs for genome-wide associations studies No presentation available
Maximo Carreras Roche, Basel Point Estimation in Two-Stage Adaptive Designs With Mid-Trial Treatment Selection No presentation available
David Lawrence Novartis, Basel The A to Z of DMC interaction in a phase II/III adaptive design with treatment selection No presentation available
Reinhard Eisebitt ClinResearch Methods to protect the blinding, including controlled emergency unblinding, in adaptive design trials with flexible randomization schemes No presentation available
Tim Friede University of Göttingen The role of DMCs in adaptive design trials: The perspective of a DMC member No presentation available
Sue-Jane Wang FDA Adaptive Design Consideration: A Regulatory Perspective on How to Maintain Validity and Integrity of Trials link
James Hung FDA Emerging Challenges in Design and Analysis of Non-inferiority Trials link
Franz König EMA No presentation available
Ralf Bender IQWiG The importance of Non-inferiority testing in benefit assessments of medical interventions link
Heinz Schmidli Novartis, Basel Estimating the placebo-effect in a non-inferiority trial: a case study No presentation available
Georg Gutjahr, Werner Brannath, Peter Bauer Bremen and Vienna Unblinded SampleSize Modification for Fisher’s Exact Test link

24.06.2010: BBS/EFSPI European Statistical Meeting on Oncology

Agenda

No recording available

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Bertil Jonsson Medical Products Agency Understanding Progression-free Survival No presentation available
Marc Buyse IDDI and University of Hasselt The Time to Progression Ratio for Phase II Trials of Personalized Medicine No presentation available
Stuart Bailey Novartis, Basel Adaptive Bayesian Designs for Phase I Oncology Trials No presentation available
Simon Wandel Novartis, Basel Bayesian Hierarchical Modelling of Clinical Response in NSCLC Subpopulations No presentation available
Claire Watkins AstraZeneca IRESSA: A Journey of Experience from Broad to Biomarker Populations No presentation available
Cong Chen Merck Optimal Cost-Effective Go-No Go Decisions in Late-Stage Oncology Drug Development No presentation available
Jonas Wiedemann Roche, Basel Oncology Dose Finding – A Case Study No presentation available

28.04.2010: BBS Seminar Epidemiology

Agenda

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Noah Jamie Robinson Roche, Basel Epidemiology: the basics and in practice (at Roche) No presentation available
Jim Young Basel Institute for Clinical Epidemiology and Biostatistics Approximate Bayesian methods for the analysis of epidemiological data link

12.03.2010: BBS Seminar

No agenda available

No recording available

Speaker Institution Title Download slides
Benda Norbert The use of adaptive designs in dose finding studies link
Brannath Werner Challenges in the application of adaptive phase II/III designs in oncology link
Fleischer Frank Statistical modeling in the context of progression-free survival link
Friede Tim Blinded sample size reestimation with count data link
Glimm Ekkehard Some lessons learned from designing and adaptive clinical trial with time-to-event as the primary endpoint link
Guthjahr Georg Multiple Type I error control in response adaptive Phase II/III designs with treatment selection link
Wang Sue-Jane U.S. FDA Draft Guidance on Adaptive Design Clinical Trials – Statistical Considerations and Issues link

12.01.2010: BBS Seminar: Enhanced Statistical Methodology

Agenda

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Leonhard Held University of Zürich A Nomogram for P Values No presentation available
Kaspar Rufibach University of Zürich Estimation of two ordered monotone regression curves No presentation available

07.12.2009: BBS Seminar: Challenges and Evaluation of Biomarkers

Agenda

No recording available

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Eric Holmgren Genentech Quantifying the usefulness of PD biomarkers in phase 2 screening trials of oncology drugs No presentation available
Martin Schumacher Novartis, Basel Class prediction with gene expression data No presentation available

26.10.2009: BBS Seminar: Operational and Implementation Considerations in Adaptive Designs

Agenda

No recording available

Speaker Institution Title Download slides
Eva Miller ICON Clinical Research Logistical Considerations in the Implementation of Adaptive Trial Designs No presentation available
Norbert Benda Novartis, Basel Considerations and Experiences in Adaptive Dose Finding No presentation available