No recording available
| Speaker | Institution | Title | Download slides |
|---|---|---|---|
| Daniel Rosenberg | Johnson and Johnson | Rare diseases: Challenges to generate Real‐World Evidence | link |
| Keith McDonald | IQVIA | Overview of the regulatory landscape for development of medicines intended for rare diseases | link |
| Laura Girardat-Rotar | IQVIA | Multi‐regional, long‐term PASS using mixed data sources | link |
| Jilles Fermont | Novartis | Collaborate to innovate: iptacopan and RWE in PNH treatment | link |
| Audrey Muller | Johnson and Johnson | Challenges for comparative designs in rare diseases | link |
| Maria Luisa Faquetti | ETH Zurich | Exploring the Applicability of the Prevalent New User Design in Rare Diseases | link |
No recording available
| Speaker | Institution | Title | Download slides |
|---|---|---|---|
| Vivian Lanius | Bayer | An Introduction to Estimands - Tell me what you want what you really, really want | link |
| Isaac Gravestock | Roche | Case Study 1: Applying the target trial emulation framework when time 0 is ambiguous | link |
| Valentine Jehl | Novartis | Case Study 2: Long term safety - how to hit a moving target | link |
| Gerd Rippin | IQVIA | Estimands in the context of External Comparator Arm studies | link |
No recording available
| Speaker | Institution | Title | Download slides |
|---|---|---|---|
| Massoud Toussi | IQVIA | What is data quality, and how data types differ in terms of data quality | link |
| Nicole Mahoney | Novartis | RWD for regulatory decisions | link |
| Clair Blacketer | Janssen | EHDEN: Data Quality Dashboard | link |
| Daniel Morales | EMA | EU Data quality framework | link |
| Dalia Dawoud | NICE | COPD case study - The Use of OMOP Common DataModel in Health Technology Assessment | link |
| Spencer James | Roche/Genentech | Data quality in Flatiron | link |
| Gracy Crane | Roche | Transcelerate - How to bridge from framework to fitness for purpose demonstration | link |
No recording available
| Speaker | Institution | Title | Download slides |
|---|---|---|---|
| Christian Schindler | SwissTPH | Overview of statistical methods used and envisaged to handle attrition bias in SAPALDIA | link |
| Audrey Muller | Actelion | Comparative effectiveness in rare disease: Handling of missing data for propensity score use in EXPOSURE | link |
| Gracy Crane | Roche | Comparative effectiveness of patients with ALK-positive non-small-cell lung cancer following progression on crizotinib: a comparison of alectinib single-arm phase II data versus ceritinib real-world data | link |
No recording available
| Speaker | Institution | Title | Download slides |
|---|---|---|---|
| Christoph Meier | University of Basel | Case study of studying the association between benzodiazepines and Alzheimer’s disease | link |
| Sigrid Behr and Daniel Rosenberg | Novartis/Actelion | Assessing long term latencies for newly marketed drugs: mission impossible for the epidemiologist | link |
| Anna Beckmeyer-Borowko | SwissTPH | The value of population-based studies and their associated biobanks in studying long term exposure effects | link |