An ECFS-CTN webinar series

With the arrival of new therapies in clinic, real world evidence (RWE) trials are becoming more common in CF. Meanwhile regulatory and HTA requirements are evolving, and often reference CF therapies in their case studies.

ECFS-CTN invites you to join us for a webinar series to learn more about RWE in cystic fibrosis, how it is synthesised and assessed, and how we can all work together efficiently to bring new medicines to people with cystic fibrosis as quickly as possible. We bring together speakers from across the spectrum of stakeholders, from patients, to ECFS projects, industry, other rare diseases, EMA and reimbursement agencies. 


The webinars are open to anybody with an interest in RWE including clinicians, research coordinators, academics, patient organisation representatives and industry.
We hope you can join us!

Webinar 1:

Introduction to RWE, the ECFSPR and the patient perspective 

Wednesday 6th October 2021, 1230-1400 CEST

  • Welcome
    Damian Downey, ECFS-CTN director
  • Introduction to RWE
    Nicholas Simmonds, Royal Brompton Hospital, London
  • CF Europe and the patient perspective
    Claire Francis and Audrey Chansard, CF Europe
  • ECFS Patient Registry and RWE
    Lutz Naehrlich, ECFS Patient Registry
  • Discussion

Webinar 2:

Real “real world” studies in CF and beyond  

Wednesday 13th October 2021, 1230-1400 CEST

  • Welcome
    Damian Downey, ECFS-CTN director
  • The sponsor perspective of performing RWE trials in CF
    Nataliya Volkova, Vertex Pharmaceuticals
  • RECOVER – real world effects of novel CFTR modulators
    Paul Mc Nally, Royal College of Surgeons in Ireland
  • Using the French Health Databases and French CF registry to define health care resource use and costs
    Isabelle Durieu, Lyon University Hospital
  • Project HERCULES: experience from Duchenne Muscular Dystrophy
    Josie Godfrey, Project Hercules
  • Discussion

Webinar 3:

The regulatory and reimbursement perspective 

Wednesday 20th October 2021, 1230-1400 CEST

  • Welcome
    Damian Downey, ECFS-CTN director
  • Opportunities and challenges of RWE to support regulatory decision-making
    Carla Jonker, Data Analytics and Methods Task Force, EMA
  • The role of RWE in CF
    Irmgard Eichler, Medical University of Vienna
  • Using RWE in health technology assessment 
    Niklas Hedberg, EUnetHTA and Chief
    Pharmacist TLV, Stockholm
  • Discussion