European network of paediatric research-European Medicines Agency meeting on rare gastrointestinal and liver diseases

• Date:
  Tuesday, 8 December 2015, All day
• Location:
  European Medicines Agency, Amsterdam, the Netherlands

The European network of paediatric research (Enpr-EMA) is organising a one-day meeting. The morning session is dedicated to the Paediatric European Digestive Diseases Clinical Research Network (PEDDCReN) to present to industry representatives their planned initiative to prospectively identify the incidence and prevalence of rare and very rare diseases in the field of paediatric gastroenterology and hepatology by establishing a European registry for these rare diseases and to discuss with industry how best to use this to work for developing research in these unique groups of patients. The afternoon session is organised together with the European Cystic Fibrosis Society and will focus on gastrointestinal outcome measures for clinical trials with new medicines targeting the basic defect in cystic fibrosis, so-called CFTR modulators.

Documents

Report on the European network of paediatric research-European Medicines Agency workshop on gastrointestinal (GI) outcome measures to evaluate CFTR modulators for the treatment of cystic fibrosis (CF)

Reference Number: EMA/26775/2016Summary:

Report on the workshop on gastrointestinal (GI) outcome measures to evaluate CFTR modulators for the treatment of cystic fibrosis (CF)

Agenda - European network of paediatric research-European Medicines Agency meeting: rare gastrointestinal and liver diseases initiative (morning session)

Agenda - European network of paediatric research-European Medicines Agency meeting: workshop on gastrointestinal (GI) outcome measures to evaluate CFTR modulators for the treatment of cystic fibrosis (CF) (afternoon sess...

Summary:

Workshop on gastrointestinal (GI) outcome measures to evaluate CFTR modulators for the treatment of cystic fibrosis (CF) - Tuesday, 8 December 2015, 13:30 – 16:30, meeting room 2E

Presentation - Intestinal Pseudo-obstruction (Gut Motility Disorders) (Nikhil Thapar)

Presentation - Chronic Intractable Diarrhoea of infancy (Nick Croft)

Presentation - Rare liver diseases (Ulrich Baumann)

Presentation - PIBD Cancer & Mortality (Lissy de Ridder)

Presentation - Why are rare diseases of interest to industry (Brihad Abhyankar)

Presentation - Rare diseases, European and UK approaches: setting the scene (Julian Thomas)

Presentation - How the EMA EU support treatment development in rare diseases (Laura Fregonese)

Presentation - Paediatric European Digestive Diseases Clinical Research Network Project (Nick Croft)

Presentation - Overview of CFTR Modifier Pipeline (Tim Lee)

Presentation - Potential of GI outcome measures in CFTR modulation (Frank Bodewes)

Presentation - The role of intestinal organoid function for evaluation of CFTR modulators (Jeffrey Beekman)