PEDDCReN Paediatric European Digestive Diseases Clinical Research - - PowerPoint PPT Presentation

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PEDDCReN Paediatric European Digestive Diseases Clinical Research - - PowerPoint PPT Presentation

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PEDDCReN Paediatric European Digestive Diseases Clinical Research Network Project PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network Aims 1. To support the development, funding and planning of high quality, trials in

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

PEDDCReN

Paediatric European Digestive Diseases Clinical Research Network Project

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

Aims

  • 1. To support the development, funding and planning
  • f high quality, trials in children with GHN

disorders.

  • Early engagement with industry in study plans
  • Support investigator led studies, (eg sign posting to

funds, feasibility identification of collaborators)

  • Model PIPS, agreed outcome measures
  • Feasibility data etc

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

Key partnerships

Led by British, Irish and Dutch Societies of Gastroenterology ESPGHAN - European Society of Paediatric Gastroenterology , Hepatology and Nutrition ENPR-EMA - European Network of Paediatric Research – European Medicines Agency

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

  • Web based secure

software

  • Electronic surveys and

databases

  • Widely used

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

PEDDCReN Expertise, resources and interest

  • 71

investigators/ centres

  • 61 Cities
  • 24 Countries

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

Register – Key points

  • Interest and Facilities

– All willing to participate in PEDDCReN – 56% willing/ able to take on phase I or II studies – 37% Dedicated paediatric clinical research facilities available on site – 34% Access to a research nurse

  • Investigator experience

– 33% had been Chief Investigators for their country – 58% had been Principle Investigators in their hospital – 27% had experience of phase I or II studies

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

Register – Key points

  • Approx 1000 NEW IBD patients per year
  • Very Rare Diseases (out of 71 centres)

– 17 units had patients with congenital enteropathy – 11 Congenital transport defect – 41 Polyposis syndromes – 28 Chronic intestinal pseudo-obstruction

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

PEDDCReN Outcome PIBD Horizon 2020 Bid

  • 6,000,000 euros
  • PEDDCReN WP 6

– Inception cohort of 300 IBD patients per year – Pan European safety monitoring of rare complication of IBD

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

PEDDCReN and industry/CROs

  • Access to experienced and motivated investigators
  • Key stakeholders – academics, clinicians, professional

societies (ESPGHAN), EMA

  • Pan European feasibility
  • Information on patient populations
  • Ability to perform feasibility and prospective

identification of patients

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

GI Liver Rare diseases proposal

  • Prospective study of incidence and prevalence and incidence
  • f rare and very diseases (Redcap)

– ? Liver – ? Intractable diarrhoea – ? Pseudo-obstruction

  • Develop national guidelines for investigations – eg infant

diarrhoea

  • Deep phenotype and genotype information

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

Rare diseases proposal

  • Date supports feasibility of trials (Redcap)
  • Plan intervention studies
  • Improve care (early identification, better quicker diagnosis, new

treatments)

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

How: Monthly E- card

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network 11/01/2016 13

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

Find patients

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

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Phenotype information collect and store biobank specimens

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PEDDCReN - Paediatric European Digestive Diseases Clinical Research Network

Summary and questions

  • Each disease is rare/very rare
  • Is there a forum for pharma to collaborate

funding across multiple diseases in this area ?

  • Is there a way to apply for funds as a group

rather than to each individually?

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