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How the Paediatric Clinical Trial Units operate in Europe:
Lets (not) go Dutch
Jos Gilissen
23 May 2018
Content:
•Our current situation•Lessons learned• The way we face Future....
Credits and disclaimer
23 paediatric specialties15 paediatric expertise centres24.000 paediatric patients yearly75% lives ≤ 50 Km driving distance
How did we do so far?
•Founded in 2008 (MCRN)•Start in 2009•From 1 to 10 +•Supports 13 Paediatric subspecialties•80% of costs paid thru external funding
The Numbers (1)
Total amount studies since 2009 210
Amount studies involving medication 90 Interventional 57
observational 33
Pharmacy driven 37
Investigator driven 53
Data collected since 2012, partly retrospective.
The Numbers (2)
Total amount studies open currently 95
Amount studies involving medication 37 Interventional 16
observational 21
Pharmacy driven 7
Investigator driven 30
Numbers for the near future
Total amount studies expected to start in 2018: 40-50
Studies involving medication ready to
start 2018/19 18 Interventional 11
observational 7
Pharmacy driven 12
Investigator driven 6
Feasibility of medicine studies
Eligibility
≈ 90% of pharmacy studies < 5 eligible patients
≈ 90% of investigator studies > 50 eligible patients
Informed Consent
Observational: ≈ 98% patient/parents give IFC
Interventional comparative drug: 75% give IFC
Interventional new drug: 85% give IFC
What have we learned so far?
CR = ‘high competitive sport’ and the investigators are the athletes.
Stimuli:• Improving Care• Career perspective• Demanded by UMC• Requested by patients• Money• Contracts
CR is a commercial business
Issues:• Paediatrics is more expensive• Less patients• Costs go for the benefits • Less funding more
competition• More Funding rules• Inexperience in negotiating
Keeping up with IT innovations
• Progress in IT is overwhelming• Electronic patient charts• It opens up a new possibilities
BUT 1:Time, skills and effort is spend in learning how to use the software.
BUT 2:Use of patient data is limited in respect of their rights, safety and privacy.
Keeping up with Regulations
• Liability issues• Reversed burden of Proof• No scandals
Investigators are burdened with bureaucratic procedures .
Not all Pharmaceutical companies have the ability to turn it in an advantage.
Example: Beneductus trailTitle: Multi-center, randomized non-inferiority trial of early treatment versus
expectative management of patent ductus arteriosus in preterm infants
Intervention: medication to try to close the PDA or no treatment.
Current practice: both treatments are applied, no conclusive SE available.
In other words: a patient in regular care is randomised by choice of hospital
Regulatory issues:
Is this a medicine study Yes or No?
Is the patient in higher danger when included in the study?
https://neonatologynetwork.eu/studies/beneductus
How do we face the future?
Thru Common Research Wealth (1)
Local: Radboudumc Technology Centre Clinical Studies
Woman and Child
(Inter) National: collaborating with research prof.
in other PRC:
Dutch Clinical Research Federation
Pedmed.
C4C, ....
Thru Common Research Wealth for (2)
•A mandatory Amalia Faesibility Proces = roadmap +
hands on support from initial research idea to the start
of a project.
•Expanding the research support team
(contract specialist, medical writers, other scientist)
• Embedding the RN in the subspecialty team to
coordinate all the research.
Providing our patients a personal track record for CS
A significant impact on healthcare
By providing a high quality infrastructure for all
paediatric clinical studies within/from the
Amalia Children’s Hospital, for (medical) student
up to professor and from initial idea for a study
to long term archiving and reuse of data.
I invite you to join us in this:
Common Research Wealth.
