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Medicines for Children Research NetworkMedicines for Children Research Network
Paediatric Clinical Trials: Principles, Practicality
and theMedicines for Children Research
Network
Andrew CollinsonCo-Director MCRN South West
Medicines for Children Research NetworkMedicines for Children Research Network
Principles – Scientific evidence
• RCTs:
– Why “randomised” (minimises confounding)– Why “controlled” (comparison group)– Why “blind” (minimises recall/ observer bias)– Applicability and validity – Power
Medicines for Children Research NetworkMedicines for Children Research Network
Principles – Children are different
• Diseases
• Physiology
• Behaviour
• Pharmacokinetics
• Pharmacodynamics
Medicines for Children Research NetworkMedicines for Children Research Network
Principles - Ethics
• A brief history of disaster:
– Nuremberg 1946 – Thalidomide 1977 – Tuskegee Syphilis Study
• And then there was…– The Declaration of Helsinki
Medicines for Children Research NetworkMedicines for Children Research Network
Practicality – Legislation and Standards
• International Conference on Harmonisation of Technical
Requirements for Registration of Pharmaceuticals for Human Use (ICH)
• ICH Good Clinical Practice (GCP)
• The EU Clinical Trials Directive
• ‘Better Medicines for Children’: European Parliament and Council Regulation
Medicines for Children Research NetworkMedicines for Children Research Network
International Conference on Harmonisation (ICH)
• Addressed:
– Need for new medicines in global market
– Differing national requirements for approval
– Need to reduce import delays
– Efficient use of human, animal and material resource – Need to reduce repeat testing
Medicines for Children Research NetworkMedicines for Children Research Network
ICH Good Clinical Practice
• ICH Standards:
– What do you think they should be?…
Medicines for Children Research NetworkMedicines for Children Research Network
‘Better Medicines for Children’: European Parliament and Council Regulation
Medicines for Children Research NetworkMedicines for Children Research Network
Practicality: Research Infrastructure
Medicines for Children Research NetworkMedicines for Children Research Network
Medicines for Children Research NetworkMedicines for Children Research Network
UK Clinical Research Network
Diabetes
Dementias
Stroke
Diabetes
Dementias
Stroke
Medicines for Children Research NetworkMedicines for Children Research Network
Medicines for Children Research Network
DirectorManager
Research nurseData entry staffAdministrator
Service support
UKCRN
MCRNCC
PortfolioDevelopment LRNx6CTU
ClinicalStudiesgroups
Regulators
Clinical expertsIndustry
ConsumersFunders
Medicines for Children Research NetworkMedicines for Children Research Network
MCRN Local Research Networks (LRNs)
Medicines for Children Research NetworkMedicines for Children Research Network
What are we trying to achieve?
“To facilitate the conduct of randomised trials and other well-designed studies of medicines for children, including those for prevention, diagnosis and
treatment”
Medicines for Children Research NetworkMedicines for Children Research Network
Clinical Studies Groups
PROACTIVE ROLES:PROACTIVE ROLES:• To identify research priorities • To propose and develop trial proposals• Representatives sit on MCRN Operational Group
REACTIVE ROLES:REACTIVE ROLES:• To help investigators to develop ideas to successful funding application• To provide subject specific and methodological advice to investigators• Representatives sit on MCRN Trial Adoption Committee
Medicines for Children Research NetworkMedicines for Children Research Network
Clinical Studies GroupsClinical Studies GroupsAnaesthesia, Intensive Care, Pain Control TBCand Cardiology
Diabetes, Endocrinology & Metabolic Medicine Prof David Dunger
Gastroenterology, Hepatology & Nutrition Dr Stephen Murphy
General Paediatrics (including Dermatology) Dr Colin Powell
Methodology Dr Peter Brocklehurst
Neonatal Prof David Field
Neurosciences Dr William Whitehouse
Pharmacy & Pharmacology Prof Ian Wong
RANII (Rheumatology, Allergy, Dr Mike SharlandNephrology, Infection & Immunity)
Respiratory & Cystic Fibrosis Prof Jonathan Grigg
Medicines for Children Research NetworkMedicines for Children Research Network
MCRN and opportunities for Industry
• Access to opinion leaders in paediatric medicine
• Standing confidentiality agreements
• Swift assessment of site feasibility
• Consistent approach to costing studies & arranging contracts
• Central sign off for R&D approval at national level
Medicines for Children Research NetworkMedicines for Children Research NetworkMedicines for Children Research NetworkMedicines for Children Research Network
MCRN Training & EducationMCRN Training & Education
The MCRN aims to ensure all staff have access to a high quality, paediatric specialised training programme,
maximising their knowledge and skills to achieve a highly qualified workforce.
• Generic UKCRN courses
• MCRN courses • Approval of external courses
Training for all staff of MCRN portfolio studies
• On-line training
Medicines for Children Research NetworkMedicines for Children Research Network
MCRN Consumer Involvement
• open and transparent
• central to all aspects of MCRN activities
• all stages and levels of research cycle
• all key National and Local MCRN committees
• reflect and contribute to good practice guidelines on involving consumers, particularly children and families, in research
• impact of MCRN consumer involvement monitored and evaluated
• findings and outputs on consumer involvement strategy should be widely disseminated.
Medicines for Children Research NetworkMedicines for Children Research Network
MCRN LRNs
Clinical Studies Groups
Clinical TrialsUnits
MCRN Portfolio
Trial Adoption
Committee
MCRN Coordinating CentreMCRN Coordinating Centre
Dr Vanessa PoustieDr Vanessa Poustie
Assistant Director
Miss Jennifer BlakeburnMiss Jennifer Blakeburn
CSG Administrator
Portfolio Development
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Current portfolio
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Medicines for Children Research NetworkMedicines for Children Research Network
Table 2. Companies who have contacted MCRN re. studies with the potential to be run through the network
Company Nature of study Status of study
Notes
GSK Gastro drug Ongoing Used for “dummy” adoption – considering whether feasible to extend study to LRN sites
MSD/Sanofi-Pasteur Vaccine study New Initial contact needs following up
MSD Renal study New Submitted for adoption MSD Hypertension study New Submitted for adoption Pfizer Diabetes study In
development May be rolled out in UK in the future
Pharmaxis Cystic Fibrosis study New Adopted by MCRN SAC 151106
Wyeth Vaccine study Ongoing Adopted by MCRN SAC 151106
Xoma Vaccine study New Initial discussions to be arranged
Contacted by physician on behalf of?
Inhaler device/asthma In development
Initial discussions to be arranged
GSK Dermatology study New Initial discussions to be arranged
Medicines for Children Research NetworkMedicines for Children Research Network