medicines for children research network paediatric clinical trials: principles, practicality and the...

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


Principles – Scientific evidence

• RCTs:

– Why “randomised” (minimises confounding)– Why “controlled” (comparison group)– Why “blind” (minimises recall/ observer bias)– Applicability and validity – Power


Principles – Children are different

• Diseases

• Physiology

• Behaviour

• Pharmacokinetics

• Pharmacodynamics


Principles - Ethics

• A brief history of disaster:

– Nuremberg 1946 – Thalidomide 1977 – Tuskegee Syphilis Study

• And then there was…– The Declaration of Helsinki


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


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


ICH Good Clinical Practice

• ICH Standards:

– What do you think they should be?…


‘Better Medicines for Children’: European Parliament and Council Regulation


Practicality: Research Infrastructure


UK Clinical Research Network

Diabetes

Dementias

Stroke

Diabetes

Dementias

Stroke


Medicines for Children Research Network

DirectorManager

Research nurseData entry staffAdministrator

Service support

UKCRN

MCRNCC

PortfolioDevelopment LRNx6CTU

ClinicalStudiesgroups

Regulators

Clinical expertsIndustry

ConsumersFunders


MCRN Local Research Networks (LRNs)


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”


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


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


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


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.


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


Current portfolio


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 network paediatric clinical trials: principles, practicality and the...

Documents