Experiences from a pediatric clinical research unit

Pediatric Clinical Research Center at the Sahlgrenska University Hospital

Jenny Kindblom , MD, PhDMedical Director PCRC

Medicines for children

• Not enough knowledge on the effect and safety ofmedicines in the pediatric population (<18 years of age)

• Treatment off-label common

• Pediatric drug development plans FDA, EMA

Pediatric Regulation 10 years (2007-2016)

50% more pediatric trials

260 new medicines authorised

Challenges

‒ Children only disorders

‒ Clinical trials (recruitment)

This is Västra Götaland

1.7 million inhabitants

49 municipalities, 24 000 km2

Major city: Gothenburg

About health carein Sweden

Free and equal access to public health care

Health interventions are financed by taxes, state funding and custom fees

Counties or regions - responsible for the health care at hospitals and local health centres

Municipalities - responsible for elderly personal care

Queen Silvia Children’sHospital

One of the nodes within Clinical Studies Sweden-and part of the SwedPedMed national network

Health Care PCRC &

pediatricpatients

Academy

Industry

Pediatricclinical trials

Large need /demand

Academic studies Industry-sponsoredClinical knowledge gaps

Complexities

ScientificEthically / morally

Practical issues

Small populations

Few children with diagnoses

Heterogeneity

How can pediatric clinical research be facilitated ?

”What research support would you need for your(academic) research?”

”What research support would you need to be ableto participate /more often/ in industry-sponsored trials?”

How can pediatric clinical research be facilitated ?

Industry-sponsored trials:

• GCP

• Agreement, legal support

• Economy, budget

• Administration

• Monitoring

• Research nurses

In academic research:

• Statistics

• Economy/Budget

• Data management / useof registers

• Research nurses

Pediatric Clinical Research Center

A resource for everyone within the health care, academia or industry who wish to pursue pediatric clinical research in Västra Götaland.

Conduct of pediatric trials

• High quality trials - expertise in conductof pediatric trials• From feasibilty to closing the trial, from discussion scientific rationale to practical and administrative issues

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!!

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Feasibility support

Start-up supportFeasibility, budget –

agreement, legal agreements, allocation of

personnelEducationGCP, conduct of pediatric trials,

scientific experience and involvment for

physicians/nurses

Maintenance supportCoordination,

administration, documentation,

monitoring

Start-up supportFeasibility, budget –

agreement, legal agreements, allocation of

personnelEducationGCP, conduct of pediatric trials,

scientific experience and involvment for

physicians/nurses

Maintenance supportCoordination,

administration, documentation,

monitoring Medical advisorProject leader

Trial coordinatorLegal representative

Research nursesMonitors

Administrative support

Educators/project leadersMedical advisor

Our mission • To facilitate clinical research

• …both academic and industry-sponsored

• …in Pediatrics, but also in all other specialities treating children (Child and AdolescentPsychiatry, Infection, ENT, Surgery…)

• …by doing as little or as muchas needed

Identifiedneeds

Start-upGetting

organisedEveryone aboard?

Let´s go!

The Swedish example

• Local facilitation is crucial !

• PCRC at the SahlgrenskaUniversty Hosital is a resource for everyone within the health care, academia or industry who wish to pursue pediatric clinical research in VästraGötaland.

Vision: be an integrated part of life at the Queen Silvia Children´s

hospital

jenny.kindblom@vgregion.se

Thank you