This document discusses the advantages and challenges of clinical research networks for pediatric GI/Hep studies. Key advantages include increased diversity that allows studies to be completed faster, as well as opportunities to study rare diseases. Challenges include complex administration, the need for consensus on study protocols, and regulatory hurdles. The document provides guidance on selecting network centers based on expertise and other factors. It also outlines various roles and responsibilities within networks, including those of clinical centers, data coordination centers, sponsors, and oversight committees.

1. Clinical Research Networks
Doin’ the Thizzle Dance
Pediatric GI/Hep Research Conference

2. Topic Choices
 Prevalence
 Impact on QOL
 Funding likelihood
 Patient/parent “investment”
 Possible geography/site differences
 Health disparities
 Knowledge deficits
 Technology advances
 Uniformity in approach feasible/ethical

3. Center Selection
 Expertise and willingness
 Innovation and writing ability/leadership
 Availability of subjects
 Geographic distribution
 Interaction of principals
 Availability of center infrastructure
 Track record/delivery on promises
 Unique quality
 Reputation and integrity

4. Research Network Advantages
 Diversity
– Geographic
– Ethnic/racial
– Age/socioeconomic/cultural
 Enrollment completed faster
 Diversity of study questions
 Broader expertise
 Rare disease study opportunities

5. Network Disadvantages
 Cumbersome administration/approvals
 Need for consensus protocol
 Regulatory delays
 Authorship/attribution
 Center “investment” and disparate
ability
 Communication/travel

6. Regulatory Hurdles
 DCC and CC IRBs
 FDA for IND
 CRADA contracts
 Data and Safety Monitoring Boards
 External advisory boards
 DCC site visits and audits
 CRC/CTRI oversight committees
 Trial registration (eg clinicaltrials.gov)

7. General Study Questions
 Epidemiology
 Natural History
 Etiopathogenesis
 Genetics
 Noninvasive predictors (imaging,
proteomics)
 QOL/standardized questionnaires
 Treatment trial
 Tissue/lab/clinical correlates

8. Network Centers/Roles
 Clinical Centers
– Protocol design
– Study objectives
– Consent
– Screening
– Registration
– Data acquisition
– Data entry
– CRFs/internal QA
– Protocol execution
– AE/SAE management
– Budgeting
– Drug dispensation
– Tissue procurement
– Manuscript preparation
 Data Coordination
– Website development
– Form development
– Biostatistical analyses
– Enrollment power
– Network QA
– Data checks
– Compliance
– Data repository
– Audits
– Brochures
– DSMB reports
– Information distribution
– Meeting preparation

9. Network roles cont’d
 Sponsor(s)
– Selection of centers
– Funding
– Budgets
– Oversight of DCC
– Oversight of CCs
– Dispute resolution
– Biorepository
– CRADA selection
– Ancillary studies
 DSMB
– External review
– Data quality
– Safety oversight
– Protocol/consent
suggestions/approval
– Interim stopping
– Oversight of SAE
management

10. Budgeting Considerations
 Capitated
 Non-capitated
 Capitated with secondary performance
basis
 Budgets for patient care,
PI/investigator time, coordinators,
supplies, equipment, travel, data entry,
research pharmacy, IRB?, GCRC?,
ancillary studies, indirect costs

11. Network Committees
 Steering
 Executive
 Database
 Treatment Trial
 Pathology
 Radiology
 Standard of Care
 Ancillary studies
 Imaging (radiology)
 Publications and Presentations
 Exemption/Exception

12. Publication Attribution
 Conventional
 Corporate
– Appendix with writing group (with
principal), Network members
 Modified Corporate