Welcome to the CTSA Consortium Child Health Oversight Committee (CC-CHOC) Annual Meeting

1. Welcome to the CTSA Consortium Child Health Oversight Committee (CC-CHOC) Annual Meeting Bonnie Ramsey, Chair 2009-2010 April 30, 2010

2. Special Introductions (1) • NIH Representatives • Barbara Alving Director, NCRR • Anthony Hayward Director, CTSA Program • Mary Purucker NCRR • Steven Hirschfeld NICHD • Alan Guttmacher NICHD

3. Special Introductions (2) New CC-CHOC Voting Members – Class of 2009 • Arkansas Center for Clinical and Translational Research (CCTR) • Richard Jacobs • NYU-HHC Clinical and Translational Science Institute • William Borkowsky • South Carolina Clinical and Translational Research Institute • Carol Wagner • UTMB Institute for Translational Sciences • Tasnee Chonmaitree • University of Cincinnati Center for Clinical and Translational Science and Training • Carole Lannon • University of Florida Clinical and Translational Institute • Desmond Schatz • University of Illinois-Chicago Center for Clinical and Translational Science • Mark Stein

4. Special Introductions (3) • CC-CHOC 2009 Fellowship Recipients • Sylvia Yoo Stanford • Justin Juskewitch Mayo Clinic Congratulations!!!

5. Election Results • Chair-Elect 2010-2011 • William Smoyer, Nationwide Children’s Hospital, Columbus, OH • New Operations Group Representatives • Jon Davis, Tufts Clinical and Translational Science Institute • Frederick Kaskel, Albert Einstein-Montefiore Institute for Clinical and Translational Research • Alex Kemper, Duke Translational Medicine Institute

6. Thank You!!! • Departing Operations Group Members • Nancy Green Columbia • Carole Marcus Children’s Hospital of Philadelphia • Bert Lubin Oakland Children’s Hospital

7. CC-CHOC 2009-2010 Year in Review

8. Mission Statement • Provide a unique national forum to identify collaborative opportunities to facilitate child health clinical and translational research • Building partnerships • Building infrastructure • Developing standard approaches • Identifying and overcoming barriers • Set priorities and enhance child health research across CTSA Consortium • Metrics of Success

9. CC-CHOC Organizational Structure

10. Building Partnerships Accomplishments (1) • Pediatric Academic Societies Alliance • 2008 - Alliance established • 2009 - CC-CHOC representative on PAS Planning Committee • 2010 - Clinical Translational Track established • Three symposia • CTSAs: Regulatory Challenges in Pediatric Research May 1, 8:00 am – 10:00 am • Translating Research into Pediatric Clinical Practice May 1, 10:15 am – 12:15 pm • CTSA’s: Changing the Environment for Pediatric Translational Research May 1, 2:45 pm – 4:45 pm • Abstract session • CTSA Supported Pediatric Clinical Translational Research May 2, 1:00 pm – 3:00 pm • Enhanced by NCRR R-13 Award 2010-2012 (S. Barkin, PI) • Workshop on Informatics • CC-CHOC Fellows

11. Building Partnerships Accomplishments (2) • Society for Clinical and Translational Science 1st Annual Meeting, April 2010 • Child Health Symposium - Life Span Research (Shari Barkin, Bill Hay, Deb Freidman) • CTSA Consortium Steering Committee (CCSC) • PI liaisons (Lisa Guay-Woodford, Ron Sokol, Jim Heubi) • Participation on SGC’s • Participation at annual meeting – October 2009 • Presentation on monthly CCSC phone calls • NIHCD • Multi-site IRB review • BPCA Administrative supplements • Integrating CC-CHOC Working Groups with KFC’s • PREW – Research Ethics • Rare Diseases • CTSA Consortium Management Group (CMG) • Tesheia Johnson appointed as CC-CHOC liaison with CMG and Administrative KFC • 1-2 volunteers from Administrative KFC will assist CC-CHOC in establishing milestones and tracking projects

12. CC-CHOC Matched to KFCs

13. Building Infrastructure Accomplishments • Distributed Biobanks for Rare Disease Research (2008 Admin Supplement) https://ctsawiki.org/wiki/display/Peds/Rare+Diseases+Workgroup • Curriculum Working Group developed core competencies for child health research • T2 WG creating inventory of existing PCRN’s • NICHD conducted workshops (Feb 2009) to develop collaboration infrastructure for product development • PREW established ethics consultative service

14. Developing Standard ApproachesAccomplishments • In partnership with NIHCD, CC-CHOC coordinated application process for administrative supplements to improve pediatric outcome measures • 48 applications (all were multi-site) • 18 awarded from 17 CTSA sites – total $8.5 million • When collaborating sites are included, 33/46 CTSA sites are participating in or benefiting from these awards • Linked to Best Pharmaceuticals for Children Act (BPCA) of 2002

15. BPCA Awards – A National Partnership with NICHD WA ME MT ND VT MN OR NH ID WI NY SD MA MI RI WY CT IA PA NJ NE NV OH IN DE IL UT MD WV CO VA KS MO CA KY NC TN AR AZ OK SC NM GA AL MS TX LA AK FL HI Participating Institutions BPCA awardees CTSAs (Sept 2009) 15

16. NICHD BPCA Funded CTSA Multi-Site Projects* * Each project must include > 3 CTSA sites 16 Topic Areas: 8-neonatology, 5-neurology, 4-hypertension/hypotension, 3-“other”; >1 topic area possible

17. BPCA AwardeesPlans for 2010 • Completion of projects • Submit abstracts to PAS – Fall 2010 • Participate in 2011 PAS Symposia and Workshops

18. Identifying and Overcoming BarriersAccomplishments • Multi-center IRB review – collaboration of PREW and NICHD • Sponsored workshops 2007-2009 to identify IRB inconsistencies • Developed proposal for a federated IRB model • Presented to SACHRP, July 2009 • Presented to OHRP, 2010 and accepted • Template memoranda of understanding (MUO’s) developed and distributed to NCS sites, BPCA admin supplement PI’s • Multi-center Scientific Review Committee • Proposed by Drugs and Devices WG (Carole Marcus) • Plan to use MUO’s similar to IRB proposal • Completed survey to understand current CTSA “landscape” regarding use of SRC’s

19. Set Priorities and Enhance Child Health • Conducted first Metrics of Success survey to establish benchmarks for child health across CTSA’s • Encouraged child health representation on CTSA External Advisory Boards • Reviewed by CCSC? • Can we assure child health representation on study sections for CTSA grants?

20. Challenges (1) • Communications • Do the quarterly webinars help you? • How can we better communicate? • Engagement of CC-CHOC membership • How to get involved • Working Groups • Key Function Committees • Operations Group • Send ideas to Chair/Chair-Elect • Promote child health research at your site • Collaborations with Pediatric Clinical/Research Networks (PCRN) – comments from 2009 PCRN R-13 Conference regarding CTSA’s • “No national mandate for pediatric research exists” • “Support for new networks is limited” • “PCRN experience with collaboration could help CTSA’s which have limited experience with collaboration” • “CTSA’s should not form their own PRCN’s”

21. Challenges (2) • Linkages across CTSA Key Function Committees • Pediatric membership on KFC’s is increasing • Conduit for communication not established • Cost-sharing models • How many sites are charging investigators? • Impact of cost-sharing on CF research • What works? What doesn’t? • Supporting young investigators in CF research • Can they afford CRC usage? • Publication citation for child health research • Most studies are multi-center network trials • Journals frequently limit citations • Networks limit citations • CTSA’s are frequently not cited in PubMed

23. Future Annual MeetingsPAS Alliance • Advantages • Annual venue for meetings (free meeting rooms) • International visibility to child health research community • Platform to present CTSA accomplishments • Enhance collaborations and partnerships with other groups (e.g., PCRN’s) • Linkage to young investigators in child health research • PAS administrative staff are extremely helpful • Challenges • Different venue each year outside Washington DC • Some child health specialties do not participate in PAS • Limited access to allied health specialties (e.g., nursing, social work, nutrition) • Competing with more established research groups for audience (e.g., neonatology, nephrology, etc) • Potentially competes with newly established Society for Clinical and Translational Science annual meeting

24. Annual Planning ProcessPAS – CC-CHOC Meeting • PAS (Chair, CC-CHOC serves as PAS liaison) • May: Draft symposia proposals • June: Submit symposia proposals • July: PAS Planning Committee (Chicago) to choose symposia and form review committee for abstracts • September: Request for abstracts released (due November) • Sept/Oct: Encourage CTSA members to submit abstracts • Sept/Oct: Ask symposia speakers • December: Abstract review and ranking • January: PAS Planning Committee (Texas) to finalize program • February: Finalize program and “track” for CTSA • CC-CHOC (Operations Group) • Fall: Develop workshop concept and speakers • January: Develop business meeting agenda • (New 2010): Review process for fellows

25. Moving Forward • Establish a planning committee • Committee Chair – OG member? • Develops symposia proposals • Manage and review abstracts • Develops R-13 supported workshop • Consider establishing a PAS liaison position • Sits on OG • Serves as member of PAS Planning Committee • Serves on CC-CHOC Planning Committee • 2-3 year term • Comments and questions about face to face meeting • Should it stay with PAS? • Is it the right length? • Longer business meeting?

26. Goals for 2010-2011

27. Two Year Goals Multi-center IRB model implemented and tested on several trials Institution specific metrics of success benchmarks established Established prioritization models for pediatric drugs and devices Establishment of a virtual biorepository for utilization by rare disease groups Contribute child health core competencies to the SGC standard curriculum 27

28. Seven Year Goals National infrastructure including data management tools, support services and policies for networks to conduct multi-center trials More emphasis on life span research with collaborations across all ages (child to adult health) to focus on prevention of adult disorders and better defining antecedents of adult disease (e.g. obesity) Additional metrics of success that include the impact of research on improved child health Increased number of young faculty developing careers in pediatric clinical and translational sciences Strong emphasis in child health across all CTSA’s 28

29. 20 Year Goals Robust child health research community with senior faculty in leadership positions and junior faculty eager to develop their careers in clinical translational science Personalized medicine with focus on prevention of childhood disorders and childhood antecedents of adult disease (e.g., obesity, hypertension, cancers). Research studies spanning the entire lifespan from children to adults (already done in CF) Established minimally invasive, cheap and valid outcome measures that are age-specific, thereby increasing the speed and efficiency of pediatric drug/device development More precise understanding of what is a “normal” child both in terms of genotype and phenotype Better health for children worldwide as a result of successful research discoveries moved into action 29

30. Thoughts from the Departing Chair (1) • Improve communication and engagement across CC-CHOC • Every CC-CHOC member should be participating on either a working group, OG or KFC • Establish a better communication linkage across committees • A concise quarterly report summarizing activities of all WG’s and KFC’s • Develop 2-3 goals or targets to benchmark from Metrics of Success Year 1 results • Encourage CC-CHOC endorsement of NICHD MUO’s for multi-site IRB review process for multi-site child health studies using CTSA resources • Consider same process for SRC reviews • Develop clear “deliverables” to encourage clinical trial networks to collaborate with CTSA’s • Educational opportunities • Informatics and biostatistics • Pilot funding • Collection and access to biologic specimens • Clinical Research Associate pool • Centralized and efficient IRB and SRC reviews

31. Thoughts from the Departing Chair (2) • Publications – we need them!!! • Consider establishing a Publication WG • Publish Best Practices • Resolve citation issue with PCRN’s • How do we remain financially sustainable? • Share revenue models • Work with Children’s Hospitals (NACRI) • Develop other funding sources (e.g., Admin Supplements)