Goals & Priorities Dr. Shari Barkin, Chair (Vanderbilt)

1. Goals & Priorities Dr. Shari Barkin, Chair (Vanderbilt) Dr. William Smoyer, Chair-Elect (Nationwide Children’s Ohio)

2. CTSA Consortium Child Health Oversight Committee (CC-CHOC) Mission • Provide a unique national forum to identify collaborative opportunities to facilitate child health clinical and translational research • Build partnerships (e.g., PAS, SCTS, NICHD) • Build infrastructure (e.g., shared resources) • Identify and overcome barriers (e.g., federated multi-center IRB review) • Set priorities and increase visibility for child health research across CTSA Consortium • Improve child health outcomes through high quality science

3. Pediatric Academic Society (PAS) Program 2011 • CC-CHOC is an alliance organization with PAS • Face-to-face CC-CHOC Meeting at PAS (Friday, April 29, 2011) • Participate on PAS Program Committee • Provided with two symposia and two abstract platform sessions • R13- multi-year conference grant: Year 2 theme- community engaged research (held in conjunction with the PAS) • April 29th: Workshop on state of the art methods and outcomes • Selection of 2-4 child health clinical and translational research fellows • PAS Symposium on the topic of state of the art methods and outcomes • Abstract Platform session highlighting community engaged research

4. R13: Year 2 Community Engaged Research April 29th Workshop • 1:00 - 1:45pm Conceptual Model of Community Engaged Research (Szylagi) • 1:45 - 2:30 Towards Best Practices in Community Engaged Research (Ross) • 2:30 - 2:45 Q&A • 2:45 - 3:00 Break • 3:00 - 3:15 Community Partner (Widman) to present on their view so attendees can see what it looks like • 3:15 - 4:15 Panel discussion (O’Lonergan moderating, discussants: Mosley, Kemper, Barkin, Coyne-Beasley, Widman): How have these best practices been used. • 4:15 - 5:15 4 Fellows present (15 min presentations) • 5:15 - 5:45 Interactive Q&A

5. Congratulations to the 2011 Child Health Clinical and Translational Research Fellows • Dr. Patel Mentor: Dr. Lee Sanders, University of Miami • Dr. Bender Mentor: Sheila Gahagan, University of California San Diego • Dr. Tyler Koep Mentor: Dr. Charlie Huskins, College of Medicine Mayo Clinic • Dr. Cassandra Neureiter Mentor: Dr. Shari Barkin, Vanderbilt

6. CC-CHOC PAS Program –2011 • Annual meeting – April 30 – May 3, 2011 Denver, CO • CC-CHOC Topic Symposia • CC-CHOC Workshops • CC-CHOC Abstract Sessions • Community-Engaged Research: Original Science: Saturday • Clinical and Translational Research: Science in Action: Sunday

7. CC-CHOC PAS Program – Symposia 2011 • CTSAs and Community Engaged Research (Chairs: Kemper and Perrin) April 30th 10:30-12:30 • Study Designs in Community-Engaged Research (Syilazyi) • Salud Con La Familia (Health with the Family): the Power of Community Engaged Research Outcomes (Barkin) • Pediatric Comparative Effectiveness Research and Community-Engaged Research (Leslie) • Community-Engaged Research Across the CTSA Consortium (Sergio Aguilar-Gaxiola, MD, PhD) • Best Pharmaceuticals for Children Act (BPCA) and Opportunities for CTSA Collaboration (Chairs: Hirschfeld and Kaskel) May 1st 1-3 • Qualified outcome measures to accelerate research into pediatric therapeutics • Presentations by 4 selected awardees

8. CC-CHOC PAS Program – Workshops 2011 • CTSA Child Health Oversight Committee: Resources and Training in Core Competencies for Clinical and Translational Research: A Guide for Residents, Fellows and Early Career Faculty (Chairs: Byrne and Mendonca) • Study Design in Clinical Research: Getting it Right (Greene) • Biorepositories, Informatics and Data Sharing Platforms (Puck) • Biostatistics in Small Subject Populations (FDA speaker) • Ethics in Pediatric Clinical Research (O’Lonergan) • Genetics and Genomics (Hobbs) • Comparative Effectiveness for Pediatric Trainees (Vassan) • CTSA Child Health Oversight Committee: Management of Clinical Research Data: Focus on Registry and Longitudinal Studies (Chairs: Byrne and Hobbs) • 4 speakers, some speakers from 1st workshop will be switched to this session

9. CTSA Consortium Child Health Oversight Committee (CC-CHOC)Quarterly Webinar:Elections & Governance February 4, 2011

10. Current Election Guidelines

11. CC-CHOC Governance 1 Voting Member from each CTSA 55 maximum, Currently 51 (not everyone has a “voting” member) Chair and chair-elect Elected at-large during annual F2F meeting Represents CC-CHOC to the CCSC (Steering Committee) NIH members Up to 1/3 of membership, appointed, multiple IC’s, intramural & extramural staff Other non-voting members include subject matter experts and informational copies

12. CC-CHOC Operations Group (OG) Conducts day-to-day business of the CC-CHOC Currently has 6 elected voting members Chair Chair-Elect 4 at-large members NIH Coordinators (2, Appointed) Other participants and ex Officio Members PI Liaisons CMG Liaison Past Chairs (optional) Workgroup chairs (optional)

13. CC-CHOC Leadership Terms and Guidelines Chair position is a 2 year commitment, with an option to serve an additional year as an ex officio member Chair-Elect (1st year) Chair (2nd year) At-large OG members serve 2 year terms, with half ending every year to provide continuity Not more than one elected member may be from a single CTSA site 2011 election will be held during the beginning of April 2011 (2011 PAS Meeting: April 29 – May 3)

14. CC-CHOC Governance proposed changes CTSA Consortium Governance Manual is flexible regarding leadership of CTSA Committees: http://www.ctsaweb.org/Docs/CTSA_Governance_Manual.pdf Current CC-CHOC Operations Group (OG) came into existence in June 2008 When N = 38 CTSAs CCEC has grown as CTSA Consortium has expanded Proposal: Number of OG members elected at-large should be increased proportionate to Consortium Add 3 positions to OG Two this year (2011) and 1 in 2012 (N = 60 CTSAs)

15. INTERACTIVE QUESTION: Would you favor an increase in CC-CHOC Operations Group elected members by 3, to include 2 in 2011 and 1 in 2012?

16. CC-CHOC Operations Group Members Roles and Responsibilities • Attend monthly teleconferences • Assist Chair/Chair-Elect in implementing CC-CHOC initiatives and priorities • Bring new ideas and initiatives to OG • Plan Spring Meeting • Review abstracts • Lead PAS sessions • Serve as liaisons to other KFC’s or CTSA Consortium activities

17. Chair and Chair-Elect Roles (1) • Close collaboration between two positions to implement new initiatives • R-13 application for Spring Conference • Metrics of Success • Help set annual goals for the CC-CHOC at f2f meeting and implement during the year • Liaison to CTSA Consortium Steering Committee • Monthly presentation on teleconferences • Annual presentation at f2f meeting • Work closely with PI liaisons on CC-CHOC • Champion collaboration of child health with other consortium committees (KFC’s, SGC’s)

18. Election Process 2011 • Electronic process in March/April • Results announced at CC-CHOC F2F Meeting April 29 • 2 (or 4) CC-CHOC OG members and Chair-Elect position are open • Submit nominations for OG member or Chair-Elect to Ashley Harris by March 15 • 1 paragraph biosketch and personal statement • Recent photograph • Ballots distributed April 1 and due April 15 • One voting member/CTSA • Questions? • E-mail Ashley Harris harris_ashley@bah.com

19. Dr. Shari Barkin

20. Dr. Mary Purucker

21. Background Scientific Management Review Boards (SMRB) Advisory body authorized by Congress Recommend changes in NIH structure to NIH Director 12/7/2010 Recommended the creation of a new IC to be a “hub” of translational science for the NIH Proposed name: National Center for Advancing Translational Science or NCATS

22. NCATS- Proposed Structure SMRB recommendations Clinical component: Clinical & Translational Science Award (CTSA) Program “Close” interaction with NIH Clinical Center Non-clinical components MLP Molecular Libraries Program RAID Rapid Access to Interventional Development TRND Therapeutics for Rare and Neglected Diseases CAN Cures Acceleration Network MLP, RAID, & TRND are existing programs that will be moved from elsewhere in NIH CAN is authorized under ACA, but not yet funded

23. NCATS-Timeline 12/7/2010 NCATS recommended by SMRB 1/14/2011 Recommendations sent to Congress by Secretary Sebelius Included proposal for new NCATS and dissolution of NCRR Congress has 180 days to decide whether to allow proposal to proceed or to take action (about mid-July) 10/1/2011 Start of FY2012 or date by which NCATS is to be stood up Unknowns include NCATS director, mission, other co-located programs, and fate of most other NCRR programs. Stay tuned.

24. Metrics of Success • Metrics developed by Child Health Research Metrics of Success Workgroup to :(1) identify consistent ways in which an institution can track its own child health research trajectory over time, and (2) measure the impact of the CTSA on child health related research. • Workgroup members:Mary Aitken, Arkansas Steven Alexander, StanfordLeon Epstein, Northwestern Alejandro Hoberman, PittsburghMargaret Hostetter, Yale Charles Huskins, MayoEllis Neufeld, Harvard Todd Nick, ArkansasAnthony Philipps, UC Davis Thomas Shanley, MichiganPeter Szilagyi, Rochester

25. Methodology (1) • Referencing NIH definitions for clinical research, the Workgroup defined “Child health research” as “all clinical and translational research involving subjects < or = 21 years of age that impacts children’s health or addresses childhood diseases. This includes maternal-fetal research, as well as studies involving infants, children and adolescents”. • Intended that only studies in which human subjects were involved should be counted (“touching people research”).

26. Methodology (2) • Metrics definitions circulated in June 2009 • Pilot project launched in October 2009. Collection of data from CC-CHOC CTSAs by March 2010 included in the database • REDCap database created by Vanderbilt Institute for Clinical and Translational Research (VICTR) to allow data entry from each CTSA site • Quantitative and qualitative data collected on each metric • Manuscript currently being written to summarize our findings

27. The Starter Metrics • Absolute CTSA grant dollars directly supportive of child health investigators as a proportion of the total CTSA budget • IRB approved studies involving pediatric subjects as a proportion of all IRB approved studies and those that are funded • Total of child health trainees and faculty who have received a mentoring grant • Number of pilot grants in child health research as a proportion of all funded pilot grants funded by the CTSA mechanism

28. Starter Metrics (2) Total inpatient and outpatient visits utilized for child health research of total GCRC days. Number of child health studies conducted in collaboration with other CTSAs Proportion of researchers in substantial CTSA leadership roles who are child health researchers Other ways child health research has benefitted from CTSA support/infrastructure Number of child health research publications that cite CTSA grant #

29. Year 2 Pilot Objective: Allow benchmarking at the level of the institution in support of child health research Next steps: • Present to the CTSA Steering committee Feb 15th • Institutions receive a REDCAP password to enter their data • Dovetail with data collection for annual reports • Submit data by April 1st.

30. INTERACTIVE QUESTION 1: Are you familiar with the ResearchMatch program?

31. INTERACTIVE QUESTION 2: How likely is your center to use ResearchMatch? 31

32. INTERACTIVE QUESTION 3: How interested are you in using/learning about ResearchMatch? 32

33. CC-CHOC Priorities for 2010-2011 (1) • Moving clinical trials to completion • Develop demonstration project in partnership with community engaged research/T2 workgroup • Contact: Shari Barkin, Alex Kemper • Establish federated multisite IRB process • Pilot underway in partnership with NCS • Contact: Steven Hirschfeld, Jon Davis

34. CC-CHOC Priorities for 2010-2011(2) Publish 2-3 CC-CHOC peer reviewed manuscripts Potential topics: Child Health Metrics, Rare Disease Biorepository (process for sharing), Peds Drug/Device Development: Position Paper Contact: Jon Davis, Jennifer Puck, Shari Barkin Impactful Science Report child health research supported by CTSAs with high translation value on a semiannual basis Contact: Shari Barkin, Bill Smoyer Disseminate CTS core competencies for child health researchers PAS Workshop Contact: Barry Byrne, Rick Kaskel, Eneida Mendonca Best Practices Contact: Bill Smoyer 34

35. CC-CHOC Organizational Structure CC-CHOC Working Groups conference calls Pediatric Drugs and Devices (Davis, Hagler) Pediatric T2 Research (Kemper, Mendonca) Metrics of Success (Barkin) Pediatric Research Ethics (Hirschfeld, O’Lonergan) Rare Diseases (Byrne, Merkel, Griggs) Pediatric – Adult Life Span (Hay, Perrin) Core Competencies (Byrne, Kaskel) Exercise Ontology (Cooper) Comparative Effectiveness Research (CER) Liaisons (Boylston-Herndon, Hsu) 35

36. What can you do to participate? • Ensure 1-2 child health research members from your CTSA attend the April 29th CC-CHOC meeting • Participate in CC-CHOC workgroups • Nominate someone from your CTSA for the CC-CHOC Operations Group Committee

37. Dr. Don Hagler Dr. Jon Davis

38. Data Workshop • Currently Cancelled because of limited time for preparation • Awaiting plans to develop new Translationals Research Center about Oct 2011 • Will attempt to reschedule in Fall after October

39. Federated IRB • Steve Hirschfeld efforts making progress • Some utilization for National Children’s study group • Neonatal research – Jon Davis

40. Discussion with FDA • Jon Davis spoke with Diane Murphy at FDA • Emphasized efforts to expand Neonatal Research • Planned NEJM article discussing barriers to Neonatal research • Discussed early input to FDA • Note Request from FDA for applications to Orphan Device Consortia Grant

41. Dr. Theresa O’Lonergan

42. Pediatric Research Ethics Working-group • The PREW is now advertising the consultation services that we are able to provide to CTSA members in the area of pediatric research. • A document is now on the PREW website and we will be sending it to various KFCs etc.

43. Pediatric Research Ethics Working Group Consultation Service The CTSA Child Health Oversight Committee (CC-CHOC) is one of the two CTSA consortium leadership groups and is focused on research in pediatric populations. The Pediatric Research Ethics Working Group (PREW) operates within the CC-CHOC. The PREW is comprised of individuals who have been chosen to represent their CTSA site in our working group. Several PREW members are also members of other working groups and key function committees. The goal of the PREW is to encourage consistency and clarity in addressing the ethical issues encountered in the conduct of clinical translational research in pediatric populations. While local CTSA sites or their institutions may have ethics resources available, the PREW can provide ethics consultation about specific issues related to research in pediatric populations. Our committee includes some the most respected and knowledgeable experts in pediatric research across a broad range of clinical and research expertise. Consultation with the PREW will also facilitate documentation of frequently encountered ethical issues and create a body of recommendations that will serve as a point of future reference. In addition to the PREW ethics consultation service we can also provide assistance in the development of research projects focused on research ethics. Several of our members have conducted this type of research and have valuable expertise. The PREW is also a source for investigators seeking collaborators for their research projects that have ethics components. To seek consultation or ethics research expertise from the PREW you may contact any of our members or the current Co-Chair of the PREW, Terri O’Lonergan (720-777-8529). See the schedule of future meetings as well as summaries of past meetings.

44. Pediatric Research Ethics Working-group • We will be presenting a TowardBest-Practices session on ethical issues in community engaged pediatric research at the CC-CHOC face-to-face meeting at PAS in 2011 • Lainie Ross will present from 3 papers a group of CTSA members have published • Followed by a panel discussion or researchers and a research partner

45. Tesheia Johnson

46. Questions? Thank you Chair: Shari Barkin shari.barkin@vanderbilt.edu Chair-Elect: Bill Smoyer william.smoyer@nationwidechildrens.org