Pediatrics Oversight Committee CTSA Informal Introductions

Bethesda, Maryland

June 23, 2008

Columbia University Medical Center

(Schools of Medicine, Public Health, Dental and Nursing)

Facilities: 1135 beds

Pediatrics: Children’s Hospital of New York with 200 beds

Training: Full range of pediatric specialties with 60 residents, 63 fellows and 155 medical student rotating annually

CTSA funding/year: K12 Program—2/8 Pilot Grant Award—2 /8 MS/POR—2/10

Irving Fellow—1 Irving Scholar—1 Imaging Pilot Award—1 Dedicated Research Staff—4

Goals:

Training, interdisciplinary collaboration, Biostatistics and IT, Community based research

Current Barriers:

Insufficient trained personnel, shortage of statistical support, communications across multiple schools and departments, too few patients in particular areas

Potential Solutions:

Standardized training and certification of research staffResearch Nurse/Coordinator—3 (ER, PICU, NICU)Multi-CTSA collaboration (NYC CTSAs)Expansion of Staff in Biostatistics and IT for courses and

consultationPrimary and Specialty Care Collaboration

Duke University Health System/Duke Translational Medicine Institute

CTSA PI: Robert M. Califf MDChair of Pediatrics: Joseph W. St. Geme III MDVoting member: Jennifer S. Li MD MHS

Facilities: 3 Hospitals with 1498 total/181 pediatric bed capacity; home care and hospice services; primary and specialty care clinics incommunities throughout the region

Pediatric department centralized at Duke Children's Hospital and Health Center, 158 faculty members, 19 comprehensive specialties

Training: 73 residents, 55 subspecialty fellows, 101 students/yr on rotation; institutional and departmental K12 awards, T32 awards in the department and in pediatric cardiology, neonatology, A/I, heme-onc, rheumatology

Research: 293 active protocols, 10 NIH sponsored pediatric networks, data coordinating center for 24 pediatric multi-center trials•Components: Duke Clinical Research Institute, Neonatal Perinatal Research Institute, Pediatric Site-Based Research Unit, Pediatric Genetics and Genomics Research Center.Pediatric Vaccine Unit, Pediatric Cell Therapy Unit, Quantitative Sciences, Community Research, Pediatric DCRU (phase 1 unit) 

Goals: “To enhance the health of children by fostering therapeutic discoveryTo enhance the health of children by fostering therapeutic discoverythrough collaborative translational and clinical research”through collaborative translational and clinical research”

•Expand the therapeutic breadth of Pediatric Phase 1 studies •Expand Community component•Develop Neurodevelopmental Outcomes Research Unit•Develop targeted cell therapies for inherited metabolic disorders, Develop targeted cell therapies for inherited metabolic disorders, brain injuries, and type I diabetesbrain injuries, and type I diabetes••Expand Expand FDA-DCRI collaboration for pediatric drug trial analysesFDA-DCRI collaboration for pediatric drug trial analyses

Current Barriers: 1) achieving the proper balance between specific pediatric needs and integration of research within the overall institutional effort, 2) ensuring that efforts in health services research are complementary to the operations of the health system, 3) recruitment and retention of faculty(too few in the pipeline), 4) delays to intellectual property issues and contracting5) $$$

Potential Solutions: develop academic and industry partnerships; coordinate localand intra-CTSA research programs; collaborative informatics, terminology, and operations

FacilitiesMayo Eugenio Litta Children’s Hospital 90 beds (16 NICU, 30 PICU, 44 general care)Mayo Clinic T. Denny Sanford Pediatric Center comprehensive, multispecialty pediatric clinicClinical Research Unit – inpatient, outpatient, & mobile units serving adults & children

Training programs PhD, Masters Degree, & Certificate programs

Research Basic, translational, & clinical research, with emphasis on cancer, aging, behavioral & developmental biology, immunology, CV & GI conditions, vaccinology & infectious diseases

Mayo Clinic

W. Charles Huskins, MD, MSc

Mayo Eugenio Litta Children’s Hospital

Mayo Clinic T. Denny Sanford Pediatric

Center

Robert M.Jacobson,

MD

GoalsExpand ranks of experienced translational investigatorsEnhance multidisciplinary translational research, particularly for musculoskeletal, gastrointestinal, & neurologic conditions

Current barriers Small but growing cadre of pediatric investigators Small but growing community with limited diversity Community engagement programs just being initiated

Potential solutions Enhance research education & training opportunitiesCollaborate in multicenter studies, particularly those involving conditions affecting children and adultsExpand community engagement programs

Mayo Clinic

Oregon Health & Science University (OHSU)

Facilities:

Doernbecher Children’s Hospital:151 beds OHSU Hospital: 373 beds. Also VA, Shriners Pediatric Department located at Doernbecher Children’s Hospital on main campus of OHSU connected to OHSU Hospital; 70 facultyTraining: Nearly full range of pediatric specialties with fellowship training-24 fellows currently in training. Medical student training: 190 students per year on rotation and electiveCTSA Research: CTRC: 22 pediatric protocols from 10 PI’s; many additional pediatric studies utilizing CTSA related services

Goals – Education & Training: Train a cadre of early career pediatric C/T researchersTranslational research in metabolic disease/nutrition/genetics, endocrinology, oncology, renal; expand into cardiology, infectious disease, neuroscience. Expand on strong base in community research

Current Barriers : Few senior mentors in C/T research, relatively small patient base in some areas, PI’s too busy trying to renew basic science grants to consider developing new C/T research collaborations

Potential Solutions : enlist assistance of senior mentors from outside department, collaborate with other institutions in research subject recruitment, form teams of basic scientists and C/T researchers especially with internal funding opportunities to incentivize new C/T research collaborations

Rockefeller University Hospital, New York, NY

First free-standing clinical research center, established 1901

Inpatient unit with 20 bed capacity + outpatient unit, both devoted solely to clinical research protocols

Organized by individual labs rather than academic departments

Training: CTSA Clinical Scholars Program grants Master’s in Clinical and Translational Science (16 scholars as of July 1, 2008)

Research: 99 active protocols in the areas of HIV, dermatology, addiction, obesity, oncology, hematology, and immunology

11 protocols include volunteers under the age of 18

None of the active protocols are focused exclusively on children,although RU has a history of pediatric research in the past.

Sandhya Vasan, M.DVoting Member

Goals:

To expand the scope of current pediatric researchTo continue to improve training for new translational investigators

Current Barriers:

Small research hospital with no pediatric department

Potential Solutions:

Recruitment of new pediatric faculty (e.g., Jean-Laurent Casanova) Collaborative studies with other CTSA centersActive tracking and feedback on K12 Clinical Scholars Program

Anthony Philipps, M.D.

University of California, Davis Medical Center•One of 5 UC Schools of Medicine •Location: Sacramento State Capital; Davis campus 20 miles w. •FacilitiesFacilities: 600 bed acute care hospital, level I Peds Trauma

•Children’s Hospital (Hospital within hosp)120 beds•Dept Pediatrics integral part of Med Center: 90 faculty•Other academic Depts in support (Radiol, Anesth, Surg, etc)•Shriners Hospital (multidisciplinary) on campus and active collaborator

•TrainingTraining: medical students, Pediatric residency program, residents from FP, Travis AFB and 5 fellowship programs•ResearchResearch:

•Research labs and clin programs >100 active protocolssome clin themes incl Frag X, Ca, Autism, Ethics, Diabetes, Vaccines

•Wet labs with themes Obesity, Asthma, Renal, Nutrition, GeneticsStem Cell/Cancer, Infect Dis.

–UCD campus collaborators: Primate Ctr, Vet and Agric Schools, Ctr Health and Tech (Telehealth), MIND Inst, and others

• GoalsGoals– Improvement in “brokering” relationships between clinicians, “bench” scientists

and industry and technology– Acceleration of multicenter and local clinical trials– Training of competent clinical research scientists for the future

• Current BarriersCurrent Barriers– Local and regional competition for, “town and gown” political problems – Fiscal crises: federal, state and local county – Faculty time and effort, P and T committee issues– Space limitations: labs, offices– Administrative barriers and red tape- internal and UC-wide:

• Contracts, liability, Intellectual property rights• IRB

• SolutionsSolutions– Partnering (Shriners. LLNL examples), web based dialogue both internal and external– Local Symposia as showcase and forum for discussion and exchange of ideas– Use of K30 program and K12 grants (2 currently In Pediatrics) coupled with CTSA

funds to develop new clinical investigators, funds for Peds Clinical coordinator

UCSF CTSI Pediatric Clinical Research Center

UCSF, Children’s Hosp of Oakland, Kaiser Oakland, SFGH, Mount ZionPCRC Advisory Committee reviews all protocolsAccess through CTSA to Bioinformatics, Statistics, “SOS” pilot funds

UCSF Independent PCRC for 30 years prior to CTSA, pioneer in pediatric HIV Currently a site of the UCSF CTSI CRC 5-bed inpatient unit with lead-lined room Outpatient unit and infusion center ICU research nursing team covering: Neonatal, Cardiac and Pediatric ICUs,

Transitional and Well Baby nurseries, L&D, OR, Imaging & Radiology

Specimen Processing Lab, shipping, EBV lines

Neurodevelopmental psychologist, full time

Nutritionist, DEXA, body composition & exercise testing

Pediatric Research Pharmacy

Poster printer

UCSF CTSI Pediatric Program Goals and Challenges

1. Integrate Research into the design of the new UCSF Children’s Hospital, currently planned to open 2012-2014.

2. Increase the pipeline of pediatric translational investigators.

3. Broaden the scope of research conducted in the PCRC.

4. Make funds and research coordinator support available for starting projects that will lead to competitive funding applications.

5. Participate in more multi-institutional studies, we hope by taking advantage of CTSA infrastructure.

J Puck, June 18, 2008

Children’s Hospital of PhiladelphiaUniversity of Pennsylvania

• 430 bed free-standing hospital (CHOP) affiliated with University of Pennsylvania

• Developed by merging 2 GCRC’s• $129,708,000 of extramural funding in FY 07• 180 current pediatric CTSA protocols• 21 pediatric divisions / departments

• Goals: Translational therapeutics; Bridging the pediatrics / adult divide; Education and investment in human capital

• Barriers: Transforming culture; Uncertain funding climate; Lack of support for pilot studies

• Solutions: Joint roles; Charge-back mechanism & institutional support; Pilot grants

• Goals for POC: Establishing strong pediatric networks for research and advocacy

Research:- 76 active pediatric protocols, from 17 sub-specialties (PCTRC) - 13 active pediatric protocols, from 5 sub-specialties (PBRN)

Training: Unified Fellows Course; 3 year program tailored to the professional development of fellows in the Department of Pediatrics.

The overall goal is to assist fellows in making a successful transition from fellowship to early faculty positions as physician-scientists, clinical researchers and academic educators in their subspecialties.

University of Pittsburgh Clinical and Translational Science InstituteParticipant and Clinical Interactions Resources Core (PCIR)

• Pediatric Clinical and Translational Research Center (PCTRC)• Pediatric PittNet (Practice Based Research Network)

Goals: - Reduce barriers to investigators

- Expand practice based research

Current Barriers: - Regulatory, IRB issues- Parental concern about participation of children in clinical research

Potential Solutions:- S. Arslanian (Dir. PCTRC), D. Perlmutter (Dept. Chair) and C. Ryan (IRB Chair) formed Advisory Committee to address local barriers- Participation in POC to address IRB barriers and facilitate pediatric research

- A. Hoberman joined PCTRC, Advisory Committee and POC to provide expertise in practice based clinical research. Developed multicenter survey to assess factors that influence parents decisions to consent to research

University of Pittsburgh Clinical and Translational Science InstituteParticipant and Clinical Interactions Resources Core (PCIR)

• Pediatric Clinical and Translational Research Center (PCTRC)• Pediatric PittNet (Practice Based Research Network)

Department of Pediatrics, Rochester NY

Clinical Facilities: - 2 hospitals, all pediatric subspecialty care centralized- 2 large primary care practices (40% of city children) - Incredible town-gown relationship

Research- Major health services research program (50% of total)- Large programs in ID, neonatology, renal, neurology, heme-onc, others - Altogether > 100 protocols

Teaching- 40 residents, 100 students, community pediatrics

Department of Pediatrics, Rochester NY Current CTSA Activities- Pediatric researchers involved in many of the 11 functions. - Pediatric faculty have leadership positions in:

-Administrative Core-Community Engagement (Co-PI on PBRN, NIH suppl.) -Research Education and Career Development-Upstate Consortium

Goals- More funding from CTSA programs- Improved centralized research office for studies (fieldwork)

Barriers- IRB, IT for EMR for PBRN, Communications, Funding

Solutions- Work collaboratively within CTSI

• Goals– Advance basic, translational, and clinical science

for children• Barriers

– Competing priorities, awareness, researcher “pipeline”, absence of industry partners, ethical concerns, small sample size

• Solutions– Skills development programs, pediatric research

infrastructure, leveraging resources, collaboration across NIH Institutes, CTSAs

Oakland Children’s Hospital (UCSF)

• Facilities:– Two outpatient units– 240 inpatient beds; Day Hospital– Pharmacy/processing laboratory– SQUID,DEXA,Nutrition Cart– Diverse patient population, Sub-

specialty programs, 5 fellowships and 75 residents

– T32 and K training programs– University affiliations: UCSF, UC

Berkeley, UC Davis– Extensive genomic, biochemistry,

cell biology laboratory resources– Focus on translational research

and training– School based programs

• Research– Seven “Centers of Excellence”– Ranked #5 Nationally in NIH funds

in 2007– 400 clinical protocols, 40 in

CRC:hemoglobinopathies, oncology, diabetes, obesity, asthma,nutrition, genomics, birth defects, cystic fibrosis neurodevelopment, cord blood cardiovascular, alcohol and substance abuse

– Active participant and leader in NIH clinical trail networks and industry studies

– Pediatric and adult studies– Community oriented programs– Minority internships

Oakland Children’s Hospital (UCSF)

• Goals– Increase translational research

projects– Support development of junior

faculty– Increase training activities– Increase collaborations with other

CTSA programs– Expand community engagement

programs– Expand minority recruitment

activities– Increase participation in NIH

multicenter trials– Continue SEPA programs– Develop community based

programs

• Barriers– insufficient funding to support

staff, junior investigators, ancillaries, or develop new programs

– Can’t accept new protocols– Can’t support junior staff projects– NIH flat budget– Future projections

• Potential Solutions– Funding from philanthropy,

industry, or medical center– Funding from NIH, foundations, or

business community

Case Western Reserve University Pediatrics in the Clinical and Translational Science Collaborative

Three hospitals with Pediatric Units (one a top-5hospital)each has training program and fellowshipsin toto in 14 subspecialties, famous in

neonatology and pulmonaryMedical students rotate at all three hospital systems in

Pediatrics Most popular specialty chosen by graduating seniors

last year. Department with >$30M in external research support

CTSA ParticipationEffective competitor for K12 slots at all the

institutions, plus more than 12 K awards outside CTSA - participate in “K community”

Many K awardees cross disciplines Training component emphasizes K to R transition.

Major CRU user at the site of largest inpatient usage – CF, sleep programs, PPRU site – One CRU located in Children’s Hospital

CTSC plans to upgrade the Pediatric Practice Based Research Network as a new leader is recruited – was active under former leader

Clinical and Translational Research Program for Pediatrics

(CTRPP)Atlanta, Georgia

Emory University - Dept of Pediatrics

Morehouse School of Medicine (MSM) – Dept of Pediatrics

Children’s Healthcare of Atlanta (CHOA)

Georgia Institute of Technology

Pediatric Clinical Interaction Sites

• Facilities

– 452 staffed beds in three hospitals– 23 satellite locations around metro Atlanta– 5 immediate care centers– 2 primary care centers– 30 pediatric specialties

– 493,00 patient visits– 227,000 unique patients– 128,000 inpatient days– 5,800 outpatient days– 34,200 surgical procedures (in-patient and out-patient)– 123,000 ED visits– 117,000 Immediate care center visits– 61,400  Primary Care Visits

• over 700 protocols• 14 protocols using adult GCRC• Building centralized inpt/outpt research unit at

Egleston• Centralized research department at CHOA• Centralized lab at Egleston supporting studies• Research pharmacist (1.0 FTE)

Research

• Establish network of centrally organized pediatric clinical interaction sites, fostering integration and collaboration.

• Encourage basic and clinical scientist interaction, enhancing discovery and clinical implementation, improving children's lives.

• Build a robust centralized research infrastructure dedicated to pediatrics, including a dedicated Pediatric Clinical Trials Office.

• Develop the Child Health Institute at MSM, incorporating research and collaborations with education, patient care, and advocacy, to build collaborative care for children with special healthcare needs.

• Expand and enhance our training program in pediatric clinical and translational research and ensure researchers have the tools to become successful and independent.

• Create a bidirectional process between the Clinical Interaction Sites and the Community Engagement program to disseminate pediatric clinical and translational research results

Goals

• Staffing research unit/hiring of qualified research nurses

• Multiple sites/geography• Multiple IRBs (Emory/CHOA/Grady)• Total research staff (IRB/grants/contracts)• Electronic data/databases• Working to integrate 2 separate research

infrastructures at CHOA/Emory

Barriers

• Funding support from other sources• Accessing Nursing Research Alliance for

qualified nurses• Sharing of resources with other programs to

optimize efficiency• Community engagement

Solutions

Facilities: Johns Hopkins Medical Institutions and the Kennedy Krieger Institute

Johns Hopkins Childrens Center integrated within Johns Hopkins Hospital

Training: Pediatrics internships, residency, subspecialty fellowships, T32 and K12 training programs

Pediatric Clinical Research Unit consisting of 7 inpatient beds, outpatient clinic, procedures unit, infant lung function laboratory, mucociliary clearance laboratory, sleep

laboratory

Johns Hopkins Institute for Clinical and Translational Research---A CTSA program in Baltimore, MD

GOAL1) Enhancing communication across the multiple programs in the ICTR2) Finding best ways to leverage research infrastructure

a. Relationships with various schoolsb. Relationships with cancer center

3) Creating an academic home for translational researchers without a single physical location4) Measuring output or translational research successes5) Identifying most productive approaches to working with other academic institutions within CTSA

CHALLENGEHaving the discipline to evaluate programs and reallocate resources

WE OFFER 1) Basic science and clinical translational forums 2) Translational Research Navigator Program3) Advanced Translation Incubator Program (ATIP)4) Community Engagement Office5) Proteomics, Genetics, and Imaging Cores

AND MUCH MORE………..

University of Chicago• Facilities: 2 hospitals (Comer Children’s and LaRabida Children’s Hospitals)

with 200 beds combined• Training: 54 peds-, 16 med/peds-, and 3 chief-residents; 42 accredited fellowship positions in 9

subspecialties and 2 positions in pediatric surgery• Research

– Laboratory-based research with emphasis on translational applications. For example,• Sue Cohn and her work on angiogenesis in cancer• Bob Daum and his work on MRSA• Mike Moskowitz and his work on the genetic basis of congenital heart disease• John Cunningham and his work on new therapies for sickle cell disease

– Therapeutic clinical trials. For example,• Participation in the Children’s Oncology Group• Ken Alexander and his work in HPV• Mike Schreiber and his work on nitrous oxygen

– Health services research (ethics and outcomes)• Becky Lipton and her epidemiological work on diabetes• Alyna Chien and pay-for-performance health services research• Lainie Ross on expanding newborn screening

– Resources: • New lab space to open in the Knapp Center for Biomedical Discovery • Establishment of Pediatric Clinical Trials Office (1/07) • MacLean Center for Clinical Medical Ethics with expertise in research ethics

– 255 active IRB approved protocols in Dept of Pediatrics

University of Chicago• Research

– Goals: continue T-1 research; expand T-2 research including both community-based participatory research [CBPR] and practice-based participatory research [PBPR]

– Current Barriers: unfunded mandates (our CTSA funding was cut administratively by 50%); inconsistent application of human subject protections across institutions; shortage of statistical support requiring outsourcing; need for external support for biobanking initiatives

– Potential Solutions: Institutional support for pediatric clinical trials office to supplement university-wide clinical trials office; strong institutional support for community-based pilot projects and for biobanking initiatives; seeking outside funding to enrich statistical center as well as collaboration within the University (Health Studies)

University of Iowa CTSA Pediatrics Key Function - 1

Paired with the Genetics/Genomics Key FunctionOnly Medical School in state, 70 Bed NICU, Numerous statewide pediatric programs

(Perinatal, Birth Defects, Nursing etc)

Four Aims1. WIKI based interactions between pediatric faculty/staff/student (Donna D’Alessandro)

2. School nurse outreach to facilitate clinical trials in the community (Ann Marie McCarthy)

3. Perinatal health projects to integrate laboratory with clinical activities including NIH Neonatal Network (Jeff Murray/Ed Bell)

4. Biorepository of data/samples on pediatric in/outpatient population (Jeff Murray)

University of Iowa CTSA Pediatrics Key Function - 2

1. Goals - better internal/external communication; training of school nurses; expand existing biorepository (currently NICU only)

2. Barriers - buy in for use of Wiki’s; resources for school nurse training; capacity building for biorepository

3. Solutions - education, new hospital EMR implementation and work with Bioinformatic key function; partner with College of Nursing and grants for school nurses; working with genetics/genomics core, financial support from the hospital

and College of Medicine to expand biorepository, partner with other CTSAs

Michigan Institute of Clinical and Health Research (MICHR)Michigan Institute of Clinical and Health Research (MICHR)

MICHR CTSAMICHR CTSA: To provide infrastructure and tangible : To provide infrastructure and tangible support to develop, help secure funding, and conduct high support to develop, help secure funding, and conduct high quality clinical and translational research projects that will quality clinical and translational research projects that will positively affect patient care and outcomespositively affect patient care and outcomes

Pediatric Research ProgramPediatric Research Program (PRP): One of 12 programs (PRP): One of 12 programs within the MICHR CTSAwithin the MICHR CTSA

Pediatric Clinical Research CommitteePediatric Clinical Research Committee: Consultative : Consultative committee of the PRP - currently consists of 17 faculty committee of the PRP - currently consists of 17 faculty members with different specialties across divisions with the members with different specialties across divisions with the Department of Pediatrics and divisions with a pediatric Department of Pediatrics and divisions with a pediatric focus in other Departments or Schoolsfocus in other Departments or Schools

1.1. To To develop and supportdevelop and support multidisciplinary clinical and multidisciplinary clinical and translational research teams in pediatric populations translational research teams in pediatric populations

2.2. To contribute to the clinical research educational and To contribute to the clinical research educational and mentoring mission of the UM CTSA mentoring mission of the UM CTSA

3.3. To serve as a To serve as a central point of contactcentral point of contact for investigators for investigators who seek to conduct biological, clinical, epidemiologic, or who seek to conduct biological, clinical, epidemiologic, or health services research with translational potential that health services research with translational potential that involves children, adolescents, and/or their families involves children, adolescents, and/or their families

Pediatric Research Program within MICHRPediatric Research Program within MICHR

• UT Southwestern Medical Center at Dallas

• 7 affiliated hospitals (3 pediatrics-only facilities; 2000 beds)

• Pediatric hospitals– Children’s Medical Center Dallas (411 beds) – teaching– Parkland Memorial Hospital (810 beds) – teaching– Texas Scottish Rite Hospital for Children (100 beds) – teaching– Children’s Medical Center Legacy (72 beds) – non-teaching

• Training– 230 medical students per year– 87 pediatric residents per year– 19 pediatric residencies or fellowships

• Pediatric clinical and translational research– Main players: Hematology-Oncology, Infectious Disease, Psychiatry, Urology– Clinical research department at CMC Dallas– New Department of Clinical Sciences at the University

Charles Quinn, MD, MS

• Goals– Advance the standing of clinical research at the university– Improve the quality and quantity or pediatric research at the university– Train investigators in clinical sciences– Develop an interactive community of clinical investigators

• Current Barriers– History and pre-eminence of basic science research– Small, scattered community of clinical investigators– Lack of integration between the CTSA/University and Children’s

• Potential Solutions– KL2/K12 Clinical Research Scholars’ Program (12/39 scholars are pediatricians)– Integrate the adult and pediatric research infrastructure where appropriate– Increase pediatric funding opportunities– Regular multi-disciplinary meetings of pediatric clinical researchers– Good ideas from this meeting

• Successes of Pediatric Program in Year 1 of UW CTSA– Pediatrics well-integrated into leadership including PCRC, Regulatory Support, Bioethics,

Informatics and Novel Methods– Center for Clinical and Translational Research established at Seattle Children’s Hospital as link

between institution and CTSA– Creating common practices between Children’s and UW – CRC functions, billing guidelines,

common application and IRB reciprocity– CTSA Pilot Program and Mentored Scholars Program for investigators focusing on childhood

disorders launched May 2008 (institutional support from SCHRI)

Seattle Children’s Hospital Research Institute (SCHRI)• Site of University of Washington CTSA pediatric program –

Bonnie Ramsey, Co-PI• $31 million in research funding• Over 400,000 ft2 of research space• 190 Investigators• Growth

– 80,000 ft2 under construction now– Building 2 complete in 3-5 years adding over

500,000 ft2 to the campus– Growth potential to 2 million ft2

Goal• Training – implement Mentored Scholars Program (4 scholars chosen) for

pre-K early faculty • Research support – implement CTSA Pediatric Biomedical Informatics and

Statistics core in Fall 2008; recruit senior biostatistical faculty• Implement cost sharing program for PCRC

Current barriers• Delays in contract negotiations• Data management support• Inability for research support staff to move across UW affiliated institutions

Potential solutions• Utilize Toyota LEAN process to optimize contract negotiations across UW

CTSA institutions• Establish common training/certification of research staff across UW

institutions

UW/SCHRI Partnership for CTSA

•University of Wisconsin-Madison•American Family Children’s Hospital: 60 bed facility opened August 2007•Adult and VA hospitals on same campus (UW-Madison main campus)•Pediatric department centralized at teaching hospital•Full range of pediatric specialties•39 pediatric residents, 15 sub-specialty fellows•Institutional and cooperative group clinical research (Children’s Oncology Group, Childhood Asthma Research and Education)

• Goals: – streamline IRB/CTRC review process– coordinate ICTR resources– provide more “pediatric friendly” services for participants in

ICTR studies• Barriers:

– still in early stages of implementing ICTR plan– some pediatric investigators lack infrastructure for efficiently

conducting studies and meeting participants’ needs• Solutions:

– implementation of ICTR plan is in progress– some pediatric researchers hold leadership roles within the

UW-ICTR and will advocate for pediatric studies and participants

Facilities: Three hospitals; Beds: 916: ; Pediatric CRCPediatric department in stand-alone children’s hospital, as part of larger academic centerTraining: Full range of pediatric specialties with fellowship training; 74 fellows; K12 mentored career developmentResearch: 26 laboratories; 52 active protocols in pediatric CRC

Shari Barkin, MD, MSHS

Kathryn Edwards, MD

Monroe Carell Jr Children’s Hospital at Vanderbilt

Vanderbilt Institute for Clinical and Translational ResearchVanderbilt Institute for Clinical and Translational Research

Vanderbilt Institute for Clinical and Vanderbilt Institute for Clinical and Translational ResearchTranslational Research

Goals: 1)Establish physical and virtual home for translational investigation; 2)Stimulate/streamline communications and remove impediments; 3)Develop a new generation of investigators; 4) Actively develop novel methodologies

Current Facilitators: Pilot funds available using the “Starbrite system”; Access to “studios” for assistance in hypothesis generation, methodology; statistical considerations, and translation.

Current Barriers: Lack of inpatient pediatric CRCPotential Solutions: Use of adult CRC and scatter beds in the

Children’s Hospital

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Institute of Clinical and Translational Science (ICTS) at WUMS

Overall Components of ICTS at WUMS Washington University in St. Louis (WUMS) BJC Health System

Barnes-Jewish Hospital (BJH)St. Louis Children’s Hospital (SLCH)

Saint Louis University (SLU)School of Public HealthCollege of Health Sciences (medical school)Graduate School

St. Louis College of Pharmacy Southern Illinois University Edwardsville (SIUE) School of Nursing University of Missouri St. Louis (UMSL) College of Nursing Community health care organizations Biomedical companies in St. Louis area

Pediatric Component concentrated at WUMS Department of Pediatrics & SLCH PORU (Patient Oriented Research Unit) PRU (Pediatric Research Unit; formerly GCRC) WUMS Pediatric & Adolescent Ambulatory Research Consortium (WUPAARC) Themes: DM, Asthma, SCD, CF, Brain Injury (neonatal & traumatic), Lung Injury

Pediatric Components of ICTS at WUMS

1. Interdisciplinary Career Development & Translational Research in Pediatrics- Aim 1: Increase number of pediatric fellows & junior faculty participating in Masters in Clinical Science Program (formerly K30). Barrier: Resources to attract fellows/faculty interested in clinical investigation

–Aim 2: Increase number of investigators with focus on child-health clinical and translational research. Barrier: Low pool of established clinical/translational investigators

- Aim 3: Create a Community Scientist Training and Research (C-STAR) program for pediatric residents. Barrier: Lack of time in residency program and ABP not receptive

- Aim 4: Encourage research partnerships between pediatric advocacy organizations and pediatric investigators. Barrier: Lack of time in residency and fellowship training programs 2. Pediatric Research Unit (PRU) Formerly the pediatric GCRC; 11th floor SLCH

Part of the Center for Applied Research Services (CARS) at WUMS

Pediatric Components of ICTS at WUMS

1. Interdisciplinary Career Development & Translational Research in Pediatrics- Aim 1: Increase number of pediatric fellows & junior faculty participating in Masters in Clinical Science Program (formerly K30). Barrier: Resources to attract fellows/faculty interested in clinical investigation

–Aim 2: Increase number of investigators with focus on child-health clinical and translational research. Barrier: Low pool of established clinical/translational investigators

- Aim 3: Create a Community Scientist Training and Research (C-STAR) program for pediatric residents. Barrier: Lack of time in residency program and ABP not receptive

- Aim 4: Encourage research partnerships between pediatric advocacy organizations and pediatric investigators. Barrier: Lack of time in residency and fellowship training programs

2. Pediatric Research Unit (PRU) Barrier: Under-funded & under-staffed

Inadequate space

Clinical and Translational Science Center Pediatrics Oversight Committee

Weill Cornell Medical College

Julianne Imperato-McGinley, MDPrincipal Investigator / Program Director

Patricia J. Giardina, MDAssociate Core Director

Clinical and Translational Resource Unit (CTRU)

Donna DiMichele, MDEducational Ombudsman / Presenter

June 23 – 24, 2008

Weill Cornell Medical College (WMC) Consortium Voting Member: Patricia J. Giardina, MD

Facilities: 12 Partners Weill Cornell Medical College (WMC) * / The New York Presbyterian HospitalMemorial Sloan-Kettering Cancer Center (MSKCC)Hospital for Special Surgery (HSS)Hunter College Center for the Study of Gene Structure and Function & RCMI programHunter College School of NursingCornell University Cooperative Extension Cornell-affiliated hospitals (6) located in underserved NYC communities

* Pediatric Department: centralized at WMC

Training: Full range of pediatric specialties with fellowship training- 4141 fellows

Medical Student Training - 100 100 students per year on rotation and elective- 3030 visiting medical students per year

Research: 9090 eligible protocols (1010 Pediatrics) Data coordinating center on site

WMC CTSC Goals Specific Barriers in Pediatrics Potential Solutions

To provide education and multidisciplinary mentorship program for trainees and C/T researchers in partnering institutions

Multi-disciplinary and diversity in level of training (pre-doctoral,

post-doctoral, MD, PhD, DSN)

Identification and recruitment of multidisciplinary mentors; Development of basic and specialized didactic core

curriculum

To foster innovative C/T research with partners in CTSC

Navigating partnering institutions diverse policies, procedures and

logistics

Enhanced TRAC representation; Sharing of complimentary

resources and the Translational Research Support Team (TREST);

Regular conferencing

To enhance new investigator C/T research

Lack of funding and infrastructure support for new investigators /

investigations

TL1 Pre- and KL2 Post-Doc training awards and Pilot awards for new

investigators; TREST Utilization

To performing C/T research in multiple pediatric disciplines

Too few patients in particular areas; Individual IRBs

Shared complimentary resources and refined IRB process for

multiple partners

Institute for Clinical & Translational ResearchHarry Shamoon MD, Associate Dean and CTSA PI Paul Marantz MD, MPH, Associate Dean Research EdBrian Currie, MD, MPH, VP for Research, Ass’t DeanFrederick Kaskel MD, PhD, Vice-chair, Pediatrics, Site Co-Director, West Campus GCRC, Director Pediatric Nephrology

CTSA direct links with “T1” ProgramsHuman genetics and genomics – new chairEpigenetics – new InstituteProteomics – Expansion of core facility from GCRC baseStem cell research – new Institute and transplant programBiomarkers – new Diabetes Center directorSystem biology – new departmentTraining development programs: K30, 12, CRTP

Continuum of Pediatric research and health care center programs:Diabetes, Cancer, Liver disease, Sickle cell disease, Neuroscience, HIV/AIDS, Developmental Disorders, Health disparities, Organ and Stem cell transplant

Goals: bridge expertise at Children’s Hospital at Montefiore, other clinical sites, with ICTR

Challenges: physical separation, integration of education and research initiative, build a cadre of pediatric researchers

Solutions: incorporate translational research experience into medical school, residency, and post-graduate medical education curricula

Harvard Clinical and Translational Sciences Center• Children’s Hosp. Boston, MGH, NICUs at partner hospitals, DFCI, plus university-wide and

community pediatrics efforts.• PI – Lee Nadler. Pediatric Representative: Ellis Neufeld• Exceptional breadth and depth of C/T research

– Multicenter networks site for pediatric heart network, renal transplantation network, asthma network, pediatric AIDS clinical trials, COG, PBMTC, neonatal studies, etc.

– CHB hosts one of the former 6 PGCRCs; four GCRCs in total; budget ~$18MM– Translational Research Program (CHB)

• Strong extramural research funding (CHB FY2007 direct cost expenditures $135M)– Federal $73M research, industry $3.5M, Foundation $12.9M, Philanthropy $20.9M

• Major pediatrics training center(s)– >300 fellows in more than 30 training programs– Existing K12 programs in pediatrics, non-malignant hematology, others– Predoctoral, K12, K30 and C/T masters programs in CTSC

• Strong pediatric participation in CTSC leadership– External pediatric oversight committee– CHB CEO on highest internal comm. – 3 pediatricians on operations comm.– Representation in all areas of CTSC

• Goals– Integrate C/T pediatric research resources across university. Participate in

national pediatrics consortium efforts. Promote training and pilot grant programs to pediatric trainees to increase number and quality of pediatric C/T researchers. Publicize the CTSC resources. Integrate peds into the 10 main CTSC areas. Transformation to PCIR in concert with the other three GCRCs.

• Current Barriers: – Very Large enterprise across Harvard community; “silos” within

programs/hospitals. Modest incremental funding. Changes in ancillary support of GCRC model.

• Proposed solutions.

– Avoid “old-think” about status quo at GCRC. Involve best and brightest C/T senior researchers in new training opportunities and expanded PCIR. Aim top candidates at training slots/pilot grants in CTSC. Establish pediatric metrics for participation in all levels of CTSC across the institution, and across all 10 areas of the CTSC project. Investigate means of covering “GCRC-like” ancillary costs for junior investigators.

Voting Member: Ram Yogev, M.D.

Alternate Member: Leon Epstein M.D.

• Children’s Memorial Hospital: 266 bed free standing tertiary children’s hospital; all sub-specialties; serves population of 8.5 million in Chicago.• Clinical Research Unit:

• Dedicated 4 bed ambulatory unit; 1400 research visits/yr• Scatter Beds; 20-25 inpatient research subjects/yr; (not including ped oncology protocols).• Personnel: director; admin assist, nurse manager;

4.5 nurses, 1.0 research coord;

1.5 research pharmacists.

Goals: 1. Integrate all pediatric research and provide the necessary

infrastructure to support new and established investigators.2. Promote research regarding the pediatric antecedents of adult

disorders.Current Barriers: 1. Pediatric research competes for resources in the larger adult CSTA.2. Inpatient beds for research are limited due to high occupancy rates.Potential Solutions:1. Independent funding for pediatric CTSAs2. Dedicated inpatient research unit

•.

Nationwide Children’s HospitalThe Ohio State University Department of Pediatrics

Facilities: – Freestanding children’s hospital with 350 bed capacity, as well as two dedicated research buildings with more than 300,000 sq ft. contiguous with the main hospital.

Training: – 33 fellowship training programs in pediatric specialties

Research: Over 700 active IRB protocols: 52 managed in clinical research core (Clinical Research Services) others are managed departmentally.

Nationwide Children’s Hospital• Goals: Clinical research staff training programs that complement research efforts –

assistance with study design, GCP to regulatory practice and quality assurance

Better/easier access to biostatistical resources

Offering specific focused resources for protection of human subjects for pediatrics and regulatory issues with gene transfer genomic analyses.

• Current Barriers: collaborations between clinical and research faculty, providing statistical support across differential studies for grant and manuscript submission and lack necessary space as a result of continuous growth

• Potential Solutions: Bench to Outcomes Seminar Series (BOSS), research faculty attending clinical division meetings, revision of website to include project types for easily desired collaboration, continued work with OSU biostatistical program to include onsite service.

The Floating Hospital for Children at Tufts Medical Center, Tufts University School of

Medicine

Jonathan M. Davis, MDChief of Newborn Medicine

Program Director, Clinical and Translational Research Center

The Floating Hospital for Children at Tufts Medical Center

•Facilities: Eight hospitals – 2,600 total licensed beds

•Pediatric Departments at two teaching hospitals

•Training: Full range of Pediatric Sub-specialties, including medical and surgical; Fellows, Pediatric Residents, and Medical Students routinely on rotation.

•Research: Clinical and translational research; NIH funded investigators in multiple areas of basic science and clinical research; Pediatric Clinical Research Center faculty working with investigators in the Institute for Clinical Research and Health Policy; full statistical services on-site.

The Floating Hospital for Children at Tufts Medical Center

•Goals: Encourage young investigators to pursue careers in academic medicine; support established investigators; enhance multidisciplinary collaboration, generation of novel research methods, academic innovation, and research education.

•Current Barriers: Need to integrate research personnel and subjects at multiple sites; too few patients in some areas; provide investigators with sufficient time and support to conduct high quality research.

•Potential Solutions: Provide support for all research related activities (administrative, study coordinators, bionutrition, laboratory, biostatistics); provide time and resources for new investigators (through grants); enhance collaboration; reduce barriers through education and computerization.

Colorado Clinical and Translational Sciences Institute (CCTSI)Ronald J. Sokol MD, CCTSI Director

Child and Maternal Health (CMH) ProgramWilliam Hay, Jr. MD, CMH Director (Co-Director, CCTSI)

Facilities: Brand new integrated Medical Campus plus 6 Hospitals including: • The Children’s Hospital, 270 beds – opened 2007 • University of Colorado Hospital – NICU, maternal-fetal program, nursery • Denver Health - 44 pediatric beds• National Jewish Medical and Research Center• Kaiser Permanente Colorado

Training: Pediatric Residency (n=70), full range of pediatric specialties with fellowship training in all (9 Sub-Board Fellowships) with 6 T32 grants (n=70). Complete Department of OB-GYN with MFM, Repro-Endo, and Basic Research Divisions. Schools of Medicine, Nursing, Pharmacy, Dentistry, Public Health, Graduate, Allied Health, Education, Engineering, etc.

Research: $89 million sponsored funding for pediatric research, 2nd NIH ranked SOM Dept. of Pediatrics, CTRC (formerly GCRC in year 45), Clinical Trials Organization (TCH), Barbara Davis Type I Diabetes Center, Perinatal Research Center, over 700 active clinical research protocols, 108,000 sq. ft. of Lab Space, 22,000 sq. ft. Clin. Research Space

CMH Research Goals:Specific Aim 1: Develop a new, broad-based, multidisciplinary organizational structure to promote integration of Child and Maternal Health Clinical-Translational Research in the CCTSI. Scaffold for initiating new collaborations among basic, clinical, and translational scientists in multiple disciplines in both the medical center and the community.Specific Aim 2: Ensure the continuity of clinical-translational research from fetus to young adult by developing and incorporating infrastructure to assist investigators through all stages of research. Provide streamlined infrastructure for longitudinal, mother-child, multidisciplinary research programs.Specific Aim 3: Build on existing community resources to create new opportunities for community translational research in child and family health research and training. Provide a national model for transformation of current Pediatric and Adult General Clinical Research Centers.

Managed by CMH Steering Committee including investigators from OB, neonatology, pediatrics, pediatric GCRC, behavioral and social sciences, bioengineering, Colorado Health Outcomes program, Medicine, Education, Nursing, Public Health, and Community programs.

Current Barriers: Lack of a primary single clinical perinatal facility (OB/MFM only at UCH, Neonatology at both UCH and TCH); not enough ELBW/extremely preterm infants; competition with non-participating neonatology--OB/MFM services; not yet developed adequate nursing and community relationships; inefficient pediatric IRB review process Potential Solutions: Develop Maternal-Fetal-Neonatal Clinical Facility and Program at TCH. Increase School of Nursing (SON) investigators and community health program leaders in leadership roles in the MCH program; provide biomedical informatics support to communities, developing and enhancing direct communication; national CTSA collaboration for rare diseases, including neonates; data sharing needs to be developed; INFOED installed in Grants Office and IRB.

University of North Carolina at Chapel Hill

Facilities: 5 Hospitals with 727 bed capacity

Area Health Education Center (AHEC) provides state research environment

UNC Pediatric Core encompasses intramural and extramural environments Training: Fellow, resident and medical student training programs

Institutional K-12, K-30 and many T-32 training programs

Research: GCRC/PCIR; Core Laboratories; Active protocols in university and community based settings

David Peden

Goals - expand pediatric capabilities in PCIR - stepwise implementation of pediatric community engagement - multilevel training to build research and mentoring capability- integration of pediatric advocates in every CTSA endeavor

Current Barriers - insufficient trained personnel; decentralized bioinformatics and statistical support; missed opportunities for enrollment and collaboration

Potential Solutions – mentor development program; facilitate greater pediatric participation in K training programs; institutional effort for centralized bioinformatics; collaborate with other institutions progress

Christus Santa Rosa Children’s Hospital

After CTSA

Audie Murphy Veterans Administration Hospital

Bartter General Clinical Research Center

Child Health Advocacy Research and Training

(CHART) CenterBefore CTSA

Facilities Adult Inpatient Unit - Bartter Clinical Research Unit Audie Murphy VA Hospital Adult Outpatient Unit – FIRST Outpatient Research Unit (off campus, free parking) Pediatric Unit - CHART Center at CHRISTUS Santa Rosa Children's Hospital 6,000 ft2 outpatient facility, free parking, weekend hours, official hospital unit Regional Clinical Research Unit – Regional campuses and off site activitiesTraining Short course (2 week) in clinical investigation (faculty, fellows, residents and staff) Masters of Clinical Investigation Program (faculty and fellows) 4 pediatric fellowship programs (2 more approved)Pediatric Research 10 protocols, 4 PIs (TODAY, HEALTHY, TrialNet, National Children Study) 5 protocols, 5 additional PIs (in process), >250 subjects

Jannine Cody, PhD, Admin. DirectorDaniel Hale, MD, Medical Director

Pediatric Goals• Increase the number of pediatric investigators via both training and recruitment • Enhance access of children in South Texas to research innovation• Build pediatric research infrastructure in the Children’s Hospital and the region• Broaden collaboration across the age spectrum• Expand the network of pediatric investigators/investigations across the regionCurrent Barriers• High clinical patient volumes and clinical demands• Limited education, low socioeconomic status, high uninsured rate in population• Socio-cultural, immigration and geographic issues• Lack of shared vision (what we can be, not what we are)• Underdeveloped local philanthropy/endowment• Small core of NIH-experience clinical pediatric investigatorsPotential Solutions• “Sell” the CHART Center to pediatric researchers throughout the region• Create (by training or recruitment) a larger core of pediatric researchers• Make clinical research more visible and a “viable career” for pediatric trainees• Focus efforts of development campaign on children’s research benefits• Collaborate across CTSAs state-wide/nationally (unified IRB and data systems)