CTSA Consortium Meeting on Development of Pediatric Drugs and Devices: Expectations and Specificatio

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CTSA Consortium Meeting on Development of
Pediatric Drugs and Devices Expectations and
Specifications
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A Fragmented System with Silos and Barriers
Discovery
Clinical Research
Translation and Adoption
Global Health
Translation
Proof of Concept in Man
Basic Discovery
Clinical Development Phase II, III
FDA Approval Evidence Based Medicine
Preclinical Research In Vivo Analysis
Practice Adoption, Practice Guidelines, Cost
Effective

• Community
• Assessment
• Care delivery
• Outcomes
• Economics

Improve Community Health Status
Global Health Service/ Research, Population/ Pu
blication
PK,PD, Toxicology
Current State Entities
Industry/Biotech
Clinical Research Organizations/AHS
HCS/Hospitals/ Practices/FQHC/AHS
Public Health/Government/ NGOs
AHS/Industry/ Biotech
Timeline
?
15-20 years
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• Pediatric Drugs and Devices Workgroup of CTSA
• Mission To deliver collaborative translational
  and clinical research so that
• safer and more effective drugs and devices can be
  developed for children
• Goals
• Initiate process for central pediatric CTSA IRB,
  harmonization of contracts,
• intellectual property, and budgeting.
• Identify industry partners to develop
  public-private partnerships for drug and
• device studies
• Develop repository for consents, case report
  forms, consultative service for
• study design

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Pediatric Drugs and Devices Workgroup of
CTSA Carole Marcus (UPenn) and Jennifer Li
(Duke)
Sudha Kashyap (Columbia) Frederick Kaskel
(Einstein) Harry Keyserling (Emory) Mary
Leonard (UPenn) Sulagna Saitta (UPenn) Michael
Shannon (Harvard) William Smoyer (Ohio State)
Gregory Yanik (Ann Arbor) Pamela Zeitlin (Johns
Hopkins) Ted Zwerdling (UC Davis)
Mark Abzug (Denver) Amira Al-Uzri (OHSU) Claire
Brett (UCSF) Susan Cohn (Chicago) Jonathan
Davis (Tufts) Julia Glade Bender (Columbia)
Donald Hagler (Mayo) Daniel Hale (San Antonio)
Alejandro Hoberman (Pittsburgh) Lilly
Immergluck (Emory)
Rosemary Higgins (NICHD) Steven Hirschfeld
(NICHD) Mary Purucker (NCRR)
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• Can we build and develop a transformative
  platform between the CTSA infrastructure and
  industry to translate drugs and device
  development to improve child health??

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Why a network vs clinical sites directly?

• Balance the necessary investment of time to
  educate, earn trust, and eventually facilitate
  pediatric subject participation in clinical
  trials
• Partner to overcome the hurdles inherent in
  pediatric therapeutic development
• Operational advantage and streamlined
  efficiencies
• Shorter start-up times

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• Next Steps??

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• To be developed in the CTSA

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ADMINISTRATIVE SUPPLEMENTS

• Notice of Availability of Administrative
  Supplements for Research on Outcome Measures for
  Pediatric Clinical Trials in support of the Best
  Pharmaceuticals for Children Act Notice Number
  NOT-RR-09-005
• Key DatesRelease Date  February 19,
  2009Receipt Date April 30, 2009Earliest
  Anticipated Start Date September 30, 2009
• Issued byNational Center for Research Resources
  (NCRR) (http//www.ncrr.nih.gov) Purpose The
  National Center for Research Resources (NCRR) and
  the Eunice Kennedy Shriver National Institute of
  Child Health and Human Development (NICHD)
  announce an administrative supplement program of
  up to 5.0 million in Fiscal Year 2009 to provide
  funds to CTSA Consortium-supported research
  projects for research on outcome measures in
  clinical and translational child health in
  priority areas as determined by the Best
  Pharmaceuticals for Children Act program
  administered by NICHD.  The administrative
  supplements are to support projects of national
  scope for established CTSA-wide consortia
  defined as the CTSA Oversight Committees or Key
  Function.

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NIH Challenge Grants program (100-200 million)

• A new RFA will be released within a week or two
  for a new, two-year program of cross-cutting,
  highly innovative projects, 1 M total per
  project.
• These will be grants to support research that are
  scientifically justifiable within the two year
  funding. They will most likely be peer-reviewed
  and will be required to comply with regular
  approach to successful award funding- highly
  meritorious, significant scientific impact.
  Awards will be in the area of 500,000 per year
  for two years.

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• CTSA Consortium Child Health Oversight Committee
  (CC-CHOC)
• Meeting in May 2009