Pediatric Drug

1
Pediatric Drug Medical Device Development
Setting Specifications Defining
ExpectationsPurpose, Vision and Expected
OutcomesFebruary 26, 2009

• Carole Marcus, MBBCh
• Co-Chair
• Pediatric Drugs and Devices Subcommittee
• CTSA Consortium Child Health Oversight Committee

2
Meeting Purpose

• To formulate specifications for a national child
  health clinical research infrastructure to
  effectively and efficiently develop drugs and
  medical devices for children

3
Lack of Pediatric Information

• 80 of listed medication labels disclaimed
  usage or lacked dosing information for children
• Physicians Desk Reference 1973 1991 surveys
• Only 20-30 of drugs approved by the FDA are
  labeled for pediatric use
• 1984-1989 survey, 1991-2001 repeat survey
• Only 38 of new drugs potentially useful in
  pediatrics were labeled for children when
  initially approved
• 1991-1997, FDA statistics

4
Need For Pediatric Drug Development
Yoon, Clin Pediatr 2006 4575
5
Need For Pediatric Medical Device Development
6
Pediatric Considerations

• Children are currently underserved by the
  medical/scientific community
• Clinical research requirements for children are
  different than those for adults

7
What CTSAs Bring To The Table

• Intellectual and academic input and experience
• Patient resources (common and rare diseases)
• Infrastructure for research
• Clinical research space
• Laboratory resources
• Clinical core facilities
• Development of central core facilities and
  informatics
• Education and training opportunities

8
Pediatrics In The CTSA

• 37 of 38 CTSAs have a pediatric component
• 34 of the 38 CTSAs sent pediatric
  representatives to the 2008 Child Health
  Oversight Committee meeting.

9
Current Problems With Pediatric Academic
Multicenter Studies

• IRB delays
• Technology transfer delays
• Scientific review delays

10
Process for Opening a Clinical Trial
Dilts, J Clin Oncol 2006 244545
11
Mean Number Of Days Per Process
Mean time (days)
58
69
100
105
189
73
251
Dilts, J Clin Oncol 2006 244545
12
Todays Goals

• The expected deliverable will be information and
  specifications on the infrastructure features and
  parameters for a national pediatric clinical and
  translational platform.

13
Workshop Expectations

• There is no expectation to achieve consensus or
  solve problems, but to provide guidance and
  information to help develop the most responsive
  infrastructure.

14
What We Are Not Going to Do!

• Discuss specific drug/device needs
• Discuss funding mechanisms
• Prioritize areas of research
• Discuss regulatory issues
• Separate meetings / activities on many of the
  above are planned by NIH.

15
Specific Topics For Discussion
16
Legal And Financial

• Use of templates and common documents for
  intellectual property issues
• Development of policies for intellectual
  property, data sharing and publication
• Development of policies for confidential
  information sharing and disclosure
• Potential mechanisms for cost sharing
• Privacy and protecting health information

17
FDA and OHRP Regulatory Support

• Regulatory support and regulatory resources
• Regulatory reporting information flow and
  responsibilities
• Interest and ability to hold INDs and IDEs

18
Availability And Development Of Core Facilities

• Analytic services
• Pharmacokinetics
• Pharmacogenomics
• Pharmacodynamics
• toxicology
• Specimen analysis
• Specimen repository
• Certification of core facilities
• GLP
• CLIA

19
Pediatric-specific Biostatistical Resources

• Longitudinal analyses
• Incorporation of growth parameters into
  statistical assessments
• Small sample sizes

20
Informatics Needs

• Use of data coordination centers
• Use of data standards for data acquisition, data
  transmission and data storage, to ensure
  interoperability
• Data security procedures

21
Pediatric-specific Ethical Issues

• Guidelines for what constitutes minimal risk
  criteria for referring protocols for 407 review
• Use of harmonized age-appropriate assent
  templates
• Guidelines for re-consenting pediatric enrollees
  in long term studies

22
Bridging the Gap From Clinical Care To Research

• Approaches to integrate participation in clinical
  trials with clinical care
• Tools to inform families about pediatric clinical
  research
• Recruitment and retention policies including
  inclusion of underrepresented populations
• Outreach efforts
• Sharing of results with study participants and
  the general public (e.g., clinicaltrials.gov)

23
Performance Metrics

• Plans to develop, implement and assess
  performance metrics
• Time to opening of study / enrollment of 1st
  subject

24
Logistical Considerations

• Need for a single point of contact for general
  information for network activities
• Need for public web site which elements to
  include

25
Building A Culture Of Product Development

• Encourage product development in addition to
  pursuit of non-product development scientific
  agendas
• Anticipated training needs for consortium
  personnel to address these needs

26
What To Expect From NIH

• Willingness to learn and adapt
• Commitment to the highest scientific and ethical
  standards
• Willingness to collaborate and cooperate with
  partners, patients, study participants and the
  public
• Interest in participating in the development of
  products with clinical benefit for children

Steven Hirschfeld, MD, PhD