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Pediatric Drug & Medical Device Development: Setting Specifications & Defining Expectations
Purpose, Vision and Expected Outcomes
February 26, 2009
Carole Marcus, MBBChCo-Chair
Pediatric Drugs and Devices SubcommitteeCTSA Consortium Child Health Oversight Committee
Meeting Purpose
To formulate specifications for a national child health clinical research infrastructure to effectively and efficiently develop drugs and medical devices for children
Lack of Pediatric Information• ~ 80% of listed medication labels
disclaimed usage or lacked dosing information for children
– Physician’s 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
3Allergy/asthma
8Steroids
13Respiratory
13Antibiotics
13Sedation/anesthestics
17Immunologic/rheumatologic
18Metabolic
21Antifungals/antiparasitics
21Anticonvulsants
25Endocrine
30Antivirals
32Analgesics
36Psychiatric
38Neurology
40Hematology
44Derm/eye/ear
44Antidotes
54Gastroenterology
57Cardiac
Percent Off-LabelClass of Medications
Off-Label Status of Medication AvailableFor Pediatric Use, by Class
Need For Pediatric Drug Development
Yoon, Clin Pediatr 2006; 45:75
Need For Pediatric Medical Device Development
Pediatric Considerations
• Children are currently underserved by the medical/scientific community
• Clinical research requirements for children are different than those for adults
What CTSA’s 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
Pediatrics In The CTSA
• 37 of 38 CTSA’s have a pediatric component• 34 of the 38 CTSA’s sent pediatric
representatives to the 2008 Child Health Oversight Committee meeting.
Current Problems With Pediatric Academic Multicenter Studies
• IRB delays• Technology transfer delays• Scientific review delays
Process for Opening a Clinical Trial
Dilts, J Clin Oncol 2006; 24:4545
Dilts, J Clin Oncol 2006; 24:4545
Mean Number Of Days Per Process
0 50 100 150 200 250 300
Start to 1st process
IRB
Contracts & grants
Scientific review committees
Receipt to open
Open to 1st subject
Receipt to 1st subject
58
69
100
105
189
73
251
Mean time (days)
Today’s Goals
• The expected deliverable will be information and specifications on the infrastructure features and parameters for a national pediatric clinical and translational platform.
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.
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.
Specific Topics For Discussion
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
FDA and OHRP Regulatory Support
• Regulatory support and regulatory resources
• Regulatory reporting information flow and responsibilities