Developing IND Protocols for Investigational Drugs at CHOP
1. Bedside Research: Protocol
Development for Investigational
New Drug Applications at CHOP
Zenobia Cofer
Research Administration Fellowship
January 20, 2015
2.
3. Investigational New Drug
• a drug that has not been to the marketplace
• previously marketed drug being studied for an
additional indication
• A previously marketed drug being studied in a
in a new or special population
4. Federal Drug Administration
• Federal Drug Administration
(FDA) requires persons/entities
(sponsor) submit an IND
application prior to
administering an IND to humans
• Sponsors can be drug
companies, a cooperative group,
or individual physicians.
• If FDA approves IND application,
study cannot proceed without
institutional approval
5. Timeline of Institutional and Federal
Regulatory Review Before Trial Start
Protocol Dev. IND/IDE SRC
IRB
FDA
Start
1-3 months 1 month
1 month
•Ideal timeframe 3-6 months
•Protocol can be stalled at any of regulatory steps
•Contact CRSO and/or institutional committees for assistance
Institutional
Federal
7. Keys of good protocol
• Gives reviewers background information regarding the condition
or target population
• Addresses current medical treatment and continued problems
with available treatment
• Justifies the use of a new drug/device to address continued
deficits in treatment
• Presents a well developed targeted plan to investigate the drug
in target population
• Requires time to develop
Protocol Dev.
8. FDA requirements for IND
Protocol
• Drug toxicity
• Previous research
(animal and clinical)
• Drug manufacturing
• Sponsor research plan
• If FDA approves the
protocol, it can still be
stalled by the IRB
9. Fellowship Projects
Purpose: Aid in protocol development
• Descriptive/Observational Study (NICU)
• Intervestigational Study I (NICU)
• Intervestigational Study II (GI&Nutrition):
biological
• Develop IND Protocol Template for
Investigators
Clinical Research Support Office
Direct Fellowship Supervisor: Deanna DiDonato
11. Protocol Development using team
dynamics
Investigator
• current treatment
• benefits of new treatment
• potential risks associated with treatment
IND/IDE Program Manager
• institutional and federal regulatory requirements
• advises investigator on regulatory obligations
• facilitates communication between different
regulatory bodies and investigators
Research Admin Fellow
• provide questions regarding methodology
• provide input on scientific design
Investigator
IND/IDE
Program
Manager
Research
Admin
Fellow/PhD
12. PhD skills relevant for Protocol
Development
• Relevant literature reviews
PUBMED
EndNote or RefWorks
• Writing skills
Succinctly, but accurately
• Can offer advice on study design
Training based on performing experiments
• Facilitate communication within the team
13. NICU Interventional Protocol
1. Complied reference list
• Original protocol had a minimal amount of citations
• No citations were after 2006
2. Wrote up background information for
protocol
3. Suggested justification for study design
based on previous literature
4. Participated in regular meetings with
NICU investigators
14. Protocol Dev. IND/IDE SRC
IRB
FDA
Start
Protocol Dev. IND/IDE SRC
IRB
FDA
Start
Protocol Dev. IND/IDE SRC
IRB
FDA
Start
NICU
NICU
NICU
Project Progress over Research
Administration Fellowship Term
Aug 2014
Nov 2014
Jan 2015
15. SRC Review of NICU protocol
• Advised investigators to justify target
population for study
• Wanted investigators to justify statistical
analysis
• Team had a meeting with SRC chair
• SRC thought background section was well
written
16. Future Directions
Protocol Dev. IND/IDE SRC
IRB
FDA
Start
NICUJan 2015
NICU Interventional Protocol
•Resubmit to the SRC in February
•Prepare concurrent submissions to the FDA and CHOP IRB
•Start trial in May 2015
NICU Descriptive Protocol
•Submission to IRB
