Bedside Research: Protocol Development for Investigational New Drug Applications at CHOP

Zenobia CoferResearch Administration Fellowship

January 20, 2015

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

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

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

*

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.

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

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 OfficeDirect Fellowship Supervisor: Deanna DiDonato

NICU

GI&Nutrition

Protocol Development using team dynamics

Investigator• current treatment • benefits of new treatment• potential risks associated with treatmentIND/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

PhD skills relevant for Protocol Development

• Relevant literature reviewsPUBMEDEndNote or RefWorks

• Writing skillsSuccinctly, but accurately

• Can offer advice on study designTraining based on performing experiments

• Facilitate communication within the team

NICU Interventional Protocol1. Complied reference list• Original protocol had a minimal amount of citations• No citations were after 20062. Wrote up background information for protocol3. Suggested justification for study design based on previous literature4. Participated in regular meetings with NICU investigators

Protocol Dev. IND/IDE SRCIRB

FDA Start

Protocol Dev. IND/IDE SRCIRB

FDA Start

Protocol Dev. IND/IDE SRCIRB

FDA StartNICU

NICU

NICU

Project Progress over Research Administration Fellowship Term

Aug 2014

Nov 2014

Jan 2015

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

Future Directions

Protocol Dev. IND/IDE SRCIRB

FDA StartNICUJan 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

Acknowledgements

Office of Postdoctoral Affairs and Research Training

Clinical Research and Support OfficeDeanna DiDonato

Questions?