clinical trials transformation initiative
TRANSCRIPT
CLINICAL TRIALS TRANSFORMATION INITIATIVE
Mobile in Clinical Trials: Developing
Novel Endpoints for use in Clinical Trials
Jennifer Goldsack
Clinical Project Manager, CTTI
September 29, 2016
CTTI Overview
To develop and drive adoption of
practices that will increase the
quality and efficiency of clinical trials
Public-Private Partnership
Co-Founded by FDA and Duke
involving all stakeholders
70+ members
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PROJECT
PORTFOLIO September 2016
Systematic approach
to evidence
generation including
use of non traditional
CT data sources &
technical innovations
Patients as
equal partners
across the
R&D
continuum
CTs designed
with a focus on
efficiency &
quality
Trials that
address emerging
public health
concerns
Safe & ethical
trials that are
streamlined
Project
Recommendations/
Findings Complete
Large simple trials GCP training
Monitoring
QbD
Recruitment
Site metrics
ABDD:
• Streamlining
HABP/VABP
trials
Opioid
Central IRB (2)
DMCs
Informed Consent
Safety reporting (3)
Active Projects Registries PG&CT Investigator ABDD: Pregnancy Testing
State of clinical trials turnover Peds
Mobile in Clinical GCP follow on Pilot studies
Trials Program: (Qualified inv) Unmet need
Mobile Devices
Legal & Reg
Novel endpoints
Stakeholders
Completed
Collaborations
Uses of electronic
healthcare data
Clinical trial survey
CV endpoints
Investigator
training course
Patient
engagement
survey
Active
Collaborations
Supporting IMPACT-
AFib
ABDD PTN
-
Mobile Clinical Trials (MCT) Program
PURPOSE:
Develop evidence-based recommendations that affect the widespread adoption and use of mobile technology in clinical trials
ANTICIPATED IMPACT:
Increase the number of clinical trials appropriately leveraging mobile technology
4 PROJECTS
Legal & Novel Mobile Stakeholder
Regulatory Issues Endpoints Devices Perceptions
*Scope: FDA-regulated clinical trials after the time of initial research volunteer consent
Developing Novel Endpoints Generated by
Mobile Technology for Use in Clinical Trials
Purpose:
This project aims to issue recommendations that clarify the pathway for developing novel endpoints*, generated using mobile technology, for use in clinical trials.
Objective:
Describe best practices for developing novel endpoints, generated using mobile technology, for use in clinical trials.
* We have defined novel endpoints as either 1) new endpoints that are not currently used, or 2) existing endpoints that can now be measured in new and possibly better ways using mobile technology.
Project Team
Team Leaders
Lauren Bataille, MJFF
Rob DiCicco, GSK
Cheryl Grandinetti, FDA
Will Herrington, Oxford
Martin Landry, Oxford
Kaveeta Vasisht, FDA
Team Members
Stephen Friend, Apple
Ashish Naryan, Northwell
Elektra Papodopoulous, FDA
Theresa Strong, FPWR
Komathi Stem, ReThynk Consulting
Ken Skodacek, FDA
Nirav 'Rav' Sheth, MC10
Andrew Trister, Sage Bionetworks
Marc Walton, Janssen
CTTI Project
Manager
Jen Goldsack,
CTTI
CTTI Executive
Committee
Champion
John Alexander,
Duke
"
"
Our Approach
Recommendations
Publication(s) of Use Cases
USE CASES:
1. Physical activity and gait / Parkinson’s disease / accelerometer 2. Physical activity / heart failure / accelerometer
3. Blood sugar level / diabetes / CGM
4. Physical activity / muscular dystrophy / accelerometer
Determine Information Needed from Existing Case
Studies (Extraction Tool)
Quick Search" of case studies to identify which novel endpoints have
already been studied to some degree
Systematic Review of Existing Case Studies
Publication of Systematic
Review
Determine Search Terms for Identifying Case Studies
Create a long list" of possible novel endpoints that we could write use
cases for
Use Case Selection (using Pugh Matrix
tool)
Use Case #1 Use Case #2 Use Case #3 Use Case #4
Publication of conceptual framework for selecting novel
endpoints for inclusion in clinical studies
Determine criteria and weightings to use to select
endpoints for use cases
Meeting Objectives
Identify how to develop novel endpoints for use in clinical trials by writing the following use cases:
Physical activity level and gait, measured using an accelerometer, as an endpoint for Parkinson's disease
Physical activity level, measured using an accelerometer, as an endpoint for heart failure
Blood sugar level, measured using a wearable continuous glucose monitor, as an endpoint for diabetes
Physical activity level, measured using an accelerometer, as an endpoint for muscular dystrophy
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Thank you.
Jen Goldsack
CONNECT WITH CTTI www.ctti-clinicaltrials.org