Resources to Support Novel Endpoint Development
Resources to Support Novel Endpoint
Development
Lindsay Kehoe, Project Manager, CTTI
July 27, 2021
Disclaimer
•
The views and opinions expressed in this presentation are those of the
individual presenter and do not necessarily reflect the views of the
Clinical Trials Transformation Initiative.
•
The presenter is an Employee of Duke University. Salary support comes
from pooled membership fees of the Clinical Trials Transformation
Initiative and from FDA Cooperative agreement.
https://www.gartner.com/en/documents/3887767
DHT-Derived Endpoints in Pivotal Trials:
Where Are We?
Peak of Inflated Expectations
Plateau of Productivity
Slope of Enlightenment
Trough of Disillusionment
Technology Trigger
V
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B
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No known clinical outcome assessments captured by a DHT have been
‘accepted’ by FDA to support an approved label claim
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As of June 2021, there are 200+ DHT-derived endpoints listed in Digital
Medicine Society’s crowdsourced
Library of Digital Endpoints
.
19 listed as “primary” endpoint in either phase 2 or phase 3
67 listed as “secondary” endpoint in either phase 2 or phase 3
•
8 DHT-Passive Monitoring COAs have an accepted Letter of Intent
•
7 qualified Clinical Outcome Assessments (COA) under the DDT
program
All are PROs
DHT-Derived Endpoints In Use Today
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Increased number of clinical trials leveraging digital health technologies.
More efficient trials
generating better quality information.
*Formerly CTTI’s Mobile Clinical Trials (MCT) Program
CTTI’s Digital Health Trials (DHT) Program*
2017
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Focus on measures that are meaningful to patients.
Select the technology after selecting an outcome assessment.
Use a systematic approach to identify key novel endpoints.
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Foster collaboration among key stakeholders.
Create technical standards for mobile technology-derived assessments.
Engage with regulators.
Include novel endpoints as exploratory endpoints in existing clinical trials and
observational cohort studies.
Think critically about how to optimally position novel endpoints in interventional trials.
Additional information at
https://www.ctti-clinicaltrials.org/projects/novel-endpoints
CTTI’s 2017 Developing Novel Endpoints Work
CTTI’s 2017 Developing Novel Endpoints Work
Steps for Novel Endpoint Development
Project Overview
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Identify gaps and barriers and solutions to achieve regulatory
acceptance for a DHT-derived endpoint
Create a glossary for DHT-derived novel endpoints
Describe the evidence needed to achieve regulatory
acceptance for a novel, DHT-derived endpoint
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Project Scope & Deliverables
*Per FDA/NIH’s BEST glossary, a clinical outcome describes or reflects how an individual feels,
functions or survives.
2021 Novel Endpoints Acceptance Project Team
Pathways for FDA Engagement for Drug Development
Example “Frameworks” for DHT-Derived Endpoint Development
2015
2017
2020
Lindsay Kehoe, CTTI Project Manager
Lindsay.kehoe@duke.edu
In this presentation, Lindsay Kehoe, Project Manager at CTTI, discusses the development of novel endpoints in clinical trials. The content covers DHT-derived endpoints, the Digital Health Trials Program, and practical approaches to enhancing clinical trial outcomes. Key topics include the use of digital health technologies, engaging patients and sites, and optimizing novel endpoint selection. For more information, visit the provided links.
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Presentation Transcript
July 27, 2021 Resources to Support Novel Endpoint Development Lindsay Kehoe, Project Manager, CTTI
Disclaimer The views and opinions expressed in this presentation are those of the individual presenter and do not necessarily reflect the views of the Clinical Trials Transformation Initiative. The presenter is an Employee of Duke University. Salary support comes from pooled membership fees of the Clinical Trials Transformation Initiative and from FDA Cooperative agreement.
DHT-Derived Endpoints in Pivotal Trials: Where Are We? VISIBILITY Peak of Inflated Expectations Plateau of Productivity Slope of Enlightenment Trough of Disillusionment TIME Technology Trigger https://www.gartner.com/en/documents/3887767
DHT-Derived Endpoints In Use Today No known clinical outcome assessments captured by a DHT have been accepted by FDA to support an approved label claim As of June 2021, there are 200+ DHT-derived endpoints listed in Digital Medicine Society s crowdsourced Library of Digital Endpoints. 19 listed as primary endpoint in either phase 2 or phase 3 67 listed as secondary endpoint in either phase 2 or phase 3 8 DHT-Passive Monitoring COAs have an accepted Letter of Intent 7 qualified Clinical Outcome Assessments (COA) under the DDT program All are PROs
CTTIs Digital Health Trials (DHT) Program* PURPOSE: Develop evidence-based recommendations that affect the widespread adoption and use of digital health technology in clinical trials for regulatory submission. ANTICIPATED IMPACT: Increased number of clinical trials leveraging digital health technologies. More efficient trials generating better quality information. DHT Program Digital Health Technologies Novel Endpoints Decentralized Clinical Trials Engaging Patients & Sites 2018 2019 2017 2018 *Formerly CTTI s Mobile Clinical Trials (MCT) Program
CTTIs 2017 Developing Novel Endpoints Work Optimizing Novel Endpoint Selection Focus on measures that are meaningful to patients. Select the technology after selecting an outcome assessment. Use a systematic approach to identify key novel endpoints. Practical Approaches to Novel Endpoint Development Foster collaboration among key stakeholders. Create technical standards for mobile technology-derived assessments. Engage with regulators. Include novel endpoints as exploratory endpoints in existing clinical trials and observational cohort studies. Think critically about how to optimally position novel endpoints in interventional trials. Additional information at https://www.ctti-clinicaltrials.org/projects/novel-endpoints
CTTIs 2017 Developing Novel Endpoints Work Steps for Novel Endpoint Development
Project Overview Purpose: Obtain reliability and acceptance of meaningful, DHT- derived novel endpoints Objectives: Identify gaps and barriers and solutions to achieve regulatory acceptance for a DHT-derived endpoint Create a glossary for DHT-derived novel endpoints Describe the evidence needed to achieve regulatory acceptance for a novel, DHT-derived endpoint Expected Impact: Increase the use of meaningful, DHT-derived novel endpoints as key endpoints in clinical trials for labeling claims
Project Scope & Deliverables IN SCOPE OUT OF SCOPE Multi- Stakeholder Meeting Interview Results Clinical Outcome Assessments (COAs)* Functional outcomes Passive and active monitoring Technology intended for use in clinical trials Surveys (ePROs) Digital therapeutics Biomarkers Team Discussion & Consensus CTTI Recommendations *Per FDA/NIH s BEST glossary, a clinical outcome describes or reflects how an individual feels, functions or survives.
2021 Novel Endpoints Acceptance Project Team Team Leaders Team Members Alicia Staley (Medidata) Elektra Papodopoulous (FDA) J rg Goldhahn (ETH Zurich) Rodrigo Garcia* (EMD Serono) Kai Langel (Janssen) Tom Switzer (Genentech) Jeremy Wyatt (ActiGraph) Xinzhi Zhang (NIH) Andrew Potter (FDA) Sue Jane Wang (FDA) Maria Ali (George Institute for Global Health) Shelly Barnes (UCB) Steven Berman (FDA) Krishna Jhaveri (Phillips) Joy Bhosai (Duke) Matthew Diamond (FDA) Katy Eichinger (University of Rochester) Jeffrey Statland (KUMC) Michelle Crouthamel (AbbVie) Phil Green (patient) Emily Cerciello (Crohn's & Colitis Foundation) Timothy Chen (Medidata) Sonja Cloosterman (Orikami) Andy Coravos (HumanFirst) Megan Doyle (Amgen) Cynthia Geoghegan (patient) Beatrix Friedeberg (Amgen) Elizabeth Kunkoski (FDA) Ingrid Oakley-Girvan (Medable) Lauren Oliva (Biogen) Colleen Rouse (Cleveland Clinic) *Now Quentin Le Masne (EMD Serono) Social Science Lead Brian Perry (CTTI) Project Manager Lindsay Kehoe (CTTI)
Pathways for FDA Engagement for Drug Development Meetings (e.g., Critical Path Innovation Meetings) IND/NDA/BLA Pathway DDT COA Qualification Pathway Outside of an individual drug development program Outside of an individual drug development program Within an individual drug development program Informal, nonbinding meetings Development of novel COAs for use in multiple drug development programs addressing unmet measurement needs Investigational New Drug (IND) submissions to FDA Potential for general CDER advice on specific methodology or technology (e.g., COAs, biomarkers) Potential to support regulatory decision- making (within the individual drug development program) Potential to result in qualification of COA
Example Frameworks for DHT-Derived Endpoint Development FDA s Roadmap to Patient- Focused Outcome Measurement in Clinical Trials Digital Medicine Society s V3 CTTI s Steps to Novel Endpoint Development 2015 2017 2020
@CTTI_Trials Lindsay Kehoe, CTTI Project Manager Lindsay.kehoe@duke.edu THANK YOU www.ctti-clinicaltrials.org
