Program: Mobile Clinical Trials (MCT)

About This Program

Project:

Novel Endpoints

overview icon Overview

Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical Trials

Mobile technology can be used in clinical trials to generate novel endpoints[1] that provide new kinds of information that were previously difficult or impossible to access. Novel endpoints derived from mobile technologies can make clinical trials more efficient and less burdensome to trial participants while contributing to meaningful, real-world understanding about patient experiences and outcomes that goes beyond the brief data “snapshots” typically gathered in clinical care or research settings.

But despite these potential benefits, novel endpoints are at present only rarely used in clinical trials. This is due to barriers arising from widespread uncertainty about how to identify and choose novel endpoints that would be useful in trials designed to support regulatory approval for new therapies, or a new indication for an existing therapy.

CTTI’s Novel Endpoints Project, a part of CTTI’s larger Mobile Clinical Trials (MCT) Program, has developed recommendations and tools to address these barriers and clarify the best pathways for identifying, selecting, and developing novel endpoints derived from mobile technologies. Along with these actionable recommendations, CTTI created four detailed use cases that show how mobile technologies can be used to collect novel endpoints in regulatory clinical trials.


[1] Definition of a novel endpoint: new endpoints that are 1) not currently used, or 2) existing endpoints that can now be measured in new ways, using mobile technology.

quick links icon Quick Links to Top Deliverables

Discover Additional Deliverables Generated From This Project

Webinar

Official Unveiling of Recommendations on Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical Trials

PowerPoint Slide of Positive Impacts

Download the .pptx slide of “Positive Impacts of Developing Novel Endpoints for Use in Clinical Trials” to use in your presentations. (Note: The Positive Impacts are also included in the first page of the Recommendations above.)

Poster

Developing Novel Endpoints, Generated using Mobile Technology, for use in Clinical Trials: A Clinical Trials Transformation Initiative (CTTI) Project (Presented at SCT 2017 on May 8)

Expert Meeting Materials

Click to view materials, including presentations & summary, for the CTTI-hosted expert meeting, Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical Trials

“By engaging with experts who have been early champions of mobile technology in trials and combining that with patient insights, CTTI has created practical recommendations and action-oriented tools that have the potential to really accelerate the use of mobile technology in clinical trials. In particular, the use cases provide a realistic pathway for incorporating novel endpoints through technology into clinical development programs. CTTI’s recommendations show we may be closer than previously believed to realizing the benefits of these novel endpoints, creating a sense of urgency to act.”

Craig Lipset Pfizer’s head of clinical innovation

in detail icon In Detail

Despite the evolution of mobile technology capable of monitoring and measuring data, it remains uncommon for such novel clinical endpoints to be used in clinical trials, especially those trials that are performed to support regulatory approval for a new therapy or indication. All stakeholders—patients and patient groups, investigators, research sponsors, regulators, and technology developers—would benefit from clarification regarding the processes for developing these novel endpoints in order to realize the benefits of incorporating mobile technology into clinical trials.

Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical Trials (2015–2017), one of four projects within CTTI’s MCT Program.

Describe best practices for identifying, selecting, and developing novel endpoints, generated using mobile technology, for use in regulatory clinical trials.

The results from the MCT Novel Endpoints Project will inform the development of novel endpoints generated from mobile technologies for use in FDA-regulated clinical trials.

CTTI hosted a multi-stakeholder expert meeting to develop four use cases describing the steps involved in developing a novel endpoint from data generated by a mobile device. This process helped CTTI to outline requirements at each step in the process, identify challenges that may occur, and propose solutions to those challenges. The use cases informed the development of more general recommendations and tools that clarify the pathway for developing technology-derived novel endpoints. The MCT Novel Endpoints Project Team has also conducted a systematic review of published clinical studies using novel endpoints in order to learn from existing approaches. This article will be posted here following publication.

CTTI convened a multi-stakeholder expert meeting to draft use cases, each developing a particular novel endpoint. Meeting attendees explored the use of accelerometers to measure treatment benefit in clinical trials of heart failure, Parkinson’s disease, and Duchenne muscular dystrophy, and the use of continuous glucose monitors to measure treatment benefit in clinical trials of diabetes. In addition to the four use cases, a meeting summary is available that highlights themes from these discussions.

A set of recommendations were distilled from the meeting findings and related discussions. The recommendations, which are expected to have short-, medium-, and long-term benefits when applied in clinical trials, are grouped into two main sections. The first, which focuses on optimizing the selection of novel endpoints, identifies the following key recommendations:

  1. Patient’s voices must drive the identification of novel endpoints;
  2. Select the mobile technology after selecting an outcome assessment; and
  3. Use a systematic approach to identify key novel endpoints.

The second section provides recommendations on practical approaches to developing novel endpoints, including:

  1. Foster collaboration among key stakeholders;
  2. Create technical standards for assessments derived from mobile technology;
  3. Engage with regulators; and
  4. Include novel endpoints as exploratory endpoints in existing clinical trials and observational studies.

These major recommendations are presented in detail, along with complementary materials that include an interactive tool for choosing among possible novel endpoints for development, a quick reference for interacting with regulatory authorities during the process of novel endpoint development, a summary of required steps in novel endpoint development, a plain-language process summary, and detailed, disease area-specific use cases.

The team has also conducted a systematic review of published clinical studies that have employed novel endpoints. The resulting paper will be posted here following publication.

Project Team Members

Role Namesort descending Affiliation
Team Member Ashish Narayan Feinstein Institute for Medical Research
Social Science Lead Brian Perry CTTI
Team Leader Cheryl Grandinetti U.S. Food and Drug Administration
Team Member Elektra Papodopoulos U.S. Food and Drug Administration
Project Manager Jen Goldsack CTTI
Executive Committee Champion John Alexander Duke University
Team Leader Kaveeta Vasisht U.S. Food and Drug Administration
Team Member Ken Skodacek U.S. Food and Drug Administration
Team Member Komathi Stem monARC Bionetworks
Team Leader Lauren Battaille The Michael J Fox Foundation for Parkinson's Research
Team Member Marc Walton Johnson and Johnson Pharmaceutical Research and Development
Team Leader Martin Landray University of Oxford, CTSU
Team Member Nirav Sheth MC10
Team Leader Rob DiCicco GlaxoSmithKline
Team Member Theresa Strong Foundation for Prader-Willi Research
Team Leader Will Herrington University of Oxford, CTSU