Detailed Steps for Novel Endpoint Development with Suggested Approaches & Considerations
Developing Novel Endpoints
New CTTI Project Aims to Increase the Use of Clinical Trial Endpoints Derived from Digital Technologies
While digital health technologies (DHT) offer clinical trial researchers the promise of more efficient and potentially accurate data, there are very few examples of digitally derived primary endpoints that can support regulatory labeling claims.
To address this issue, CTTI is conducting a new project that will build on previous novel endpoint work to create additional resources and recommendations to drive the use of digitally derived, functional outcomes as key endpoints in clinical trials to support regulatory decision-making.
Once the project is complete, stakeholders can look forward to resources such as:
- Best practice recommendations on the “standards of evidence” needed to qualify a DHT-derived novel endpoint
- Glossary of terms for digital health endpoints
- Summary of interview results
- Expert meeting summary
CTTI will conduct glossary term evaluations, literature reviews, and landscape assessments – as well as in-depth interviews and multi-stakeholder expert meetings – to collect data that will inform the new recommendations and resources.
These solutions will serve to increase the use of meaningful DHT-derived novel endpoints in clinical trials for labeling claims and help to advance the benefits of running digital health trials, including the capture of more meaningful and higher quality research data.
CTTI Update: Advancing the Use of Mobile Health Technology to Transform Clinical Trials
Mobile technology has the potential to revolutionize clinical trials, but putting mobile clinical trials (MCTs) into action requires consideration of issues such as data integrity, legal and regulatory barriers, scientific and technological challenges, and perspectives of potential trial participants, investigators, and site staff. For the past two years, CTTI’s MCT Program has systematically explored barriers to MCTs, gathered evidence, and convened multiple stakeholders to tackle these issues.
By developing best practices on how to effectively integrate mobile technologies into clinical trials for regulatory submissions, CTTI intends to promote quality, efficient, and more cost-effective research. Recent developments include the following:
- CTTI’s Recommendations for Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical Trials were released in June and are prominently featured in a recent mHealth Action Plan from the Duke-Margolis Center for Health Policy.
- CTTI’s recommendations are accompanied by several practical tools and case examples that describe the process of novel endpoint development for MCTs.
- A survey of potential research participants, as well as in-depth interviews with site investigators on issues related to MCTs, are underway. Analysis of these results will begin this fall and will inform recommendations to overcome barriers to the use of mobile technology in clinical trials as perceived by key stakeholders.
- CTTI presented “Turning Opportunity Into Reality: Real Data and Practical Guidance for Incorporating Mobile Technology in Clinical Trials” at the DPharm Disruptive Innovations Conference in September 2017. The presentation included—as it relates to mobile technology—the patient perspective, tips for dealing with large volumes of data, and engaging with the FDA during trial design, execution, and monitoring.
- Two expert meetings were held in 2017, one on legal and regulatory issues and one on scientific and technological challenges affecting MCTs. Official CTTI recommendations and resources on these topics will be issued in early 2018
Update on CTTI’s Mobile Devices Project: Webinar Recording Now Available
CTTI Team Leaders Provide Update on Project Exploring how Mobile Technology Can Be Effectively Leveraged to Improve Clinical Trials
Mobile technologies, including both medical sensors and consumer products, offer exciting new possibilities for clinical research but present substantial challenges as well. Find out how CTTI’s Mobile Devices team is exploring how these technologies can be used to improve patient engagement, remote monitoring, and the collection of new kinds of high-quality, objective data capable of supporting rigorous analysis and regulatory submission.
In a webinar now available as a recording on the CTTI website, project team leaders Cheryl Grandinetti (FDA), Chris Miller (AstraZeneca), and Barry Peterson (Philips) provide an update on CTTI’s Mobile Devices Project, which focuses on scientific and technological challenges affecting the selection and use of mobile technologies in clinical research. The presenters share preliminary evidence gathered from research sponsors and clinical investigators experienced in using mobile devices in clinical trials. Issues addressed in the webinar include:
- Challenges affecting data management, validation, analysis, and security in mobile technologies;
- Scientific and technical considerations in mobile device selection; and
- Approaches to managing and reporting safety signals and adverse events.
After additional evidence and insights from this project are gathered, the team will release recommendations designed to help overcome challenges that are currently hindering the wider use of mobile technologies in clinical trials.
The Mobile Devices Project is one of four distinct efforts taking place through CTTI’s larger Mobile Clinical Trials Program. The MCT Program is developing evidence-based recommendations to guide the widespread adoption and application of mobile technologies in clinical trials. The three other projects within the program are focused on:
- Legal and regulatory issues
- Development and use of novel endpoints for use with mobile technologies
- Stakeholder perceptions regarding the use of such technologies
CLICK HERE to view recordings of other CTTI-hosted webinars.
Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical Trials
SEPTEMBER 29, 2016 TO SEPTEMBER 30, 2016
CTTI Project: Developing Novel Endpoints
Meeting Objectives
Identify how to develop novel endpoints for use in clinical trials by writing the following use cases:
- Parkinson’s Disease: Physical activity level and gait, measured using an accelerometer, as an endpoint for Parkinson's disease
- Heart Failure: Physical activity level, measured using an accelerometer, as an endpoint for heart failure
- Diabetes: Blood sugar level, measured using a wearable continuous glucose monitor, as an endpoint for diabetes
- Muscular Dystrophy: Physical activity level, measured using
Meeting Location:
DoubleTree Silver Spring Hotel by Hilton, 8727 Colesville Rd, Silver Spring, Maryland
Meeting Presentations
- Mobile in Clinical Trials: Developing Novel Endpoints for use in Clinical Trials by Jennifer Goldsack
- Clinical Trial Endpoints from Mobile Technology: Regulatory Considerations by Elektra J. Papadopoulos
- Assessing and Supporting Outcome AssessmentsCategories, Context, and Evidence: Mobile Technology Novel Endpoints Workshop by Marc K. Walton
- Performance Outcome Assessments: Identifying Endpoints that are Meaningful and Measurable by Stephen Joel Coons
- Developing Performance Outcome Measures for Use with Regulatory Agencies: The Aging In Motion (AIM) Coalition Experience by Cynthia A. Bens
- Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical Trials: The Use Case Approach by Jennifer Goldsack
The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.