The Patient Technology Initiative aims to enable and accelerate the adoption of patient-facing digital technologies (PT) in clinical trials. PT presents opportunities to transform drug development and the patient experience. New or improved measurements for known and novel endpoints are foundational to the opportunities of PT. To highlight the importance of this opportunity, the Patient Technology Initiative published the Novel Digital Endpoints Validation Pathway Paper in Digital Biomarkers. This peer-reviewed paper provides a use case proposal and considerations when developing and obtaining regulatory acceptance for a novel digital endpoint to assess sleep in patients with rheumatoid arthritis (RA)
To encourage patient-centric collaboration in the space, the Patient Technology Initiative also completed a first of its kind exercise in knowledge sharing. Leveraging TransCelerate’s membership, the initiative team collected member company experience implementing PT in clinical trials. The resulting insights and findings from that knowledge sharing exercise can be found in a team-authored white paper, “A Shared Perspective of Patient Technology Implementation in Clinical Trials.”
To further assist the goal to enable and accelerate the adoption of PT in clinical trials, the Patient Technology Toolkit was developed. This Toolkit was developed based on foundational research published in the Patient Technology manuscript.
What problems are we trying to solve?
Though the potential benefits of using PT are widely discussed, they are not yet fully realized because:
A variety of regulatory, technical, cultural, and operational obstacles make it difficult for sponsors to progress novel PTs from pilot phase to scale
Lack of communication between stakeholders (both internal and external) limits learning over time, resulting in sub-optimal PT implementation
Risks and unknowns associated with PT may be overwhelming and study teams often struggle to navigate them
The Patient Technology Toolkit was designed to:
Help decision-makers and study teams approach PT implementation strategically with long-term goals and scalability in mind
Facilitate the flow of knowledge and feedback between internal and external clinical trial stakeholders with respect to PT
Help study teams identify, prevent or mitigate potential risks associated with PT adoption
Not Sure Where to Get Started?
Check out these common scenarios identified by clinical study teams, and how the toolkit can be leveraged to address technical challenges
How is the Toolkit Used?
Navigate Jim's use of the toolkit by clicking on the icons for each step.
Jim
Jim is interested in using a digital patient-facing technology in his Rheumatoid Arthritis trial to measure sleep. He needs to understand:
Jim uses the PT Vendor and Sponsor Engagement considerations to continually monitor, discuss and improve his and the vendor’s approach to implementing patient technology
Implementation Framework
PT Discussion Guide & *Patient Considerations
Share Information
Jim first uses the Implementation Framework to help him understand the end-to-end process, develop strategy, identify potential risks, and plan for implementation.
He then uses the PT Discussion Guide to help engage with internal and external stakeholders, and *Patient Considerations to ensure the patient perspective is appropriately taken into account during the implementation of the Patient Technology.
He consults the Regulatory Landscape Tool to understand the potential regulatory impact on his project.
Jim then returns to the Implementation Framework to ‘de-risk’ technologies and implementation strategies through rapid, iterative testing.
During the trial, Jim uses the *PT Site Feedback Questionnaire to learn from sites how implementation could be improved in the future.
Jim debriefs his implementation experience with his internal team and vendor, sharing the current learnings across his organization and with the vendor to improve and scale future PT implementation.
Regulatory Landscape Tool
*PT Site Feedback Questionnaire
Implementation Framework
Implementation Framework
Jim first uses the Implementation Framework to help him understand the end-to-end process, develop strategy, identify potential risks, and plan for implementation.
PT Discussion Guide
He then uses the PT Discussion Guide to help engage with internal and external stakeholders, and *Patient Considerations to ensure the patient perspective is appropriately taken into account during the implementation of the Patient Technology.
Regulatory Landscape Tool
He consults the Regulatory Landscape Tool to understand the potential regulatory impact on his project.
Implementation Framework
Jim then returns to the Implementation Framework to ‘de-risk’ technologies and implementation strategies through rapid, iterative testing.
PT Site Feedback Questionnaire
During the trial, Jim uses the *PT Site Feedback Questionnaire to learn from sites how implementation could be improved in the future.
Share Information
Jim debriefs his implementation experience with his internal team and vendor, sharing the current learnings across his organization and with the vendor to improve and scale future PT implementation.
*Solutions within the Patient Technology Toolkit that were updated to account for modern clinical trial approaches (e.g., decentralized clinical trials, patient inclusion and diversity, and other topics).
Novel Digital Endpoints Consideration Paper
Novel Digital Endpoints Consideration Paper
POV Manuscript
Implementation Framework
PT Discussion Guide
*Patient Considerations
Regulatory Landscape Tool
*PT Site Feedback Questionnaire
Vendor Engagement & Vendor-Influenced Tools
Novel Digital Endpoints Consideration Paper
Novel Digital Endpoints (NDEs) are an evolving field and have the potential to unlock many opportunities within the drug development lifecycle. The NDE Sub-team has developed an NDE opinion paper that summarizes key considerations (complemented with a case study) on how to develop and navigate the path to Health Authority approval to use digital tools for NDE development in a clinical trial. Download now—Novel Digital Endpoints Consideration Paper
Our presenters followed Digital Medicine Society’s (DiMe) V3 framework and Clinical Trials Transformation Initiative’s (CTTI) framework in a hypothetical case to validate a sleep endpoint. They identified key considerations and challenges and described approaches for developing novel digital endpoints for use in medical product development. Additionally, they highlighted the importance of regulatory strategy in the development and validation of novel digital endpoints.
Watch now – Developing a Novel Measurement of Sleep in Rheumatoid Arthritis: Study Proposal for Approach and Considerations Public Webinar
POV Manuscript
In our manuscript, the PT Initiative describes the potential for patient-facing digital technologies (also called “Patient Technology”) to serve a variety of functions in clinical trials, such as capturing clinical endpoints, engaging patients, and facilitating remote study conduct. However, these technologies are not yet accepted as mainstream research tools.
To understand the barriers and facilitators affecting PT adoption, the TransCelerate Patient Technology Initiative conducted a series of surveys, interviews, and focus groups with approximately 600 subject matter experts (SMEs), including pharmaceutical company representatives, clinical trial investigators, and clinical trial participants. We identified several challenges that were common across the industry and outlined several recommendations to address them.
TheImplementation Framework outlines a possible approach to Patient Technology implementation that encourages a mindset of strategic thinking, rapid learning, and patient-centric design. It can be used by study or strategy teams to:
Get started or move forward with PT implementation
Incorporate an iterative, rapid-learning approach to PT
Consider patient and site perspectives during program design
Enable PT programs to build toward a company’s unique strategic vision
Mitigate common risks associated with PT implementation
For additional information, see our Implementation Framework FAQs.
PT Discussion Guide
The Discussion Guide provides a list of core questions to consider during the implementation of PT in clinical trials. It enables users who are new to PT to identify risks, ask the right questions, and engage relevant stakeholders early in the PT evaluation and implementation process. The guide covers considerations related to:
Budget & Resourcing
Feasibility & Quality
Legal & Regulatory
Process & Strategy
Scalability & Support
Site Impact
Technology & Infrastructure
The Discussion Guide can be accessed as a standalone tool or as an integrated supplement to the Implementation Framework.
*Patient Considerations
The *Patient Considerations Guide provides a list of core questions and considerations to think about during the implementation of PT in clinical trials, including any considerations that may be applicable to decentralized trials. It was developed by asking a diverse set of patients what they want sponsors to think about when deciding to implement patient-facing digital technology. The Patient Considerations Guide enables study teams to think critically about how their decisions will impact patients. The document covers considerations related to the topics of:
Patient Benefits
Patient Burden
Patient Compliance
Patient Consent and Comprehension
Patient Experience
(NEW) Patient Inclusion and Diversity
Patient Safety
Patient Willingness to Participate
Privacy
Technology Maintenance
Technology Support
User Experience Design
Regulatory Landscape Tool
The Regulatory Landscape Tool provides an overview of US and EU Health Authority regulation and guidance related to digital technologies and their use in clinical trials. It covers:
Medical device classification and associated requirements
Related regulations and guidance pertaining to clinical trial conduct
Pathways for obtaining Health Authority feedback
Policies and initiatives that might reflect or impact Health Authority positions on the use of digital technologies
For additional information, see our Regulatory Landscape Tool FAQs.
*PT Site Feedback Questionnaire
The *PT Site Feedback Questionnairecaptures actionable feedback on sites’ experiences with patient-facing technology and sites’ impressions of patient experience using technology over the course of a clinical trial. This structured feedback tool can help:
Identify and mitigate potential issues as the study progresses
Overcome potential burden added in the decentralized clinical trial environment, such as handling of technology interruptions, the impact on data management, and training patients on technology
Inform future study design based on user feedback
Accelerate adoption of the same or similar patient-facing technology(ies)
Using Vendor Perspectives to Implement Patient Technology in Clinical Trials An engaging and insightful infographicdescribing key findings from surveys and interviews with patient technology vendors. Includes findings related to industry challenges and methods to improve vendor and sponsor relationships.
Vendor & Sponsor Engagement Considerations The Vendor & Sponsor Engagement Considerationsare a list of core questions by topic to think about during and after the implementation of PT with a vendor in clinical trials. The considerations were developed with vendor input and are designed to assist the facilitation of more routine two-way feedback discussions on the deployment of PT. The topics and ideas in the document can help enable open discussion environments between parties where learnings can be shared, documented and leveraged for future studies.
