Patient Engagement
CTTI Holds Meeting to Discuss Engaging Stakeholders in Trial Design
The Clinical Trials Transformation Initiative (CTTI) held a multi-stakeholder Expert Meeting on April 4 to discuss high-value approaches and situation-specific considerations for meaningful engagement of internal and external stakeholders in clinical trial design and implementation. Engaging all stakeholders during the earliest stages of study development is an important feature of quality by design (QbD). Aligned with CTTI’s QbD approach, the Engaging Stakeholders in Trial Design Project team is developing an engagement roadmap and recommendations to enable clinical trial designers to meaningfully and effectively engage all stakeholders across the trial design process. The project team is also collating resources surrounding the engagement of stakeholders to increase the efficiency of trial design to benefit patients faster.
Experts from academia, clinical research organizations, patient advocacy groups, regulatory agencies, and the pharmaceutical industry discussed the challenges and opportunities, key strategies, and metrics for assessing holistic stakeholder engagement across the continuum of clinical trials. The following key themes were emphasized during the meeting:
- It is important to engage key stakeholders – including patients, site staff, and regulatory agencies – very early in the design of clinical trials. When planning study timelines, identify all internal and external stakeholders and the appropriate time and approach to solicit their input.
- Stakeholder engagement should be an iterative process throughout the life cycle of clinical trials. Internal and external stakeholders should be engaged, as appropriate, from the beginning of clinical trial design through the dissemination of results.
- Bring stakeholders and functional groups together to identify gaps between teams and brainstorm solutions to increase the quality and efficiency of trials. Effective communication and collaboration across all stakeholder groups is crucial to create a community around designing high-quality studies that meet the needs of patients and that generate reliable evidence with fewer amendments.
- Advanced methodologies and tools, such as artificial intelligence and machine learning, can help study designers develop innovative, high-quality trials and streamline processes. With databases of real-world and clinical trial data, artificial intelligence and machine learning can be used to inform the design of trials, explore potential treatments for patient subgroups, assess feasibility of enrollment, and identify sites.
- A collection of resources for designing clinical trials, including recommendations on how and when to meaningfully engage all stakeholders, is needed to help study designers plan innovative clinical trials more efficiently and in alignment with regulatory guidance.
Engaging Stakeholders in Trial Design Expert Meeting
April 4, 2023
CTTI Project: Engaging All Stakeholders in Clinical Trial Design
Meeting Objectives:
- Review two clinical trial ‘models’ where stakeholder engagement was well-executed
- Discuss and explore opportunities, barriers, and best practices for study designers to engage all stakeholders in trial design
- Identify situation-specific considerations for ensuring engagement is appropriately equitable, effective, and feasible
Meeting Location: Washington, D.C.
Meeting Materials:
- Welcome Remarks and Opening Comments
- Introduction to the Clinical Trials Transformation Initiative (CTTI)
- Engaging Stakeholders in Trial Design Project Overview
- Meeting Objectives
- Session 1: Models for Success
- Review of two well-executed trials
- Key milestones and interactions that supported success
- Discussion: What does a well-engaged trial look like?
- Methods and milestones
- Roadblocks to engagement
- Session 2: Challenges and Opportunities
- Panel discussion: opportunities, barriers, and best practices for engaging all stakeholders in trial design
- Building the value proposition of engaging stakeholders in trial design
- Timing of stakeholder engagement
- Operationalizing stakeholder feedback
- Simplifying the process
- Measuring the impact
- Panel discussion: opportunities, barriers, and best practices for engaging all stakeholders in trial design
- Session 3: Break Out Sessions
- Timing of stakeholder engagement
- Simplifying the stakeholder engagement process
- Measuring the impact of stakeholder engagement in the design of clinical trials
The views and opinions expressed in these presentations 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.
Patient Engagement Collaborative Announces Eight New Members
Today, the U.S. Food and Drug Administration (FDA) and the Clinical Trials Transformation Initiative (CTTI) announced eight newly selected representatives for the Patient Engagement Collaborative (PEC). The group of 16 patients, caregivers, and patient group representatives will meet with the FDA several times a year to discuss topics such as communication, transparency, and the best ways for patients to engage the FDA about medical product regulation. Those selected to the PEC are expected to serve for two years.
The eight new representatives are:
- Kim Hindery
- Yasmin Ibrahim
- Seth Morgan
- Cara O’Neill
- Ceciel Rooker
- Christina Sisti
- Trina Stelly
- Lauren Youngborg
The representatives were selected from 75 applications received in response to a Federal Register notice published in September 2022. These new members of the PEC include patients who have personal disease experience, caregivers who have personal experience supporting someone with a health condition, and representatives from patient groups who have direct or indirect disease experience. The selection committee, which included patient advocates, FDA staff from multiple centers, CTTI staff and several outgoing PEC members, worked to identify individuals with diverse perspectives and experiences who could meaningfully contribute and express the patient voice.
“CTTI is delighted to welcome the new members of the PEC and we sincerely appreciate the contribution of those outgoing members and extend our deepest thanks for their dedication and service,” said Sally Okun, CTTI’s executive director. “Including patients as equal partners in clinical research is a foundational value of CTTI. Discussions with the PEC contribute to advancing our vision of Transforming Trials 2030, in particular our first pillar: ‘to make clinical trials patient-centric and easily accessible’.”
The PEC, a collaboration established by FDA and CTTI in 2018, is a group of patient organizations and individual representatives who discuss ways to enhance patient engagement. FDA and CTTI collaborate to involve representatives with a variety of perspectives including patients, caregivers and representatives from diverse patient organizations and communities. The PEC is run by the FDA’s Patient Affairs, which is dedicated to providing an inviting, welcoming and meaningful experience for patient communities to engage with the FDA.
“Understanding patients’ experiences is critical to support medical product regulation and help ensure safe, effective and innovative medical products are available for patients. The PEC is an important opportunity for individuals to share their experience with the FDA. The FDA listens closely to feedback from individuals in the PEC and uses the insight from the PEC to enhance engagement opportunities and provide a platform for patients’ voices to be heard,” said Andrea Furia-Helms, M.P.H., Director of Patient Affairs, Office of Clinical Policy and Programs, FDA.
Going forward, the PEC will continue the dialogue around how patient perspectives can inform and enhance the clinical trials enterprise. Previous topics have included: creating new ways to collaborate with patient communities; making patient engagement more systematic; improving transparency through education and outreach; and enhancing communication between the FDA and patient communities.
The FDA and CTTI wish to thank the outgoing members for their service to the PEC over the past several years:
- Ronald Bartek
- Anne Hall
- Elizabeth Joniak-Grant
- Isabelle Lousada
- Rick Phillips
- Philip Posner
- Lynne Quittell
- Adrienne Shapiro
The PEC has worked tirelessly to incorporate the patient voice in the FDA’s medical product regulatory programs. For example, the PEC has:
- Provided enhanced information to patient communities (e.g., for patients webpage).
- Created materials to help patients understand FDA’s mission and patient engagement activities.
- Worked to better understand COVID-19’s impact on clinical trials for other diseases and conditions through informal discussions.
- Worked to understand patient concerns regarding COVID-19 vaccines.
- Met with the European Medicines Agency’s Patients’ and Consumers’ Working Party in 2021 to exchange ideas around enhancing patient engagement, particularly incorporating youth perspectives and in 2022 to discuss COVID-19 communication.
Since 2008, CTTI has included patient advocates on its Executive Committee, Steering Committee, and project teams and, today, nearly all of its more than 30 sets of evidence-based recommendations and associated frameworks and tools mention inclusion of patients as a critical part of the clinical trials process.