CTTI News

Patient Engagement Collaborative Announces Eight New Members

CTTI News | December 11, 2024

Topics Included: Patient Engagement

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 caregivers, patients, and patient group representatives meets with the FDA several times a year and discusses a variety of topics such as improving communications, education, and patient engagement related to medical product regulation. Those selected to the PEC are expected to serve for two years.

The eight new representatives are:

  • Bernard Coley
  • Lisa Craine
  • Rebecca Esparza
  • Syreen Goulmamine
  • Heather Guidone
  • Carter Hemion
  • Toni Jaudon
  • Mary Anne Meskis

The representatives were selected from 75 applications received in response to a Federal Register notice published in July 2024. These new members of the PEC include caregivers who have personal experience supporting someone with a health condition, patients who have personal disease experience, and representatives from patient groups who have direct or indirect disease experience. The selection committee, which included 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.

The PEC, a collaboration established by the FDA and CTTI in 2018, is a group of patient organizations and individual representatives who discuss ways to enhance patient engagement. The FDA and CTTI collaborate to involve representatives with a variety of perspectives including caregivers, patients, and representatives from diverse patient organizations and communities. The PEC is run by the FDA’s Public Engagement Staff, which is responsible for communicating, educating, and engaging with external partners including academia, caregivers, consumers, health care providers, patients, and trade associations on the agency’s policy priorities.

Going forward, the PEC will continue the dialogue around how patient perspectives can inform the clinical trials enterprise and provide information and resources to enhance PEC members’ knowledge about the FDA and CTTI activities to share with patient communities. Previous topics have included: increasing awareness of existing engagement mechanisms among patients and patient groups; increasing patients’ education to empower them to act as representatives of their broader community; enhancing communication between the FDA and patient communities; and promoting global collaboration of patient organizations.

The FDA and CTTI wish to thank the outgoing members for their service to the PEC over the past several years:

  • Kim Hindery
  • Yasmin Ibrahim
  • Seth Morgan
  • Cara O’Neill
  • Ceciel Rooker
  • Christina Sisti
  • Trina Stelly
  • Lauren Youngborg

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.

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Holiday Greetings from CTTI: Celebrating a Year of Progress

CTTI News | December 10, 2024

Topics Included:

As 2024 draws to a close, we reflect on a busy year of collaboration at the Clinical Trials Transformation Initiative (CTTI). Thanks to your shared commitment to advancing clinical research, we have developed critical new resources to assist with registration and reporting of clinical trials as well as assessing the ability to embed trial elements into clinical practice.

To showcase these achievements and more, we are thrilled to share our first-ever digital annual report, Modernizing Trials Together. This interactive report celebrates the resources we published in 2024 and highlights our collective impact on the clinical trials landscape.

At CTTI, we believe that actionable solutions underpin meaningful behavior change. That’s why in 2024 we focused on creating foundational tools and assessments like the Embedding Trials Feasibility Survey and the ClinicalTrials.gov report that empower stakeholders to improve processes.

Equally important, though, is our role as a connector in the clinical trials community. Through events like our new CTTI Dinners, panel discussions, public workshops, and Steering Committee meetings, we have fostered open dialogue among industry leaders, patients, regulatory agencies, and researchers. These gatherings are more than just opportunities to meet colleagues — they are the building blocks of future collaborations to make clinical research more impactful, inclusive and practical.

Looking to 2025, we are energized by the biological, computational, and technological advances that are redefining what’s possible in medical product development. CTTI will continue to anticipate the corresponding needs on the clinical development side — ensuring we have the tools for efficient, high quality clinical trials.

We wish you a joyful holiday season and a healthy, productive year ahead.

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New CTTI Project Aims to Identify Challenges and Solutions to Strengthen Proportionate Enrollment in Clinical Trials 

CTTI News | December 3, 2024

Topics Included: Access to Clinical Trials

In December 2024, CTTI launched the Collective Strategies to Enhance Proportionate Enrollment project to improve communication, accountability and clarity across clinical trial partner groups. The project focuses on addressing persistent challenges that hinder the enrollment and retention of participants who reflect the populations most affected by the disease under study. 

Through this effort, CTTI aims to foster unified, action-focused collaboration among sponsors, contract research organizations, sites, patient organizations and other key groups involved in clinical research. By strengthening coordination and aligning expectations, the project will help identify practical strategies to improve how trials are planned and executed. 

To inform this work, CTTI is gathering evidence through qualitative methods, including interviews with individuals involved in clinical trial operations, to better understand group-specific needs and challenges. These insights will shape a set of recommendations and tools that clarify roles, outline implementation considerations and support proportionate enrollment across the trial lifecycle. 

Ultimately, this project will support the generation of high-quality, reliable data and the development of medical products that are safe and effective for all populations. These efforts align with CTTI’s Transforming Trials 2030 vision to create a more efficient, patient-centered and representative clinical trial enterprise. 

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CTTI Leads Discussion on Digital Health Trials at ETH Digital Biomarkers Summit

CTTI News | November 27, 2024

Topics Included: Data Collecting and Reporting, Innovative Trials, Site Planning

In November 2024, Lindsay Kehoe, senior project manager at CTTI, presented at the Digital Biomarkers Summit held at ETH Zurich. The event gathered experts from academia, healthcare, and industry to discuss the transformative potential of wearable technologies in healthcare. Among the many topics covered, a key focus was the translation of digital innovations, such as wearable devices for sweat and breath, into clinical implementation to revolutionize healthcare. Digital technologies enable real-time, continuous monitoring of patient data, providing researchers and healthcare providers with deeper insights into patient health, improving trial design, and enhancing data accuracy.

In her workshop, Setting Up Digital Health Trials, Kehoe offered actionable insights on designing successful, fit-for-purpose digital health trials. She outlined key benefits, such as improving trial efficiency, fostering patient-centric research, and ensuring more reliable data. Kehoe also addressed common questions and challenges in implementing digital health trials and shared practical solutions for overcoming them. A central theme of her talk was the importance of communication and collaboration, emphasizing that “collaboration is key to bringing cutting-edge innovation into trials, addressing unmet needs, and improving trial efficiency.”

Dr. Jörg Goldhahn, a CTTI member and Professor at ETH Zurich, echoed Kehoe’s point, noting, “New technology offers great potential for novel biomarkers that can be used remotely, closer to ground truth, and on a continuous basis. However, intensive interprofessional collaboration is required to take these technologies from proof-of-concept into clinical use.”

Kehoe also discussed the importance of equipping researchers with the right tools and resources to successfully implement digital health strategies. She introduced essential resources from CTTI’s Digital Health Trials Hub, designed to guide researchers through key aspects of trial design, including selecting measures and appropriate technologies, managing data securely, developing novel endpoints, and collaborating effectively with clinical sites, patients, and regulators for seamless trial execution.

A notable example of how CTTI’s Digital Health Trials Recommendations are being put into practice is at ETH Zurich, where the institution has integrated these recommendations into its Digital Trial Intervention Platform within the Translational Science Advanced Studies program. This platform enhances the flexibility and scalability of clinical research and aims to optimize digital clinical study designs. These initiatives underscore the real-world impact of CTTI’s resources, demonstrating how we can take digital tools from bench to bedside and drive innovation in clinical research.

For those interested in seeing how CTTI’s resources are being applied in other research settings, the Case Study Exchange offers a range of real-world examples, showcasing how different organizations are integrating innovative tools and approaches into clinical trials.

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New Publication Shares Findings & Recommendations for Embedding Clinical Trial Elements into Routine Clinical Practice

CTTI News | November 15, 2024

Topics Included: Data Collecting and Reporting, Innovative Trials, Site Planning

A new CTTI publication published in the Journal of Clinical and Translational Science highlights the qualitative research findings that informed recommendations to facilitate the integration of clinical trials into clinical practice. By integrating key elements of clinical trials—such as patient identification and data collection—into everyday practice, research efforts can be streamlined to reduce duplication of activities. However, several administrative, cultural, and data-related challenges must be addressed to fully realize these benefits. 

The publication identifies barriers to embedding clinical trials into routine practice, including potential disruptions to workflow, challenges in obtaining buy-in from staff, the need for robust data and research infrastructure, and the necessity for a culture shift to value and support research. 

To address these barriers, recommendations are provided based on in-depth interviews and insights from two expert meetings hosted by CTTI. Key suggestions include ensuring healthcare data sources are research-ready, aligning trial designs with existing clinical workflows, preparing sites for trial integration, and securing support from leaders at regulatory, funding, and health system levels to champion the value of embedding trials in routine practice. 

Integrating trial elements into everyday clinical practice does not have to be an “all or none” approach. Even incorporating a few elements can bring significant benefits. By addressing the identified barriers and following these recommendations, long-term improvements in operational efficiency and patient outcomes can be achieved.

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Recent CTTI Publication Reviews Current Applications and Future Opportunities for Disease Progression Modeling

CTTI News | October 17, 2024

Topics Included: Innovative Trials, Regulatory Submissions + Approvals

A new CTTI publication, published in Clinical Pharmacology & Therapeutics, reviews current applications of disease progression modeling (DPM) and opportunities to advance the awareness and value of DPM in clinical trials. Use of modeling and simulation during drug development, otherwise known as model-informed drug development (MIDD), can help researchers make informed decisions when planning and executing clinical trials. DPM is one form of MIDD that integrates multiple types of data from various sources – including translational, clinical trial, and real-world data – to optimize trial design and execution and support regulatory decision-making. The potential of DPM has yet to be fully realized.  

To advance the use of disease progression modeling for decision making in clinical trials and medical product development, CTTI assembled a diverse project team, conducted a scoping literature review to identify current DPM applications, and shared these results at a multi-partner Expert Meeting with representatives from academia, data/tech organizations, government, the medical product industry, patients, and trade/professional organizations. They discussed the value of disease progression modeling and identified key areas that need to be addressed to incorporate DPM approaches more effectively in medical product development and regulatory decision-making. 

The paper presents the scoping review results, opportunities to increase uptake identified at the Expert Meeting, and proposes key questions that medical product developers and regulators may use to inform clinical development strategy and optimize trial design with disease progression models.

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New CTTI Project Aims to Offer Clarity Around Implementing Flexible Clinical Trial Approaches While Maintaining Data Quality

CTTI News | October 2, 2024

Topics Included: Innovative Trials

Implementing less rigid, more flexible approaches to clinical trials can increase patient and site access to research, improve participant satisfaction, facilitate real-world data use, enroll more diverse patient populations, and enable more generalizable trial results. Ultimately, these flexible trial approaches help sponsors bring new therapies to patients faster. However, despite their numerous advantages, questions remain about how to maintain data quality when adopting these strategies. High-quality trial data must be fit-for-purpose, credible, and reliable to support regulatory decision-making without jeopardizing participant safety or the integrity of trial results.

To address these questions, CTTI has started a new project to gather diverse perspectives on the advantages and disadvantages of flexible trial approaches. This includes methods such as embedding trials in clinical practice, incorporating decentralized trial elements, and allowing for setting adjustments during trial participation. Through multi-partner roundtable discussions, in-depth interviews, and surveys, CTTI will identify critical concerns and considerations for maintaining data quality when using flexible trial approaches. CTTI will outline case examples of flexible approaches in trials, identify common themes and responses from partners around these case examples, and consider a toolbox that outlines the strengths of implementing flexible approaches and provides recommendations for how to maintain data quality when incorporating them.

Pamela Tenaerts, CTTI project team lead and representative from Medable, emphasized the potential of flexible methodologies, stating, “Embracing more flexible methodologies for data collection enables us to better represent the populations we’re looking to serve while providing a better experience for participants.”

CTTI’s work in this area will help to increase the use of flexible approaches in clinical trials, resulting in improved trial access, participant satisfaction, enrollment of diverse patient populations, and trial efficiency.

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Recording of Hybrid Public Workshop Now Available: AI in Drug and Biological Product Development v2

CTTI News | September 23, 2024

Topics Included: Data Collecting and Reporting, Ensuring Quality, Innovative Trials, Patient Engagement, Recruitment, Regulatory Submissions + Approvals, Safety, Site Planning

The recording for the hybrid public workshop on AI in Drug and Biological Product Development convened and hosted by the U.S. Food and Drug Administration (FDA) and Clinical Trials Transformation Initiative (CTTI), held August 6, is now available. Over 9,000 registrants from diverse backgrounds and perspectives joined to explore the responsible use of AI in drug development. 

During the workshop, we learned from experts as they discussed guiding principles for the responsible use of AI in the development of safe, effective, and high-quality drugs. Drawing on real case examples, experts discussed their rationale for particular approaches, shared their methods for evaluating success, recounted challenges and obstacles, explored options for scaling and wider applicability, and outlined considerations for moving forward. Presentations can also be found here. 

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CTTI’s Trials in Clinical Practice Resources are Valuable for Those Looking to Design and Conduct Randomized Trials that Integrate into Routine Clinical Practice

CTTI News | September 23, 2024

Topics Included: Data Collecting and Reporting, Innovative Trials, Site Planning

The FDA recently issued new guidance titled “Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice Guidance for Industry.” Those looking to put concepts from the guidance into action will find a range of valuable resources in CTTI’s Trials in Clinical Practice Toolkit. From recommendations and a site feasibility tool to case examples, these resources provide organizations and individuals with actionable operational and trial design considerations noted in the guidance. 

CTTI Senior Project Manager Lindsay Kehoe provides further insight in the recent Clinical Leader article, “Putting It Into Practice: Why We Need Embedded Clinical Trials,” highlighting the need to incorporate elements like randomization and informed consent from trials into clinical practice to bridge the gap between research and practice. She underscores the potential for this integration to lead to a boost in knowledge, reduction in duplication of resources, and ultimately, enhanced patient care. Kehoe suggests that integration of trial elements is not all or none, benefits can be seen regardless of their number, and emphasizes the need for data to be relevant and reliable. She concludes that the success of this integration hinges on several factors: access to appropriate data, alignment of trial design with clinical workflow, preparedness of sites, clear accountability, and increased awareness of the value of research. 

The new guidance also emphasizes following a Quality by Design approach to streamline trials and proactively address risks of important errors. CTTI has developed a comprehensive Quality by Design toolkit, including recommendations, a principles document, and various resources that can help support implementation. 

More information about CTTI’s Trials in Clinical Practice & Quality by Design work is available on CTTI’s website.

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New Publication by CTTI, DCRI, the Duke Margolis Institute for Health Policy, and Protas Investigates Practices to Improve Evidence Generation System in Clinical Trials

CTTI News | September 17, 2024

Topics Included: Access to Clinical Trials

A new publication developed in collaboration by CTTI, the Duke Clinical Research Institute (DCRI), the Duke Margolis Institute for Health Policy, and Protas published in Trials highlights the current barriers to clinical trial transformation, areas of improvement, and actions needed to transform clinical evidence generation in the United States to better align with clinical care demands. A modern clinical trial infrastructure should prioritize trial accessibility, answer relevant study questions reliably, respond to public health emergencies rapidly, and more. 

The article identifies three key barriers that are currently impeding the transformation of the clinical trial evidence generation system – gaps in policy, inefficient infrastructure, and a lack of research prioritization. The United States’ health and data system has a fragmented infrastructure that can hamper the progress of clinical trials, especially at locations not accustomed to participating in research. Taking advantage of tools that increase trial efficiency will help significantly improve trial activation and participation. Regulatory support and appropriate policies are needed to implement a modern clinical trial infrastructure.  

Lastly, clinical trials are often deprioritized in health care settings because incentives to participate in trials are often limited and/or misaligned with clinical care activities. Health system leadership should encourage integration of trials more routinely into care delivery and help advance a learning health care system, especially as systems implement reforms to improve their clinical data infrastructure. 

Reliable and better evidence can be generated if there is continued movement towards more simple trial designs, regulatory clarity, the creation of a sustainable systemwide infrastructure, and coordinated leadership to improve the research and health care culture. 

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