New CTTI Steering Committee Positions Open for Patient/Caregiver Representative(s)

One of the ways that the Clinical Trials Transformation Initiative (CTTI) engages with and integrates patients into our work is by having patient/caregiver representatives on our Steering Committee. We are currently accepting applications for this position until 11:59 p.m. ET, Tuesday, April 2. We are looking for patients or caregivers who are self-starters, have personal experience and familiarity with clinical trials, and can work with stakeholders across the research community. 

During their three-year term, CTTI Steering Committee patient/caregiver representatives contribute ideas for projects, participate in their conduct and analysis, and are committed to disseminating CTTI recommendations and encouraging adoption of system changes that will improve the efficiency and quality of clinical trials. 

To learn more about this opportunity and apply, please visit the patient/caregiver page on our website.  You can also meet our current patient/caregiver representatives. 

Recording Now Available: CTTI Unveils New Tool for Embedding Clinical Trials into Clinical Practice

A recording of CTTI’s Feb. 29 webinar unveiling a new tool that aids in assessing the feasibility of embedding clinical trial elements into clinical practice is now available. 

The one-hour webinar included a welcome from CTTI Director of Projects Sara Calvert; a project overview from CTTI Senior Project Manager Lindsay Kehoe; a panel discussion focused on stakeholder perspectives related to the practical use of the tool; and a request for input on how to optimize the tool’s use.  

Embedding elements of clinical trials, such as patient identification, informed consent, and data acquisition, into routine care reduces duplication of trial and care activities and promotes the development of a learning health care system. This can naturally lead to better decision-making,  treatment options, and outcomes for patients.  

This tool is a part of CTTI’s Embedding Trials in Clinical Practice work, which aims to enable health care settings to participate in embedded trials and assists sponsors with their design and conduct.  

View the slide deck to learn more. 

CTTI to Launch New Embedding Trials Feasibility Assessment Tool

CTTI will host a free public webinar on Thursday, Feb. 29 at 12:00 p.m. EST to unveil a new tool for assessing the feasibility of embedding clinical trial elements into clinical practice.  

The webinar will include a welcome from Sara Calvert; a project overview from Lindsay Kehoe; a panel discussion that will focus on stakeholder perspectives related to the practical use of the tool; and an opportunity for you to help CTTI optimize its use. 

Embedding elements of clinical trials, such as patient identification, informed consent, and
data acquisition, into routine care reduces duplication of trial and care activities and promotes the
development of a learning health care system. This can naturally lead to better decision-making,
treatment options, and outcomes for patients. 

However, integrating interventional clinical trials into clinical practice is complex, and operational direction is needed. Supporting recently released recommendations, CTTI created a new tool that aims to aid sponsors involved in early planning and design of a clinical trial by providing insight into site readiness and feasibility of integrating trials into clinical practice. 

Register for the free 60-minute webinar. 

Report Now Available: Mitigating Clinical Study Disruptions During Disasters and Public Health Emergencies

The report on the two-day virtual public meeting, “Mitigating Clinical Study Disruptions During Disasters and Public Health Emergencies (PHEs),” convened by the U.S. Food and Drug Administration (FDA) in collaboration with the Clinical Trials Transformation Initiative (CTTI), is now available. 

The meeting, which was held on October 18 and 19, 2023 with 720 global attendees, discussed recommendations provided by the FDA during the COVID-19 public health emergency to mitigate disruption of clinical studies, including recommendations detailed in the guidance entitled “Conduct of Clinical Trials of Medical Products During the COVID-19 Public Health Emergency, Guidance for Industry, Investigators, and Institutional Review Boards.” The meeting also featured speakers and panelists from across the clinical trials enterprise, including representatives from Medtronic, the National Cancer Institute (NCI), the National Institutes of Health (NIH), Roche, and many more, who shared the experiences, best practices, and strategies they used to effectively navigate disruptions to clinical studies during the COVID-19 public health emergency.  

The meeting fulfilled a requirement under section 3605 of the Food and Drug Omnibus Reform Act of 2022 (FDORA). For more information about this meeting, visit CTTI’s website to view the resources including the final report, the presentation slides and the recording of the meeting. 

CTTI and FDA Share Strategies for Improving Timely, Accurate, and Complete Registration and Reporting of Summary Results Information for Applicable Clinical Trials on ClinicalTrials.gov

Timely, accurate, and complete registration and reporting of summary results information for applicable clinical trials on ClinicalTrials.gov allows access to current research and evidence for all partners in the clinical trials enterprise, including patients, providers, sponsors and investigators, regulators, payers, and health system leaders. Despite the regulatory requirements for applicable clinical trials and the importance of fostering transparency while increasing knowledge of potential new treatments in development, multiple publications have reported gaps in clinical trial registration and results information submission to the data bank.  

To address this issue, the Clinical Trials Transformation Initiative (CTTI) collaborated with the Food and Drug Administration (FDA) on a project to understand the barriers to timely, accurate, and complete registration and reporting of summary results information for applicable clinical trials on ClinicalTrials.gov. The project involved in-depth interviews and a survey to identify the range of barriers and their salience by type of stakeholder, root causes influencing barriers, and potential solutions and helpful practices to implement moving forward. After the relevant themes were assessed, CTTI surveyed 92 individuals who represented 84 unique organizations and condensed this information into a report containing strategies and recommendations for improving registration and reporting. 

Some of the strategies include: 

  • Use a centralized/dedicated approach to meeting ClinicalTrials.gov requirements  
  • Take a proactive, rather than reactive, approach to complying with ClinicalTrials.gov regulatory requirements 
  • Escalate to upper levels of leadership for PIs/study teams that are non-responsive to the administrative unit’s communication about compliance  
  • Inform PIs/study teams  
    • about the possibility of civil money penalties for non-compliance with ClinicalTrials.gov regulatory requirements;  
    • that submitting results information on ClinicalTrials.gov is separate from publishing results; and 
    • that, per ICMJE, reporting results information on ClinicalTrials.gov does not preclude publishing results in journals 
  • Provide education, resources, guidance, and support about meeting ClinicalTrials.gov requirements to PIs/study teams and other research personnel 

See the full report for a complete list of strategies. 

When describing challenges with registering and reporting summary results information from applicable clinical trials, respondents cited several major challenges: 

  • A lack of understanding on the part of the Principal Investigator (PI) and their study team regarding  
    • the type of trial that must be registered,  
    • which trial results must be submitted,  
    • and when they should be registered.  
  • Administrative groups also reported challenges relating to non-responsive PIs and study teams, which hinders both timely registering and reporting of results.  
  • For reporting, PIs and study teams responding to the survey also expressed concerns about waiting until all data are analyzed before reporting results information on ClinicalTrials.gov to prevent discrepancies between ClinicalTrials.gov data and published results. 

For more information, see CTTI’s Project page: Challenges Meeting U.S. ClinicalTrials.gov Reporting Requirements. 

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 meets with the FDA several times a year and discusses a variety of topics such as improving communication, 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:  

  • Anna Ball 
  • Kelly Barta 
  • Ricki Fairley 
  • Liam McCarthy 
  • Andrea Meredith 
  • Patrick Moeschen 
  • Olga Torres 
  • Kathryn Washington 

The representatives were selected from 75 applications received in response to a Federal Register notice published in July 2023. 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 thrilled once again to have eight new members of the PEC who will share their insights on patient engagement and research. CTTI’s Transforming Trials 2030 pillars envision a system that representative, accessible, and directly informed by patient preferences and experiences. Having patients as partners, especially through the PEC, is critical to our work. We also wish to thank the outgoing members for their invaluable perspectives during their term,” said Morgan Hanger, CTTI’s Executive Director. 

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. 

Patient community experiences and opinions are essential to inform FDA’s regulatory work. The FDA listens closely to PEC members to integrate different perspectives into meaningful patient engagement and communication opportunities.” 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 the clinical trials enterprise and provide information and resources to enhance PEC members’ knowledge about FDA and CTTI activities to share with patient communities. Previous topics have included: providing enhanced information to patient communities to clarify FDA’s role; updating the PEC Framework; working to better understand COVID-19’s impact on clinical trials; creating new ways to collaborate with patient communities; promoting global collaboration of patient organizations; 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: 

  • Carol Abraham 
  • Julie Breneiser 
  • Sneha Dave 
  • Maria De Leon 
  • Sharon Lagas 
  • John Linnell 
  • James Pantelas 
  • Traceann Rose 

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. 

Congratulations and Best Wishes to CTTI Executive Director Sally Okun on Her Retirement

Today, Sally Okun, who has served as CTTI’s Executive Director since October 2021, is retiring after five decades in healthcare.  

“On behalf of the entire CTTI community, we congratulate Sally on her exceptional career and on her retirement,” said CTTI’s incoming executive director, Morgan Hanger. “Sally’s tenacity and gift for storytelling have had a remarkable impact on bringing person-centered research and care into the mainstream. As we move into a phase of AI-driven innovation, her vision for ethical, patient-led data stewardship is providing an invaluable foundation for CTTI. We are so grateful for her 50 years of contributions to healthcare and to the success of this organization.”  

During her 20 years as a nurse and palliative care specialist in the community setting, Sally worked with hundreds of patients and caregivers. At the online patient research network PatientsLikeMe, where she served in a variety of roles for 12 years including as vice president of policy and ethics, she led the national conversation on patient centricity and created an evidence base to support expanded use of patient-generated health data. More recently at CTTI, Sally has initiated enterprise-wide measurement, expanded our commitment to diversity in clinical trials, and empowered the team to be independent and creative.  

When Sally announced her retirement in August, she committed to ensuring a seamless transition to her successor. In October, CTTI announced Morgan Hanger’s promotion to Executive Director upon Sally’s retirement, and over the past two months they have doubled down on that commitment. With the support and guidance of the CTTI Executive Committee Chairs and team, Sally and Morgan have relied on their shared values of connection, openness, and integrity to stay focused on what matters: the health and growth of our team; exciting, relevant work; and partnership with our members and the FDA.  

“It has been a wonderful experience working with entire CTTI team in preparation for my retirement,” said Okun. “I am confident that the organization will flourish through the leadership of my dear friend and colleague Morgan Hanger.” 

Please join us in thanking Sally for her service to CTTI and wishing her well in her retirement. 

Holiday Greetings from CTTI: Reflecting on 2023 & Embracing the Future

Throughout this year, we’ve had the pleasure of crossing paths with many of you at diverse locations around the world – from San Francisco and Boston, to the nation’s capital and the Big Apple, to Montreal and Riyadh. We shared our work at more than two dozen meetings, conferences, and events, and it’s been truly gratifying to see our community actively engaged in our work on a global level. 

In 2023, CTTI has redefined possibilities around some of the clinical trials enterprise’s most pressing challenges and made strides toward creating a more effective and efficient research process. We’re proud to share the results of those efforts in our 2023 CTTI Annual Report. 

As we usher in a new year with CTTI’s next generation of leadership, our strategy is to build on CTTI’s key strengths by offering new engagement and implementation opportunities with our members that are focused, flexible, and poised for impactful change, including: 

  • Last Mile Implementation: CTTI is well-known for launching products and recommendations to improve the quality and efficiency of clinical trials. Yet, we recognize that more is needed to drive sustainable and scalable change. Therefore, we will test innovative approaches to make CTTI recommendations easier to implement in a range of settings, including our website. 
  • Engagement Diversification: CTTI’s flagship multi-year project archetype is important and we appreciate that in-depth learning and meaningful multi-stakeholder consensus takes time. We are eager to forge different modes of engaging, including more dynamic conversations with members and with the FDA. With the increased accessibility of powerful AI tools, research is changing rapidly, presenting interesting opportunities to learn together by convening in new and interesting ways. 
  • Dynamic Collaborations: There’s growing attention from private and public sectors as the U.S. government (including OSTP, NIH, ARPA-H and the FDA) intensifies its focus on clinical trials modernization and readiness. We’re delighted by the new and growing initiatives from digital platform companies to bring the research experience closer to patients wherever they are. CTTI has a crucial role to play, and we’ll continue to partner with our members, related efforts, and organizations around the world to harness these moments for impact.  

To our CTTI community, we thank you. While we all come from different parts of this complex enterprise, our collective culture is strong and our commitment steadfast. As our organization transitions from one leader to the next, we will carry CTTI’s legacy forward together by building on the work of leaders and members, past and present, to shape the future of clinical trials that we all want for ourselves and our loved ones.  

As we set our sights on 2024 and beyond, look to CTTI to pick up the pace of innovation and transformation in respect to our mission. We wish you a wonderful holiday season and a prosperous new year! 

Recording of Virtual Public Workshop Now Available: Enhancing Diversity in Clinical Studies

A recording for the virtual public workshop to enhance clinical study diversity convened by the U.S. Food and Drug Administration (FDA) and Clinical Trials Transformation Initiative (CTTI), held November 29 and 30, is now available.  

Day 1 of the virtual public workshop opened with welcoming remarks from Karen A. Hicks, Deputy Director of the FDA’s Office of Medical Policy (OMP). Following the opening remarks, Patrizia Cavazzoni, Director of the FDA’s Center for Drug Evaluation and Research (CDER), delivered the keynote address. Following the keynote, multiple speaker presentations and panel discussions were held, including topics such as an overview of clinical study diversity, establishment of clinical study enrollment goals, approaches to support the inclusion of underrepresented populations in clinical studies, and more. The first day concluded with closing remarks from Karen Hicks.    

Day 2 of the workshop began with opening remarks from Mathilda Fienkeng, Director of the FDA’s Division of Medical Policy Development at the OMP. Following the opening remarks, further presentations and panel discussions were held on approaches to support the inclusion of individuals with mental illness in clinical trials, how to use digital health tools to increase clinical study diversity, community engagement, and next steps to enhance clinical study diversity with respect to the FDA’s overall mission. The second and final day of the workshop concluded with closing remarks from Hicks. 

View the full slide deck to learn more about the methods to support the inclusion of various underrepresented populations in clinical studies.  

This workshop fulfilled a requirement under section 3603 of the Food and Drug Omnibus Reform Act of 2022 (FDORA). 

CTTI Publication Explores Stakeholder Reflections on Implementing the National Institutes of Health’s Policy on Single Institutional Review Boards

A new CTTI publication, published in the Journal of Ethics and Human Research, explores stakeholder reflections on implementing the National Institutes of Health’s policy on single Institutional Review Boards (sIRB). CTTI interviewed 34 stakeholders with various roles in the single IRB process to gather perspectives about their experiences implementing the sIRB model, focusing specifically on the six goals of the NIH sIRB policy. Some stakeholders suggested that using the sIRB model has streamlined and reduced inefficiencies associated with local IRB model. However, new inefficiencies were created due to unclear roles, lack of systems and processes, and increased workloads for some stakeholders. 

These stakeholder responses suggest that standardizing the sIRB process and implementing new systems that support the NIH’s goal for enhancing and streamlining the IRB process are needed. For example, standardizing the roles and responsibilities of reviewing IRBs and relying institutions, improving the compatibility of IRB software, and having a central repository for institutional information may improve the efficiency of the sIRB process. CTTI utilized the findings of these interviews to inform an sIRB evaluation framework.