Projects

CTTI Holds Meeting to Discuss Strategies for Advancing the Use and Acceptance of Disease Progression Modeling (DPM) in Clinical Trial Design & Decision Making

Posted on by Hannah Faulkner

The Clinical Trials Transformation Initiative (CTTI) held a multi-stakeholder expert meeting on March 6 to discuss strategies for advancing the use and acceptance of disease progression modeling (DPM) in clinical trial design and decision making. Meeting attendees discussed DPM and its current applications; explored opportunities, barriers, and best practices for advancing the use of DPM in clinical trial decision making; and proposed relevant metrics for monitoring and evaluating the recognition, value, and consistent use of DPM. CTTI will be using the ideas generated during this meeting and collecting case examples of DPM application in order to identify and catalog examples of how DPM can provide value for clinical trials – with the ultimate goal of developing and disseminating recommendations that address DPM best practices. 

The maturity and potential impact of model-informed drug development (MIDD) is recognized, but knowledge sharing and clear regulatory expectations are needed to realize its widespread use. FDA’s paired meeting program and the Fit-for-Purpose initiative are examples of programs that foster engagement and transparent communication but additional communication and pre-competitive collaboration is needed between the modeling industry, regulatory agencies, and the clinical community to advance consistent application of MIDD. 

During the meeting, attributes that allow for a successful application of DPM in a given context were discussed, leveraging the results from the CTTI DPM project team scoping review and expert panel presentations.  

  • Meeting attendees also discussed essential next steps for advancing the recognition,
    value, and use of DPM approaches, including the need to:
    ► Establish best practices and provide illustrative case examples.
    ► Develop a common language for disease progression modeling.
    ► Highlight the value and impact of DPM for a variety of stakeholders.
    ► Facilitate communication across stakeholders.
    ► Create metrics to monitor the changes in DPM acceptance and use within organizations and across the clinical trials enterprise (CTE). 

Overall, meeting attendees highlighted that the benefit of CTE-wide adoption of DPM would be faster, higher-quality drug development programs, and more diverse clinical trials. 

Disease Progression Modeling Expert Meeting

March 6, 2023

CTTI Project: Using Disease Progression Modeling to Advance Trial Design and Decision Making

MEETING OBJECTIVES:

  • Discuss disease progression modeling (DPM) and its current applications 
  • Explore opportunities, barriers, and best practices for advancing the use of disease progression modeling to aid in decision making 
  • Brainstorm relevant metrics to monitor and evaluate the recognition, value and consistent use of disease progression modeling  

Meeting Location: Mayflower Hotel, Washington, D.C.

Meeting Summary

Meeting Agenda

List of Meeting Attendees

Full Presentation Set            

Meeting Materials:

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.

CTTI Publication Investigates Organizational Practices to Promote Diversity and Inclusion in Clinical Trials

Posted on by Hannah Faulkner

A new CTTI publication, published in the Journal of Clinical Pharmacology & Therapeutics, investigates organizational-level practices for enhancing diversity and inclusion in clinical trials as one component of advancing health equity and improving health outcomes. When clinical trial participants do not reflect the populations that will use the medical product, it can limit our understanding of the safety and efficacy of the investigational medical product, restrict the availability of evidence-based treatment guidelines for vulnerable populations disproportionally burdened by disease, and delay access to innovative and potentially life-extending therapies.  

CTTI interviewed 36 senior-level leaders at 20 organizations that conduct clinical trials to gather perspectives on strategies to support diversity and inclusion in clinical research. Based on these interviews and a 2-day meeting with 53 experts, CTTI identified four key action areas to implement sustainable, organizational-level practices: commitment, partnerships, accountability, and resources. To improve equitable access and increase diversity in clinical trial populations, CTTI suggests making diversity and inclusion in clinical trials an organizational priority and defining responsibility for these efforts, establishing and maintaining bi-directional community partnerships, and allocating organizational resources that support diversity and inclusion in clinical research. The CTTI Diversity Project Team is using the findings from this research to develop recommendations and a tool for institutions to use to initiate or enhance their diversity and inclusion efforts. 

Patient Engagement Collaborative Announces Eight New Members

Posted on by Hannah Faulkner

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. 

CTTI Publication Investigates Use of Preventive Statins among Older Patients with Cardiovascular Disease

Posted on by Hannah Faulkner

A new CTTI publication, published in the Journal of the American Geriatrics Society, investigates the use of preventive, high-intensity statins among patients 75 and older diagnosed with atherosclerotic cardiovascular disease (ASCVD). The study, which used pharmacy and medical claims data from a commercial health plan, found substantial underuse of statins among insured ASCVD patients. Less than half of older adults with ASCVD, who are at the highest risk of vascular events like heart attack or stroke, are taking statins. Only a minority of older patients are taking high-intensity statins, and very few are taking non-statin ASCVD treatments. Notably, women and patients who had a diagnosis of cerebrovascular disease (CeVD) or peripheral arterial disease (PAD) were less likely to be prescribed a statin. This paper was based on exploratory work done by CTTI with Duke and HealthCore to assess patient use of ASCVD therapies, with a focus on high-intensity statins, to identify areas where future clinical trials and/or quality improvement initiatives can be targeted to address treatment gaps between clinical practice and guideline recommendations. 

Recording Now Available: CTTI Webinar Unveiling New Recommendations on Embedding Clinical Trials into Clinical Practice

Posted on by Hannah Faulkner

A recording of CTTI’s December 15 webinar unveiling the new recommendations on embedding clinical trials into clinical practice is now available. 

The one-hour webinar includes a welcome from Sally Okun, CTTI Executive Director; a keynote
presentation from Robert M. Califf, U.S. Food & Drug Administration; a project overview from Matthew Roe, AstraZeneca; and a panel discussion moderated by Lindsay Kehoe, CTTI Senior Project Manager. The panel discussion, which focused on stakeholder perspectives related to the
integration of the new recommendations, included: Sam Bozzette, National Institutes of Health;
Lara Jehi, Cleveland Clinic; Holly Robertson, Medidata; and Alandra Weaver, Crohn’s & Colitis
Foundation. 

The goal of creating these recommendations for embedding elements of clinical trials into care is to reduce trial inefficiencies and enable faster, more representative, evidence generation that improves patient care. The recommendations were developed for trials of drugs, devices, and biologics in close collaboration with stakeholders across the clinical trials enterprise. The first phase involved gathering input through qualitative, in-depth interviews with designers and implementers of interventional clinical trials that are embedded into clinical practice. The recommendations were drafted and refined with input from a multi-stakeholder project team and through Expert Meetings. 

View the slide deck to read more.