CTTI was founded over 15 years ago in the fall of 2007, holding our first Executive Committee meeting on March 4, 2008. Since then, CTTI has continued to work towards our mission of developing and driving the adoption of practices that increase the quality and efficiency of clinical trials. CTTI’s progress toward our bold Transforming Trials 2030 vision is due to the invaluable effort, support, and collaboration of our founders, members, staff, and stakeholders. As we celebrate our 15th anniversary, we would like to thank the more than 700 organizations and nearly 3,000 individuals whose work and commitment have helped us to advance and transform the clinical trials enterprise. We look forward to your continued support as we work together to shape the future of clinical trials.
CTTI News
CTTI at 15: Reflections on Progress & the Future of Transforming Trials 2030 from CTTI Leadership
By: Sally Okun, CTTI Executive Director
Take a moment and think back to 2007. In that year Apple released its first iPhone, which has since sold more than 1.5 billion units worldwide. NASA launched the Phoenix Mars Lander, which entered the Martian atmosphere within nine months and shortly thereafter confirmed that the soil it ‘tasted’ contained water ice. And An Inconvenient Truth won the Oscar for best documentary and brought the topic of climate change to the forefront of mainstream culture.
And in 2007, the Clinical Trials Transformation Initiative was founded with the ambitious mission to improve the quality and efficiency of clinical trials and by the spring of 2008 held its first meeting of the Executive Committee. Since then, CTTI has had an influential role in advancing incremental change in clinical trials by working collaboratively with stakeholders from across the clinical trials enterprise, including industry, academia, patients and patient advocacy groups, biotechnology, professional societies, and government agencies. Together with its member organizations, this public-private partnership of the U.S. Food and Drug Administration and Duke University has completed 50 projects, publicly launched 105 recommendations and tools, and published 88 peer-reviewed articles. And then in 2020, CTTI set its sight on the future with its bold vision for Transforming Trials by 2030.
Now with CTTI’s 15th anniversary upon us, I sat down with three members of CTTI’s leadership team whose tenures with the organization range from 12 years to less than two and asked them to reflect on CTTI’s enduring impact, share their insights on CTTI projects they are most proud of and think about their aspirations for CTTI in the coming five years:
- Sara Calvert, Director of Projects, has been with CTTI for 12 years and holds much of the organization’s institutional memory. Sara reflected on the first project she took on as a project manager at CTTI – to address challenges of adopting single Institutional Review Board (sIRB) review. Listening to Sara describe the evolution of this work was fascinating. It began in 2010 as the Central IRB Project and in 2013 shifted to Central IRB Advancement. By 2017 the work, reflecting a change in nomenclature, focused on Single IRB Adoption and eventually in 2018 it evolved to Single IRB Evaluation. Sara is pleased that the work of these completed projects continues to guide investigators and others newly introduced to the use of sIRBs in their research. And she noted the that this important body of work was recently cited in the U.S. Government Accounting Office Report to congressional requesters titled IRBs: Actions Needed to Improve Federal Oversight and Examine Effectiveness.
- Zach Hallinan, Director of Strategic Engagement and Communications, spoke with pride about his role as the project manager for CTTI’s Quality by Design (QbD) portfolio to which he was assigned upon joining the organization nearly 7 years ago. As he spoke, a pattern emblematic of CTTI projects emerged – they address complex issues; they take time; and they may pivot or expand their scope in response to new insights, changing regulatory requirements, legislative mandates or shifts in practice across the clinical trials enterprise. In this case, what started as a project on improving monitoring procedures in 2010 changed as the project team recognized monitoring is just one component of an overall quality approach that is needed for trial planning, conduct, and oversight. By 2018 CTTI’s QbD Adoption project was underway with the objective of creating tools and resources to support implementation of the critical to quality elements for improving clinical trial quality and efficiency. Before long, the University of California Irvine used CTTI’s resources to develop their Critical to Quality Design Studios and shared this work in CTTI’s Case Study Exchange. Additionally, Zach noted that CTTI members recently ranked the QbD portfolio as the highest priority for dissemination across the clinical trials enterprise.
- Morgan Hanger, Director of Strategic Programs, joined CTTI less than two years ago and quickly recognized CTTI’s potential to be a catalyst for enduring and transformational change. Morgan’s interest in driving implementation toward lasting impact has positioned CTTI more intentionally to convene our colleagues from across the clinical trials enterprise to explicitly align our goals and activities in certain key areas. While we are still in the early stages of this convergence work, Morgan believes this orientation will be transformative for driving outcomes and shared accountability across all the organizations and related efforts working to improve trials. Morgan also proudly shared the progress made on the first activity she took on when joining CTTI in January 2022 – to advance the development of metrics for monitoring and evaluating progress on the five pillars of CTTI’s Transforming Trials 2030 vision. A project team and expert panel are currently being convened to take the results of the evidence gathering work to the project’s next phase.
Universally, Sara, Zach and Morgan cited CTTI’s role in ensuring that patients and patient advocates always have an equal voice on CTTI project teams and at expert meetings. They all expressed pride that CTTI was at the forefront of engaging with patients and patient advocates as true partners from the organization’s earliest days and that legacy continues today.
The impact of CTTI’s work will be not measured in units sold or in discoveries on a distant planet, but rather in bringing to the forefront of mainstream awareness translatable knowledge and actionable evidence generated through clinical trials that are:
- truly person-centric and easily accessible by all who want to participate in them;
- transparently integrated into the healthcare received by all;
- always designed with quality and representativeness at their core;
- maximally leveraging all available data while never compromising on individual privacy, autonomy, and choice.
As members of CTTI’s leadership team, we wish to acknowledge and collectively celebrate the thousands of individuals, organizations and team members who are foundational to CTTI’s work and without whom the accomplishments of the last 15 years could not have been achieved. When the time to celebrate our 20th anniversary arrives, we trust that — together with the clinical trials enterprise — we will be well on our way towards the transformative change we envision for 2030.
CTTI Holds Meeting to Discuss Strategies for Advancing the Use and Acceptance of Disease Progression Modeling (DPM) in Clinical Trial Design & Decision Making
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.
REPORT NOW AVAILABLE: ICH M11: Clinical electronic Structured Harmonised Protocol (CeSHarP)
The report of the public webinar on “ICH M11: Clinical electronic Structured Harmonised Protocol (CeSHarP),” organized by the International Council for Harmonisation (ICH) and convened by the Clinical Trials Transformation Initiative (CTTI), is now available. More than 1,700 people registered for the webinar, which was held on January 26, 2023.
The ICH M11 Expert Working Group (EWG) members, who are updating the guideline, explained the benefits and opportunities that will be created for stakeholders with the electronic exchange capabilities of an internationally harmonised structured protocol template. The EWG members presented an overview of the M11 Guideline and the associated protocol template and technical specification. In closing, EWG members answered common questions that were submitted by participants during the registration process.
View the recording and slide deck of the webinar to learn more.
For additional information on the ICH M11 Guideline, please see the ICH Guidelines site, M11 Draft Guideline, M11 Template, and M11 Technical Specification.
CTTI Publication Investigates Organizational Practices to Promote Diversity and Inclusion in Clinical Trials
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.
Recording of Public Webinar Now Available: New ICH M11 Harmonised Guideline, Protocol Template, and Technical Specification
A recording for the International Council for Harmonisation’s public webinar, held January 26th, “New ICH M11 Harmonised Guideline, Protocol Template, and Technical Specification,” is now available.
The webinar opened with welcoming remarks from Jacqueline Corrigan-Curay of the U.S. Food & Drug Administration (U.S. FDA). Following the opening remarks, members of the M11 Expert Working Group—including Janice Maniwang of the U.S. FDA, Noémie Manent of the European Medicines Agency (EMA), and Mitzi Allred of Merck—provided detailed overviews of the M11 Guideline, Protocol Template, and Technical Specification. Following the presentations, the speakers also addressed questions that had been submitted during webinar registration. The webinar concluded with closing remarks from Ronald Fitzmartin of the U.S. FDA.
View the full slide deck to learn more about the M11 Guideline, Protocol Template, and Technical Specification. The U.S FDA public consultation period opened on Thurs., Dec. 22, 2022 and will close on Tues., Feb. 21, 2023. Comments may be submitted on Regulations.gov.
For additional information on the ICH M11 Guideline, please see the ICH Guidelines site, M11 Draft Guideline, M11 Template, and M11 Technical Specification.
ICH Public Webinar Will Provide an Update on the Latest Developments Concerning the M11 Harmonised Guideline and Protocol Template
The International Council for Harmonisation (ICH) M11 Expert Working Group (EWG) is organizing a public webinar to describe the latest developments on the ICH M11 Guideline. The EWG is holding this webinar on Thursday, Jan. 26, 2023 from 10:00 a.m. – 12:00 p.m. EST. Registration is open for this event, and registrants are invited to submit questions about this webinar in English using this form by Friday, Jan., 20, 2023. All information and presentations will be provided in English.
Click here to register for the webinar.
The webinar will include the following speakers from the ICH M11 committee:
- Janice Maniwang, U.S. Food and Drug Administration (FDA)
- Noémie Manent, European Medicines Agency (EMA)
- Mitzi Allred, Merck
During the webinar, these speakers will describe the latest developments on the M11 Guideline, Protocol Template, and Technical Specification; explain the benefits and opportunities created by the M11 Protocol Template; explain how the Protocol Template was developed; and explain the purpose of the technical specifications in supporting the exchange and storage of clinical trial data. There will also be a Q&A session at the end of the webinar during which the speakers will address a selection of questions submitted during registration.
For additional information on the ICH M11 Guideline, please see the ICH Guidelines site, M11 Draft Guideline, M11 Template, and M11 Technical Specification.
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.
CTTI Publication Investigates Use of Preventive Statins among Older Patients with Cardiovascular Disease
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.
CTTI Thanks Our Amazing Community for its Contributions to Our Work This Year
In 2022, our organization embraced innovation at the intersection of science, culture, disciplines and people. We believe it’s the crossroads of where change and creativity occur and it has led us to reaffirm our commitment to doing clinical research differently. Through diverse collaboration and unique approaches, we are making great progress in our efforts to create solutions that change how clinical trials are designed and run and, ultimately, improve the health of people.
Before this year draws to a close, we want to take a moment to express our sincere gratitude for the knowledge, ideas and talent you’ve contributed to our work. We are grateful for the ways you push us to work smarter. And we are privileged to work with the many diverse experts and stakeholders through whom we explore challenges and create solutions that we couldn’t arrive at alone.
Some of the accomplishments you’ve helped make possible this year include:
- Releasing new and enhanced sets of recommendations and resources with the Digital Health Trials Hub and Embedding Clinical Trial Elements into Clinical Practice.
- The launch of two new projects including Disease Progression Modeling and Engaging Stakeholders in Trial Design, as well as continued progress on Increasing Diversity in Clinical Trials and ClinicalTrials.gov Reporting Challenges.
- Five publications from the Informing ICH E6 Renovation, Master Protocols, and HABP/VABP Studies projects, as well as the IMPACT-AFib Study and a statin use analysis.
- The Measurement Initiative Project, which aims to assemble a parsimonious set of metrics to help us monitor, evaluate, and analyze progress towards the Transforming Trials 2030 vision.
We are proud to have you as part of this amazing community. Although the road to transforming clinical trials by 2030 is long and complicated, it is through our collaboration that we will reveal sustainable, lasting change that improves health care for all.
We look forward to working with you in 2023 and wish you the best this holiday season and in the New Year!