Presentation: Building Upon Quality by design and Risk-based Monitoring Implementation Momentum
Date: Wednesday, June 18, 8:30 - 9:30 a.m.
Presenters: Gerrit Hamre (Duke University), Morgan Hanger (CTTI), Sameera Ibrahim (Bristol Myers Squibb), and Alissa Munn (Food and Drug Administration/CDER)
Topics Included: Clinical Trials Landscape
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In September 2023, CTTI launched the Measuring Trials Transformation (MTT) Project to track progress toward CTTI’s Transforming Trials 2030 (TT2030) vision. Introduced in 2021, this vision outlines how clinical trials should evolve by 2030 to become more efficient and inclusive, focusing on five key pillars: patient-centeredness, integration with health processes, trials designed with a quality approach, data-driven approaches, and improving population health. To measure progress toward these goals, the MTT project created the Metrics Framework, a parsimonious set of metrics that correspond to key domains of progress within each of the TT2030 pillars. This framework is designed to assess the transformation of clinical trials by creating specific benchmarks for understanding the state of clinical trials both today and in the future.
Since its launch, CTTI’s Measuring Trials Transformation team has made significant progress in refining the Metrics Framework by reviewing more than 100 public comments and incorporating feedback to improve the set of metrics. From the broader Framework, the team identified nine high-priority metrics to focus on for data collection and analysis. The team is currently analyzing original trial documentation from CTTI’s AACT database of Phase 3 interventional trials conducted at U.S. sites between 2018 and 2024 to be able to answer key metrics.
Kelly Avery, MTT Project Team Leader and Operations Administrator for Research at Mayo Clinic, said, “The MTT project at CTTI is focused on moving us into the future. We’re striving to truly understand the ‘state of clinical trials, and to do that, we must first establish a baseline. By creating this baseline, we’ll gain the clarity needed to guide our next steps and drive meaningful progress.”
To support the successful execution of the project, CTTI has partnered with ClinConnect, a patient advocacy platform with valuable expertise in clinical trial analysis. ClinConnect developed best-in-class technology to manage and promote a searchable database for patients and caregivers, making it simple and straightforward for everyone to understand the totality of their clinical trial options.
Robert Maxwell, Founder of ClinConnect, highlighted the value of the collaboration, saying, “Working with the MTT team has been a highly rewarding experience. We’re aligned on the key challenges and moved by the progress we’ve made. The ClinConnect team thrives when we’re given the freedom and trust to address these issues head-on, and the MTT team has been excellent here. We look forward to continuing this collaboration and making a lasting impact for patients worldwide.”
CTTI deeply appreciates ClinConnect’s partnership and the invaluable insights they have shared, which have been instrumental in driving the success of this project. CTTI also extends our appreciation to the MTT Project Team, whose ongoing dedication and expertise are essential to the success of this effort.
Looking ahead, CTTI plans to release the full Metrics Framework and initial findings on our website. These findings will serve as an important reference for discussions at the inaugural State of Clinical Trials: Charting the Path Forward meeting on May 22, 2025.
CTTI is eager to continue this vital work and engage in meaningful conversations that will help shape the future of clinical trials.
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Today, CTTI announces the appointment of three new members to its Executive Committee, which is responsible for making recommendations about the overall direction and strategy for the organization. The new Executive Committee members, whose three-year term runs from 2025-2028, include:
“CTTI is honored to welcome these three leaders to our Executive Committee,” said Morgan Hanger, CTTI Executive Director. “From their different seats as patient advocates, regulators, and research sponsors, all have truly unique expertise in leveraging innovation to generate and use the evidence that individuals need to make decisions, which is at the heart of our work.”
“The newly appointed Executive Committee members bring a wealth of experience as well as a demonstrated commitment to transformation,” said CTTI Executive Committee Chair Mark McClellan. “We look forward to collaborating with these leaders as they contribute their unique insights to our ongoing efforts to enhance and modernize clinical trials.”
The 19-member Executive Committee includes distinguished U.S. and international thought leaders in academia, government, industry, and patient advocacy who are experienced at envisioning and facilitating improvements in health care and medical research systems. Additionally, CTTI wishes to express sincere gratitude to the following outgoing Executive Committee members for their 20 years of collective service ranging from 2018-2024: Donna R. Cryer, Global Liver Institute; Pat Furlong, Parent Project Muscular Dystrophy; and Steven K. Galson, Boston Consulting Group.
CTTI envisions a future where clinical trials are more efficient, inclusive, and impactful by 2030—setting a shared aspiration for everyone in the research ecosystem. While progress may unfold at different paces across regions, populations, therapeutic areas, and trial types, this vision serves as both a roadmap and a call to action. To support this effort, CTTI launched the Measuring Trials Transformation project, developing a streamlined Metrics Framework and identifying high-priority metrics to track progress toward trial transformation.
Guided by this vision, CTTI is shaping its priorities, encouraging widespread adoption of forward-thinking practices, and collaborating with individuals and organizations to drive meaningful advancements.
While our vision applies broadly to clinical trials, CTTI’s efforts primarily align with the NIH definition—research studies that prospectively assign participants to one or more interventions (which may include placebos or controls) to evaluate their effects on health-related biomedical or behavioral outcomes. However, we anticipate that our work will also benefit other clinical studies, including those without prospective assignment. Recognizing that clinical trials exist within the broader landscape of evidence generation, we remain committed to fostering progress that enhances the accessibility, quality, and relevance of research worldwide.
The need for better, more efficient clinical trials has never been clearer. CTTI unites experts from across the clinical trials enterprise—including clinicians, ethics board representatives, healthcare system leaders, patients, payors, regulators, and sponsors—to identify challenges, exchange ideas, and develop actionable solutions. Through a collaborative, research-driven approach, CTTI creates evidence-based recommendations and tools that drive real change. These solutions are designed to enhance the quality and efficiency of trials today while advancing a bold vision for the future: Transforming Trials 2030.
Building Upon Quality by design and Risk-based Monitoring Implementation Momentum
In September 2023, CTTI launched the Measuring Trials Transformation (MTT) Project to track progress toward CTTI’s Transforming Trials 2030 (TT2030) vision. Introduced in 2021, this vision outlines how clinical trials should evolve by 2030 to become more efficient and inclusive, focusing on five key pillars: patient-centeredness, integration with health processes, trials designed with a quality approach, data-driven approaches, and improving population health. To measure progress toward these goals, the MTT project created the Metrics Framework, a parsimonious set of metrics that correspond to key domains of progress within each of the TT2030 pillars. This framework is designed to assess the transformation of clinical trials by creating specific benchmarks for understanding the state of clinical trials both today and in the future.
Today, CTTI announces the appointment of three new members to its Executive Committee, which is responsible for making recommendations about the overall direction and strategy for the organization.
Ensuring the safety and protection of our study participants is without question the highest priority of any research endeavor, and at CTTI, we understand the complexities involved. Thoroughly and... Engaging patients and patient organizations as partners early and often in the research process leads to clinical trials with greater feasibility, enhanced recruitment and retention, and more relevant outcomes.... "Quality" in clinical trials is defined as the absence of errors that matter-that is, errors which have a meaningful impact on the safety of trial participants or credibility of... here’s the creating diversity in clinical trials excerpt.
Ensuring the safety and protection of our study participants is without question the highest priority of any research endeavor, and at CTTI, we understand the complexities involved. Thoroughly and efficiently incorporating human research protection goes beyond risk mitigation; it demands timely and accurate safety data reporting, appropriate IRB and informed consent practices, and continuous communication with data monitoring committees.
CTTI's patient safety resources provide comprehensive solutions, featuring practical tips, best practices, and expert guidelines to help you navigate these complexities.
Engaging patients and patient organizations as partners early and often in the research process leads to clinical trials with greater feasibility, enhanced recruitment and retention, and more relevant outcomes. Meaningful patient group partnership, however, takes careful planning, commitment, and strategy.
CTTI’s resources can help to build a patient engagement framework that sets your research up for more relevant research aims, accelerated study enrollment rates, improved protocol design, and stronger dissemination of results.
“Quality” in clinical trials is defined as the absence of errors that matter—that is, errors which have a meaningful impact on the safety of trial participants or credibility of the results (and thereby the care of future patients). Once considered primarily in the monitoring process of a clinical trial, we now know that quality considerations must be holistic, woven deeply into the fabric of the research lifecycle from study design all the way through to delivery and dissemination of results. But without the right resources, it can be challenging to know where to start.
CTTI’s solutions offer straightforward processes for building studies with end-to-end quality and embedding a culture of quality within an organization. Use them to empower teams, foster critical thinking, prevent costly errors, ensure data is fit-for-purpose, and minimize patient burden wherever possible.
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Enrolling diverse populations leads to more accurate and generalizable trial results by enhancing and expanding our understanding of the safety and efficacy of investigational medical products. In addition, inclusion of all the populations who are affected by a condition can improve access to innovative and potentially life-extending or life-improving therapies, develop trust in clinical trial results, and facilitate uptake if the medical product is approved. Representative clinical trials increase the availability of evidence-based treatment guidelines for populations that are disproportionately burdened by disease.
At the start of the Clinical Trials Transformation Initiative (CTTI) Diversity Project in 2019, many efforts to increase clinical trial diversity focused on reactive, study-level strategies narrowly aimed at supporting the recruitment and retention of underrepresented populations. The far-reaching consequences of a lack of diversity in clinical trials were clearly underscored by the COVID-19 pandemic, leading individuals and organizations across the clinical trials ecosystem to call for deep, systemic change. These CTTI recommendations seek to build on the growing recognized need for long-term, transformative strategies that are rooted in a deep organizational commitment to developing clinical trial research infrastructure that is more responsive to the needs of historically underrepresented populations.
Target Audience: Organizations that design and conduct clinical trials involving FDA-regulated medical products*, such as medical product companies, patient groups, academic and nonacademic research institutions, contract research organizations, and sites.
Scope: Inclusion of women and people from racial and ethnic groups who have been historically underrepresented in clinical trials in the United States. Collectively these groups are referred to throughout these recommendations as diverse populations†.
Clinical trial diversity is an important component of a broad organizational culture and commitment to diversity, equity, and inclusion (DEI). While these recommendations are not intended to directly address broader workforce development and DEI initiatives, some recommendations may apply to, or be supportive of, such efforts.
These recommendations and maturity model are applicable for organizations at various stages in their development of organization-wide strategies for improving equitable access and participation of diverse populations in clinical trials. The recommendations describe best practices and the supporting maturity model is intended to guide organizations, including those starting to build organizational-level strategies, in the assessment of the current state of development and identification of their ultimate desired progress.
The Clinical Trials Transformation Initiative (CTTI), a public-private partnership co-founded by Duke University and the U.S. Food and Drug Administration, seeks to develop and drive adoption of practices that will increase the quality and efficiency of clinical trials. Bringing together organizations and individuals from across the ecosystem—representing academia, clinical investigators, government and regulatory agencies, industry, institutional review boards, patient advocacy groups, and other groups—CTTI is transforming the clinical trials landscape by developing evidence-based solutions to clinical research challenges. Many regulatory agencies and organizations have applied CTTI’s more than 20 existing recommendations, and associated resources, to make better clinical trials a reality. Learn more about CTTI projects, recommendations, and resources at http://www.ctti-clinicaltrials.org.
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2. Clark LT, Watkins L, Piña IL, et al. Increasing Diversity in Clinical Trials: Overcoming Critical Barriers. Current Problems in Cardiology. 2019;44(5):148-172.
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5. Corneli A, Hanlen-Rosado E, McKenna K, et al. Enhancing Diversity and Inclusion in Clinical Trials. Clin Pharmacol Ther. 2023;113(3):489-499.
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9. Wright JT, Jr., Dunn JK, Cutler JA, et al. Outcomes in hypertensive black and nonblack patients treated with chlorthalidone, amlodipine, and lisinopril. JAMA. 2005;293(13):1595-1608.
10. National Academies of Sciences E, and Medicine; Policy and Global Affairs; Committee on Women in Science, Engineering, and Medicine; Committee on Improving the Representation of Women and Underrepresented Minorities in Clinical Trials and Research. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. In: Bibbins-Domingo K, Helman A, eds. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington (DC)2022.
11. Chastain DB, Osae SP, Henao-Martinez AF, Franco-Paredes C, Chastain JS, Young HN. Racial Disproportionality in Covid Clinical Trials. N Engl J Med. 2020;383(9):e59.
12. Webb Hooper M, Nápoles AM, Pérez-Stable EJ. COVID-19 and Racial/Ethnic Disparities. JAMA. 2020;323(24):2466-2467.
13. Woodcock J, Araojo R, Thompson T, Puckrein GA. Integrating Research into Community Practice - Toward Increased Diversity in Clinical Trials. N Engl J Med. 2021;385(15):1351-1353.
14. Feldman S, Ammar W, Lo K, Trepman E, van Zuylen M, Etzioni O. Quantifying Sex Bias in Clinical Studies at Scale With Automated Data Extraction. JAMA Netw Open. 2019;2(7):e196700.
15. Flores LE, Frontera WR, Andrasik MP, et al. Assessment of the Inclusion of Racial/Ethnic Minority, Female, and Older Individuals in Vaccine Clinical Trials. JAMA Netw Open. 2021;4(2):e2037640.
16. Loree JM, Anand S, Dasari A, et al. Disparity of Race Reporting and Representation in Clinical Trials Leading to Cancer Drug Approvals From 2008 to 2018. JAMA Oncol. 2019;5(10):e191870.
17. Kennedy-Martin T, Curtis S, Faries D, Robinson S, Johnston J. A literature review on the representativeness of randomized controlled trial samples and implications for the external validity of trial results. Trials. 2015;16:495.
18. Gray DM, 2nd, Nolan TS, Gregory J, Joseph JJ. Diversity in clinical trials: an opportunity and imperative for community engagement. Lancet Gastroenterol Hepatol. 2021;6(8):605-607.
19. Bentley-Edwards KL, Jordan Fleming P, Doherty IA, Whicker DR, Mervin-Blake S, Barrett NJ. The 5Ws of Racial Equity in Research: A Framework for Applying a Racial Equity Lens Throughout the Research Process. Health Equity. 2022;6(1):917-921.
20. Levitan B, Getz K, Eisenstein EL, et al. Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI’s Patient Groups and Clinical Trials Project. Therapeutic Innovation & Regulatory Science. 2017;52(2):220-229.
21. Fouad MN, Acemgil A, Bae S, et al. Patient Navigation As a Model to Increase Participation of African Americans in Cancer Clinical Trials. Journal of Oncology Practice. 2016;12(6):556-563.
22. U.S. Food and Drug Administration. Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials Guidance for Industry. https://www.fda.gov/media/157635/download.
