Quality

A Viewpoint on the Current State of Clinical Trials on the Path to Transformation from Dr. Robert Califf

CTTI Project: State of Clinical Trials

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Webinar Presenter:

Dr. Califf, who served as an original co-chair of CTTI and was instrumental in its creation, is a prominent cardiologist and clinical researcher whose career as a physician, teacher, researcher, and regulator spans more than three decades. Dr. Califf has provided leadership for numerous high-impact clinical trials and has been at the forefront of innovation in clinical research methods.

Webinar Information:

During this webinar, Dr. Robert Califf, former Commissioner of the US Food and Drug Administration (FDA) and the Fortin Professor of Cardiology at the Duke University School of Medicine, examined the current state of the clinical trials enterprise. Specifically, he addressed recent developments that have major implications for the clinical research enterprise in the United States, including the opportunities enabled by growing access to digital sources of data, the increasing importance of patient-reported outcomes, and the power of patient engagement in reshaping approaches to clinical research. As the recent head of FDA, Dr. Califf was directly involved in cross-agency efforts to modernize the national system for conducting clinical research by articulating a large, flexible, and widely shared approach to evidence generation (known as “EvGen”) that could leverage rapidly expanding sources of digital health data to produce high-quality, actionable information for patients and healthcare providers.

A particularly important aspect of these “EvGen” efforts hinges on effectively engaging patients and their advocates as partners in research, particularly as digital technologies and patient-reported outcomes data present new potential for the rapid and seamless acquisition of health data that is truly representative of broad and diverse populations. Ultimately, it is hoped that this new national research infrastructure will enable more rapid, representative, affordable, and generalizable clinical trials that can provide the high-quality evidence to enable a true learning health system. Many of these activities draw upon insights that were developed through pioneering investigations sponsored by CTTI, whose efforts will continue to shape national approaches to the creation and implementation medical knowledge.

Report Now Available: Virtual Public Workshop to Enhance Clinical Study Diversity

Posted on by Hannah Faulkner

The report of the two-day virtual public workshop to enhance clinical study diversity, convened by the U.S. Food and Drug Administration (FDA) in collaboration with the Clinical Trials Transformation Initiative (CTTI), is now available. The workshop was held on November 29 and 30, 2023 with more than 2,600 global attendees. 

The workshop solicited input on strategies and considerations for increasing the enrollment of historically underrepresented populations in clinical studies and encouraging clinical study participation that reflects the prevalence of diseases or conditions among demographic subgroups. Speakers and panelists included representatives from the FDA and other federal agencies, academia, medical device and pharmaceutical sponsors, patients and patient advocacy groups, and research study teams. 

The workshop fulfilled a requirement under section 3603 of the Food and Drug Omnibus Reform Act of 2022 (FDORA). View the recording and slide deck to learn more. 

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

Posted on by Hannah Faulkner

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. 

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

Posted on by Hannah Faulkner

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).