Recommendations for using Real-World Data to Plan Eligibility Criteria and Enhance Recruitment
Novel Endpoints
Recording Now Available: The Fastest Path to Effective COVID-19 Treatments: Using Master Protocol Studies Public Summit
A recording for CTTI’s public summit, The Fastest Path to Effective COVID-19 Treatments: Using Master Protocol Studies, held Jan. 13, is now available. The summit, conducted as a webinar, opened with a welcome from Janet Woodcock, Operation Warp Speed; a call-to-action from Robert M. Califf, Verily and Google Health; and included three panel discussions focused on solutions related to scaling master protocols.
The first panel, Practical Solutions to Setting Up Master Protocol Sites, moderated by Pamela Tenaerts, CTTI, with panelists Derek Angus, REMAP-COVID; Laura Esserman, I-SPY-COVID; and Manizhe Payton, ACTIV-2, examined key challenges and potential solutions to starting up new sites including contracting, competing trials, staffing, and IRB submission.
The second panel, Increasing Participant Enrollment in Master Protocols, moderated by Esther Krofah, Faster Cures, with panelists Kousick Biswas, Veterans Health Administration; Dan Cooper, UC Irvine; and Martin Landray, RECOVERY Trial, examined the key challenges limiting participant enrollment including competition with other trials, burden on staff, and discussed potential solutions to overcome hurdles such as co-enrollment.
The final panel, Lessons for the Future, moderated by Mark McClellan, Duke-Margolis Center for Health Policy, with panelists Sam Brown, Intermountain Health System; Adrian Hernandez, PCORnet; and Saye Khoo, AGILE, discussed policy changes needed to improve future pandemic preparedness.
The public summit also addressed the status of COVID-19 clinical trials, including results from a recent CTTI analysis of data from ClinicalTrials.gov, and findings from a pre-summit survey of those involved in COVID-19 treatment master protocols, specifically those involved in setting up new sites or recruiting participants at existing sites.
View the full slide deck for the public summit to learn more from each presenter.
For additional information on CTTI’s master protocols work, please refer to this web page.
This public summit is part of a collaborative effort with the Duke-Margolis Center for Health Policy at Duke University and FasterCures, of the Milken Institute.
CTTI to Host Jan. 13 Public Summit, The Fastest Path to Effective COVID-19 Treatments: Using Master Protocol Studies, Featuring Research & Healthcare Leaders
CTTI will host a public summit, The Fastest Path to Effective COVID-19 Treatments: Using Master Protocol Studies, on Wednesday, January 13. The summit, conducted as a webinar and moderated by CTTI Executive Director Pamela Tenaerts, will include a welcome from Janet Woodcock, Operation Warp Speed; a call-to-action from Robert M. Califf, Verily and Google Health; remarks from Mark McClellan, Duke-Margolis Center for Health Policy; and a panel discussion moderated by Esther Krofah, FasterCures. The panel discussion will focus on solutions related to scaling master protocols, including:
- Overcoming barriers to starting up sites
- Increasing participants at existing sites
- Using the COVID experience to inform our preparedness for future pandemics
“As we’ve learned in the global pandemic, the faster we have reliable answers, the better,” said Tenaerts. “As we look to successfully accelerate progress for COVID-19 treatments, the need for collaborative trials that supply reliable insights has never been greater and in many cases, master protocol studies can provide the solution.”
This public summit will also address the status of COVID-19 clinical trials, including results from a recent CTTI analysis of data from ClinicalTrials.gov and other research, and examine the use of common platforms to optimize the design of master protocols for multiple therapies attempting to treat the same diseases or specific health problems.
The free public summit will be held from 10:30am until noon EST.
This public summit is part of a collaborative effort with the Duke-Margolis Center for Health Policy at Duke University and FasterCures, a Center of the Milken Institute.
CTTI Paper Highlights Clinical Criteria that Can Help Identify Patients at High Risk of HABP/VABP
Although treatment of possible nosocomial pneumonia is common with patients in the intensive care unit (ICU) receiving respiratory support, more than half of those treated do not fit the standard clinical definitions of hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP). A new CTTI paper available online in CHEST® Journal, accompanied by an insightful editorial, “Heeding the Prophetic Call,” outlines how the use of simple clinical criteria may help to identify high-risk patients earlier and aid future research to improve prevention and treatment.
The paper highlights the Prospective Identification of Pneumonia in Hospitalized Patients in the ICU (PROPHETIC) study, a large, contemporary, prospective cohort clinical trial designed by CTTI that made several key observations.
“HABP/VABP is associated with high mortality and morbidity and may be caused by multidrug-resistant pathogens,” said John Farley, director of the Office of Infectious Diseases at the FDA. “Conducting clinical trials in HABP/VABP is challenging, and data to understand the patient population is critical to improve trial feasibility.”
The study sought to define the incidence of HABP/VABP in an ICU population and identify characteristics associated with the development of HABP/VABP to inform the design and conduct of future clinical trials.
The study determined that 32 percent of 4,613 prospectively identified high-risk patients received antibiotics for possible HABP/VABP. It was also determined that only 12 percent of the aforementioned high-risk patients fit the FDA Guidance standard clinical definition of HABP/VABP.
“These findings indicate that the burden of HABP and VABP is significant and there is also some concern about antibiotic overprescription in this high-risk population,” said Vance Fowler, professor of medicine at Duke University. “Receiving antibiotics is itself a risk factor for developing pneumonia, carries risks of adverse events, and may preclude eligibility for HABP/VABP clinical trial enrollment.”
Additionally, the manuscript highlights the common characteristics and treatment exposures researchers identified that were associated with increased odds of developing HABP/VABP in high-risk patients.
“Application of the study results to prospectively identify patients at highest risk for HABP/VABP may help to facilitate the conduct of innovative and efficient clinical trials,” said Pamela Tenaerts, executive director of CTTI. “This will help to promote development of optimal preventive, diagnostic, and treatment strategies to improve management of this disease.”
Learn more about CTTI’s work on HABP/VABP Studies.
New CTTI Project Aims to Improve Clinical Evidence with Practical Approaches for Embedding Trials Into Health Care
Integrating trials that are intended for medical product review into clinical health care offers the promise of data that reflect real-world populations and settings, and improve efficiency by avoiding duplication of activities that already occur in clinical care. However, there are currently no guidelines or strategies to help clinical trial stakeholders operationalize this fit-for-purpose integration.
To remedy this issue, CTTI is starting work on a new project that will result in data, resources, and recommendations aimed at bridging the gap between trials and clinical care settings to improve evidence generation. This project aims to:
- Identify when elements of interventional clinical trial integration into clinical settings would be feasible and the associated benefits and risks
- Identify barriers and potential solutions to incorporating interventional trials into clinical care
- Describe operational approaches to incorporating interventional trials into clinical care
CTTI will develop the new recommendations by conducting in-depth interviews that identify barriers and solutions with a wide variety of clinical trial and healthcare experts, compiling case studies on innovative operational approaches, and organizing a multi-stakeholder meeting to discuss strategies.
By taking a multifaceted approach, CTTI aims to create resources that will help clinical trial stakeholders overcome the barriers to integrating interventional research into real-world settings to create more relevant results.
Recording Now Available: CTTI’s New Robust Set of Resources to Assist with Designing and Running Master Protocols
A recording is now available of the public webinar held on Tues., Oct. 13, to launch CTTI’s new set of resources that can be used to collaborate, communicate, and design master protocol studies. The webinar was led by Abby Bronson, Edgewise Therapeutics; Marianne Chase, Massachusetts General Hospital; Daniel Millar, Janssen R&D; and Nick Richardson, FDA, CDER.
While the interest in master protocol studies is growing, many organizations—particularly non-traditional drug developers such as patient advocacy groups and other nonprofits—lack the experience to design and implement this kind of study. In response to these challenges, through a high-level landscape review, a series of expert meetings, and a public open comment opportunity, CTTI developed a set of resources that include frameworks, case studies, and other interactive materials to assist in the planning and development of master protocols.
“With the success of master protocols in COVID-19 trials, there’s never been a better time to rethink the way you’re doing clinical trials in other disease areas,” said Pamela Tenaerts, executive director at CTTI. “In many cases, a well-designed and well-planned master protocol approach is an ideal way to get answers to scientific questions faster and serve patients better. Our new set of resources give sponsors, non-profit groups, and others a starting point and pathway for designing and conducting a successful master protocol study from start to finish in your disease area.”
The specific resources that CTTI released in conjunction with this webinar include:
- Value Proposition Guide: outlines key considerations for presenting the value proposition of the master protocol to stakeholders such as collaborators and vendors
- Study Simulation Tool: provides an overview of key modules that should be simulated in order to optimize master protocol design
- Protocol Development Map: describes the unique aspects of developing a master protocol from the academic or nonprofit organization perspective
- Operations Partner Assessment Tool: outlines considerations for engaging and selecting vendors as partners in master protocols
- FDA Engagement Tool: describes the formal mechanisms that facilitate early interaction with the FDA and provides a general timeline for development of an FDA engagement strategy
Both the webinar and latest resources can be found on the CTTI website.