OCE Insights is an occasional department developed for The ASCO Post by members of the Oncology Center of Excellence (OCE) at the U.S. Food and Drug Administration (FDA). In this installment, the OCE’s Steven Cunningham, MD, MLA, FACS, Clinical Reviewer on the Gastrointestinal Cancers Team, Division of Oncology 3; Donna Rivera, PharmD, MS, FISPE, Associate Director for Pharmacoepidemiology and Oncology RWE; Kirsten Goldberg, MA, Associate Director for Communications; Richard Pazdur, MD, Director; Kelly Norsworthy, MD, Associate Director for Research Mentorship; and Jennifer Gao, MD, Associate Director for Education, explore initiatives aimed at increasing awareness of and facilitating the conduct of pragmatic trials using approved oncology therapies.
What is Project 5 in 5?
Project 5 in 5 is a new crowdsourcing initiative launched on May 5, 2024, by the OCE to convene interested parties—including industry, cooperative groups, academia, advocacy organizations, and government agencies—to ultimately identify five important and clinically relevant questions using FDA-approved oncology therapies that can be answered through the use of pragmatic clinical trials over the next 5 years. As described by OCE Director Richard Pazdur, “These trials should be trials that answer five clinically important questions and will be completed in 5 years” following pragmatic principles.
Why did OCE initiate Project 5 in 5?
This initiative is important to enhance patient-centric clinical trial innovation. A key goal of the project is to raise awareness of, as well as to facilitate, the initiation and completion of pragmatic trials.
What are pragmatic trials?
Traditional prospective randomized controlled trials in oncology, which are typically associated with extensive monitoring, assessments, tests, and clinical follow-up visits that can be burdensome to trial participants, investigators, and trial sponsors. Pragmatic trials are designed to efficiently inform decision-making on the benefits, burdens, and risks of health interventions in representative populations by including elements that (1) are partially or fully integrated into routine clinical practice and/or (2) streamline trial design and conduct.
The differences between traditional clinical trials and pragmatic clinical trials span a continuum and not a dichotomous framework. Along this continuum, more pragmatic clinical trials incorporate design elements that are more reflective of routine clinical practice, take advantage of more functional efficiencies, such as fewer and simpler eligibility criteria, and have more flexibilities in trial delivery and outcome measurement. The degree of flexibility is tailored to the trial context with a focus on the safety of patients and the integrity of trial data.
Studies conducted in real-world settings can reduce the burden of traditional clinical trials, not only for trial participants (ie, patients), but also for providers and trial sponsors. The idea is to increase access, facilitate rapid enrollment, and reduce attrition. Furthermore, due to conduct in routine clinical practice, pragmatic trials have greater potential to produce evidence that is broadly applicable to populations affected by the clinical questions tested.
Where can I get more information about pragmatic clinical trials?
Please see the following available resources:
–OCE Project Pragmatica web page (fda.gov/about-fda/oncology-center-excellence/project-pragmatica)
–PRagmatic Explanatory Continuum Indicator Summary (www.precis-2.org)
–Rethinking Clinical Trials, NIH Pragmatic Trials Collaboratory (rethinkingclinicaltrials.org).
How will the FDA identify the five ideas for pragmatic clinical trials?
A multidisciplinary team at FDA Oncology with expertise in the relevant diseases will review and share the most popular ideas submitted via external crowdsourcing that meet the outlined specifications.
What happens after the five ideas are selected? Who can participate in conducting the trials?
While OCE is serving as the coordinating group, it will not be involved in the implementation or conduct of the clinical trials. Instead, OCE’s role is to inspire innovation by crowdsourcing ideas from the public, to facilitate open discussion, and then to select five crowdsourced ideas for pragmatic trials to share with the oncology community, including cooperative groups, academic researchers, government agencies, industry, advocacy organizations, and other interested parties who may be eager and able to implement pragmatic clinical trials to answer the proposed questions. All in the oncology community are encouraged to participate.
How can I participate?
Share your ideas on the Project 5 in 5 crowdsourcing web page (https://shareyourvoice.ideascalegov.com/c/), which will be available from May 5 to July 5, 2024. Describe an important and clinically relevant question that can be answered through the use of a pragmatic clinical trial over the next 5 years in your disease area of interest, using oncology therapies that are FDA-approved in that disease area. An example of a clinically relevant question could be: “What is the median overall survival for patients with <specific type of cancer> treated with drug X vs drug Y?” We look forward to hearing your ideas!
DISCLOSURE: The authors reported no conflicts of interest.
