FDA Catalyst–using FDA’s Sentinel Initiative for large scale pragmatic randomized trials
Novel Endpoints
CTTI Article Finds that Patients and IRBs are Amenable to Early Enrollment Strategy
A CTTI article recently published in JAMA Network Open shows that an early enrollment strategy for research on healthcare-associated pneumonia is acceptable to patients, investigators, and institutional review boards (IRBs). This strategy has the potential to speed enrollment in trials for critical new antibiotic therapies by allowing patients to be approached and consented to before being diagnosed with pneumonia.
Through its ABDD HABP/VABP Studies work, CTTI conducted qualitative interviews with 52 stakeholders—including patients at risk for pneumonia, caregivers, study investigators and coordinators, and IRB representatives—as part of formative research to assess the acceptability of the approach.
The study found that patients and caregivers had no concerns about patients being approached early and having their records monitored before they developed pneumonia. They believed that patients would be able to understand consent information before diagnosis, and shared their preferences for opt-out procedures.
IRB representatives were also supportive of an early enrollment strategy, and investigators and study coordinators indicated that the approach would not be burdensome.
Experts Explore Ways to Promote Use of Real-World Evidence in Clinical Trials: Latest CTTI Expert Meeting Summary Now
The use of real-world evidence (RWE)—derived from sources such as electronic health records (EHR) and claims data—has the potential to provide a more complete picture of patient experiences, reduce costs, and offer unique insights into important scientific questions. At the same time, there are relatively few examples to inform efforts to integrate RWE into clinical trials, as well as a lack of consensus among researchers about valid approaches for leveraging these data sources.
To address this issue, CTTI convened an expert meeting in June 2018 to discuss best practices and needed resources for advancing the use of RWE in randomized clinical trials. The meeting (see one-page summary) included investigators, patients, regulators, technology experts, sponsor representatives, and other groups.
While participants agreed that the use of RWE could be a major step forward in clinical trials, they stressed the importance of recognizing and planning for its limitations, including inconsistent data reliability and quality. They also agreed that direct interaction with participants and the integration of various real-world data sources is critical for improving the depth and breadth of available data.
As the next step in its Real-World Evidence work, CTTI will develop recommendations and resources for incorporating RWE into randomized trials for regulatory submission.
CTTI Study Shows More Efforts Are Needed to Stimulate Pediatric Antibacterial and Antifungal Drug Trials
In an article recently published in Pediatrics, CTTI researchers assess the impact of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) on pediatric antibacterial and antifungal drug trials.
The study, which evaluated pediatric trials conducted between 2007 and 2017, found that nearly two-thirds of pediatric antibacterial and antifungal drug trials were conducted under BPCA or PREA. These trials were more likely to collect pharmacokinetic data and report results than non-BPCA/PREA trials.
However, the overall number of pediatric antibacterial and antifungal drug trials was low, representing less than 1 percent of pediatric trials overall. These trials also rarely enrolled infants up to 30 days old.
The findings show that, while federal legislation is likely having an impact on pediatric antibacterial and antifungal drug trials, more efforts are needed to stimulate these trials and improve the reporting of results.
This study was conducted as part of CTTI’s ABDD Peds Trials Project, which focuses on creating efficient, evidence-based processes to accelerate the development of safe and effective pediatric antibacterial drugs.
CTTI Celebrates Clinical Trials Day at SCT Annual Meeting
CTTI will commemorate Clinical Trials Day (which will be celebrated on May 21 this year) by leading a number of presentations at the Society for Clinical Trials (SCT) Annual Meeting May 20-23 in Portland, Ore. CTTI will discuss its findings on using mobile technologies for data capture, selecting and developing novel endpoints for use in clinical research, incorporating real-world evidence in randomized clinical trials, and more.
CTTI Executive Director Pamela Tenaerts will also share lessons learned since CTTI’s inception in 2007 and reflect on 10 years of transforming the clinical trials enterprise. See a full schedule of CTTI presentations below.
Poster: Selecting, Developing and Incorporating Technology-Derived Novel Endpoints into Clinical Trials
Date: Mon., May 21, 3:30-4:00pm
Related CTTI Project: MCT Novel Endpoints
Poster: One Decade of Impact. One Vision Ahead. Optimizing the Power of Public-Private Partnership: Lessons Learned from the Clinical Trials Transformation Initiative
Date: Tues., May 22, 3:30-4:00 p.m.
Poster: Use of Qualitative Data Analysis Software to Facilitate a Systematic Literature Review
Date: Tues., May 22, 10:15-10:45 a.m.
Related CTTI Project: MCT Novel Endpoints
Presentation 1: The Challenges and Opportunities of Mobile Technology: A Qualitative Examination of Investigators’ Experiences and Recommendations
Presentation 2: Mobile Technology and Clinical Trials: Patient Perspectives and Opportunities to Reduce the Burden of Participation
Date: Wed., May 23, 9:30-10:30 am
Presenter: Virginia Nido (Genentech – a member of the Roche Group)
Related CTTI Project: MCT Stakeholder Perceptions
Presentation: Designing Study Protocols When Mobile Devices Are Used for Data Capture: Recommendations from the Clinical Trials Transformation Initiative
Date: Wed., May 23, 9:30-10:30 am
Presenter: Tom Switzer (Genentech – a member of the Roche Group)
Related CTTI Project: MCT Mobile Technologies
Presentation: Understanding the Clinical Trial Enterprise’s Newest Trend: Incorporating Real-World Evidence into Randomized Clinical Trials
Date: Wed., May 23, 9:30-10:30 am
Presenter: Scott Evans (SCT)
Related CTTI Project: Real-World Data
CTTI Releases 2017 Annual Report: One Decade of Impact. One Vision Ahead.
CTTI’s 2017 Annual Report, released today, celebrates a decade of CTTI impact and highlights our activities and accomplishments over the past year. The report includes reflections from past and present CTTI leaders, case studies on the use of CTTI recommendations and resources, and a glimpse into what lies ahead for CTTI and the clinical trials enterprise.
Since its founding in 2007, CTTI has completed more than 25 projects with the aim of increasing the quality and efficiency of clinical trials. The resulting recommendations were downloaded more than 26,000 times over the past year.
CTTI issued five new sets of recommendations in 2017, many of which draw on recent advances in technology and data sciences to bring about improvements for clinical trials. Read the report to learn more about our latest recommendations and resources, which offer:
- Practical guidance for using mobile technology to develop viable novel endpoints for clinical trials.
- Best practices for assessing and designing registries for use in clinical trials so that the data can meet expectations for FDA review of new products.
- Actions that can be taken to strengthen the investigator site community and create an environment that sustains long-term investigator engagement.
- Guidance on planning for and making decisions about pregnancy testing in potential trial participants.
- Suggested ways to address the unique challenges of conducting pediatric antibacterial trials.
As described in the report, these recommendations and resources are being implemented by organizations across the clinical trial spectrum, including IQVIA, monARC Bionetworks, and the Pulmonary Fibrosis Foundation.
As we reflect on the past year—and the past decade—we are grateful for the engagement and commitment of our members, who ensure that we continue to address the latest trends, top barriers, and leading opportunities in clinical research.