Author: Lorri Bingham

Five New Members Join CTTI Executive Committee

Posted on by Lorri Bingham

CTTI is pleased to announce that its Executive Committee will welcome five new distinguished thought leaders. These experts hold impressive track records in implementing change at many levels of the health care and research system, from regulatory bodies to thoe drug development pipeline.

  • Steven K. Galson has spent more than 20 years in government service at regulatory agencies.
  • Michael Kolodziej is an expert on payment reform, personalized medicine, and practice care delivery transformation in oncology.
  • Theodore Lystig provides leadership and guidance in the use of robust statistical and research design methods.
  • Rod MacKenzie focuses on global clinical development and advancement of medicines.
  • Pierre Meulien works to improve and accelerate the drug development process across Europe.

“These new Executive Committee members are leaders in their fields,” said CTTI Executive Director Pamela Tenaerts. “Their guidance and passion to improve clinical trials will help ensure CTTI can achieve its goals in a rapidly evolving ecosystem.”

The five new Executive Committee members join a group of thought leaders whose knowledge and experience enables CTTI to make meaningful improvements in clinical research.

“These experts will broaden and strengthen the committee’s knowledge in a number of key areas, from clinical research design to quality assurance to drug development,” said CTTI Executive Committee Chair Mark McClellan. “We are confident that they will make a significant contribution to CTTI’s work to move the clinical trials enterprise forward.”

Steven GalsonMichael KolodziejTheodore LystigRod MacKenziePierre Meulien

   Steven Galson                 Michael Kolodziej           Theodore Lystig           Rod MacKenzie           Pierre Meulien

CTTI Webinar Highlights the Importance & Benefits of Quality by Design

Posted on by Lorri Bingham

A recent CTTI webinar brought together stakeholders from across the clinical trials enterprise for an overview of Quality by Design (QbD), existing CTTI resources, and how QbD is being implemented in the real world.

QbD is defined as the absence of “errors that matter”—or, those errors that could jeopardize the ability to 1) protect patients during the trial, and 2) obtain reliable results and meaningful information from the trial. CTTI started working on QbD in 2011, with a focus on addressing ineffective clinical trial monitoring. It was determined that monitoring should be viewed as only one component of an overall quality framework. From there, CTTI went on to develop a broad set of evidence-based recommendations and resources to help drive adoption of QbD.

During the webinar, CTTI walked stakeholders through its QbD Toolkit, which is split into three sections that allow users to:

  • Learn about QbD;
  • Introduce QbD to their organizations via workshops and printable resources; and
  • Adopt QbD within their organizations.

CTTI also reviewed other available resources, such as the QbD Principles document, a key tool for QbD implementation.

Jean Mulinde, a senior policy advisor for the FDA, then spoke about why quality is important to clinical trials.

“QbD should be implemented at the clinical trial level and may be one component of an organization’s overarching quality management system,” Mulinde said. “Implementing QbD serves to focus the protocol and all of the operational plans necessary to implement the protocol on critical processes and data from the outset of a trial.”

Representatives from both academic and private organizations also shared their successes in QbD implementation. Hamid Moradi, a faculty member and researcher at University of California-Irvine, said that his organization hosted a CTTI-led QbD workshop to increase buy-in, which received a positive response from attendees. Moradi also discussed the QbD working group established at UC Irvine.

“This team will be a resource to investigators to help them better design and conduct their trials using QbD principles,” he said.

Currently, CTTI is working on additional QbD resources to support implementation, which it aims to announce within the next year.

Now Available: Summary of the March Patient Engagement Collaborative Meeting

Posted on by Lorri Bingham

The FDA, in collaboration with CTTI, convened the second in-person meeting of the Patient Engagement Collaborative (PEC) in March at the FDA’s White Oak Campus in Silver Spring, Md. A summary of the meeting is now available.

The primary objective of the PEC’s most recent meeting was to discuss a “how-to guide” to help patients and patient groups navigate engaging with the FDA. Ideas for new resources included a patient ambassador training program and informational marketing campaigns to meet patients where they live and work.

The PEC, a joint initiative of the FDA and CTTI, has 16 members who were chosen from nearly 200 patients and patient advocates. The members’ discussions help to inform the FDA’s efforts to engage patients meaningfully in medical product development and other regulatory discussions.

This effort is facilitated by provisions in the 21st Century Cures Act of 2016 and the Food and Drug Administration Reauthorization Act of 2017.

Article Outlines Lessons Learned from First Large Pragmatic Trial Using Sentinel

Posted on by Lorri Bingham

A new article in Clinical Trials, co-authored by CTTI, outlines lessons learned from the planning phase of the implementation of a randomized controlled trial to improve treatment with oral anticoagulants in patients with Atrial Fibrillation (IMPACT-AFib) trial, the first large pragmatic clinical trial that used the Sentinel infrastructure. This work is part of CTTI’s larger effort to advance trials that use real-world data sources.

The FDA’s Sentinel System uses electronic health records (EHR) and insurance claims from 18 health plans and delivery systems covering about 45 million individuals enrolled in commercial health plans, in addition to 22 million Medicare recipients.

The IMPACT-AFib trial, launched in 2017 by collaborators including CTTI, identified patients with atrial fibrillation who were at high risk of stroke, and randomized them to either an early or delayed intervention. The Sentinel System helped the trial team assess the size of the potential trial population, examine baseline measurements, and determine there were significant gaps in care, which showed there was a need for the trial.

In selecting participants for the trial and conducting other planning analyses, the trial team used Sentinel’s common data model and reusable analytic programs, which accelerated the planning and budgeting for the trial while keeping member-level data safe.

The IMPACT-AFib team also considered limitations introduced by Sentinel, including the potential for misclassification due to the use of administrative claims data. Other lessons learned for developing health plan-based clinical trials include using a single IRB, involving patient representatives in protocol design, and timing the start of the trial and mailing of materials around open enrollment and other health plan initiatives.

Learn more about CTTI’s other work to increase the appropriate use of real-world data sources and real-world evidence in clinical trials for regulatory submission.

Webinar Now Available: CTTI’s New Recommendations for Engaging Patients and Sites in Mobile Trials

Posted on by Lorri Bingham

A recording is now available of the public webinar on Thurs., Feb. 21, to launch CTTI’s new recommendations for incorporating patient and site perspectives in mobile clinical trials. The webinar was led by patient advocate Cindy Geoghegan and Virginia Nido of Genentech, a member of the Roche Group.

CTTI conducted qualitative and quantitative research to better understand the perceptions of patients and site personnel, and convened experts and leaders across the clinical trials enterprise to develop actionable recommendations and resources.

“Patients are attracted to the possibilities of greater convenience and greater access to clinical research—fewer visits, less time away from work and family, and less burden on their caregivers,” Geoghegan said. “Our goal was to enhance the experience for both patients and hard-working site staff.”

The resulting recommendations are designed to help research sponsors engage patient and site perspectives in planning mobile trials, maximize value and minimize burden for study participants, and address challenges for investigative sites.

“One of the key messages is the importance of planning,” Nido said. “We are new to this as an industry, and we need to plan for and address these new challenges in order to maximize the benefits of mobile technology in clinical trials.”

This is the final of four sets of recommendations CTTI has released as part of its Mobile Clinical Trials (MCT) Program. In 2017, CTTI announced recommendations for developing novel endpoints generated by mobile technologies and, in 2018, it unveiled new solutions for using mobile technologies for data capture in clinical trials, and recommendations for using decentralized clinical trials. Additionally, they build on previous CTTI work—including our Quality by Design and Patient Groups and Clinical Trials recommendations—that emphasizes the involvement of many stakeholders, including patients and sites, in trial design.

CTTI Unveils New Recommendations for Incorporating Patient and Site Perspectives in Mobile Trials

Posted on by Lorri Bingham

CTTI released new recommendations and resources today for engaging patients and sites when planning clinical trials using mobile technologies. These recommendations have the potential to help sponsors, CROs, and other stakeholders gain the full benefits of using mobile technologies in clinical trials, including remote participation, increased protocol adherence, and participation by a more diverse population.

While buy-in from stakeholders is critical to the success of mobile trials, little was known about patient and site perspectives until CTTI conducted surveys and interviews with patients and investigative site personnel to assess perceived benefits and barriers.

Engaging Patients and Sites Recommendations

The resulting recommendations have the potential to help research sponsors:

  • Engage patients and sites in protocol design, technology selection, and pilot testing
  • Maximize value and minimize burden for study participants by setting patient expectations, protecting privacy, returning data, enhancing patient-site interactions, and providing technical support
  • Address challenges for investigative sites, from contracting and budgeting to evaluating site readiness and implementing effective and streamlined training

“CTTI’s recommendations and resources are designed to take our understanding of patient and site needs in mobile clinical trials to the next level,” said Bill Wood, associate professor at the School of Medicine at the University of North Carolina at Chapel Hill. “With these new insights, sponsors and researchers can plan and conduct mobile trials that work better for everyone involved, streamlining the clinical research process and accelerating the development of critical new therapies.”

The recommendations are the fourth set generated through CTTI’s Mobile Clinical Trials (MCT) Program, following recommendations on developing novel endpoints generated by mobile technologies, using mobile technologies for data capture in clinical trials, and planning and conducting decentralized clinical trials. Additionally, they build on previous CTTI work—including our Quality by Design and Patients Groups and Clinical Trials recommendations—that emphasizes the involvement of many stakeholders, including patients and sites, in trial design.

CTTI and FDA Workshop Will Explore How Best to Include Patient Perspectives in Clinical Trials

Posted on by Lorri Bingham

CTTI will convene a public workshop, “Enhancing the Incorporation of Patient Perspectives in Clinical Trials” in collaboration with the FDA on Mon., March 18, from 9:00 a.m. to 5:00 p.m. at the Tommy Douglas Conference Center in Silver Spring, Md.

 

The workshop will seek ideas for best practices and key considerations for enhancing the incorporation of patient perspectives on clinical trial access, design, conduct, and post-trial follow-up. It will also gather input from patients, caregivers, industry, academic researchers, and expert practitioners on the challenges and barriers to patient participation in clinical trials. CTTI and the FDA are especially interested in the perspectives of patients, caregivers, researchers, and others who have participated in clinical trials and are willing to share their experiences.

 

This workshop meets an FDA commitment that is part of the sixth authorization of the Prescription Drug User Fee Act (PDUFA VI). After the event, a published report on proceedings and recommendations from the workshop will be made publicly available.

 

Register for the workshop by March 11. Registration is free and based on space availability, with priority given to early registrants. If you are unable to attend the workshop in person, you can watch the live webcast.

 

Don’t miss this opportunity to make your voice heard and shape the future of clinical research!

CTTI to Launch Recommendations for Engaging Patient and Site Perspectives in Mobile Clinical Trials

Posted on by Lorri Bingham

CTTI will host a public webinar on Thurs., Feb. 21, to announce new recommendations and resources for incorporating patient and site perspectives when planning and conducting mobile clinical trials.

“Acceptance and adoption by key stakeholders is critical to the widespread use of mobile technologies in clinical trials,” said CTTI Executive Director Pamela Tenaerts. “However, very little was known about patient and sites’ perspectives until our work shed light on what they see as significant benefits and barriers. Our new recommendations account for these perspectives and provide a valuable resource for optimally deploying mobile clinical trials.

“Further, they build on previous CTTI work—including our Quality by Design and Patient Groups and Clinical Trials recommendations—that emphasizes the involvement of many stakeholders, including patients and sites, in trial design. Engaging these groups early and often is the key to running better trials.”

CTTI conducted surveys and in-depth interviews to better understand the perceptions of patients and clinical investigators, and convened experts and leaders across the clinical trials enterprise to develop actionable recommendations and resources.

The resulting recommendations are designed to assist research sponsors in:

  • Engaging patient and site perspectives in planning clinical trials using mobile technologies, including protocol design, technology selection, and pilot testing
  • Maximizing value and minimizing burden for study participants, including setting patient expectations, protecting privacy, returning individual data, enhancing patient-site interactions, and providing technical support
  • Addressing challenges for investigative sites, from contracting and budgeting to evaluating site readiness and implementing effective and streamlined training

This is the final of four sets of recommendations CTTI has released as part of its Mobile Clinical Trials (MCT) Program. In 2017, CTTI announced recommendations for developing novel endpoints generated by mobile technologies and, in 2018, it unveiled new solutions for using mobile technologies for data capture in clinical trials, and recommendations for planning and conducting decentralized clinical trials.

The free webinar will begin at noon ET and will be led by patient advocate Cindy Geoghegan and Virginia Nido of Genentech, a member of the Roche Group.

Now Available: Summary of the Patient Engagement Collaborative Inaugural Meeting

Posted on by Lorri Bingham

The FDA, in collaboration with CTTI, convened the inaugural meeting of the newly formed Patient Engagement Collaborative (PEC) in August at the FDA’s White Oak Campus in Silver Spring, Md. A summary of the meeting is now available.

 

The PEC is a joint initiative of the FDA and CTTI. Its 16 members, selected from nearly 200 patients and advocates, are helping expand the FDA’s efforts to achieve meaningful patient engagement in medical product development and other regulatory discussions. This effort is facilitated by provisions in the 21st Century Cures Act of 2016 and the Food and Drug Administration Reauthorization Act of 2017.

 

The objectives of the PEC’s first meeting were to agree on the group’s vision and goals and to discuss opportunities to enhance communications and resources for interactions between the FDA and the patient community. Among the meeting’s key takeaways, the PEC agreed that patient engagement can inform the research and development continuum from beginning to end, and that a systematic approach by the FDA to including the patient voice in the regulatory decision-making process will be important.

 

The PEC will meet several times a year, both in-person and by teleconference, to continue to discuss how to enhance patient engagement in regulatory decision-making.

 

CTTI Article Finds that Patients and IRBs are Amenable to Early Enrollment Strategy

Posted on by Lorri Bingham

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.