CTTI News

CTTI Unveils New Resources to Facilitate Implementation of Single IRB for Multicenter Trials

Posted on by Lorri Bingham

CTTI has announced new resources to facilitate the implementation of single IRB (sIRB) in multicenter trials. The tools walk research institutions, sponsors, and IRBs through considerations for determining engagement, and provide a library of available sIRB resources. They are enhancements to CTTI’s extensive work on the topic dating back to 2010, which was featured in a recent issue of Contemporary Clinical Trials Communications.

“For nearly a decade, CTTI has been at the forefront of the sIRB movement, championing the thoughtful implementation of sIRBs for multicenter clinical trials to enhance patient safety, accelerate study start-up, and improve trial conduct,” said Pamela Tenaerts, executive director of CTTI. “We are pleased to unveil new resources to address some of the most pressing sIRB challenges and information gaps that remain for researchers. These tools augment CTTI’s already robust set of resources to collectively make sIRB adoption more seamless for the clinical trials enterprise.”

CTTI’s new tools include:

  • A set of Engagement Materials including:
    • An Engagement Overview, which provides an introduction to the Engagement Materials, information about who has the authority to make engagement decisions, required information to assess, and responsibilities of an engaged institution.
    • An Engagement Flowchart, which guides institutions through a set of questions for determining if its employees or agents are performing activities that constitute “human subjects research” on the institution’s behalf as defined by the Office for Human Research Protections (OHRP).
    • An Engagement Scenarios Guide, which illustrates examples of institutional involvement in research that would be considered “engaged” versus “not engaged.”
    • Engagement Definitions for terms used in the Engagement Materials.
  • A Resource of Resources that offers a library of available sIRB guides, templates, and tools to help stakeholders to optimally prepare, engage, and implement sIRB.

More information on these new resources is available via a recent CTTI-hosted webinar that features Nichelle Cobb (University of Wisconsin-Madison) unveiling the tools and showcasing their utility. The webinar also includes an overview of CTTI’s previous and ongoing work on sIRB for multicenter clinical trials, as well as results of recent semi-structured interviews to assess current perceptions of sIRB benefits, process challenges and solutions, and informational needs for using an sIRB process for FDA-regulated studies.

CTTI and FDA Workshop Explores Patient Partnerships at Every Step of Research Process

Posted on by Lorri Bingham

While many share the vision that patient engagement should be embedded throughout the research enterprise, Donna Cryer, president and CEO of the Global Liver Institute, and CTTI Executive Committee member, goes one step further: “I hope one day it’s simply called ‘research’.”

 

Cryer keynoted a recent public workshop, “Enhancing the Incorporation of Patient Perspectives in Clinical Trials,” convened by CTTI in collaboration with the FDA. The workshop provided an opportunity for patients, investigators, and other groups to have their voices heard and, ultimately, help shape better clinical trials. A full report on proceedings from the workshop and a summary of public comments are now available.

 

The workshop was divided into three main sessions:

 

  • Enhancing awareness and access to clinical trials
  • Designing and conducting patient-centered trials
  • Post-trial communication and engagement

 

Wrapping up the day’s event, a panel of multi-stakeholder thought leaders summarized their key takeaways and perspectives on the future of patient engagement:

 

Cryer said she was inspired by the idea that researchers should let patients push them to a place where they are uncomfortable, letting the patients chart the course. “It’s different from how medicine has been taught. It’s different from how research has been constructed,” she said. “But it’s an endeavor worth taking on.”

 

Michael Kurilla, clinical innovation director at the National Center for Advancing Translational Sciences, noted that the increased trend toward the use of wearables and digital media holds the potential to lessen the burden of clinical trial participation. “Use of wearables will stimulate new ideas and directions in terms of clinical trial research that wouldn’t have previously been considered,” he said.

 

Craig Lipset, former head of clinical innovation at Pfizer, said that patient engagement is still anecdotal and not consistent across the drug development industry. A key component that’s needed to make patient engagement consistent is empathy. “Everyone thinks they are empathetic, but that’s different from incorporating empathy into how you are designing and engaging,” he said. In addition to a call for greater consistency, Lipset also listed 5 other themes of critical importance: understanding and meeting the needs of participations before, during and after participation; strategies that can better bridge divide between research and health care; concierge-level support for participants that anticipates all the steps required to get to a study visit; remote and decentralized trials; and making return of results to participants a standard deliverable from sponsors.

 

Theresa Mullin, associate director for strategic initiatives at the FDA’s Center for Drug Evaluation and Research (CDER), discussed the need for companies to develop long-term relationships with patients. When companies develop partnerships, and communicate with patients transparently, patients will be generous with their time and insights.

 

Peter Saltonstall, president and CEO of the National Organization for Rare Disorders, said that it is important to “keep the patient at the absolute center of the conversation and build every single thing around the patient.” He cited the need for a new culture―from enrolling patients to returning results. “That culture isn’t fully developed yet and needs to be encouraged.”

 

Pamela Tenaerts, executive director of CTTI, said she is hopeful about the future of patient engagement. Key to the solutions discussed throughout the workshop is the idea of people working together. Organizations in the research ecosystem need to continue to facilitate communication between sponsor companies and patients.

 

John Wilbanks, chief commons officer for Sage Bionetworks, outlined two potential futures for patient engagement. In one future, patient engagement could be thought of solely in terms of recruitment and participation in traditional trials that are designed “for” and not “with” patients. In another future, patients would unite to demand more from investors in clinical research. The changes needed for true engagement require deep discomfort. “It’s really hard to add a little bit of change to a totally broken system. You have to actually change the system,” he said. “It will be slower and more expensive to center patients in this system that was built to center researchers and investors.”

 

Panelists concluded the workshop by expressing their deep gratitude to the patient representatives who attended the meeting and encouraging all attendees to continue moving toward true patient partnerships in clinical research.

 

This workshop met an FDA commitment that is part of the sixth authorization of the Prescription Drug User Fee Act (PDUFA VI).

CTTI Article Sheds Light on Participants’ Views of Mobile Clinical Trials

Posted on by Lorri Bingham

While adoption of mobile technologies presents many opportunities to enhance the quality and efficiency of clinical research, questions remain on patients’ preferences surrounding the use of mobile technologies in clinical trials. To help trial sponsors and others maximize the potential offered by mobile technologies, CTTI surveyed a group of potential research participants and recently published findings from the survey in Contemporary Clinical Trials Communications.

The survey results, which include responses from 193 individuals ages 23 to 83, shed light on the respondents’ perceptions of and willingness to participate in mobile clinical trials. In the survey, participants were presented with two similar hypothetical clinical trial scenarios: 1) relying on participants’ use of wearable and other mobile technologies outside of the clinic setting to collect study endpoints, and 2) relying on traditional in-clinic follow-up appointments with study staff to collect study endpoints. About 81 percent of respondents said they were willing to participate in the hypothetical mobile clinical trial, while only about 51 percent were willing to participate in the similar traditional clinical trial.

If given the option of participating in either scenario, the majority of respondents (76 percent) said they would prefer to participate in a mobile trial over a traditional one, citing greater convenience, fewer in-person visits, and perceived greater data collection accuracy offered by the mobile technology. Respondents were willing to use a variety of technologies―including mobile apps, wearable devices, and ingestible sensors―provided they were comfortable, convenient, and easy to use.

The survey also collected data about the aspects of mobile clinical trials that cause participants concern. Those who are less familiar with technology or do not use a smartphone said they are less likely to participate in a mobile clinical trial. Nearly half of respondents reported that they would not participate in a trial if there were no guarantees that their data would remain confidential, while others expressed concern about seeing a doctor less frequently in a mobile clinical trial setting.

CTTI’s findings contributed to the development of recently-announced recommendations and resources that help the research enterprise maximize the opportunities of mobile technologies to advance the development of new medical products. Related work and resources can be found as part of CTTI’s broader Mobile Clinical Trial program.

CTTI Launches New Tool to Help Identify High-Value Patient Engagement Opportunities

Posted on by Lorri Bingham

As part of its commitment to promote the engagement of patients as equal partners in clinical research, CTTI launched a new tool today at the 2019 DIA Global Annual Meeting that will help sponsors and patient groups identify high-value opportunities to collaborate.

The free web-based tool walks users through a three-step prioritization process:

  1. Identify relevant engagement activities. Patient groups and sponsors first identify all potential opportunities to collaborate, working from a comprehensive list developed by CTTI and adding their own fit-for-purpose activities.
  2. Evaluate benefits and investments. After reviewing factors identified by CTTI research, sponsors and patient groups rate the benefit and investment of each activity.
  3. Identify mutually beneficial activities. Research sponsors and patient groups compare and discuss their respective priorities and decide on opportunities that are of high value for each. A benefit/investment matrix helps visualize priorities and support discussion.

The tool is part of CTTI’s Patient Groups & Clinical Trials work, which encourages sponsors, investigators, and other stakeholders to engage with patient groups early and often for better and more efficient clinical trials.

“This new prioritization tool builds on the foundational work CTTI has already completed and ensures that collaboration is focused on areas where the greatest benefit can be achieved for everyone involved, given limited resources,” said CTTI Executive Director Pamela Tenaerts. “With an easy, three-step process, the tool helps create and identify high-value opportunities for patients and sponsors to engage in the clinical research enterprise.”

Linda Brennan, director of community partnerships at the Cystic Fibrosis Foundation, and Jaye Bea Smalley, director of patient advocacy and life cycle management for inflammation and immunology at Celgene Corporation, will publicly introduce the tool in the “Identifying High-Value Patient Engagement Opportunities: A Collaborative Three-Step Process for Sponsors and Patient Groups” DIA 2019 session at 8 a.m. PT.

CTTI to Focus on Three Key Areas of Clinical Research Improvement and Innovation at DIA 2019

Posted on by Lorri Bingham

At this year’s DIA 2019 Global Annual Meeting, CTTI will share insights from three key areas of its work dedicated to enhancing the quality and efficiency of clinical trials. At the event, which will take place in San Diego from June 23-27, CTTI will present its recommendations and resources for enhancing patient engagement, investigator qualification, and the use of mobile technology in clinical research. Please join us at the following presentations:

Presentation: Measuring the Impact of Patient Engagement Activities in Medicines R&D: A Way to Sustain Cultural Change

Date & Time: Tues., June 25, 2:00 – 3:15 p.m. PT (GMT-07:00)

Presenter: Jaye Bea Smalley (Celgene Corporation)

Related CTTI Project: Patient Groups & Clinical Trials

Presentation: Identifying High-Value Patient Engagement Opportunities: A Collaborative Three-Step Process for Sponsors and Patient Groups

Date & Time: Wed., June 26, 8:00 – 9:15 a.m. PT (GMT-07:00)

Presenters: Linda Brennan (Cystic Fibrosis Foundation) and Jaye Bea Smalley (Celgene Corporation)

Related CTTI Project: Patient Groups & Clinical Trials

Presentation: Improving Trial Quality by Better Preparing Site Teams

Date & Time: Wed., June 26, 8:00 – 9:15 a.m. PT (GMT-07:00)

Presenters: Sabrina Comic-Savic (The Medicines Company), Christine Hildebrand (Amici Clinical Research), Jimmy Bechtel (SCRS), Janette Panhuis (Population Health Research Institute, McMaster University), Ronnie Todaro (Parkinson’s Foundation)

Related CTTI Project: Investigator Qualification

Presentation: Demystifying Technology Selection in Mobile Clinical Trials

Date & Time: Wed., June 26, 8:00 – 9:15 a.m. PT (GMT-07:00)

Presenters: Aaron Coleman (Fitabase), Barry Peterson (Wearable Devices, Independent Consultant), Isaac Rodriguez-Chavez (FDA/CDER), and Thomas Switzer (Genentech―a member of the Roche Group)

Related CTTI Project: MCT Mobile Technologies

Presentation: A New Path Forward for Using Decentralized Clinical Trials

Date & Time: Wed., June 26, 4:15 – 5:30 p.m. PT (GMT-07:00)

Presenters: Gail Adinamis (GlobalCare Clinical Trials), Michael O’Brien (The Avoca Group), Laura Podolsky (Science 37), and Penny Randall (IQVIA)

Related CTTI Project: MCT Decentralized Clinical Trials

CTTI Unveils New Database of Feasibility Studies on Mobile Technologies in Clinical Research

Posted on by Lorri Bingham

Mobile technologies present exciting opportunities for researchers to monitor participants outside of the clinical setting. However, with new solutions entering the market daily, it can be challenging to decide which technology option will be most effective for a particular trial.

To help stakeholders address this issue, CTTI has established a searchable online database of pilot studies that assess the feasibility of various mobile technology solutions. The Interactive Database of Feasibility Studies for Mobile Clinical Trials, part of CTTI’s Mobile Clinical Trials Program, is the first publicly available resource that contains a catalogue of published feasibility studies related to the use of mobile technologies in clinical research.

A CTTI manuscript recently published in npj Digital Medicine, describes how this tool will enable sponsors and researchers to capitalize on information gained from 275 previous pilot studies to reduce risk and optimize the effective use of wearables and other devices in clinical trials. The database will allow users to search by therapeutic area, technology type, participant characteristics, and other variables to explore a range of issues surrounding mobile technology, such as sensor performance and algorithm development.

“We are excited about the opportunities presented by this new resource,” said CTTI Executive Director Pamela Tenaerts. “CTTI is continually striving to provide relevant recommendations surrounding the rapidly changing field of mobile technology. The database aligns nicely with CTTI’s comprehensive set of resources that will help stakeholders adopt mobile technologies to make clinical trials more effective.”

Resources from CTTI extend beyond the database—it also has created a set of recommendations and a tool that can be used to select mobile technologies for data capture in clinical trials.

CTTI plans to regularly maintain and update the database to give online users easy access to the most current knowledge on the use of mobile technologies in clinical trials. Viewers can also help grow the resource by submitting a paper to the database.

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.