Expert Meetings

Real-World Data and Evidence in the Evaluation of Medical Products

JUNE 12, 2018 TO JUNE 13, 2018

CTTI Project: Real-World Data

Meeting Scope:

  • Real-World Data (RWD) are data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.
  • Real-World Evidence (RWE) is the clinical evidence regarding the usage, and potential benefits and risks, of a medical project derived from analysis of RWD.
  • The focus of this meeting and project is to explore the appropriate use of electronic health records and payment claims (RWD) in randomized clinical trials (RCTs) to generate RWE to support regulatory decision-making.

Meeting Objectives:

  • Present findings from evidence gathering activities.
  • Identify barriers and potential solutions to generating RWE for regulatory submissions from these RWD sources.
  • Describe what recommendations and resources CTTI should develop to equip change agents to increase appropriate use of RWD in RCTs, including to support regulatory submissions.

Meeting Location:

DoubleTree by Hilton Hotel Bethesda-Washington DC 8120 Wisconsin Ave., Bethesda, MD 20814

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

  1. Introduction to the Clinical Trials Transformation Initiative (CTTI) by Pamela Tenaerts
  2. Issue, Project Overview, and Meeting Objectives by Gerrit Hamre
  3. Qualitative Interview Findings by Jack Sheehan
  4. Use of RWD in Pre-Study Planning and Study Set Up Session Introduction by Jane Perlmutter
  5. Use of RWD in Pre-Study Planning & Study Set up: Health Plan Perspective by Kevin Haynes
  6. Use of RWD in Pre-Study Planning & Study Set up: Manufacturer Perspective by Ben Gutierrez
  7. Using the Cystic Fibrosis Patient Registry to Support Clinical Trials Planning by Aliza Fink
  8. Use of RWD in Study Recruitment and Enrollment: EHR Enabled Platform by James Hamrick
  9. Patient & Site Identification Using an EMR Data System by David Thompson
  10. Real-World Treatment Responses in Advanced NSCLC Patient Subgroups by Michael Lu
  11. Real World Data Use in Embedded Pragmatic Clinical Trials by Sarah Leatherman
  12. Use of Real-World Data within ADAPTABLE, The Aspirin Study by Brad Hammill
  13. Day One Recap and Discussion of Priority Insights by Khair ElZarrad

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Patient Groups and Clinical Trials Expert Meeting

JANUARY 21, 2015 TO JANUARY 22, 2015

CTTI Project: Patient Group Engagement

Meeting Objectives

  • Define consensus principles of Patient Group engagement from the 3-way (Industry, Patient Group, and Academia) stakeholder perspectives using case studies of success and barriers
  • Share evidence and key findings from a 3-way stakeholder assessment of Patient Group engagement in the Clinical Trial process
  • Gain understanding of the FDA Rules of Engagement and various Conflict of Interest (COI) issues
  • Solicit feedback on evidence to inform recommendations that can further enhance the value to industry, academia and patient groups of mutually beneficial partnerships that engage the patient groups as fully as possible in the complete spectrum of the clinical research enterprise
  • Solicit feedback on benchmarking, metrics, and value of patient engagement in clinical trials for research sponsors

Meeting Location:

Fairmont Hotel, Washington, DC

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

DAY 1 – JANUARY 21 , 2015

DAY 2 – JANUARY 22 , 2015

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Stakeholder Perceptions of the Use of Mobile Technology in Clinical Trials

JANUARY 09, 2018 TO JANUARY 10, 2018

CTTI Project: Digital Health Trials

Meeting Objectives:

  • Present findings from CTTI evidence gathering activities examining the perspectives of investigators and potential research participants on the use of mobile technology for collection of objective data in clinical trials.
  • Discuss how this and additional evidence presented may be used to provide direction to the research enterprise for the appropriate utilization of mobile technology in clinical trials.
  • Identify products that CTTI should develop to equip the clinical trials enterprise to address the barriers, preferences, and needs of investigative site personnel and potential research participants in regulatory submission trials using mobile technology.

Meeting Location:

Hyatt Regency Bethesda 1 Bethesda Metro Center, Bethesda, MD 20814

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

  1. Welcome and Background by Annemarie Forrest
  2. Keynote Presentation by John Wilbanks
  3. Findings from the MCT Stakeholder Perceptions Survey of Potential Clinical Trial Participants by Angie Botto-van Bemden, Cindy Geoghegan, and Les Jordan
  4. Key Considerations for Patient Engagement in Clinical Trials by Suzanne Schrandt
  5. Ethical and Privacy Considerations of Mobile Technology by Camille Nebeker
  6. Findings from MCT Stakeholder Perceptions Investigator Interviews by Bill Wood

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Statistical Issues Think Tank II

NOVEMBER 19, 2014

CTTI Project: Unmet Need

Meeting Objectives

  • To provide an update on the current status of statistical methodologies for the design and analysis of antibacterial drugs
  • To discuss ongoing challenges in the development and adoption of innovative methods
  • To generate strategies to propel antibacterial drug development forward.

Meeting Location:

Bethesda North Marriott Hotel & Conference Center, Bethesda, MD

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

Session 1: Current status of drug development and ongoing challenges

Session 2: Current research and additional opportunities for the future

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Qualifying Investigators to Conduct Sponsored Clinical Trials

DECEMBER 13, 2017 TO DECEMBER 14, 2017

CTTI Project: Investigator Qualification

Meeting Objectives:

  • Report evidence gathered on:
    • Critical tasks associated with clinical investigators’ conduct of clinical trials.
    • Gaps and redundancies in training for preparing clinical investigators to conduct clinical trials.
    • Suggested knowledge and skills necessary for the quality conduct of clinical trials.
  • Evaluate proposed framework of characteristics within control of clinical investigator sites that define the quality conduct of a clinical trial.
  • Discuss how preparing clinical investigators for the quality conduct of a clinical trial could be optimized.
  • Identify the recommendations and tools that sponsors and investigators could implement to better prepare clinical investigators for the quality conduct of a clinical trial. Also, pinpoint the barriers—and solutions—to implementing these recommendations.

Meeting Location:

Hyatt Regency Bethesda 1 Bethesda Metro Center, Bethesda, MD 20814

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

  1. Welcome and Review of Aligned CTTI Work by Annemarie Forrest
  2. The CTTI Investigator Qualification Project and Meeting Goals by Jennifer Goldsack
  3. A Changing Ecosystem – A Brief Review of ICH GCP Renovations Planned and Currently Under Way by Theresa Mullin
  4. Proposed Framework of Characteristics that Define the Quality Conduct of a Clinical Trial by Janette Panhuis
  5. Evidence Gathering: Our Approach to Data Collection by Teri Swezey
  6. Critical Tasks that our Evidence Suggests Should be Well Executed to Drive the Quality Conduct of a Clinical Trial by David Ciavarella
  7. Perceived Risks to the Quality Conduct of Clinical Trials by Kate Haratonik
  8. Knowledge and Skills our Evidence Suggests may be Needed to Perform Critical Tasks and Mitigate Risks to Quality Trial Conduct by Catherine Dillion
  9. Evaluating Current Approaches to Preparing Investigators and Their Delegates by Patricia Hurley
  10. Building a Learning Ecosystem by Tina Chuck
  11. Case Study of an Investigator Mentoring Program by Emily Lemons
  12. Delivering Effective Training to Investigators and Their Delegates by Tina Chuck
  13. An Investigator's Reflections on Training by Christine Hildebrand
  14. FDA's Perspective on GCP Training by Bridget Foltz

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Scientific and Technological Issues Surrounding the Use of Mobile Devices in Clinical Trials

JUNE 15, 2017 TO JUNE 16, 2017

CTTI Project: Digital Health Trials

Meeting Objectives

  • Present findings from evidence gathering activities
  • Discuss how this evidence may be used to provide direction for the appropriate utilization of mobile devices for objective data capture in clinical trials. Specifically,
    • possible solutions to key data challenges
    • scientific and technological considerations
  • Identify the change agents who will drive adoption of mobile devices for capturing objective data in clinical trials, including for the purposes of regulatory submission
  • Describe what products CTTI should develop to equip change agents to include mobile devices for data capture in clinical trials, including for the purposes of regulatory submission

Meeting Location:

DoubleTree by Hilton Hotel Crystal City 300 Army Navy Drive | Arlington, VA

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

1. The CTTI MCT Mobile Devices Project and Meeting Goals by Jennifer Goldsack
2. Human Adaptation to Information: A Challenge for the Next Decade by Robert Califf
3. Mobile Clinical Trials and CDRH's Regulation of Devices by Dharmesh Patel
4. Recommendations from CTTI’s MCT Novel Endpoints Project by Jennifer Goldsack
5. Presentation of Evidence on Data Challenges (Panel Presentation)
6. Presentation of Evidence on Scientific and Technological Issues (Panel Presentation)

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Optimizing Operational Efficiencies for Data Collection in HABP/VABP Trials

NOVEMBER 12, 2013

CTTI Project: Streamlining HABP/VABP Trials

Meeting Background:

The goal of this meeting is to seek practical solutions to streamline the operational processes and build efficiencies for data collection in HABP/VABP trials.

Meeting Location:

Arlington, Virginia

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

  • Introduction by Pamela Tenaerts

Session 1: Challenges in data collection for HABP/VABP trials

Session 1 Goal: Understanding the issues surrounding data collection in HABP/VABP trials

Session 1 Focus: Why reducing excessive non-critical data collection in HABP/VABP trials would improve data quality, and benefit investigators, sponsors, reviewers, and patients

Session 2: Regulatory requirements for AE data collection in registration trials

Session 2 Goal: Discuss different regulatory requirements and the challenges of meeting these requirements for data collection for HABP/VABP registration trials that are globally conducted, and evaluate whether future work is needed to harmonize approach to AE collection

Session 2 Focus: AE collection and adjudication of relatedness and seriousness; Reporting of SAE’s and SUSARS and evaluation

Session 3: Strategies to simplify data collection using a QbD approach

Session 3 Goal: To simplify the recording, monitoring, and review to make HABP/VABP trials more feasible and economical

Session 3 Focus: Reduction in the amount of data collected by identifying data that are critical to quality and those that may be essential to determine the benefit/risk of the treatment for registration, and those may be less informative

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Strengthening the Investigator Community Project

APRIL 05, 2017

CTTI Project: Investigator Community

Meeting Objectives

  • Present findings from CTTI’s Strengthening the Investigator Site Community Project: Expert Interviews and Survey
  • Receive feedback on identified challenges experienced by principal investigators and strategies to overcome these challenges
  • Identify essential elements necessary to strengthen and grow the community of productive, experienced site investigators
  • Develop strategies and best practices to promote the growth and strengthening of the community of experienced site investigators
  • Identify barriers to strategy implementation and propose solutions

Meeting Location:

Sheraton Silver Spring Hotel 8777 Georgia Ave., Silver Spring, MD

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

1. Issue, Project Overview, and Meeting Objectives by Diana Foster
2. One and Done Survey Design and Findings by Christopher Fordyce
3. Active Investigator Interview Findings by Terri Hinkley Key
4. Elements for Site Investigator Success by Matthew Roe
5. Fiscal Responsibility and Discipline by Kaitlin Malone
6. Optimizing Trial Execution and Conduct by Robin Douglas
7. Investigator Perspective: My Approach / Why Do I Remain Involved? by David Whellan
8. PCORnet by Matthew Roe
9. A Primer in FDA Resources for Clinical Investigators by Bridget Foltz
10. FDA Observations Related to Investigator Participation by David Burrow

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Meeting on the Long-Term Safety and Efficacy of Extended-Release and Long-Acting Opioid Analgesics for Chronic Pain

AUGUST 12, 2013 TO AUGUST 13, 2013

Meeting Background:

The purpose of this meeting, hosted by CTTI, is to facilitate a broad scientific discussion about optimal ways to generate additional evidence to help inform the safe and effective use of extended-release and long-acting opioid analgesics.

Meeting Objectives:

  • Engage multiple stakeholders in discussions around approaches to develop more evidence on the long-term safety and efficacy of extended-release and long-acting opioid analgesics for the treatment of chronic non-cancer pain
  • Review and discuss a proposed study to evaluate the long-term efficacy of opioids for the treatment of chronic non-cancer pain
  • Discuss strategies to assess the long-term safety of extended-release and long-acting opioid analgesics for the treatment of chronic non-cancer pain

Meeting Location:

Sheraton Silver Spring Hotel, Silver Spring, MD

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

Session 1: Evidence for the Efficacy of Opioid Analgesics for Chronic Pain

Session 2: Review of Proposed Efficacy Study

Session 3: Continued Discussion of Efficacy Study

Session 4: Existing Data Sources to Evaluate Opioid Analgesic Safety

Session 5: Gathering Additional Evidence on Opioid Analgesic Safety

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.