Study Design

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.

Obtaining Novel Endpoint Reliability and Acceptance Expert Meeting

JULY 27, 2021 TO JULY 28, 2021

CTTI Project: Developing Novel Endpoints

Meeting Objectives:

  • Identify the barriers and discuss solutions to the adoption of DHT-derived endpoints into pivotal trials
  • Discuss the data needed to prove that a DHT-derived endpoint is ready for a pivotal trial
  • Explore how collaboration and other new efforts can advance the adoption of DHT-derived endpoints

Meeting Location:

Virtual Meeting

Meeting Agenda

Meeting Summary

List of meeting attendees

Pre-Meeting Materials

Full Presentation Set - Day 1

Full Presentation Set - Day 2

Meeting Materials:

Day 1

Day 2

The views and opinions expressed in these presentations 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.

The Use of Antibacterial Drugs Developed via Streamlined Approaches for Serious Infections Where There is Unmet Need: Understanding Patient and Physician Perspectives and Considerations to Take Forward

MARCH 01, 2016

CTTI Project: Unmet Need

Meeting Objectives

  • Present perspectives from patients, caregivers and physicians on antibacterial drugs developed using streamlined approaches
  • Identify focus group themes and discuss topics which should be further explored or where draft recommendations could be made
  • Obtain feedback to improve labeling, risk communication, public understanding and stewardship

Meeting Location:

Sheraton Silver Spring Hotel, Silver Spring, MD

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

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.

Increasing Diversity in Clinical Trials Expert Meeting

OCTOBER 12 & 21, 2021

CTTI Project: Diversity

Meeting Objectives:

  • Present findings from project evidence generation: in-depth interviews with key decision-makers.
  • Refine a maturity model for organizational-level strategies to increase diversity in clinical trials.
  • Identify specific multi-stakeholder, portfolio-level strategies to increase the participation of underrepresented racial and ethnic minorities and women in clinical trials.

Meeting Location: Virtual Meeting

Meeting Summary

Meeting Agenda

List of Meeting Attendees

Pre-Meeting Materials

Meeting Materials:

Day 1

 

Day 2

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.

Master Protocol 2020 Expert Meeting

APRIL 20, 2020 TO APRIL 21, 2020

CTTI Project: Master Protocol Studies

Meeting Overview:
The purpose of the meeting was to continue to drive the development of publicly available resources to support the successful design and implementation of master protocol studies. Specific meeting objectives included the following:

  • Refine CTTI tools to support the development of emerging master protocol studies in diverse therapeutic areas
  • Identify specific capacity building efforts to support cross-institutional collaboration in response to COVID-19 and other major public health threats

Meeting Materials:

Meeting Agenda

Meeting Summary

Full Presentation Set - Day 1

Full Presentation Set - Day 2

Expert Meeting on Large Simple Trials (LSTs)

MAY 13, 2013 TO MAY 14, 2013

CTTI Project: Large Simple Trials

Meeting Background:

The purpose of this meeting is to develop recommendations to facilitate and promote the adoption of LST designs for regulatory submissions or other purposes.

Meeting Objectives:

  • Discuss findings from a survey of practices
  • Discuss strategies that companies are using to implement LSTs
  • Discuss the challenges to LSTs

Meeting Location:

Hilton Washington DC/Rockville Hotel and Executive Center, Rockville, MD

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

Day 1 - Session 1: Landscape of LSTs

Day 1 - Session 2: Models of Approaches to LSTs 

Day 2

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.

Master Protocol 2019 Expert Meeting

OCTOBER 22, 2019 TO OCTOBER 23, 2019

CTTI Project: Master Protocol Studies

Meeting Overview:
The purpose of the meeting was to explore and develop strategies to address common challenges related to the design and implementation of master protocols, including:

  • Mapping the pre-planning, planning, and execution processes of a master protocol trial.
  • Strategizing solutions to common roadblocks related to the pre-planning, planning, and execution of a master protocol study.
  • Identifying and prioritizing the creation of tools to support the adoption of emerging best practices outlined by CTTI’s roadmap.

Meeting Materials:

Meeting Agenda

Meeting Summary

Roadmap

Participant List

Full Presentation Set - Day 1

Full Presentation Set - Day 2

Welcoming Remarks & Meeting Overview

Session I: Landscape Review

Session II: Building Capacity for Innovation

Session III: Small Group Breakout Sessions

Session IV: Tool Identification

Session V: Next Steps & Closing Comments

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.

ICH E6 Guideline for Good Clinical Practice – Update on Progress

MAY 18, 2021 TO MAY 19, 2021

CTTI Project: Informing the Update of ICH E6

On behalf of the International Council for Harmonisation (ICH), the ICH E6 Good Clinical Practice (GCP) Expert Working Group (EWG) held a free public web conference to provide an update on the progress to revise this important and impactful guideline. This web conference was convened by the Clinical Trials Transformation Initiative (CTTI).

The EWG held two similar meetings on Tuesday, May 18, 2021 (8-11 a.m. EDT) and Wednesday, May 19, 2021 (6-9 pm JST) to reach a broad global audience across time zones. As you will note on the agenda, the same topics were presented each day with speakers from different regions to represent the global effort. All information and presentations was provided in English.

Recorded presentations of the public web conferences are available above.

Meeting Location:

Virtual Meeting

Meeting Agenda - May 18

Meeting Agenda - May 19

Meeting Summary

Meeting Materials:

Video: ICH Guideline Development Process

Watch May 18 Web Conference

Watch May 19 Web Conference

 

Presentations:

Session 1 – General Introduction

Session 2 – E6(R3) GCP Expert Working Group (EWG) Vision & Engagement

Session 3 – Principles & Stakeholder Reflections

Full Web Conference Slide Deck

 

Additional Meeting Details:

ICH E6 GCP is the international ethical, scientific, and quality standard for the conduct of clinical trials for the development of new drugs and biologics involving human participants that are intended to support regulatory applications. Due to the wide impact of this important guideline, the ICH Management Committee is taking this unique step to provide a status update on the revisions to the guideline.

In this web conference, members of the ICH E6 EWG discussed the work-in-progress to develop principles and annexes for ICH E6 GCP (third version or E6(R3)) that are intended to be responsive across clinical trial types and settings and to remain relevant as technology and methodologies advance. The draft, work-in-progress principles that were made public by the ICH on April 19, 2021, are designed to be flexible and applicable to a broad range of clinical trials. View the principles on the ICH website. The EWG is not taking public comments on the principles at this stage. However, once the ICH E6 guideline achieves step 3 of the ICH guideline development process, the EWG will invite and consider public input.

The EWG also discussed its plans and approaches to update the guideline in general and its engagement efforts with a variety of stakeholders that greatly enriched the discussions of the EWG. As part of these continued efforts to engage with stakeholders, the web conference included presentations from stakeholders on their vision and aspiration for clinical trial design and conduct that are responsive to the needs of the community.

View the full conference agenda here. For additional information on the ICH E6 revision efforts, please see the Reflection Paper on Renovation of Good Clinical Practice and the Concept Paper. For further information on the engagement approach, please see the published outline of the ICH E6 engagement proposal. Other materials, including the current guideline, "ICH E6(R2): Guideline for Good Clinical Practice," the business plan, work plan, an expert list, and reports of prior public engagements are available on the ICH website: https://www.ich.org/page/efficacy-guidelines.

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.

CTTI Informed Consent Project Expert Meeting

MARCH 10, 2015 TO MARCH 11, 2015

CTTI Project: Informed Consent

Meeting Objectives

  • Present findings and conclusions from the project literature review and expert interview series
  • Solicit feedback and develop consensus on proposed recommendations to enhance the informed consent process

Meeting Location:

DoubleTree by Hilton Hotel Washington DC, Silver Spring, MD

Meeting Materials:

Meeting Summary

Meeting Agenda

List of meeting attendees

Meeting Presentations:

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.