Innovative Approaches for Gene Therapy Long-Term Follow-Up

Webinar

Date: May 6, 2025

Innovative approaches to long-term follow-up (LTFU) in gene therapy (GT), including Registries and Platform Trials. While GTs may offer transformative health benefits, long-term safety monitoring is often essential and can span years or decades. Our panelists will explore scientific and logistical challenges, along with emerging solutions.

Panelists:

  • Dr. Barbara Konkle (Washington Center for Bleeding Disorders, University of Washington, Bloodworks NW) on the World Federation of Hemophilia’s Gene Therapy Registry, a global effort to track long-term outcomes in hemophilia.
  • Dr. Amy Moskop (Medical College of Wisconsin, CIBMTR) on the CIBMTR registry and its role in GT LTFU.
  • Dr. Avery McIntosh (Pfizer) on safety study design, platform protocols, and master protocol approaches, drawing from recent publications.

Moderator: Dr. Carolyn Riley Chapman, MRCT Center.

Related Resources:

MRCT Center:

Introduction:

Avery McIntosh, PhD:

Barbara A. Konkle, MD:

Amy Moskop, MD, MS:

Advancing Pediatric Platform Trials: A Conversation with Dr. Danny Benjamin

Podcast

Published: April 2024

Podcast Episode: Advancing Pediatric Platform Trials – A Conversation with Dr. Danny Benjamin

Recorded during the MRCT Center’s October 2024 conference, Advancing Pediatric Platform Trials: Streamlining Development, Maximizing Impact, this keynote conversation features Dr. Danny Benjamin, Kiser-Arena Distinguished Professor of Pediatrics at Duke University Medical Center.

Discussion Highlights:

  • The historical evolution of pediatric drug development and key regulatory milestones
  • The role and achievements of the Pediatric Trials Network (PTN)
  • How platform trials can increase efficiency and reduce burden in pediatric research
  • Ethical and operational challenges unique to pediatric platform trials
  • Real-world examples demonstrating the value of shared trial infrastructure
  • Future directions to drive innovation, collaboration, and regulatory alignment

🔗 Listen to the full episode: EP 1: Advancing Pediatric Platform Trials – A Conversation with Dr. Danny Benjamin

More about the Advancing Pediatric Platform Trials: Streamlining Development, Maximizing Impact conference:

The MRCT Center hosted a 2-day hybrid meeting in Washington DC in October 2024 to examine the potential benefits, challenges, and opportunities of platform trials for pediatric populations. In addition to consideration of overarching issues, three diseases – pediatric oncology, major depressive disorder (MDD), and multidrug-resistant tuberculosis (MDR-TB) – were chosen to represent different conditions, epidemiology, settings, therapeutic challenges, and patient populations, and to illuminate different potential approaches and solutions. 

With ~80 pediatric and platform trial experts from Europe, the UK, Asia, Australia and the US in attendance, the discussants were successfully identified knowledge gaps and practical challenges that impact pediatric platform trial planning and execution. Each of the three subgroups recommended actionable approaches to address identified issues, and the respective groups continued to meet and take concrete steps, furthering the momentum created at the workshop, with the MRCT Center remaining actively involved with each disease-specific subgroup to facilitate the next steps.

Framework: Post-trial, Continued Access Responsibilities to Investigational Significant-Risk Devices – Scenarios that require further consideration

Framework

Date: April 25, 2025

The Post-Trial, Continued Access Responsibilities to Investigational Significant-Risk Device Framework: Scenarios that Require Further Consideration outlines five key milestones, specific scenarios, and considerations to support organizations in making equitable and transparent decisions regarding continued access to investigational significant-risk devices. A companion framework addressing investigational medicines is also available. Both frameworks are intended to be used alongside the Principles of Post-Trial Responsibilities – Continued Access, a set of 12 foundational principles that define the shared obligations of stakeholders in ensuring appropriate post-trial, continued access.

To learn more about the Post-Trial Responsibilities – Continued Access, click here.  

Advocating for collaboration among key partners to promote diversity in clinical studies amid policy challenges in the United States of America

Publication

Published on: April 25, 2025

Published in: Trials

In a commentary published in Trials“Advocating for collaboration among key partners to promote diversity in clinical studies amid policy challenges in the United States of America”, Barbara Bierer and colleagues underscore the urgent need for sustained, cross-sector collaboration to protect and advance diversity in U.S. clinical research. Given the importance of representativeness of the participant population, the authors highlight practical, unified strategies—emerging from the 2023 Stanford Think Tank—to ensure inclusive participation across the clinical research enterprise.

This article is Open Access, available under the terms of a Creative Commons Attribution 4.0 International License.

Advancing Pediatric Platform Trials: Streamlining Development, Maximizing Impact

Meeting Summary

Published: April 2024

The MRCT Center hosted a 2-day hybrid meeting in Washington DC in October 2024 to examine the potential benefits, challenges, and opportunities of platform trials for pediatric populations. In addition to consideration of overarching issues, three diseases – pediatric oncology, major depressive disorder (MDD), and multidrug-resistant tuberculosis (MDR-TB) – were chosen to represent different conditions, epidemiology, settings, therapeutic challenges, and patient populations, and to illuminate different potential approaches and solutions. 

With ~80 pediatric and platform trial experts from Europe, the UK, Asia, Australia and the US in attendance, the discussants were successfully identified knowledge gaps and practical challenges that impact pediatric platform trial planning and execution. Each of the three subgroups recommended actionable approaches to address identified issues, and the respective groups continued to meet and take concrete steps, furthering the momentum created at the workshop, with the MRCT Center remaining actively involved with each disease-specific subgroup to facilitate the next steps.

We appreciate the financial support of several companies, without whom this workshop would not have been possible. The workshop was supported in part by voluntary contributions from AstraZeneca, Johnson & Johnson, and Sanofi. Our thanks to all the individuals who contribute their time and expertise to the planning and conduct of this workshop and without whom this event would not have been possible. We are delighted to share the comprehensive workshop report, a detailed summary of the meeting proceedings.

Related Resource:

Podcast Episode: Advancing Pediatric Platform Trials – A Conversation with Dr. Danny Benjamin

Recorded during the MRCT Center’s October 2024 conference, Advancing Pediatric Platform Trials: Streamlining Development, Maximizing Impact, this keynote conversation features Dr. Danny Benjamin, Kiser-Arena Distinguished Professor of Pediatrics at Duke University Medical Center.

Discussion Highlights:

  • The historical evolution of pediatric drug development and key regulatory milestones
  • The role and achievements of the Pediatric Trials Network (PTN)
  • How platform trials can increase efficiency and reduce burden in pediatric research
  • Ethical and operational challenges unique to pediatric platform trials
  • Real-world examples demonstrating the value of shared trial infrastructure
  • Future directions to drive innovation, collaboration, and regulatory alignment

🔗 Listen to the full episode: EP 1: Advancing Pediatric Platform Trials – A Conversation with Dr. Danny Benjamin

Global Development of Clinical Research Workforce: Tools and Resources

Webinar

Date: April 3, 2025

Dr. Lembit Rago, Secretary-General of the Council for International Organizations of Medical Sciences (CIOMS), delivered a compelling keynote on CIOMS initiatives to define and synthesize professional competencies essential for clinical research and emphasized the importance of cooperative agreements to harmonize workforce development efforts.​

The session also highlighted the Joint Task Force for Clinical Trial Competency (JTF) Framework as a foundational tool for training and professional development, addressing the challenges and opportunities in recruiting and retaining a diverse and capable workforce.​

Sally Armstrong, CEO of PRAXIS Australia, shared how PRAXIS has incorporated the JTF Framework into their educational offerings—including courses, workshops, and immersive onsite training programs.​

Susan Landis, Executive Director of the Association of Clinical Research Professionals (ACRP), discussed ACRP’s “Partners Advancing the Clinical Research Workforce” initiative, which uses the JTF Framework to support a diverse and qualified clinical research workforce through training programs, resources, and strategic collaborations.​

A recording of the webinar and the presentation slides are available here:​

On-demand YouTube webinar recording: Global Development of a Clinical Research Workforce: Tools and Resources

Related Resources:

Data Literacy in Clinical Research: Enhancing Trust and Transparency

Webinar

Date: April 3, 2025

A key aspect of health literacy—especially in clinical research—is understanding what data is collected, how it is protected, and how it is used during and after a study. Truly informed consent requires transparency about data practices so that participants can make educated decisions.

The MRCT Center and PHUSE are collaborating to develop a suite of resources, including videos and infographics, to enhance data literacy in the clinical research context.

During this webinar we discuss the importance of data literacy, explore practical applications for research teams, and unveil new materials designed to support participants and the broader research community.

Key Topics:

  • Advancing Data Literacy as a Component of Health Literacy: Explore how understanding research data supports informed decision-making and aligns with broader health literacy efforts.
  • Applying Plain Language and Visual Tools to Data Communication: Learn how the MRCT Center and PHUSE have used plain language and infographics to make clinical research data more accessible.
  • Empowering Participants with Transparent and Actionable Information: Identify ways to integrate data literacy resources into your clinical research processes to support research trust and engagement.

Related Resources:

MRCT Center Clinical Research Glossary

PHUSE – The Global Healthcare Data Science Community

PHUSE Working Group: Educate the General Public on Data Privacy and Data Sharing

Data Literacy: MRCT Center and PHUSE Resources

Flyers and Videos

Developed: April 2025 

Developed by: MRCT Center and PHUSE

The MRCT Center is pleased to collaborate with the PHUSE Data Transparency team on materials to educate the general public on Data Privacy and Data Sharing. These materials were presented at a webinar on April 3, 2025. The recording and slides are now available on-demand here.

These infographics and videos are freely available and designed to explain how data is used and protected in clinical research.

MRCT Center-led Infographics:

PHUSE-led Videos:

Joint Publications:

We are also thrilled to share some publications about this work:


​*You can learn more about PHUSE’s data transparency work here.

Resources

Comment: A global call to action for disability inclusion in health research

Publication

Published on: March 26, 2025

Published in: Nature Medicine

Abstract: Barbara Bierer and Willyanne DeCormier Plosky co-authored an article, led by Anna M. Anderson (University of Leeds), in Nature Medicine titled, “A global call to action for disability inclusion in health research.”

Disabled people are often excluded from health research despite being the largest underserved group in many countries, having worked to enshrine legal mandates for fair treatment and equal opportunity, and driving innovations that benefit everyone. The article describes the research cycle, points at which disabled people currently face barriers, and actions that can be taken by the research community to better involve disabled people during those points in the research cycle. This call to action is made by authors from different countries around the world as part of the Disability Inclusion in Research Collaboration (DIRECT) to emphasize the critical importance of collaboration, learning, and support across countries on this issue.

Workforce Development: Opportunities to Engage and Retain Talent in Clinical Research

Webinar

Presented on: February 25, 2025

The MRCT Center, on behalf of the Convergence Project hosted a virtual event focused on workforce development strategies to support a more dynamic and competitive clinical research workforce. This meeting brought together community leaders, policymakers, and researchers to discuss how to create and support pathways into the clinical trial workforce (e.g., part-time degree programs, community colleges, fellowships). Key topics included workforce development, targeted recruitment, and actionable steps to drive systemic improvements, and meeting attendees had the opportunity to pose questions to panelists leading programs in Georgia, North Carolina, South Dakota, and other states across the country.

Related Resources