May 6, 2025

1:00 pm ET - 2:00 pm ET

Location:

Innovative Approaches for Gene Therapy Long-Term Follow-Up: Leveraging Registries and Platform Trials

Webinar

Please join us on Tuesday, May 6, from 1-2 pm ET for a webinar that will explore different approaches for realizing Gene Therapy (GT) Long-Term Follow-Up (LTFU). GTs have the potential for long-lasting and transformative health benefits, but there are also long-term health risks. Therefore, the FDA recommends long-term safety monitoring of recipients of certain types of GTs. As these LTFU studies can last years (even up to a lifetime), they pose significant scientific and logistical challenges. We are grateful for our three distinguished panelists, who will discuss innovative approaches for GT LTFU. 

Dr. Barbara Konkle, a hematologist at the Washington Center for Bleeding Disorders, Professor of Medicine Emeritus in Hematology-Oncology at the University of Washington, and the Medical Director of the Bleeding Disorders Laboratory at Bloodworks NW, will share information about the goals and development of the World Federation of Hemophilia’s Gene Therapy Registry (GTR). The GTR is a prospective, observational, and longitudinal registry designed to collect long-term data on people with hemophilia who receive GT. 

Dr. Amy Moskop is an Assistant Professor in the Department of Pediatrics and the Division of Hematology/Oncology/BMT at the Medical College of Wisconsin and the Scientific Director of the Gene Therapy Program at the Center for International Blood and Marrow Transplant Research (CIBMTR). Dr. Moskop will provide an overview of the CIBMTR registry and its intersection with GT LTFU. 

Dr. Avery McIntosh, Director, Biostatistics, Internal Medicine and Infectious Disease at Pfizer, will share practical considerations for GT LTFU safety studies and provide an overview of the potential benefits of platform and master protocol approaches. Dr. McIntosh has co-authored a paper on this topic, published in 2024 (doi:10.1002/cpt.3087). He has co-edited a book, Development of Gene Therapies: Strategic, Scientific, Regulatory, and Access Considerations, also published last year.

Dr. Carolyn Riley Chapman, who leads the Cell and Gene Therapies project at the MRCT Center, will moderate.

Bioethics Collaborative

May 8, 2025

10:00 am ET - 11:00 am ET

Location:

A Shared Language for Clinical Research: How Technical Organizations are Embracing Plain Language and Implementing the MRCT Center Clinical Research Glossary 

Webinar

A common misconception is that easy-to-understand, plain language information is only necessary in limited situations. However, adopting health literacy best practices benefits everyone who encounters simplified, thoughtfully designed scientific information even in highly technical areas like the data sciences.

In this webinar on Thursday, May 8, from 10 – 11 AM ET, leaders from the Society for Clinical Data Management (SCDM) and the Clinical Data Interchange Standards Consortium (CDISC) will join the MRCT Center to explain why plain language information is essential in the data sciences and how the organizations have embraced a culture of health literacy. This approach benefits both industry members, as well as patients, participants, and their caregivers.

Join us to learn how integrating health literacy and plain language resources improve communication and understanding in clinical research.

Key Topics/Objectives:

By the end of the webinar, attendees should be able to:

• Understand the connection between health literacy and data sciences, and why this is a priority for many organizations.
• Explain how health literacy and plain language resources have been integrated into organizational initiatives.
• Identify opportunities to implement health literacy best practices within their own organizations.

We appreciate your interest and look forward to an engaging discussion.

Bioethics Collaborative

June 24, 2025

3:00 pm ET - 4:00 pm ET

Location:

A Framework for AI Adoption and Oversight in Clinical Research

Webinar

Please join us on Tuesday, June 24, from 3:00-4:00 pm ET for a webinar to launch the MRCT Center and WCG’s Review Framework for Protocols Involving Artificial Intelligence (AI). This framework supports Institutional Review Boards (IRBs) in reviewing research where AI is part of the intervention. The framework was developed through a multi-disciplinary task force, and these actionable tools offer both (1) structured guidance grounded in U.S. regulations and (2) ethical considerations to assist IRBs in evaluating benefits and risks while ensuring oversight and transparency across the AI development lifecycle. This session introduces a practical decision-making resource intended to help IRBs determine when and how to apply appropriate and proportionate oversight to protocols involving AI.

Key Topics:

• Introduction to the IRB review framework focusing on aligning review practices with existing regulations and ethical standards.
• Structured questions IRBs can use to assess risk, benefits, and ethical considerations when AI is used as a research intervention.
• An expert panel discussion on navigating ethical considerations and the question of “AI Exceptionalism” during IRB review.

Bioethics Collaborative

June 26, 2025

1:00 pm ET - 2:00 pm ET

Location:

Patient-Centered Long-Term Follow-Up for Gene Therapies

Webinar

Please join us on Thursday, June 26, from 1:00-2:00 pm ET, for a webinar in which we will discuss patient-centered approaches in the specific context of Gene Therapy (GT) Long-Term Follow-Up (LTFU) studies.

GTs have the potential for long-lasting and transformative health benefits, but there are also possible long-term health risks. Therefore, the FDA recommends long-term safety monitoring of recipients of certain types of GTs to minimize and better understand any health risks. Although important, these LTFU studies can last years and may pose significant burdens on patients, which is why it is critical to consider patient needs and preferences in the design and conduct of LTFU for GTs. Our three panelists will offer different perspectives on how we can put patients at the center of LTFU for GTs. 

Bioethics Collaborative