Focus Area: Clinical Trials & Research

Joint Task Force for Clinical Trial Competency (JTF) Global Biannual Meeting

Meeting

June 22, 2026 @ 9:00 am – 11:00 am

The Joint Task Force for Clinical Trial Competency (JTF), anchored at the MRCT Center, develops and disseminates standards and practices for the global clinical research workforce. By fostering a cohesive and collaborative approach, the JTF ensures that professionals have the competencies to conduct clinical trials ethically and effectively.

Our international team of investigators, educators, and clinical research professionals has developed and/or utilizes a framework that defines the knowledge, skills, and attitudes necessary for conducting safe, ethical, and high-quality clinical research.

Join us on June 22, 2026, 9:00-11:00 AM ET for the Joint Task Force for Clinical Trial Competency (JTF) Biannual Global Meeting to hear about global perspectives on implementing the JTF Framework. Agenda specifics will be posted soon.

To learn more about the JTF Framework: https://mrctcenter.org/clinical-trial-competency/framework/overview/

All registrants will receive slides and a meeting summary after the meeting. This event is free.

Meeting of The Research, Development, and Regulatory Roundtable (R3)

November 5, 2026 @ 1:00 pm – 3:30 pm

The Research, Development, and Regulatory Roundtable (R3) is a forum to discuss pre-competitive issues in drug and device development, regulatory oversight of clinical trials, and human subjects research. Meetings convene policymakers, legal counsel, academicians, industry representatives, and global regulators. The R3 is a cooperative endeavor coordinated by the MRCT Center and Ropes & Gray LLP.

This hybrid meeting is open to sponsors of the Research, Development, and Regulatory Roundtable.

Topic: TBD

For more information, please email at MRCT@BWH.HARVARD.EDU

Deliverables

Projects

Meeting of The Research, Development, and Regulatory Roundtable (R3)

September 3, 2026 @ 1:00 pm – 3:30 pm

The Research, Development, and Regulatory Roundtable (R3) is a forum to discuss pre-competitive issues in drug and device development, regulatory oversight of clinical trials, and human subjects research. Meetings convene policymakers, legal counsel, academicians, industry representatives, and global regulators. The R3 is a cooperative endeavor coordinated by the MRCT Center and Ropes & Gray LLP.

This hybrid meeting is open to sponsors of the Research, Development, and Regulatory Roundtable.

Topic: TBD

For more information, please email at MRCT@BWH.HARVARD.EDU

Deliverables

Projects

Use of AI in Clinical Trials – Exploration of Key Legal and Regulatory Issues (continued)

June 8, 2026 @ 9:30 am – 11:30 am

The Research, Development, and Regulatory Roundtable (R3) is a forum to discuss pre-competitive issues in drug and device development, regulatory oversight of clinical trials, and human subjects research. Meetings convene policymakers, legal counsel, academicians, industry representatives, and global regulators. The R3 is a cooperative endeavor coordinated by the MRCT Center and Ropes & Gray LLP.

This hybrid meeting is open to sponsors of the Research, Development, and Regulatory Roundtable.

Topic: Use of AI in Clinical Trials – Exploration of Key Legal and Regulatory Issues (continued). This is the continuation of a discussion first introduced at the April 9, 2026 R3 meeting.

For more information, please email at MRCT@BWH.HARVARD.EDU

Deliverables

Projects

Use of AI in Clinical Trials – Exploration of Key Legal and Regulatory Issues

May 5, 2026 @ 1:00 pm – 3:30 pm

The Research, Development, and Regulatory Roundtable (R3) is a forum to discuss pre-competitive issues in drug and device development, regulatory oversight of clinical trials, and human subjects research. Meetings convene policymakers, legal counsel, academicians, industry representatives, and global regulators. The R3 is a cooperative endeavor coordinated by the MRCT Center and Ropes & Gray LLP.

This hybrid meeting is open to sponsors and select guests of the Research, Development, and Regulatory Roundtable.

Topic: Use of AI in Clinical Trials – Exploration of Key Legal and Regulatory Issues

For more information, please email at MRCT@BWH.HARVARD.EDU

Deliverables

Projects

Ethical, Legal, and Social Issues (ELSI) in Human Somatic Gene Therapy Clinical Research: A Scoping Review

Publication

Presented on: November 14, 2025

Published in: Human Gene Therapy

Carolyn Chapman, Mena Shaikh, Ava Glazier, Andrew Creamer, and Barbara Bierer published Ethical, Legal, and Social Issues (ELSI) in Human Somatic Gene Therapy Clinical Research: A Scoping Review in Human Gene Therapy. Dozens of gene therapies have been approved, and hundreds more are in development, prompting the need to better characterize the ethical, legal, and social implications (ELSI) of this emerging therapeutic class. The authors conducted a scoping review to map these issues across the literature, identifying themes related to risk–benefit assessment, engagement and communication, justice and access, ethical trial design, and the influence of financial and regulatory decision-making. The article also discusses potential approaches to address these ELSI as gene-therapy research expands.

View article

Ethical, Legal, and Social Issues (ELSI) in Human Somatic Gene Therapy Clinical Research: A Scoping Review

Carolyn Riley Chapman, Mena Shaikh, Ava Glazier, Andrew Creamer, and Barbara E. Bierer Human Gene Therapy 202536:21-22, 1387-1404

AI Digital Twins and Synthetic Data: Practical Use Cases for Clinical Research

Webinar

Presented on: November 18, 2025

This webinar focused on real-world examples and lessons learned from deploying synthetic data and digital twins across therapeutic areas.

Key Topics:

  • Reducing the size of control arms 
  • Enhancing Bayesian statistical analysis 
  • Supporting single-arm trials 
  • Optimizing the design of future trials 

Panelist: Daniele Bertolini, Machine Learning Scientist at Unlearn.ai.

Moderator: Barbara Bierer, Faculty Director at the MRCT Center

A Framework for AI Adoption and Oversight in Clinical Research
2025-11-18 Use Cases DigiTwins and SynData_CombinedDownload
AI Digital Twins and Synthetic Data: Application to Clinical Trials
Framework for Review of Clinical Research Involving AI
A Framework for AI Adoption and Oversight in Clinical Research

EC/IRB Guide to Understanding Post-Trial Continued Access

Guidance

Date: November 13, 2025

The EC/IRB Guide for Understanding Post-Trial Continued Access aims to assist Ethics Committees (EC) and Institutional Review Boards (IRB) in interpreting their role under Paragraph 34 of the Declaration of Helsinki. Paragraph 34 requires sponsors and researchers to arrange post-trial provisions for “participants who still need an intervention identified as beneficial and reasonably safe.” Exceptions to this must be approved by an EC or IRB, necessitating an understanding of when post-trial, continued access is applicable.

This resource outlines principles and criteria for evaluating when continued access is appropriate. It provides tools and questions to guide equitable decisions, ensuring ethical and transparent approaches to post-trial, continued access decisions. Additional resources and frameworks are available through the MRCT Center’s Post-Trial Responsibilities: Continued Access project page to support ethics committees.

Ethics Committees Post Trial Continued Access ResourceDownload
Continued Access to Investigational Products: Guiding Equitable and Fair Decisions
Framework: Post-trial, Continued Access Responsibilities to Investigational Significant-Risk Devices – Scenarios that require further consideration
Framework: Post-trial, Continued Access Responsibilities to Investigational Medicines
Principles of Post-Trial Responsibility: Continued Access to an Investigational Product
Post Trial Responsibilities: Continued Access

Mitigating Financial Toxicity for Participants in Clinical Trials

Webinar

December 9, 2025 @ 10:00 am – 11:00 am

Mitigating Financial Toxicity for Participants in Clinical Trials
Tuesday, December 9 | 10:00–11:00 AM ET – and now available ON-DEMAND

Hosted by the MRCT Center, in collaboration with Equitable Access to Clinical Trials (EACT), a project convened by LUNGevity Foundation.

Clinical trials are vital to advancing medical knowledge and care, yet participation can impose significant financial burdens on participants and their families—from travel and time away from work to uncovered medical and ancillary expenses.

This webinar examined the sources and impact of these costs and highlighted emerging strategies to reduce financial hardship for research volunteers. Presenters also introduced tools, checklists, and other resources developed through the EACT Project, a collaborative forum advancing financial neutrality in clinical research participation.

Panelists

Moderator
Dr. Barbara Bierer, Faculty Director, MRCT Center

November 30, 2022

Representation in Research ›

Long-Term Follow-Up for Gene Therapies: Toolkit Release

Webinar

Presented on: November 4, 2025

Long-term follow-up (LTFU) studies of gene therapy recipients are crucial for understanding the overall benefit-risk profile of these innovative products. However, LTFU studies are challenging to design, conduct, and execute, and pose significant burdens on both patients and sponsors.

In September 2024, the MRCT Center launched an LTFU Working Group. The committee comprises patients, as well as representatives from patient advocacy organizations, industry sponsors, academic medical centers, clinical research organizations, and human oversight protection organizations, each bringing diverse perspectives and complementary scientific, medical, regulatory, and ethical expertise.

On November 4, 2025, the MRCT Center released the Toolkit for Supporting the Design, Conduct, and Reporting of Long-Term Follow-Up Studies as a draft for public comment. The Toolkit provides practical guidance regarding best practices for LTFU studies for both investigational and approved gene therapies. It aims to balance the generation of critical long-term safety and efficacy data with the need to reduce burdens placed on participants, caregivers, sponsors, and investigators.

This webinar introduced the Toolkit’s 𝘀𝘁𝗿𝘂𝗰𝘁𝘂𝗿𝗲 𝗮𝗻𝗱 𝗰𝗼𝗻𝘁𝗲𝗻𝘁𝘀, including:
🔹 𝗚𝘂𝗶𝗱𝗶𝗻𝗴 𝗣𝗿𝗶𝗻𝗰𝗶𝗽𝗹𝗲𝘀
🔹 𝗖𝗼𝗻𝘀𝗶𝗱𝗲𝗿𝗮𝘁𝗶𝗼𝗻𝘀 𝗮𝗻𝗱 𝗥𝗲𝗰𝗼𝗺𝗺𝗲𝗻𝗱𝗮𝘁𝗶𝗼𝗻𝘀
🔹 𝗟𝗼𝗼𝗸𝗶𝗻𝗴 𝗙𝗼𝗿𝘄𝗮𝗿𝗱

It also highlighted additional practical resources:
🔹 𝗞𝗲𝘆 𝗱𝗲𝘀𝗶𝗴𝗻 𝗲𝗹𝗲𝗺𝗲𝗻𝘁𝘀 of LTFU studies for FDA-approved gene therapies
🔹 𝗜𝗻𝘁𝗲𝗿𝗻𝗮𝘁𝗶𝗼𝗻𝗮𝗹 𝗿𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 𝗴𝘂𝗶𝗱𝗮𝗻𝗰𝗲
🔹 𝗚𝗹𝗼𝘀𝘀𝗮𝗿𝗶𝗲𝘀 𝗮𝗻𝗱 𝗯𝗮𝗰𝗸𝗴𝗿𝗼𝘂𝗻𝗱 𝗶𝗻𝗳𝗼𝗿𝗺𝗮𝘁𝗶𝗼𝗻 on types of LTFU studies

𝗠𝗼𝗱𝗲𝗿𝗮𝘁𝗼𝗿:
Carolyn Riley Chapman, PhD MS – Lead Investigator, Brigham and Women’s Hospital; Member of the Faculty, Harvard Medical School

𝗣𝗮𝗻𝗲𝗹𝗶𝘀𝘁𝘀:
Durhane Wong-Rieger, PhD – President and CEO, Canadian Organization for Rare Disorders
Barbara Isquith Arone, MS – Vice President, Medical Affairs Category Lead, IQVIA
Patrick Cullinan, PhD – Head of Medical Writing and Transparency, Adverum Biotechnologies

2025-11-04 LTFU Toolkit release webinar_finalDownload
2025_LTFU-Toolkit_For-Public-Comment_11-04-2025Download
Cell and Gene Therapies

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/postapproval-methods-capture-safety-and-efficacy-data-cell-and-gene-therapy-products

Patient-Centered Long-Term Follow-Up for Gene Therapies
Innovative Approaches for Gene Therapy Long-Term Follow-Up