Focus Area: Clinical Trials & Research

Clinical Research Competencies to Support Patient Partner Engagement

Webinar

May 7, 2026 @ 9:00 am – 10:00 am

Join the MRCT Center, CANTRAIN, and EUPATI to learn the results of an effort to integrate patient partnership into the existing Joint Task Force (JTF) Framework for Clinical Trial Competency.

The initiative, entitled “JTF-Patient Partner Project (P3) – Co-Creating Clinical Research Competencies to Support Effective Patient Partner Engagement Activities,” united a representative group of patient and caregiver partners, academic researchers and study staff, industry representatives, and others, to imagine what patient partnership within the JTF Framework would cover and include.

The updates proposed in JTF-P3 include a supplementary addendum focused on integrating patient partners into the study team. The results provide a blueprint that is both operational and aspirational, supporting more skilled, inclusive, and equitable clinical research teams to achieve results faster through more responsive and impactful trials.

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

  • Explain what patient partners and partnerships are, and why patient partner inclusion within study teams is so important
  • Explain the JTF-P3 process
  • Describe the JTF-P3 updates to the original JTF Framework
  • Identify next steps in the process of meaningful patient partner integration into study teams

This webinar will be offered twice, with the same content presented by different regional panelists. One registration and one Zoom link cover both sessions. Register once and join whichever session best fits your schedule.

  • Session A — May 7, 9:00 AM – 10:00 AM EDT (Boston/Ottawa); 3:00 PM – 4:00 PM CEST/SAST (Brussels/Cape Town); 6:30 PM – 7:30 PM IST (New Delhi)
  • Session B — May 7/8 8:00 PM – 9:00 PM EDT (Boston/Ottawa); 9:00 AM – 10:00 AM JST (Tokyo, May 8); 10:00 AM – 11:00 AM AEST (Melbourne, May 8)

Deploying Digital Twins and Synthetic Data in Evidence Generation

Webinar

Presented on: March 19, 2025

What does it take to deploy digital twins and synthetic data in clinical evidence generation — and what do regulators expect when you do?

In this third webinar in the MRCT Center’s Digital Twins and Synthetic Data series, a multidisciplinary panel examines the real-world application of these technologies across the clinical trial lifecycle. The discussion covers evidence quality and validation, regulatory benchmarks, model transparency, and the evolving landscape of FDA and EMA expectations. Panelists draw on experience spanning machine learning, FDA policy development, and drug development leadership to offer practical, grounded perspectives on what adoption looks like today — and where the field is headed.

Topics include:

  • Defining digital twins and synthetic data: key distinctions and appropriate uses
  • Reducing control arms and enhancing statistical power in randomized and single-arm trials
  • Applications across rare disease, oncology, and common conditions
  • Machine learning vs. traditional statistical approaches: complementary, not competing
  • Regulatory acceptance: FDA draft guidance, EMA qualification of PROCOVA, and engagement strategies
  • Model evaluation benchmarks and performance validation across development phases
  • Cultural and organizational barriers to adoption — and how to address them

Panelists: Daniele Bertolini, Principal Machine Learning Scientist, Unlearn.AI | Tala Fakhouri, VP Consulting AI & Digital Policy and Real World Evidence, Parexel | Karen Smith, Board Director, Context Therapeutics, Skye Bioscience, and Sangamo Therapeutics

Moderator: Barbara Bierer, Faculty Director, MRCT Center

A Framework for AI Adoption and Oversight in Clinical Research
2026-03-19b Evidence Generation DigiTwins and SynData_FINALDownload
AI Digital Twins and Synthetic Data: Practical Use Cases for Clinical Research
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

Characterization of Key Information Sections in Informed Consent Forms Posted on ClinicalTrials.gov 

Publication

Published on: August 14, 2023

Published in: Journal of Clinical and Translational Science

Abstract: Barbara Bierer and MRCT Center colleagues Walker Morrell, Ava Glazier, and Deborah Zarin, together with Luke Gelinas of Advarra and Tony Tse of the National Institutes of Health, published Characterization of Key Information Sections in Informed Consent Forms Posted on ClinicalTrials.gov in the Journal of Clinical and Translational Science. The 2018 revised Common Rule requires informed consent forms to open with a “concise and focused” key information (KI) section, but provides no guidance on content or format. Analyzing federally funded treatment trial ICFs posted on ClinicalTrials.gov, the authors found that three-quarters included an identifiable KI section averaging one page—yet readability showed little improvement over full ICFs, and a meaningful minority omitted core content such as the main reasons to join or not join a study. The authors call for consensus-based guidelines to help researchers and IRBs develop KI sections that are both compliant and genuinely useful to participants.

Gelinas L, Morrell W, Tse T, Glazier A, Zarin DA, Bierer BE. Characterization of key information sections in informed consent forms posted on ClinicalTrials.gov. Journal of Clinical and Translational Science. 2023;7(1):e185. doi:10.1017/cts.2023.605

View Article

Reimagining Informed Consent

Reimagining Informed Consent

The complexity of current informed consent models limit, rather than support, patient comprehension. Informed consent forms (ICFs) often reach dozens of pages long, exceed the general reading levels of clinical trial participants, and are focused on regulatory compliance and liability concerns rather than participant understanding of their rights and options as clinical trial participants.

The MRCT Center seeks to reimagine informed consent processes, moving beyond compliance-focused documentation toward innovative, participant-centered approaches. This initiative is exploring disruptive solutions within the informed consent development timeline, from structural changes to pre-IRB approval innovations to post-IRB approval, personalized consent pathways that adapt a “fit-for-purpose” consent model.

Our goal is to fully re-envision the informed consent process with global partnerships, while respecting participant autonomy throughout the research process. This work will develop in parallel project workstreams dedicated to structural changes to the ICF, as well as to changes in content and process. To learn more or get involved, please email Blythe Chen.

OBJECTIVES

  • Scope current field of informed consent and identify strategies for improvement
  • Develop and validate models of new informed consent processes, incorporating new technologies
  • Develop multi-stakeholder consensus on improved informed consent processes in multi-national academic and industry clinical trials, aligned with regulatory and ethical expectations.
  • Create tools and resources to enable adoption of innovative informed consent approaches.

KeY MILESTONES

  • March 2026: Project launch
  • September 2025-March 2026: Convened the subject matter experts to scope the opportunities for reinventing informed consent processes in clinical trials.
  • October 2025: Organized an interactive brainstorming session during the MRCT Center’s Annual Symposium on the Reimagining the informed Consent Process, beginning the project workstream.

project Leadership & sTAFF

  • Barbara Bierer, MD, Faculty Director, MRCT Center
  • Sarah White, MPH, Executive Director, MRCT Center
  • Blythe Chen, MPH, Research Assistant II, MRCT Center

Project Resources

Investigator Oversight in Decentralized Trials: New Resources and Practical Tools

 

Webinar

May 20, 2026 @ 12:00 pm – 1:00 pm

Join us on Wednesday, May 20, from 12:00 PM to 1:00 PM ET for a webinar introducing new tools to support investigator oversight in trials with decentralized elements. Developed by the MRCT Center and Medable, in collaboration with the PI-Oversight in DCT task force, these resources reflect cross-sector input and address the evolving responsibilities of investigators in DCT settings. This work builds on the MRCT Center’s prior guidance for IRB/EC review of decentralized trials and continues to expand the DCT project portfolio with practical, ethics-grounded tools.

Moderator: Barbara Bierer, Faculty Director, MRCT Center

IRB/EC Considerations for DCT Review
Ethical Considerations for Decentralized Clinical Trials: Tools, Resources and Best Practices

Deploying Digital Twins and Synthetic Data in Evidence Generation

Webinar

March 19, 2026 @ 1:00 pm – 2:00 pm

This webinar will examine the use of digital twins and synthetic data in evidence generation, focusing on (1) their emerging applications across the clinical trial lifecycle, including study design, control arm reduction, and single-arm trials, and (2) regulatory expectations and experience. Panelists will discuss how evidence quality, validation approaches, performance benchmarks, and model interpretability shape decision-making, and reflect on how practical and regulatory considerations influence the adoption of these approaches in clinical research. The discussion will draw on use cases, including synthetic controls and power enhancement strategies, and will include a moderated discussion and time for audience Q&A. 

Panelists:

Moderator: Barbara Bierer, Faculty Director, MRCT Center

Deploying Digital Twins and Synthetic Data in Evidence Generation
AI Digital Twins and Synthetic Data: Practical Use Cases for Clinical Research
AI Digital Twins and Synthetic Data: Application to Clinical Trials
A Framework for AI Adoption and Oversight in Clinical Research
Framework for Review of Clinical Research Involving AI

2025 Annual Symposium

Meeting

Presented on: October, 22, 2025

Presented at: Ropes & Gray and Hybrid

2025 Annual Symposium: Recordings now On-Demand

The MRCT Center held its Annual Symposium on October 22, 2025, hosting 100 people in Boston and 580 people virtually. Sessions focused on concrete implementation challenges in global clinical trials, including how to modernize trial operations, reduce inefficiencies, alleviate site and participant burden, reimagine informed consent as an enabling participant-centered process, and adopt the responsible use of AI. The MRCT Center Long-Term Follow-Up Toolkit was introduced.

The panel recordings are now available on demand.

Keynote | John F. Crowley (BIO)

John F. Crowley, President & CEO, Biotechnology Innovation Organization (BIO)
A call for regulatory modernization, trial simplification, and cross-sector partnership to sustain innovation and expand equitable access to medicines worldwide.

Panel | Clinical Trial Reform: The Need for Change and Progress

Kevin Bugin (Amgen); Karen Hartman (Mayo Clinic); Bridgette René McCullough (ACIRAH Health); Ann Meeker-O’Connell (Novartis)
Practical reforms to reduce protocol complexity and site burden, standardize systems, accelerate start-up, and expand patient access through adaptive and decentralized trial designs.

Session | Meeting the Moment in an Evolving Research Environment

Sarah White, Executive Director, MRCT Center
Repositioning the MRCT Center to remain nimble and opportunistic amid regulatory, technological, and operational uncertainty through focused work on access, innovative trial design, data use and protection, patient-centered approaches, responsive forums, global capacity building, and artificial intelligence.

Panel | Rethinking Informed Consent with Artificial Intelligence

Michael Cohen-Wolkowiez (Duke University); Megan Doerr (Sage Bionetworks); Cristin Freeman (Bristol Myers Squibb)
Real-world applications of AI to enhance informed consent, focusing on participant-centered, scalable, and feasible innovation, while respecting ethical and regulatory expectations.

Panel | Cell & Gene Therapy Long-Term Follow-Up (LTFU): Key Deliverables and the Path Forward

Lara Gehl (Bristol Myers Squibb); Daniel Kavanagh (WCG); Pamela Tenaerts (Medable)
Practical applications of the MRCT Center’s LTFU Toolkit to harmonize data, reduce participant burden, integrate decentralized methods, and meet long-term safety and regulatory requirements.

MRCT Center Updates | Select Initiatives

Willyanne DeCormier Plosky; Sarah White; Sylvia Baedorf Kassis; Lisa Koppelman; Barbara Bierer (MRCT Center)
Implementation updates on Representation in Research, capacity building through the TRACE Project, workforce competency standards (JTF), pediatric research, and the European Health Data Space.

Save the date: The 2026 MRCT Center Annual Symposium will take place on Wednesday, October 21, 2026, in Boston. An agenda will be shared in the coming months.

Navigating the MRCT Center’s Long-Term Follow-Up Toolkit for Gene Therapies

Podcast

Published: January 30, 2026

Podcast Episode

Listen to a podcast discussion with Barbara Bierer and Carolyn Chapman as they discuss the Toolkit for Supporting the Design, Conduct, and Reporting of Long-Term Follow-Up (LTFU) Studies for Gene Therapies (Version 2.0)

Available now on Spotify, YouTube, Apple Podcasts, and all streaming platforms. Subscribe to the MRCT Center Podcast to see the full library of conversations.

Toolkit for Supporting the Design, Conduct, and Reporting of Long-Term Follow-Up Studies for Gene Therapies

Long-Term Follow-Up for Gene Therapies: Key Deliverables and the Path Forward 

Video

Presented on: October 22, 2025, at the MRCT Center Annual Symposium

Long-Term Follow-Up (LTFU) studies are critical for understanding the long-term safety and risk/benefit profile of innovative cell and gene therapies. Study participants, patients, treating clinicians, companies, regulators, payers, and society-at-large all derive different value from LTFU studies. However, these studies present unique challenges due to their unprecedented duration. For sponsors, they are challenging and expensive to design, conduct, and operationalize. For patients, LTFU participation can be burdensome in terms of time, expense, and opportunity costs.

In this recorded panel, which was held during the MRCT Center’s Annual Symposium in October 2025, Dr. Carolyn Chapman describes the aims of the Center’s LTFU Working Group, which was launched in September 2024 and benefited from the perspectives of 25 members from outside the Center.  Dr. Chapman also previews the release of the LTFU Toolkit, which was collaboratively developed by the Working Group. The Toolkit offers practical guidance to support best practices for LTFU studies for both investigational and approved gene therapies.

In this session, Dr. Chapman leads a discussion with three distinguished industry leaders on a variety of issues addressed in the LTFU Toolkit. These include integrating decentralized elements to reduce burden, refining endpoint selection, harmonizing data for interoperability across LTFU studies, and minimizing loss to follow-up through participant engagement and clear communication. They also highlight the need for flexibility in LTFU study designs to meet evolving patient needs, and the importance of keeping new and current participants informed about protocol changes and any key findings. Looking ahead, the group calls for ongoing dialogue on data harmonization and sharing, results transparency, and innovative approaches to streamline the collection of long-term safety information.

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

Panelists:

  • Pamela Tenaerts, MD, MBA, Chief Medical Officer, Medable
  • Daniel Kavanagh, PhD, RAC – Senior Scientific Advisor, Gene Therapy, Vaccines, & Biologics, WCG
  • Lara Gehl, Executive Director, Global Trial Management – Bristol Myers Squibb
2026_LTFU-Toolkit-Version02_01-23-2026Download
Webinar: November 4, 2025
2025-11-04 LTFU Toolkit release webinar_finalDownload
Cell and Gene Therapies
Patient-Centered Long-Term Follow-Up for Gene Therapies
Innovative Approaches for Gene Therapy Long-Term Follow-Up

Toolkit for Supporting the Design, Conduct, and Reporting of Long-Term Follow-Up Studies for Gene Therapies

Toolkit

Released on: January 23, 2026

Long-term follow-up (LTFU) studies of gene therapy (GT) 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.

On January 23, 2026, the MRCT Center released version 2.0 of the Toolkit for Supporting the Design, Conduct, and Reporting of Long-Term Follow-Up Studies (CGT), revised and improved based on feedback of a draft that had released for public comment.  

The MRCT Center CGT Toolkit is comprehensive and provides background information, practical resources, and recommendations to support best practices for LTFU studies for both investigational and approved gene therapies—balancing the generation of critical long-term safety and efficacy data with the need to reduce burdens placed on participants, caregivers, sponsors, and investigators. 

Compared to the draft version, the updated Toolkit v2.0 includes two new resources, an Executive Summary and a Patient Resource, which have also been released as standalone versions. Additions included new and/or revised charts, tables, and resources. The Toolkit explores ideas for how LTFU studies could be improved in the future, raising questions that the field should discuss and address.

The Toolkit enables easy navigation to various sections and subsections via multiple clickable, interactive toolbars. The sections are as follows, with the core elements in bold font:

  • Executive Summary (new in v2.0)
  • Introduction and Background
  • Types of LTFU studies for GTs
  • Flowcharts
  • Guiding Principles
  • Considerations and Recommendations for the Design, Conduct, and Reporting of LTFU Studies for GTs
  • Looking Forward
  • Key Design Elements of LTFU Studies for FDA-approved GTs
  • Regulatory Guidance Relating to LTFU of GTs
  • Patient Resource: Long-Term Follow-Up Studies After Gene Therapy (new in v2.0)
  • Compiled Glossary of Scientific LTFU-Related Terminology
  • Easy-to-Understand (Accessible) LTFU-Related Definitions from the MRCT Center’s Clinical Research Glossary
  • Appendices
  • List of Acronyms and Abbreviations Used
  • References Cited

Please email us at mrct@bwh.harvard.edu with any questions or suggestions regarding the Toolkit.

2026_LTFU-Toolkit-Version02_01-23-2026Download

Navigation Highlights

The Toolkit incorporates several interactive features designed to support intuitive navigation and ease of use:

  • Clickable Table of Contents for rapid access to major sections and subsections.
  • Right-hand vertical navigation bar on every page, enabling quick movement between tools within the document.
  • Interactive table of LTFU study types, mirrored by color-coded tabs that remain clickable throughout the associated pages.
  • Secondary navigation bar at the top of each page within the Considerations & Recommendations section. This feature highlights your location within the nine subsections, allows you to jump directly between subsections by clicking the dots, and includes a grey diamond icon that returns you to the full list of subsections.
Navigating the MRCT Center’s Long-Term Follow-Up Toolkit for Gene Therapies
Long-Term Follow-Up for Gene Therapies: Toolkit Release
MRCT Center Annual Symposium: Panel 3 – Cell and Gene Therapies Long-Term Follow-Up (LTFU).
Panelists:
Pamela Tenaerts, MD, MBA, Chief Medical Officer, Medable
Daniel Kavanagh, PhD, RAC – Senior Scientific Advisor, Gene Therapy, Vaccines, & Biologics, WCG
Lara Gehl, Executive Director, Global Trial Management – Bristol Myers Squibb
𝗠𝗼𝗱𝗲𝗿𝗮𝘁𝗼𝗿:
Carolyn Riley Chapman, PhD, MS – Lead Investigator, Brigham and Women’s Hospital; Member of the Faculty, Harvard Medical School
Patient-Centered Long-Term Follow-Up for Gene Therapies
Innovative Approaches for Gene Therapy Long-Term Follow-Up
Cell and Gene Therapies