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
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
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:
Daniele Bertolini, Principal Scientist, Unlearn.AI
Tala Fakhouri, Vice President: Consulting, AI & Digital Policy, Real-World Research, Parexel
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.
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.
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.
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.
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.
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.
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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
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.
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:
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.
From Policy to Practice: Implementing the EHDS for Responsible Data Sharing
The European Health Data Space (EHDS) establishes a common framework for sharing electronic health data across all 27 EU member states in a secure, standardized manner, advancing individuals’ ability to access and control their own health data and enabling certain health data to be used for approved secondary uses.
In May 2022, the European Commission proposed EHDS to harmonize electronic health data sharing and governance across all EU member states. The EHDS Regulation, finalized and entered into force for implementation on March 26, 2025, is the inaugural initiative among nine planned European sector-specific data spaces. As a new regulatory framework, EHDS introduces significant opportunities that encompass two core components: (1) patient rights regarding access and control of their electronic health data, supported by the necessary infrastructure; (2) provisions for access and secondary use of health data for research, innovation, policy development, and other approved uses. The implementation of these components requires harmonization of data governance mechanisms across diverse health systems of EU member states.
To support stakeholders in understanding and navigating this evolving landscape, the MRCT Center has partnered with Vivli to co-host a series of online, invitation-only forums for interested pharmaceutical and biotechnology companies.
These forward-looking sessions address and discuss EHDS implementation and its accompanying data governance, harmonization, and compliance pieces for secondary use of data, as well as allow for collaboration opportunities to develop and submit public commentaries on EHDS draft implementation guidelines. Through this collaborative approach, we aim to identify and address opportunities and challenges, ensuring EHDS fulfills its promise of advancing personal health, innovation, and clinical research across Europe.
Regulation (EU) 2025/327 of the European Parliament and of the Council of 11 February 2025 on the European Health Data Space and Amending Directive 2011/24/EU and Regulation (EU) 2024/2847 (Text with EEA Relevance). 11 Feb. 2025, http://data.europa.eu/eli/reg/2025/327/oj/eng.
Convene and engage global stakeholders to collaborate on EHDS developments, coordinate efforts, and foster dialogue on data sharing and governance.
Guide policy and framework development toward a harmonized system for secondary use of health data, ensuring alignment with ethical, regulatory, and compliance standards that advance innovation in clinical research and healthcare.
KeY MILESTONES
November 2025: Hosted the EHDS workgroup forum to review TEHDAS2 guidance and stakeholder insights, and submitted three comments to the second wave of TEHDAS2 Public Consultations, on draft guidelines addressing data minimization, opt-out implementation, and notification of significant findings.
September 2025: Hosted two EHDS workgroup forums, first featuring Elina Drakvik (Sitra), presenting EHDS rules for secondary data use, Finland’s implementation efforts, and ongoing work of the TEHDAS2 draft guidelines. The second forum, members prioritized and strategized on the TEHDAS2 draft guidelines for comment submission.
July 2025:Held the EHDS Workgroup forum, where Aneta Tyszkiewicz, Associate Director, Digital & Data for the Science Policy and Regulatory Affairs team at EFPIA, provided an overview of the EHDS regulation and EFPIA’s active engagement in this area.
May 2025:The EHDS Workgroup Forum convened industry members to discuss the practical implications of EHDS for industry and research organizations and how to transform uncertainty into actionable, pragmatic next steps.
March 2025:Launch of Workgroup Webinar Series “From Policy to Practice: Implementing the EHDS for Responsible Data Sharing.” The forum convened experts from industry, legal, and regulatory backgrounds to discuss the European Health Data Space (EHDS) and its implications for the sharing of clinical trial data.
project Leadership & sTAFF
Barbara E. Bierer, MD, Faculty Director, MRCT Center
Liz Graham, MPH, MS, Senior Clinical Research Data Manager, Vivli
Lisa Koppelman, MSW, LICSW, MPH,Team & Program Director, MRCT Center
Rebecca Li, PhD,CEO Vivli
Alex Senra, Executive Assistant, Vivli
Olchey Tchavyntchak,Research Assistant, MRCT Center
Comments provided to: TEHDAS2, coordinated by the Finnish Innovation Fund SITRA
Description:The MRCT Center and Vivli co-host an ongoing, invite-only forum focused on the European Health Data Space (EHDS), bringing together stakeholders to track developments and coordinate responses. The MRCT Center submitted responses to three TEHDAS2 guidance documents offered for public consultation to Health Data Access Bodies:
This meeting was presented to the Bioethics Collaborative. The Bioethics Collaborative is a forum to propose, share, and discuss ethical challenges in multi-national clinical trials. Meetings convene individuals from academia, industry, patient/participant groups, ethics committees, government, and others.
Abstract: Since funding and other resources for clinical research are limited, decisions must be made about which research projects to pursue, which not to pursue, and how to prioritize among the studies that are chosen. The principle of “unmet medical need” is often acknowledged as a guiding consideration in this context, and there have been calls for community input into prioritization and the choice of the study question. Further, addressing unmet medical needs, particularly in the context of the global burden of disease, is important for public health but may not, and likely will not, maximize market opportunity or financial profits – a dynamic that is particularly salient for private industry sponsors. Should prioritization then rest solely or principally with the funder? How should such entities balance economic obligations toward shareholders with the public good?
One salient principle of distributive justice is “prioritarianism,” the idea that research that stands to benefit the worse-off or those who are already underprivileged should be given priority over research that stands to benefit people in better situations: the well-being of the most disadvantaged is prioritized. Even prior to this, however, questions arise over how to understand the expected goods of research, who the beneficiaries might be, and how the well-being of different possible beneficiary groups should be measured. Further downstream, issues arise over who should engage in prioritization decisions, and in particular, whether the research community should rely solely on high-level, centralized prioritization mechanisms (e.g., industry sponsors, NIH, non-profit funders), or whether individual institutions, local communities, and/or patients and their allies might have some role to play in ensuring that studies are appropriately prioritized at a local level. How should these various voices be heard, should they be represented, and how can—or should—balance be achieved, and if so, what processes should be considered? The March Bioethics Collaborative will seek to address these and other issues in connection with the ethics of research priority-setting.
