The authors press for follow‑up models that reduce patient and caregiver burden, improve retention, and leverage decentralized methods and real‑world evidence. Achieving this vision will require sustained collaboration across disciplines and meaningful engagement with patient communities.
Chapman et al., Patient-centered long-term follow-up for gene therapies aligns with ethics and science, Molecular Therapy (2025), https://doi.org/10.1016/j.ymthe.2025.04.040
Barbara Bierer and Willyanne DeCormier Plosky co-authored an article, led by David Gerber (UT Southwestern), in the Journal of Clinical Oncology titled, “Financial Toxicity in Cancer Clinical Trials: An Issue in Need of Clarity and Solutions.” This work was spearheaded by the Equitable Access to Clinical Trials (EACT) Project, hosted by Lungevity, of which the MRCT Center is one participating organization. The article illustrates some of the non-medical costs that cancer trial participants often pay out-of-pocket, such as an average of $600 US for travel, which are an additional stressor during a difficult time for participants and their families. The authors then detail financial support approaches to address these costs, and special considerations for sponsors, contract research organizations, and sites. Finally, the article highlights recently introduced federal legislation that could significantly ameliorate some of the barriers for sponsors trying to implement financial support approaches and for participants trying to access them.
Published in:The Journal of Empirical Research on Human Research Ethics
Barbara Bierer and Willyanne DeCormier Plosky co-authored an article, led by Emily Nguyen (University of Virginia) and David Resnik (NIEHS/NIH), in the Journal of Empirical Research on Human Research Ethics titled, “An Analysis of Institutional Review Board Policies for Enrollment of Adults with Impaired or Uncertain Decision-Making Capacity.” Some people (at some times) may have uncertain or impaired decision-making capacity, due to medication side-effects, cognitive or developmental disabilities, psychiatric disorders, dementia, physical or emotional trauma, or stress. Unless it is determined that they lack capacity and require a legally authorized representative, people with uncertain or impaired decision-making have the right to decide for themselves, with assistance such as supported decision-making (if desired), whether they would like to participate in clinical trials. However, they must first be offered an equal opportunity to participate. We explore in this paper how this equal opportunity, legally supported by Section 504 of the Rehabilitation Act, may be affected by Institutional Review Board (IRB) policies that are oriented towards exclusion rather than inclusion.
A common misconception is that plain language is only needed in select situations. In reality, applying health literacy best practices—such as using clear, thoughtfully designed language—benefits everyone, including professionals working in highly technical fields like data science.
In this on-demand webinar, originally presented on May 8, 2025, leaders from the Society for Clinical Data Management (SCDM), the Clinical Data Interchange Standards Consortium (CDISC), and the MRCT Center explore the essential role of plain language tools, such as the Clinical Research Glossary, in advancing understanding and engagement. They share how their organizations are fostering a culture of health literacy that supports both industry professionals and the broader research community, including patients, participants, and caregivers.
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.
Join us on June 24 from 12 – 1 PM ET to learn about the MRCT Center’s Clinical Research Glossary, a collaboratively developed plain language resource designed to enhance understanding of clinical research terminology. Discover how you can participate in the annual Public Review—an essential step toward making these terms a CDISC global standard. Your involvement ensures the glossary remains accurate, inclusive, and effective for informed decision-making in clinical research.
Key Topics:
Why your voice matters in Public Review
How to easily participate and submit feedback
The role of Public Review in shaping global clinical research standards
The terms and definitions going through Public Review
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 involvedwith each disease-specific subgroup to facilitate the next steps.
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, so it is critical to consider patient needs and preferences in the design and conduct of LTFU for GTs.
Three panelists will offer different perspectives on how we can put patients at the center of LTFU for GTs:
George Eastwood serves as Executive Director for the Emily Whitehead Foundation (EWF). The EWF funds groundbreaking research for pediatric cancer patients while simultaneously advocating for increased access to innovative therapies and supporting patients on their treatment journey. Mr. Eastwood will provide an overview of the Foundation’s work on addressing concerns about patient loss to follow-up in GT LTFU studies.
Dr. Jessica Scott is CEO of Legacy Health Strategies. As a patient engagement leader, she works to drive the culture change of increasing patient perspectives in the health care industry, which in turn will propel mutually beneficial outcomes for organizations and the patients they serve. She will share perspectives on how patient-centric approaches can be applied to GT LTFU.
Tesha Samuels has journeyed from a Sickle Cell patient to a beacon of hope, advocacy, and activism. Her story is a testament to the power of patient voices in shaping better, more compassionate healthcare systems. Her nonprofit, Journey to ExSCellence, is dedicated to empowering individuals affected by Sickle Cell Disease. Ms. Samuels will share perspectives on GT LTFU as both a patient herself and an advocate for others.
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.
