Topic: Medical Need or Market Opportunity: Setting Research Priorities
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.
This meeting is open to sponsors of the Bioethics Collaborative. For more information about the Bioethics Collaborative and how to become a sponsor, click here.
Episode 2 of the Trials Beyond Borders podcast is now available on all streaming platforms. In this episode, Racquel Bruton, Associate Director and Clinical Trials Diversity Portfolio Lead at Biogen, and Willyanne DeCormier Plosky, Program Director at the MRCT Center, discuss how sponsor companies are enhancing representation in clinical trials, particularly outside the US. Racquel highlights Biogen’s longstanding commitment to reaching underrepresented populations and explores how the MRCT Center’s roadmap can help organizations develop effective diversity action plans. Tune in to learn practical insights on operationalizing diversity strategies.
Description: A recent Frontiers in Pediatrics article, co-authored by Barbara Bierer and Elisa Koppelman of the MRCT Center, emphasizes the importance of directly involving children and adolescents in pediatric research design and execution, highlighting the benefits of incorporating their perspectives alongside those of parents and guardians. The authors provide theoretical, ethical, and practical recommendations for systematically engaging young participants to enhance the relevance and effectiveness of clinical studies.
Description: In the Health Affairs article “Towards A National Action Plan To Improve Representation In Clinical Trials,” co-authored by the MRCT Center’s Barbara Bierer, the authors address the critical need for increased diversity in clinical research to enhance health outcomes for underrepresented populations. They propose a comprehensive national strategy that includes policy reforms, community engagement, and the integration of diverse perspectives in trial design and implementation.
Carolyn Chapman, MRCT Center Member of the Faculty and Lead Investigator, will moderate and co-sponsor a webinar, “What’s up with Long-Term Follow-Up: ethical, regulatory, operational challenges,” with the Pediatric Gene Therapy and Medical Ethics (PGTME) Working Group at the Division of Medical Ethics, NYU Grossman School of Medicine. The webinar is part of PGTME’s fifth annual Lunchtime Lecture Series.
Key Topics:
Why and How Long-Term Follow-up (LTFU) studies are conducted for Gene therapies
Ethical, Regulatory, and Operational Challenges of LTFU studies
Maximizing the value and minimizing the burden of LTFU studies
Published on: November 18, 2024 and updated on May 30, 2025
The “Exit Survey Inclusive of the LGBTQIA+ Participant Perspective” is one tool in the LGBTQIA+ Inclusion by Design in Clinical Research Toolkit and the third of three tools in the section of the Toolkit directed more toward participants. It is important for participants in clinical research activities to give feedback about their experiences. This feedback helps research teams and organizations better understand the participant’s experience of the research activity and learn where they can improve on efforts to empower research participants and the participants’ supporting families, friends, and communities. One way to gather feedback is through a survey, which may be given to participants periodically (e.g., once a month), and/or (as an exit survey) at the end of research activities. In this tool, we aim to show examples of the topics that participants may be asked about in a survey or interview or, if not asked, that the participant may wish to share with the research team in a patient portal, email, or other format.
Please note that the Exit Survey Inclusive of the LGBTQIA+ Participant Perspective has been comprehensively updated in the 2.0 version, particularly in questions 21c-f and in the footnotes for those questions and response options. We have also included a new appendix to this tool, which provides a summary of Key Resources and Recommendations for SOGI Data Collection. These resources and recommendations are drawn from documented best practice and are meant to support researchers designing surveys and research participants that are considering how they might give feedback on their participation experience.
Published on: November 18, 2024 and updated on May 30, 2025
The “Participant Questionnaire from the LGBTQIA+ Participant Perspective” is one tool in the LGBTQIA+ Inclusion by Design in Clinical Research Toolkit, and the second of three tools in the section of the Toolkit directed more toward participants. There are numerous questions that participants may want to ask as they move from thinking about possible trials and sites to starting the process of enrolling and participating in a trial. Some of these questions may be covered by informational materials given to participants during the informed consent process and study visits, and some may not be. This list is to help participants prepare so that they can get the answers that they need and feel comfortable before continuing with the trial. The Participant Questionnaire tool is divided into sections that include questions to ask the research team, questions you may want to ask family, friends, and others you trust, and questions to ask yourself. It includes questions that anyone might want to ask and adds questions that LGBTQIA+ people (and/or their accompanying friends and family) might also want to ask.
Published on: November 18, 2024 and updated on May 30, 2025
The “Site Feasibility Decision Tree from the LGBTQIA+ Participant Perspective” is one tool in the LGBTQIA+ Inclusion by Design in Clinical Research Toolkit, and the first of three tools in the section of the Toolkit directed more toward participants. This tool is meant to empower potential LGBTQIA+ participants to assess whether a site may be trustworthy and welcoming. This tool is structured in tiers, with checkpoints in between. The first tier is potential capacity, where we provide prompts, or “determination factors” based on what IS happening at a site (or in the area nearby) that LGBTQIA+ people can use in considering whether the site is potentially a good place to participate in a study. In the second tier we focus on “historical capacity,” or the things that the site HAS/HAD done to support the well-being of LGBTQIA+ people that may inspire confidence that the site is trustworthy.
Details: The Joint Task Force for Clinical Trial Competency (JTF), anchored at the MRCT Center, is dedicated to developing and disseminating standards and practices for the clinical research workforce. The JTF fosters a cohesive, global approach to ensuring that professionals have the necessary competencies to conduct clinical trials effectively and ethically.
The public is invited to attend virtual biannual meetings, which happen twice a year.
Key topics discussed at this meeting:
Duke University’s Workforce Engagement and Resilience Program
Community of Practice & JTF Competency Framework:
The Research Professionals Network Workshops
Developing Team Sciences Competencies for Clinical Research Professionals
Accreditation of Academic Programs in Clinical Research
Promotion of the JTF Core Competency Framework in Asia and Africa
Deploying the JTF Framework across the world: Translations and Applications
Details: The Joint Task Force for Clinical Trial Competency (JTF), anchored at the MRCT Center, is dedicated to developing and disseminating standards and practices for the clinical research workforce. The JTF fosters a cohesive, global approach to ensuring that professionals have the necessary competencies to conduct clinical trials effectively and ethically.
The public is invited to attend virtual biannual meetings, which happen twice a year.
Key topics discussed at this meeting:
Integrating JTF Framework into Takeda’s R&D’s Knowledge Development Academy (KDA)
Update on Translations
JTF Competency Survey in Low and Middle Income Countries: Initial Analysis of Results
Clinical Research Core Competencies: An Adaptation of the JTF Framework to Switzerland
