Public Comment
Submitted on: February 7, 2024
Submitted to: U.S. Department of Justice, Civil Rights Division
Focus Area: Clinical Trials & Research
Gene Therapies: Probing the Ethics
Meeting Summary
Presented on: December 14, 2023
Presented at: Cambridge, MA and online
The MRCT Center recently initiated a project on cell and gene therapies (CGTs), led by Carolyn Riley Chapman, PhD MS, Lead Investigator in the Division of Global Health Equity at Brigham and Women’s Hospital and Member of the Faculty at Harvard Medical School. In collaboration with diverse stakeholders, the project will identify and characterize the unique ethical, regulatory, and logistical challenges related to CGT research and development; we will then develop potential solutions or approaches to help address those issues. This meeting, held in conjunction with the Bioethics Collaborative, served, in part, as the project launch.
While the MRCT Center Bioethics Collaborative meetings are usually open only to invited guests and individuals from the organizations that sponsor the forum, this session was presented in conjunction with the 2023 MRCT Center Annual Meeting and was open to the public.
Related Resources
Cell and Gene Therapies
Cell and Gene Therapies
There is justified excitement about the emergence and potential of cell and gene therapies (CGT): a number of CGTs have recently received market approval and hundreds more are in preclinical and/or clinical development. As a new therapeutic class, there is still much to learn about the benefits and risks of CGT, necessitating complex decision-making and long-term follow-up in many trials. Many CGTs also have complex manufacturing and/or supply chain challenges. For these reasons and because of the need for specialized clinical expertise, trial sites are often geographically limited. The CGT project aims first to ascertain and characterize the ethical, regulatory, and logistical issues that are arising in the specific context of CGT research and development. We will then work to address and mitigate these challenges through a collaborative, multi-stakeholder approach.
OBJECTIVES
- Identify and characterize ethical, regulatory, and logistical challenges arising in the context of global research and development of cell and gene therapies.
- Employ a multi-stakeholder approach to co-develop actionable and practical mechanisms for addressing these challenges to support efficient, safe, and respectful clinical development of CGT products.
KeY MILESTONES
- June 2025: Held webinar: “Patient-Centered Long-Term Follow-Up for Gene Therapies”
- June 2025: Carolyn Chapman spoke at the June 2025 Bridging the Gap webinar, “Ethical Issues in CGT Trials: Benefits, Risks, Long Term Follow-Up and Beyond,” sponsored by Azenta Life Sciences and the Emily Whitehead Foundation
- May 2025: Published “Patient-centered long-term follow-up for gene therapies aligns with ethics and science” in Molecular Therapy
- May 2025: Two poster presentations at ASGCT meeting in New Orleans, Louisiana
- May 2025: Held webinar: “Innovative Approaches for Gene Therapy Long-Term Follow-Up”
- December 2024: Carolyn Chapman spoke at the Institute for Advanced Clinical Trials for Children (I-ACT)’s webinar, “Science Applications and Ethics of Cell and Gene Therapy”
- December 2024: “What’s Up with Long-Term Follow-Up: Ethical, Regulatory, & Operational Challenges” webinar, co-hosted by the MRCT Center and NYU’s Pediatric Gene Therapy and Medical Ethics (PGTME) Working Group
- September 2024: Launch of the Long-Term Follow-Up Working Group and of the Strategic Advisory Committee
- May 2024: Poster presentation at ASGCT meeting in Baltimore, Maryland
- December 2023: MRCT Center Bioethics Collaborative, “Gene Therapies: Probing the Ethics”
project Leadership & sTAFF
- Carolyn Riley Chapman, PhD MS, MRCT Center. Lead Investigator, Brigham and Women’s Hospital and Member of the Faculty, Harvard Medical School.
- Barbara E. Bierer, MD, Faculty Director, MRCT Center.
- Sarah White, MPH, Executive Director, MRCT Center.
- Emina Berbic, Program Manager, MRCT Center
Project Resources
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Meeting Summary
February 14, 2024
Gene Therapies: Probing the Ethics ›
Promoting Global Clinical Research in Children – A Project Overview
We are delighted to announce the launch of an interactive webpage dedicated to our Promoting Global Clinical Research in Children project. With our workgroup activities now complete, we are focused on presenting our findings and actively engaging in international projects. We offer a comprehensive suite of resources tailored for stakeholders who are directly involved or have an interest in pediatric clinical research.
Please visit this interactive summary page or the Promoting Global Clinical Research in Children project page for tools, publications, and materials supporting pediatric clinical research worldwide.
Is It Time to Retire the Concept of Deidentification?
December 12, 2024 @ 9:00 am – 11:30 am
In the US, research with data only qualifies as human subjects research and triggers protections, such as ethics board review and informed consent, when those data are identifiable, defined as “information for which the identity of the subject is or may readily be ascertained by the investigator or associated with the information” (45 CFR 46.102(e)(5)). The process of deidentification of data involves removing explicit identifiers such that the data cannot be readily linked to an individual; deidentified data is no longer considered protected health information subject to the protections of the Privacy Rule, implemented in response to the Health Insurance Portability and Accountability Act of 1996 (“HIPAA”). Deidentified data, therefore, can be used without consent, without review by an IRB or ethics committee, and without oversight by a Privacy Board.
In the approximately 30 years since the passage of HIPAA and even in the 5 years since the issuance of the Common Rule, technology has advanced. The concept of deidentification may no longer be relevant. Numerous studies have demonstrated that it is relatively easy to re-identify individuals from seemingly deidentified data sets, particularly when these data are triangulated with publicly available data sources. The increasing use and sophistication of Artificial Intelligence (AI) further exacerbates this situation, raising questions about whether any types of data are beyond re-identification or fail to meet the regulatory definition of “identifiable.” Other countries (e.g., EU/EEA, China, India, and others) and even US States (e.g., California) have adopted this position and, unlike the US, have substantiated data protections and personal rights to privacy through the law.
Eliminating the concept of deidentification[1] (a concept that the HHS Office of Human Research Protections promises to review periodically) would have profound effects on clinical research, including impact on IT infrastructure, data repositories, secondary use of data, consent paradigms, and scientific discovery. But given the current ease of reidentification, might it be time to retire the concept of deidentification in the service of privacy, autonomy, and respect for persons?
[1] The HHS Office of Human Research Protections promises to review the concept of identifiability periodically, although that has not yet occurred in the five years since the effective date of the 2019 Final Common Rule. If OHRP changes its definition, FDA is likely to reconsider its interpretation of identifiability.
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.
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Impact of Dobbs on Reproductive Health: Unintended Consequences for/on Research
September 10, 2024 @ 10:00 am – 12:30 pm
Topic: Impact of Dobbs on Reproductive Health: Unintended Consequences for/on Research
In 2022, the United States Supreme Court ruled, in Dobbs vs. Jackson Women’s Health Organization, against a federal right to abortion. The effects of this ruling continue to reverberate across the research ecosystem. Clinical trials often involve the administration of investigational therapies that pose reproductive risks. Because of this, participants are routinely asked to undertake measures that could pose privacy risks to them in states where elective termination of pregnancy is illegal. These measures include documentation of the results of pregnancy tests and reporting of incidental pregnancies and their outcome, among others. These risks may also extend to site staff in states where providers are prohibited from advising or facilitating elective termination of pregnancy. In addition, research on various aspects of pregnancy is itself at risk of being stifled for similar reasons. What should the research community’s response be to this situation? Is there an obligation to educate participants about these risks as part of the informed consent process? Who should bear this responsibility? What mechanisms are available to help shield participants and other stakeholders from undue risk while permitting the collection of important pregnancy data? To what extent, if any, do Certificates of Confidentiality provide sufficient legal protections? Will this ruling impact innovation in reproductive health research in the US more generally? At the next Bioethics Collaborative, we will focus on these and other questions related to the ongoing effects of the Dobbs rulingon clinical research.
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.
Reciprocity in Research: Does Justice Support Community Investment?
June 18, 2024 @ 9:30 am – 12:00 pm
Topic: Reciprocity in Research: Does Justice Support Community Investment?
Abstract: It is generally agreed that sponsors and other entities undertaking research in limited-resource settings incur certain duties of reciprocity. These obligations ensure that host communities are treated fairly and not exploited, given the burdens and risks of research undertaken by community members. However, the expectations, content, and limitations of these obligations are not defined. The next Bioethics Collaborative will be devoted to understanding and assessing different approaches to satisfying the ethical contorts of reciprocity for research undertaken in these settings.
Click here to view the Meeting Summary.
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.
Comments on the Public Health Service Policies on Research Misconduct Notice of Proposed Rulemaking
Public Comments
Submitted on: January 4, 2024
Comments provided to: The Office of Research Integrity
Biannual Global Meeting of The Joint Task Force For Clinical Trial Competency November 2023
Proceedings and Presentations
Proceedings from: Joint Task Force for Clinical Trial Competency Biannual Global Meeting on November 14, 2023
Held at: Virtual Meeting
Conference Slides
Comment on the NPRM Request of the Office for Civil Rights (OCR) re: Discrimination on the Basis of Disability in Health and Human Services Programs or Activities
Public Comment
Submitted on: November 11, 2023
Comments provided to: Office of Civil Rights, U.S. Dept. of Health and Human Services
The MRCT Center submitted a response to the Notice for Proposed Rulemaking (NPRM) request of the Office for Civil Rights (OCR) for comments on the draft, “Discrimination on the Basis of Disability in Health and Human Service Programs or Activities. The MRCT Center applauds OCR’s considerations of the rights of people with disabilities and OCR’s effort to clarify and strengthen Section 504 of the Rehabilitation Act of 1973. The MRCT endorsed several key areas in the proposed rule Center presented some suggestions for further clarification.