Human challenge studies are a type of research study in which participants are intentionally exposed to a pathogen. These study designs have a long and complicated history, figuring in notorious cases of research abuse (Willowbrook, Guatemala STI experiments) but also as important components in treatment advances for conditions that tend to disproportionately impact the world’s worst-off, such as dengue and malaria. The potential for human challenge studies to be leveraged ethically in relation to Sars-CoV-2 has been a matter of recent and significant debate in the bioethics community.
Governments, companies, academic research institutions, and the public are increasingly aware of the value of data, prompting a reevaluation of who “owns,” controls, and benefits from the use of an individual’s data. Discussion at the Bioethics Collaborative focused on models of data ownership and stewardship in research. The meeting scope included data collected during clinical research and health data from the medical record that are used for research.
The roles of sex, gender, and sexual orientation in clinical research are important but underexplored. Discussion at the Bioethics Collaborative focused on two types of ethical issues related to sex, gender, and sexual orientation in clinical research. First, while sexual orientation and gender identity (SOGI) are social constructs, disease risk, manifestation, and/or response to treatment may vary by SOGI characteristics.
It is important to understand that any variation in disease risk, manifestation, or response, if it exists, is likely related to other underlying differences (e.g., hormonal status). Nevertheless, collecting and analyzing data on sex, gender, and sexual orientation may point to differences that need to be understood biologically to provide safe and effective interventions across diverse populations. Second, research should be respectful and inclusive of individuals of diverse sexes, genders, and sexual orientations. The principles of respect for persons and justice must be considered in addition to that of biologic relevance.
In November 2017, the MRCT Center published the Post-Trial Responsibilities: Continued Access to Investigational Medicines Guidance Document and Toolkit. This framework outlined a case-based, principled, stakeholder approach to evaluate and guide the ethical responsibilities related to providing continued access to an investigational medicine at the conclusion of a patient’s participation in a clinical trial.
Over the past 5 years, sponsors and investigators have utilized the MRCT Center guidance to create their own internal PTR policies. While the ethical principles and main consensus points remain valid, the application of the ethical principles and implementation of consensus points continue to challenge clinical research stakeholders and study participants. It is therefore timely to revisit and update the framework. This meeting served as a launch for revisiting the MRCT Center’s 2017 Guidance Document. Starting in January 2023, the Center will be convening a monthly task force to (1) define the current issues associated with continued access to investigational drugs and maintenance of implantable devices after trial completion and (2) identify practical and actionable resources and tools needed by stakeholders.
Some state privacy laws apply to specific types of data that might be used in research. Other state privacy laws, sometimes referred to as omnibus privacy laws, regulate the collection and use of personal data throughout the economy. Meeting attendees reviewed and discussed both types of laws in several states, including their applicability to organizations involved in research, exemptions for research activities, and overall impact on research.
Topics: 1) Legal and Regulatory Challenges with Diagnostic Tests in the United States (US), including Laboratory Developed Tests (LDTs), and 2) The Impact of Emerging US State Privacy Laws on Research
There has been an increase in sustained interactions between sponsors of clinical research or their designees and potential and enrolled study participants and their families. Sponsor interactions with study participants and their families raise certain legal and regulatory concerns when sponsors perform the functions of investigators, such as taking medical histories or securing records from third-party health care providers, or when interactions contribute to inaccurate participant perceptions regarding who is responsible for their participation in a study, among other things.
Topics: Part I: Direct Interactions Between Sponsors and Research Participants: A Legal Perspective; Part II: Rethinking the Workforce for Decentralized Clinical Trials: Who is Engaged and Regulatory Requirements
We live in a destabilizing world, where natural disasters, wars, pandemics, and other disruptive events are becoming more common. In clinical research, such disruption is detrimental to research participants and to the advances in scientific knowledge that will benefit future patients. Emergency responses, however, can be fragmented and inefficient. The COVID-19 pandemic has impressed this point on the research community, raising awareness of the need to plan ahead and adapt in response to disaster. To date, most of the attention has focused on actual responses to the pandemic – primarily setting up new, pandemic-focused trials, or calling for changes to clinical trial infrastructure in anticipation of the next pandemic. However, other types of emergencies are often left out of these discussions.
At this meeting of the MRCT Center Bioethics Collaborative, we hope to explore sponsors’ and investigators’ ethical obligations to plan for emergencies in advance. What obligations do they have to participants? How much preparation is necessary? How much is realistic? What obligations do research teams on the ground in disruptive and potentially dangerous situations have to research participants and to maintaining research protocols? Answering these questions and more will allow the clinical research community to develop the guidelines and infrastructure necessary to prepare for future emergency situations.
The MRCT Center is pleased to announce our upcoming webinar, “Promoting Global Clinical Research in Children: Informing the Future,” the conclusion of the five-part virtual conference series on Advancing International Pediatric Clinical Research.
This event will showcase select resources and materials from the pediatric clinical research initiative, including information from previous webinars.
Topic: Gene Therapies: Probing the Ethics (open to all)
There is understandable excitement about the potential for cellular products and gene therapies to transform the therapeutic landscape for many medical conditions. Although a number of cell and gene therapy therapies have already been approved and are available on the market, hundreds more are in various stages of research and development. However, this new class of therapeutics raises distinctive ethical issues that deserve careful consideration. Cell and gene therapies may have a transformative impact on research participants, manifesting not only in extended benefit but also in the need for additional safety monitoring far beyond what is typical in clinical research. Further, individuals can generally participate in only one of several clinical trials investigating possible treatments for the same condition and may need to forego alternative treatments that are developed in the future. Finally, there are issues of access to these experimental therapies. These features of cell and gene therapies can raise the stakes, making questions about how to apply traditional bioethical principles of autonomy, beneficence, non-maleficence, and justice more challenging. How can researchers and developers of cell and gene therapies design trials with due consideration for the distinct ethical issues raised by these therapies? Given the significance of the choice to receive cell and gene therapies and how this may impact and/or limit future options, what are best practices for the informed consent process? What key information should be disclosed to potential participants and caregivers and what measures should be taken to ensure comprehension? How does the acuity, instability, and chronicity of the disease impact these decisions? In this session, we will consider these ethical issues and others, assessing them in light of the need for scientifically robust research on cell and gene therapies while striving to do justice to research participants, patients, and their families.
This meeting is part of the MRCT Center’s 2023 Annual Meeting in Boston on December 13-14 and is open to all registrants at no cost. Guests are welcome to attend the entire program or individual sessions. While in-person attendance is encouraged, virtual participation is welcome.
