Tag: Bioethics Meeting

Topic: Ethics of Rare Disease Research

Abstract

Research in rare diseases presents several ethical challenges which have to date been under-explored. Decisions about whether to initiate rare disease research in the first place can be complicated by economic realities, given that the eventual market for rare disease therapies may be quite limited. How should companies and research funders generally balance economic and feasibility considerations with the needs of rare patient groups? Other challenges characterize the conduct of rare disease research. Some of these involve issues related to privacy and confidentiality. The smaller the patient population, the easier it may be to identify participants and individuals within clinical trial datasets, even when efforts are made to protect privacy. Others involve how to be fair toward rare disease patients eager to participate in research, which include determining where to run trials and provide fair access. Finally, determining the scope of post-trial obligations toward rare disease populations, and what is owed to rare disease patients who appear to benefit from therapies that lack efficacy in wider populations, can be complicated. How should sponsors and other research stakeholders think about these issues?      

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.

Location: Virtual

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.  

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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.

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?

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[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. 

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 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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