Ethical Framework Needed for Gene Therapy Clinical Trials

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An ASGCT poster focused on special ethical considerations of gene therapy human research and how to address them.

Carolyn Riley Chapman, PhD, MS, Lead Investigator, Division of Global Health Equity (DGHE), Multi-Regional Clinical Trials (MRCT) Center, Department of Medicine, Brigham and Women’s Hospital, and Harvard Medical School

Carolyn Riley Chapman, PhD, MS

Credit: Brigham and Women's Hospital

The growing prevalence of gene therapies in the investigational and clinical fields necessitate discussion and strategy to navigate the complex ethics of this mode of treatment.

A poster presented by Carolyn Riley Chapman, PhD, MS, Lead Investigator, Division of Global Health Equity (DGHE), Multi-Regional Clinical Trials (MRCT) Center, Department of Medicine, Brigham and Women’s Hospital, and Harvard Medical School, at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland, outlined some ethical considerations with gene therapies.

“There are some characteristics of gene therapy clinical trials that make it somewhat unique. And part of that is due to the types of diseases that they're often targeting, including rare diseases or diseases that don't have a lot of alternative options, pediatric diseases, so there's that aspect,” Chapman told CGTLive® during the ASGCT meeting. “But then there's also characteristics of gene therapies. Oftentimes, they're one dose with persistence and durability, which can preclude people from taking other types of therapies. So that can make it a higher stakes decision as well. And then because the special manufacturing that's required oftentimes is not available in certain sites, that can bring up ethical issues about equitable access even to drug trials.”

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Chapman’s poster outlined several ethical considerations for gene therapy trials that should be addressed when designing and conducting these trials. These considerations include community engagement; risk/benefit analysis; fair subject selection; informed consent; respect for prospective and enrolled participants; and upholding scientific validity and rigor, maximizing data quality and sharing.

The poster also emphasized the importance of considering the differing perspectives of different stakeholders involved with gene therapy trials, including patient and community perspectives, when designing clinical trials. Other contextual factors that may impact ethical design include the availability of alternative therapies, natural history data, and clinically meaningful endpoints; durability of the gene therapy product and the potential for genomic change; the size and geographic distribution of the target population; and the route of administration.

Chapman shared that the MRCT is conducting a systematic review of ethical, legal, and social issues in gene therapy clinical research and is planning a qualitative interview study to better understand different stakeholder perspectives. The MRCT plans to create empirically informed guidelines for gene therapy investigators to create and conduct ethical clinical trials.

“I've recently joined the MRCT Center at Brigham and Women's Hospital and Harvard and that is an organization that's housed within an academic institution. But it functions sort of like a think tank and a convener of different stakeholders in the clinical research sphere. And so, we try to use that experience as a convener to both better characterize challenges that that are being faced in different aspects of clinical research... and trying to figure out how we can leverage that position to contribute in this area," Chapman said. “One of the ideas is to help potentially create a decision-making framework for researchers, oversight, and patient advocates, to think about these special medical issues in the country in this context.”

REFERENCE
Chapman CR. Delivering on the Ethics of Gene Therapy Trials. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #925
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