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College of Health Professor Awarded NIH Grant to Research Governance and Ethics of Gene-Based Therapies

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September 02, 2024

Michael Gusmano to analyze evidentiary standards for emerging genetic technologies for safety, effectiveness, and cost

Michael Gusmano, a professor in the College of Health’s department of community and population health, has been awarded funding from the National Institutes of Health’s (NIH) Ethical, Legal and Social Implications genetic and genomic research program to investigate the ethical and policy changes facing the gene therapy ecosystem. 

“Gene therapy edits a person's genes to treat or prevent disease,” said Gusmano. “Sometimes the therapies replace an existing gene. Other times the intervention may inactivate a gene that leads to disease. In other cases, the therapy involves introducing a new gene that can help to treat a disease.”

In 2017, the U.S. Food and Drug Administration (FDA) approved the first gene based therapy, and since then, hundreds of gene therapy clinical trials are in development or are currently testing the safety and effectiveness of additional gene therapies for a wide range of disorders.

For example, CAR-T Cell treatments use genetically modified T cells to treat certain blood cancers. “First, T cells are collected from the patient's blood and are genetically modified to produce chimeric antigen receptors (CARs) on their surface,” Gusmano explained. “CAR-Ts are special receptors that bind to antigens on the surface of cancer cells. After a few weeks, the CAR T cells are grown in a lab and infused back into the patient's bloodstream. When it works properly, the CAR-Ts latch onto the cancer cells and kill them.”

Gusmano and his team will be asking many different types of questions through interviews with payers, regulators, and physicians.

“We are interested to know what kind of evidence is being used to determine whether these new therapies are safe and effective,” he explained. “To what extent are they being approved on an accelerated pathway by the FDA? There’s a lot of pressure to get the drugs to the market sooner. And if so, are they requiring— and are companies conducting— post-marketing surveillance of those drugs?”

He continued, “What do physicians who treat the kinds of patients who may benefit from some of these new gene-based therapies think about them? Are they willing to use them? Do they think the evidence is strong enough?”

Access to the treatments, as well as their enormously high cost, are key issues under consideration.

A one-time infusion may cost several million dollars, which, in theory, may cure or offer substantial benefit to the patient. “Sometimes the rationale for the huge price,” Gusmano said, “is that the lifetime cost of treating that same patient in a less effective way would be greater. Therefore, this is cost saving.”

“How much should these things cost, and what’s fair? What’s unfair?” asked Gusmano. “What are the implications for private payers? What are the implications for public payers?”

Gusmano’s team will begin the three-year, mixed methods study later this summer or in the fall and will start by surveying physicians. They have put together a national advisory committee comprised of experts in gene-based therapy, as well as regulatory matters and policy, who will offer advice on data collection instruments, preliminary findings, and their recommendations. 

Data collection will take place in year 2, followed by data analysis and the production of articles, reports, and recommendations in year 3. The project will aim to understand the perspectives of physicians, insurers, and regulators from across the country.

“I think gene-based therapies are really going to be a major part of the future of the pharmaceutical industry,” Gusmano said, “and there’s a lot of open promise that they will represent highly effective treatments. I still have a lot of concerns about their safety and effectiveness and the evidentiary standards being used to evaluate them.”