FDA advisory committee unanimously agrees donor liver portable system is safe, effective
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The FDA’s Gastroenterology and Urology Devices Panel, Medical Devices Advisory Committee voted unanimously that a portable system for near-normothermic continuous perfusion of donor livers with perfusate was safe and effective.
The system will be prepared by hospital pharmacy and include compatible packed red blood cells.
The panel addressed three questions related to the premarket approval of the device Organ Care System Liver System (TransMedics). The entire 14-member panel voted that the device is safe, all voted that the device is effective and voted 12 to 1 with 1 abstaining that the benefits of the OCS Liver System outweigh the risks.
The meeting focused on the results of the PROTECT Trial, a prospective, multicenter, open-label trial of 300 transplant recipients from 20 U.S. transplant sites. Investigators randomly assigned patients to the OCS Liver System or standard cold, static storage(control). Enrollment closed on October 15, 2019. Follow-up at 30 days was completed on November 19, 2019, 6-month on March 28, 2020, and 12-month final follow-up on October 15, 2020. Researchers followed patients for a minimum 30 days post liver transplant and up to 24 months post-transplant. At 6 and 12 months, researchers collected data regarding graft survival, incidence of ischemic biliary complications, diagnosis method, liver graft-related serious adverse events at 6 months, liver graft related rehospitalization after initial discharge, primary reason/diagnosis for hospitalization and length of stay. The primary endpoints were safety and effectiveness of the OCS Liver System.
Safety, effectiveness
Despite caveats related to the PROTECT trial, all panel members said there was enough evidence to indicate the OCS Liver System was safe for its indicated use.
Panel member, Jason Connor, PhD, president and lead statistical scientist at ConfluenceStat LLC, said although he said yes to all three questions, the design of the trial made it difficult assess the evidence. He said it was a ‘solution in search of a problem’. He hopes the sponsor obtains more evidence to determine how to make the OCS more applicable for donor livers.
Members of the panel felt the sponsor offered strong evidence that indicated the device was effective.
“This is the beginning of the data not the end,” Richard Lange, MD, panel member, from Texas Tech University Health Sciences Center, said.
Panel member, Colleen M. Gallagher, PhD, executive director of The University of Texas, MD Anderson Cancer Center, voted yes for the first two questions and no to the third. She does not think the benefits were proven with the trial. She said a post approval trial may prove the benefits, more livers may be needed in a trial.
“I am hopeful that this technology and what follows it will address the difficult and challenging problems in the world of transplantation,” Mark Talamini, MD, MBA, panel member, from Northwell Health, said.
Post-approval studies
Panel member, David Asis, MD, from the Yales School of Medicine, said a post marketing study of marginal populations will be important for patients to get the most benefit from this technology.
If the FDA chooses to issue premarket approval for the OCS Liver System, the FDA advisory committee proposed the sponsor continue to follow patients in the PROTECT trial up to 2 years post-transplant and to continue to follow-up pre-market cohorts, though there may be limitations with the design.
In addition, the FDA panel recommends a new enrollment study to better understand safety and effectiveness of the technology on DCD donor organs, donor organ transplantability criteria and device malfunctions. They also suggest a long-term assessment of clinically meaningful outcomes.
“That would make for an interesting study to understand the benefits starting at the time of listing all the way to post transplant,” Jennifer Lai, MD, panel member, from the University of California, San Francisco, said.
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