UR CTSI Joins Effort to Expand Experimental Treatment Access to Desperately Ill Patients

Gravely ill patients who have exhausted conventional treatment options will now have a better chance to try cutting-edge treatments thanks to a $4.8 million national effort led by the University of Michigan. The University of Rochester Clinical and Translational Science Institute (UR CTSI) is one of four institutions involved in the effort, which aims to help more hospitals offer experimental options to their patients via the U.S. Food and Drug Administration’s Expanded Access process.

Patients at some major medical centers already receive experimental therapies through the Expanded Access – or “compassionate use” – process, but in many cases, patients who might benefit are not connected to the potentially new treatments. The four partner institutions in the Transforming Expanded Access to Maximize Support and Study (TEAMSS) consortium will reach out to hospitals to help clinicians understand how they can bring patients forward for potential Expanded Access treatment after they’ve exhausted other options and suitable clinical trials are not available.  

Expanded Access, which is more regulated than federal “right to try” legislation and has a decades-long track record, allows medical teams and hospitals to work with the FDA and pharmaceutical, biotech and medical device companies to provide ethical, regulated and rapid access to certain products, before the full FDA approval process is complete.

“The beauty of this project is that it will reliably harness all of the data generated during Expanded Access use,” said Joan Adamo, Ph.D., director of the Office of Regulatory Support at the UR CTSI, who will lead the TEAMSS effort at the University of Rochester. “That information can now be consistently used to further product development and approval, as well as maintain patient safety.”

As a test case, the consortium has partnered with HbO2 Therapeutics to provide its oxygen-carrying drug, Hemopure, to patients via Expanded Access. Michigan Medicine and Duke University’s medical center already offer the product under an Expanded Access cohort protocol that allows rapid treatment of critically ill patients without seeking FDA approval each time. Now the consortium will expand that protocol to include patients at the University of Rochester and University of Texas Southwestern Medical Center.

The consortium aims to build a national framework for more efficient, consistent and widespread use of Expanded Access and to develop best practices for collecting data from Expanded Access that can later be used in conventional FDA approval processes.  Ultimately, their goal is to help more hospitals offer patients one more chance.

Learn more about the TEAMSS project in Michigan Medicine News.

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The TEAMSS project is supported by program award 1U01TR002488-01 from the National Center for Advancing Translational Sciences of the National Institutes of Health.

Michael Hazard | 7/27/2018