In February 2012, Kaiba Gionfriddo became the first infant to receive an experimental 3D-printed airway/tracheal splint that opened his airways and saved his life. Nineteen additional young patients have since been implanted with the device, which was developed by researchers at the University of Michigan and provided to the patients under the Food and Drug Administration’s (FDA’s) Expanded Access program.

All of the patients had tracheobronchomalacia, a rare condition characterized by weak airway walls that can collapse and make it difficult to breathe.

In the years since the successful implants, University of Michigan researchers have continued to refine the airway splints. The airway splint technology is just one of the numerous exciting therapeutic breakthroughs that can provide access to investigational drugs and devices to vulnerable patients through the expanded access process, but there has been a gap in robust infrastructure to support the process. Michigan has taken the lead in improving the expanded access process that made these devices and many other options available to desperately ill patients.

These efforts received a boost in July of last year, when a University of Michigan-led national consortium was awarded $4.8 million for the Transforming Expanded Access to Maximize Support and Study (TEAMSS) project. Funds were provided by the National Center for Advancing Translational Sciences of the National Institutes of Health.

Our TEAMSS partners are Duke University, the University of Rochester, and the University of Texas Southwestern Medical Center. Like the University of Michigan, Duke University and the University of Rochester are national Clinical and Translational Science Award (CTSA) Program hubs, committed to speeding the translation of research discovery into improved patient care.

Partnering to increase access and enhance care

The FDA Expanded Access program allows desperately ill patients to try investigational medicines and devices when all other treatment options have been exhausted. It is one of two federal pathways to investigational treatments outside clinical trials. The other pathway, the recently enacted federal Right to Try Act, applies only to patients seeking permission to try experimental medicines and biologics—not medical devices. Even more important, from the University of Michigan’s perspective, the FDA Expanded Access program provides better protections for patients. For this reason, our TEAMSS project focuses on the long-standing FDA pathway.

FDA approves around 99% of requests for expanded access, but, on average, fewer than 2,000 physicians apply each year—often because patients or their physicians are not aware of the expanded access option. A major TEAMSS goal is to build awareness and enhance care for the most vulnerable patients.

With TEAMSS, we are creating the foundation for an integrated, nationwide approach to expanded access. Our plan is to develop a network for cohort-based expanded access programs and disseminate best practices across the larger 55-member national CTSA consortium.

One of our first steps is to assess expanded access efforts at each of the four TEAMSS partners and share successful approaches. At the University of Michigan, for example, changes to the expanded access process and infrastructure since 2015 have resulted in significant year-over-year increases in patient access to investigational products. Last year, the university processed 92 expanded access cases enabling over 600 patients to gain access to investigational products via the FDA pathway. This year, we expect to process more than 100 requests and to see corresponding increases in patient access.

Through the TEAMSS project, we also will:

  • Reach out to community hospitals and physician offices to help providers understand how FDA Expanded Access can be another option for patients who have exhausted all other alternatives.
  • Work with industry partners to develop demonstration projects for treatments in oncology, infectious disease, acute and critical care, and other medical specialties. TEAMSS will be partnering with biotech and pharmaceutical companies to demonstrate the value of the network.
  • Create a database of information generated by FDA Expanded Access use and tap that information to ensure patient safety and advance product development, as appropriate.
  • Publish these practices and disseminate the information to the broader community so others benefit from the knowledge gained through the TEAMSS effort.

Ultimately, we expect to demonstrate that the FDA Expanded Access program has the potential to play an important role in advancing treatment and improving care—and that a network-based approach to expanded access can be instrumental in harnessing that potential.

Blog post contributor Kevin Weatherwax is managing director of the Michigan Institute for Clinical and Health Research at the University of Michigan Medical System (Michigan Medicine). He is co-principal investigator of the TEAMSS grant and co-chair of the Expanded Access Oversight Committee at Michigan Medicine.