Indiana CTSI assists team delivering breakthrough pancreatic cancer therapy to patients prior to FDA approval
Collaborative work enabled patients to receive a breakthrough treatment early, accelerating access to innovative cancer care for Hoosiers.
A remarkable team effort across the IU Simon Comprehensive Cancer Center, IU Health, and research and regulatory partners at the Indiana CTSI helped bring a promising new treatment option to Indiana patients with metastatic pancreatic cancer months before it became commercially available.
In May, the FDA authorized Expanded Access, often referred to as compassionate use, for daraxonrasib, an investigational targeted therapy for metastatic pancreatic cancer. Expanded Access provides a pathway for patients with serious or life-threatening conditions to receive promising investigational therapies outside of a clinical trial when no comparable or satisfactory treatment options are available. For patients who have exhausted standard treatments, these programs can offer access to potentially life-extending care while therapies are still under review.
Recognizing the potential impact for patients, multidisciplinary teams moved quickly to establish an Expanded Access protocol across Indiana. The Indiana CTSI assisted with regulatory navigation and coordination, helping facilitate the expanded access pathway and supporting the infrastructure to implement Expanded Access at IU Simon Comprehensive Cancer Center.
The initiative was led by:
- Anita Turk, MD | Principal Investigator, IU Melvin and Bren Simon Comprehensive Cancer Center
- Michael G. House, MD, FACS | Director, IU Melvin and Bren Simon Comprehensive Cancer Center Pancreatic Cancer Program
- Christine Sego Caldwell, JD, MA, CIP | IU Associate Director, Regulatory Knowledge and Support, Indiana CTSI
- Mona Geinosky, RN, MSN, CCRP | IU Health Statewide Director
- Chris Feagans | IU Health Project Manager
These leaders worked alongside numerous clinical research, contracting, regulatory, operational and support team members across IU Health and the IU Simon Comprehensive Cancer Center to bring this innovative therapy to patients in record time.
In early June, Turk initiated efforts to bring the treatment to Indiana patients. Within days, the team engaged with program partners and began securing the approvals, agreements and regulatory reviews necessary to launch the Expanded Access program. By July, approvals were in place for the IU Simon Comprehensive Cancer Center and additional IU Health sites, including Ball and Bloomington, creating a framework to expand access as broadly as possible across the state.
Those efforts quickly translated into patient care. The first patient was consented on August 5 and began treatment less than two weeks later.. Ultimately, eleven patients at the IU Simon Comprehensive Cancer Center received daraxonrasib through the Expanded Access program before the FDA formally approved the therapy and made it commercially available on August 26.
“This achievement reflects the dedication and expertise of teams from across IU Health and the IU Simon Comprehensive Cancer Center,” said Turk. “The speed and coordination required to launch this program would not have been possible without tremendous collaboration from everyone involved.”
The statewide infrastructure established through this effort ensured access was available beyond Indianapolis and demonstrated a commitment to bringing innovative cancer therapies to Hoosiers regardless of geography.
This accomplishment highlights the power of collaboration across clinical care, research, and regulatory teams. Most importantly, it provided patients facing a difficult diagnosis with earlier access to a potentially life-extending therapy, reinforcing IU Health’s commitment to advancing innovative treatments and accelerating hope for patients across Indiana.
