Cancer treatment centers nationwide are working rapidly to provide patients access to a breakthrough experimental medication for pancreatic cancer through a special early access program while awaiting anticipated FDA approval.
Federal regulators approved the expanded access program on May 1, coming less than three weeks after Revolution Medicines announced that their once-daily medication, daraxonrasib, showed remarkable results by doubling patient survival times in clinical testing for advanced pancreatic cancer – one of the most fatal cancer types with extremely low five-year survival statistics.
Revolution Medicines had requested federal permission to distribute the treatment at no cost to patients with previously treated pancreatic cancer that had metastasized throughout the body.
“The public caught wind of the FDA announcement… which has triggered a deluge of patient requests,” said Dr. Daniel King, medical oncologist at the Zuckerberg Cancer Center of Northwell Health. “Cancer centers are all figuring out how to engage with our own institutions, opening up the protocols to provide access.”
Medical specialists indicate that obtaining approval and administering the expanded access program will be time-consuming and will demand cancer facilities to allocate significant resources beyond their standard procedures.
The medication became one of the initial treatments accepted by federal regulators last year under their new fast-track review system, and could receive approval within one to two months following submission of a complete application.
The Redwood City, California-based Revolution Medicines did not provide immediate response to requests for comment.
CEO Mark Goldsmith, during a conference call last week, refused to predict how many patients might pursue early access to the treatment and provided no timeline for complete FDA submission. “There’s a full-throttle effort to do it,” he stated.
Former Nebraska U.S. Senator Ben Sasse recently disclosed that he has Stage 4 pancreatic cancer and informed the CBS news program “60 Minutes” that he is taking the Revolution drug.
The medication targets a genetic alteration present in approximately 90% of pancreatic cancers and demonstrated in clinical testing the ability to extend median survival to 13.2 months versus 6.7 months for patients receiving chemotherapy.
“Doubling survival compared to best available chemotherapy is a big deal,” said Dr. Gulam Manji, co-director of the pancreas center at Columbia/New York-Presbyterian. “It is not a cure, but I think that this drug is a new breakthrough we can build on.”
During his decade-long career, Manji recalled requesting compassionate use of an experimental treatment for only one other patient. During a recent clinic day, the Columbia specialist reported that seven patients inquired about beginning treatment with daraxonrasib.
Providing them access involves much more complexity than prescribing an FDA-approved medication, according to him and other cancer experts.
“Patients are already aware of the press release and are already calling,” said Dr. Vincent Chung, pancreas cancer specialist at City of Hope. “The challenge now is how to proceed.”
The program demands that doctors submit individual patient requests to Revolution Medicines, Chung explained, and if the company determines they qualify as good candidates, all documentation must then be forwarded to the FDA. Hospital oversight committees will need to monitor the patients.
“Given the volume, I am not sure what will happen on the FDA side. I’m sure they don’t want to have 10,000 applications at once,” Chung said, noting that the agency might instead establish a broader enrollment system.
Manji indicated his understanding that cancer centers will not need to gather comprehensive patient data under the expanded access program, but must report severe side effects or other complications.
The FDA did not provide response to requests for comment. The expedited voucher program was promoted as a major accomplishment by Dr. Marty Makary, who stepped down as FDA Commissioner on Tuesday following weeks of disagreements with Trump administration officials.
Approximately 67,000 Americans will receive pancreatic cancer diagnoses this year, with 53,000 expected to die from the disease, according to the American Cancer Society.
“We are doing this as a service to our patients,” Chung said. “I’m hoping of course that the FDA is going to review the data and then there is an approval much sooner than is typical.”
