When compared to chemotherapy, a drug targeting oncogenic RAS mutations extended overall and progression-free survival for patients with metastatic pancreatic cancer, according to a study published May 31 in The New England Journal of Medicine.
The third-leading cause of cancer death in the U.S., researchers have spent decades attempting to improve pancreatic cancer outcomes. Most recently, healthcare organizations have turned to AI and robotics to improve the detection, prediction and treatment. Now a single pill may completely transform how health systems provide pancreatic cancer care.
“For a disease that has long been marked by limited treatment options and often devastating outcomes, seeing a therapy double overall survival and progression-free survival and triple the response rate compared with standard-of-care chemotherapy […] is unprecedented,” Anna Berkenblit, MD, chief scientific and medical officer of the Pancreatic Cancer Action Network, said in a June 1 statement.
The study results were met with a standing ovation when presented at the American Society of Clinical Oncology’s annual meeting.
“I’ve gone to this meeting for 25 years and it is rare to see the plenary session receive a standing ovation; it’s very moving,” William Oh, MD, director of precision medicine at New Haven, Conn.-based Yale Cancer Center and Smilow Cancer Hospital and service line medical director of the Smilow Cancer Hospital at Greenwich (Conn.) Hospital, told Becker’s. “All oncologists know how disappointing the treatments for pancreas cancer have been. That spontaneous standing ovation really summarizes just how difficult it has been to treat.”
The drug, called daraxonrasib and manufactured by Revolution Medicines, has yet to receive FDA approval, though the agency did grant expanded access to the drug through a May 1 “safe to proceed” letter.
Excitement around daraxonrasib has created a swell of patient demand, which health system leaders are now preparing to meet. Without FDA approval, that process requires organizations to join an expanded access program with the drug manufacturer, Daniel King, MD, PhD, told Becker’s.
A gastrointestinal medical oncologist, Dr. King is director of research and development for the Center for Genomic Medicine at New Hyde Park, N.Y.-based Northwell Health Cancer Institute.
“Right now healthcare systems are wrestling with requests from not only our own patients, but from patients all around, even overseas. They have no options left and they’re looking for this as a salvage,” he said. “Many of them come with sort of desperate emails asking to access the drug. We have to say simply, ‘The drug is available through this EAP and we have to apply for each individual patient.’ It’s a process.”
Beyond patient approval, the current drug distribution pathway also takes a nonstandard route. Though daraxonrasib comes in an easy-to-use pill form, the drug manufacturer’s EAP will use a specific specialty pharmacy for drug distribution that is not part of a healthcare system, Dr. King said. Additionally, health systems will not receive money to help distribute the drug or collect safety data.
While health system leaders weigh these considerations as they decide whether to participate in the EAP, Dr. King foresees downstream benefits alongside potential pressures in the near future.
“Most patients get treated in communities that are not part of academic sites. Those health system leaders may have less familiarity with navigating an EAP,” he said. “What that ultimately may translate to is that for the majority of healthcare leaders in America, their patient populations will have less access to this drug, which may put more pressure on academic healthcare centers, who are now going to get more patients from the community.”
“Something that’s favorable is that it’s a pill and distributed more easily. One constraint for healthcare administrators is that their infusion rooms are very busy. It is conceivable that, because [this drug] is an oral therapy, infusion chairs will no longer be needed for these patients. It may actually let up a little bit of demand for pancreas cancer patients who normally would have been in infusion care for 46 hours. Now these patients go to the lab, take the pill and go home. That’s something to keep in mind if you’re running an infusion center, more infusion room space may help you offset some of the [initial] cost.”
Organizational and operational pressures aside, the drug is proven to be “a true game changer,” according to Dr. Oh.
“Part of what is really exciting about this drug is the mechanism by which it works. RAS is a cancer gene that is also important in other cancers and was considered untargetable or untreatable for decades,” he said. “[The study results suggest] mechanistically that this will work in lung cancer and other cancers that are driven by RAS.
“It’s not curing these patients; what makes oncologists excited is that it’s the first step. It’s not enough to melt away the cancer, but in oncology, incremental benefits are — especially for very difficult-to-treat cancers like pancreas cancer — a big deal.”
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