Expect the unexpected
Payers need increased transparency and access to specialists, and must pay “more and more” attention to the drug-development pipeline, Saltzman said.
FDA’s priority reviews and other hastened approval mechanisms mean that payers have to “expect the unexpected,” Saltzman said, citing the previous afternoon’s surprise FDA approval of Kite/Gilead’s CAR-T therapy axicabtagene ciloleucel (KTE-C19) for non-Hodgkin lymphoma—a month earlier than expected, despite lingering concerns about cost and toxicity.
Investigational CAR-T (chimeric antigen receptor T-cell) immunotherapies are definitely on the Emerging Therapies Workgroup’s radar. CAR-T cells are the patient’s own cells, genetically engineered at a remote lab to recognize and attack tumor cells. Blood is taken from the patient, cryopreserved and shipped to the specialty lab for genetic reprogramming and amplification, then shipped back to the treating hospital for infusion into the patient.
“Vein-to-vein,” the process can take between 16 to 22 days, Lin said.
The expense of CAR-Ts’ highly-personalized manufacturing process—an anticipated $475,000 per treatment for Novartis’s tisagenlecleucel (Kymriah), a treatment for children and young adults with relapsed acute lymphoblastic leukemia (ALL)—has made headlines.
The Institute for Clinical and Economic Review (ICER) recommends that payers develop “internal sophistication in knowledge about gene therapies,” Lin said. “They recommend early dialogue with manufacturers of promising new gene therapies about making clinical studies as robust as possible—particularly if randomized, controlled trials will not be performed.”
Payers should also work with clinicians, patient groups, regulators, and manufacturers to develop robust patient registries, Lin added.
They should also explore and negotiate outcomes-based payment agreements, she said.