Tisagenlecleucel (Kymriah), Novartis' newly approved CAR T-cell therapy, will be put on the market with a price of $475,000 for a single infusion, an amount that is within the range anticipated by oncologists and that Novartis characterized as well below a price level that could be justified on cost.
“While both external and Novartis’ quantitative assessments of these values indicate that a cost-effective price could be $600,000 to $750,000, we recognize the importance of this paradigm-shifting therapy and are setting the price at $475,000 for this one-time treatment,” Dana Cooper, a spokesman for Novartis, said in an interview with OncLive.
The FDA approved tisagenlecleucel for pediatric and young adult patients with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. The approval of the immunocellular therapy follows the advice of the FDA’s Oncologic Drugs Advisory Committee, which voted 10-0 in July to recommend approval of tisagenlecleucel for pediatric ALL.
The approval of tisagenlecleucel was based on phase II results from the single-arm, international ELIANA trial of 63 patients who received a single dose of tisagenlecleucel. The overall remission rate was 82.5% (95% CI, 70.9-91.0) in treated subjects. Forty patients (63%) had a complete remission (CR) and 12 (19%) had a CR with incomplete hematologic recovery.
The $475,000 price for a single infusion of tisagenlecleucel does not include pre-infusion treatment cost, drug administration and hospitalization costs, or costs associated with adverse events and follow-up care. When those costs are added, the average total cost for 1 year of tisagenlecleucel is estimated to be $547,000, a figure that is based on experience from tisagenlecleucel clinical trials, Cooper said.
Prior to the FDA approval, outside estimates for what Novartis’ CAR T-cell therapy might cost ranged as high as $700,000. Cooper noted that the price Novartis has set for tisagenlecleucel is still below the cost of an allogeneic stem cell transplant.
“The only potentially curative option for these pediatric and young adult patients is allogeneic stem cell transplant, which costs $500,000 to $800,000 for the first year,” Cooper said. “Additionally, stem cell transplant economic burden in subsequent years is high. With the potential to extend survival and improve quality of life, [tisagenlecleucel] can deliver significant societal value.”
Cooper said fewer inpatient and outpatient visits for treatment and fewer hospitalizations are likely to result from treatment with CAR T-cell therapy, which he said would allow parents and other family members and caregivers “to resume a normal life, including the possibility to return to work.”
Although the price of the therapy is high by any standard, Cooper stated that Novartis is “committed to doing everything we can to ensure that children and young adults who can benefit from [tisagenlecleucel] have access to the therapy and we are working with payers to ensure they fully understand the value of [it] and provide coverage for patients. While payers are developing their coverage policies for [tisagenlecleucel], we will offer an access program in the United States for eligible uninsured or underinsured patients.”
Tisagenlecleucel is a genetically modified autologous T-cell immunotherapy. Each dose is a customized treatment created with a patient’s own T-cells, which are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein—a chimeric antigen receptor or CAR—that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.
Novartis estimates that the turnaround time for processing the cells is 22 days, and initially the FDA has approved one manufacturing site for conducting this work: Novartis’ Morris Plains, New Jersey, laboratory. Whereas the time involved in processing is considered critical because of the need for rapid treatment of patients, Novartis has a cryogenic process that enables it to freeze and store samples of patients’ blood cells for processing at more convenient times and earlier during the treatment cycle.
“[Tisagenlecleucel] is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease," Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “Not only does [tisagenlecleucel] provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”
The price may be high for CAR T-cell therapy, but it is important to weigh the costs against the potential benefits for patients, Gwen Nichols, MD, chief medical officer for the Leukemia & Lymphoma Society, said in an interview with OncLive. When children and young adults have their whole lives at stake, it’s easier to justify the expense of this new treatment.
Without CAR T-cell therapy, the costs of care, including a potential additional bone marrow transplant, are already “outrageously expensive,” Nichols said. “The real question is going to come when this therapy is poised to be expanded into other patients who are older, and providing the chance for 60 or 70 years of [additional] life is not what you’re talking about, and then we’re really going to have some tough value questions that we should be prepared to ask.”