When evaluating costs for medical technologies like CAR-T, one should focus on “overall value” rather than dollar figures, argued Stuart Goldberg, MD, of the John Theurer Cancer Center at Hackensack University Medical Center in New Jersey and chief scientific officer at Cota, a health care data and analytics company.

“In the case of the recent approval of CAR-T for pediatric leukemia, the therapy yields a high likelihood of cure,” Dr Goldberg said. “From a value standpoint, measures such as QALY [quality-adjusted life years] are favorable.

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“For CAR-T in pediatric leukemia, the value equation (even with a half-million-dollar initial charge) is favorable,” he added. “Yet as CAR-T technologies expand to lymphoma and myeloma, where the chance of cure is lessened and (due to the age of patients) the years of life gained may be low, the dollar charge for the CAR-T therapy will need to be adjusted.”

“Indication-based” pricing, where similar treatments are priced differently based on disease indication and outcomes, may become more common in the future, Dr Goldberg predicted. 

“This idea is still new but gaining traction,” he said.

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Meanwhile, Novartis has struck an “outcomes-based pricing” deal with the US Centers for Medicare and Medicaid Services (CMS) to charge patients for treatment with CTL019 only if they see a response within 1 month.1,2

Toxicity Concerns

Cytokine release syndrome triggered by activation/expansion of patients’ T cell populations and neurotoxicity are concerns for CAR-T therapies.4

“The persistence of anti-CD19 CAR-Ts in a subset of patients can result in ongoing tumor surveillance but also the off-tumor but on-target toxicity of prolonged B-cell aplasia,” noted Noelle Frey, MD, of the hospital of the University of Pennsylvania in Philadelphia, in a recent paper.4 “Durability of remissions with anti-CD19 directed CAR-Ts are variable across studies, driving efforts to minimize both CD19-positive and CD19-negative relapses.”

“CAR-T therapy is a new modality; hematologist-oncologists will need to familiarize themselves with the potential benefits and risks of the treatment,” said Dr Goldberg.

Initially, CTL019 will be limited to pediatric leukemia patients treated at specialized centers. That will allow time for clinicians to learn about CAR-T safety profiles and patient monitoring, Dr Goldberg said.

CAR-T administration requires “considerable expertise,” Dr Goldberg said, as reflected by the FDA’s requirement that a risk mitigation and evaluation strategy (REMS) program be established at centers administering CAR-T immunotherapies.

The REMS program includes certification of centers, training of physicians and staff on administering CAR-Ts and potential toxicities, and on-site availability of treatments for CAR-T cytokine release syndrome and neurologic toxicities.

That will limit availability to centers of excellence, at least for the time being, similar to the situation with bone marrow transplantation. Transplantation centers collaboratively maintain databases like the Center for International Blood and Marrow Transplant Research to track and improve patient outcomes, and similar efforts will be needed for CAR-T immunotherapy.

“It will be essential that the outcomes of patients treated with CAR-T be tracked and analyzed,” Dr Goldberg said. “Each center will perform only a few procedures per year and if each center’s data remain in isolation it will take years to fully evaluate effectiveness and toxicities. The lessons learned in the early days of bone marrow transplant can be instructive for CAR-T.”

While clinicians and patients keep an eye on new therapies’ prices and toxicities, Dr Goldberg urges a focus on treatments’ net values to patients and health care systems: “It is not the cost but rather the value that will be the focus in evaluating these new technologies.”


  1. Novartis receives first ever FDA approval for a CAR-T cell therapy, Kymriah ™ (CTL019), for children and young adults with B-cell ALL that is refractory or has relapsed at least twice [news release]. Basel: Novartis; August 30, 2017. https://www.novartis.com/news/media-releases/novartis-receives-first-ever-fda-approval-car-t-cell-therapy-kymriahtm-ctl019. Accessed September 2017.
  2. The Lancet. CAR T-cells: an exciting frontier in cancer therapy. Lancet. 2017;390(10099):1006. doi: 10.1016/S0140-6736(17)32395-4
  3. Szabo L. New CAR-T drug for leukemia, while effective, is ‘going to cost a fortune.’ STAT News website. https://www.statnews.com/2017/08/23/car-t-leukemia-drug-cost/. Published August 23, 2017. Accessed September 2017.
  4. Frey N. The what, when and how of CAR T cell therapy for ALL. Best Pract Res Clin Haematol. 2017 Aug 3. doi: 10.1016/j.beha.2017.07.009 [Epub ahead of print]