Andrew L. Pecora, MD
Chimeric antigen receptor (CAR) T-cell therapies could spawn a new and lucrative industry in anticancer immunotherapy, resulting in single-infusion treatments costing hundreds of thousands of dollars, and even attracting medical tourism from abroad, where regulatory permissions are likely to come more slowly than in the United States.
That is the picture that is emerging in interviews with oncology experts as the FDA considers whether to approve the first genetically engineered T-cell therapies. CAR T-cell therapies are a new form of immunotherapy treatment that not only breaks fresh ground on innovation in medicine but also tests the boundaries on what patients and payers are willing to pay and what doctors and medical institutions are willing to charge.
In July, the FDA’s Oncologic Drugs Advisory Committee unanimously recommended that the agency approve Novartis’ tisagenlecleucel (CTL019) for the treatment of pediatric and young adult patients aged 3 to 25 years with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (ALL). The FDA also is considering Kite Pharma’s application for axicabtagene ciloleucel (KTE-C19; axi-cel) as a treatment for transplant-ineligible patients with r/r non-Hodgkin lymphoma (NHL).
Just a handful of tertiary care centers would be able to provide CAR treatments in the beginning, making medical tourism a possibility, said Andre Goy, MD, MS, chairman, director, and Lymphoma Division chief at John Theurer Cancer Center (JTCC), Hackensack Meridian Health, in New Jersey. “Refractory lymphoma has no treatments, and when you have a technology that’s potentially serviceable for patients who are otherwise sent to hospice or die, people will come from very far away,” he said.
Estimates for treatment costs vary widely, from several hundred thousand dollars to as much as $700,000 per patient, oncology experts said in interviews. CAR T-cell engineering is a high-tech, labor-intensive process; it involves collecting the patient’s blood mononuclear cells, shipping them to a factory for refinement and enrichment ex vivo for T-cell growth, programming them with an anti-CD19 CAR transgene, testing, and finally returning those supercharged cancer-fighting cells to the clinical site where they can be reinfused into the patient. In the case of tisagenlecleucel, a separate clinical team is assigned to each blood shipment and follows it throughout the manufacturing process to ensure optimal quality and safety. All in all, manufacturing time for CAR T-cell therapies can stretch several weeks.
In addition to the costs of manufacturing the therapy, there are supportive care costs and potential expenses from additional lines of treatment, which may cause total medical costs to rise further.
Estimates Reach $700,000
In the months leading up to potential FDA approvals, many stakeholders in this epic development in immunotherapy care have been unwilling to publicly discuss the bottom-line costs of care, although the issue reportedly has been foremost in negotiations and talks that had already begun to take place between medical institutions and payers. “We won’t be able to provide a live interview at this time, and any commercial/pay/cost questions won’t be addressed until the therapy is approved and launched,” said a spokeswoman for Novartis in response to a query from OncLive®
. Novartis did provide written answers to questions about manufacturing specifics and offered to address other less-critical issues similarly.
Sensitivity to the cost issue extends to payers as well. Anthem, one of the largest healthcare companies with revenues of $85 billion in 2016, also declined to discuss CAR T-cell marketing. “CAR T-cell therapy has not yet been reviewed through the Anthem medical policy process,” the company said in a statement. “We anticipate developing a policy pending FDA approval. Anthem is committed to the ongoing evaluation of the safety and efficacy of therapeutic services to ensure clinically appropriate use based on clinical purposes.”
JTCC wants to be a part of the CAR T-cell launch and has been a testing site for the therapy that Kite Pharma pioneered. The center stands equally ready to help commercialize Novartis’ tisagenlecleucel and has been in talks with payers, according to Goy. He said payers are very concerned about where the price ceiling on this therapy is going to end up.
Goy said the point must be made in these discussions that a round of CAR T-cell therapy, although costly, might enable patients to avoid a lot of unsuccessful therapy that also builds up the medical tab for payers. “Yes, of course they’re nervous, but this is something that instead of giving therapies that don’t work, repeatedly, you can give something that gives a little mileage. Everything’s relative.”