Late in 2017, we witnessed the approval of two Chimeric antigen receptor (CAR) T-cell products, passionately referred to as the CAR-Ts. CAR-Ts are genetically modified T cells, where a patient's own T cells are manipulated ex vivo to recognize a specific antigen or protein on the malignant B-cell surface. The cells are infused back into the patient to elicit a very specific T-cell response against the malignant B-cells. CAR T-cells have been studied most extensively in hematologic malignancies, confirmed by the approved indications for Kymriah™️ and Yescarta™️, although there is ongoing work in solid tumors as well. While the CAR T-cell therapy fills an unmet need in hematologic malignancies, they are not without risk. Side effects are substantial in certain patients and include signs of the cytokine release syndrome such as fever, hypotension, altered mental status, and seizures, with some patients requiring intensive care. Both CAR-T cell products are monitored under the FDA Risk Evaluation and Mitigation Strategies (REMS) program.
As with any new technology, our first concern will usually be the question of cost. With the cost of Kymriah and Yescarta listed at $475k and $373K respectively, this is only the beginning. Additional costs will be incurred for the initial apheresis, followed by a round of chemotherapy for lymphodepletion and infusion of the CAR-T cells approximately 2 weeks later. In both cases, the CAR-T cells are infused in certified facilities where adequate training has been provided by the manufacturer. The additional costs could match the cost of the CAR T-cells. Additional costs are incurred by the patient and care givers to travel to the certified facility.
While the cost burden of treatment options is one that we will be continually be battling and questioning, I would contend that the CAR T-cell treatment will bring a focus to another disconnect in the health care system, being patient care coordination and navigation. Patient care coordination and navigation needs to go far beyond that of the physician-patient relationship. CAR T-cell therapy will require an active coordination between the physician, patient, and the payer. While the physician-patient relationship remains intact, the payer needs to establish processes to become an active participant in the process. As noted, the CAR T-cells must be infused at a certified facility. That facility may or may not be within the payer’s immediate provider network. Medical necessity may not pose the biggest barrier to access this treatment option, but rather the reimbursement for the treatment process may prove to be the biggest barrier, especially to an out-of-network provider. The national payers will more than likely have national network coverage, however, there will be issues with the regional players. More than likely, the certified facilities will have a set reimbursement level, which must be proposed and negotiated with the payer. Under current processes today, the negotiation process can be very long and drawn out. Payers must be educated on the CAR T-cell treatment process and be timely in their responses in order to prevent treatment delays.
Along the line of certified facilities being the in the provider network, member benefit contracts will also need to be reviewed, and in some cases modified. In many benefits, patients will have a higher liability when using a non-network provider. Most HMO plans will not provide coverage for services received out-of-network. In cases where the patient has little choice as to where services can be accessed, the patient should not be penalized.
These are exciting times where innovation is presenting us with new effective treatment options. As expected, these treatment options are costly. While we deal with cost burden issues on a daily basis, we now also need to focus our attention to the need to coordinate care and help the patient navigate through a complicated treatment process. Patient navigation is only starting to gain attention within the larger oncology practices. These practices represent a closed system with their patients. CAR T-cell treatment opens up the treatment process to potentially multiple providers and multiple facilities. Are we up for the challenge to work together to allow the CAR T-cell treatment option to fulfill an unmet need? —Winston Wong