Excitement over chimeric antigen receptor (CAR) T-cell therapies has been matched only by the frustration at figuring out how to pay for these unique, expensive, and hard-to-administer treatments. An early value-based agreement
between Novartis and CMS collapsed that covered the first therapy, tisagenlecleucel (Kymriah), which lists at $475,000. Federal policymakers are in the midst of a National Coverage Analysis that will produce a decision memo in February 2019. In the meantime, the science races ahead, with more treatments and more indications expected, as well as the prospect that CAR T-cell therapy will advance from autologous to allogeneic treatments.
So, what can payers and institutions do in the meantime? At least one Medicaid program isn’t waiting, as a pair of speakers discussed Wednesday at the Academy of Managed Care Pharmacy Nexus 2018 in Orlando, Florida. Therese Mulvey, MD, is the director of Quality Safety and Value at Massachusetts General Hospital in Boston and Stephanie Tran, PharmD, a clinical consultant pharmacist for the University of Massachusetts Medical School covered the clinical and financial challenges that CAR T-cell therapy presents and how the commonwealth has addressed them with a carve-out initiative through the pharmacy benefit.
First, Mulvey reviewed data from the ZUMA-1 trial
for axicabtagene ciloleucel (Yescarta), approved to treat diffuse large B-cell lymphoma (DLBCL) at a cost of $373,000 and the JULIET trial
for tisagenlecleucel (Kymriah), approved initially for pediatric acute lymphoblastic leukemia (ALL). As she explained, the patients in the trials who had success with these therapies had run out of options, and the results were astounding. Because these are “living drugs,” they keep working after a single treatment. “These are drugs that are very expensive, but if the patients have a response, and the responses are durable, the folks won’t require additional treatment.”
She contrasted this with multiple myeloma, which now has many treatments options and much improved survival rates, can cost $2.2 million per patient over the first 3 years. Clinical trials for CAR T-cell therapy in multiple myeloma are ongoing, Mulvey said.
A big challenge for CAR T-cell therapy is toxicity, notably cytokine release syndrome (CRS), which Mulvey said is usually manageable and reversible but can emerge up to 50 days after the infusion. And of course, the other toxicity is financial. The price tag for CAR T-cell therapy, “has definitely shattered oncology pricing norms,” Mulvey said. (Some estimates have put the total price including inpatient costs at $1 million.)
Medicare and Medicaid frameworks are simply not designed for this, and that’s a problem, given that roughly 50% of the DLBCL population will be in Medicare (and CMS is pushing more of them into Medicare Advantage). Among the pediatric ALL population, 40% are eligible for Medicaid. Given the need for inpatient infusion, the possibility of CRS, and the administrative burden involved, Mulvey asked rhetorically, “Why should a facility or a provider take the time to do this work?”
She called on payers and policymakers, including the National Comprehensive Cancer Network, to develop policies to grant equal access, to ensure that patients who are poor or live in remote areas can be reimbursed for travel. There’s a need to address the timing of payments, because the risk to institutions is great. A life-saving therapy “should not bankrupt the system.”
Tran then gave a detailed overview of how Massachusetts’ Medicaid program, MassHealth, designed a “carveout” with CMS approval to cover CAR T-cell therapies. A carveout is considered when it became clear that CAR T-cell treatments were so costly that they rose above the “outlier” policy that reimbursed 85% of normal adjudicated rates. The decision was made to develop a carveout in November 2017; providers were notified in February 2018, and the carveout became effective in March 2018. CMS gave its approval on June 22, 2018, with an effective date of March 1, 2018.
Under the policy, providers bill separately for the therapy, which is not included in the normal adjudicated rate. But treatment centers are still at risk for some ancillary costs, including the initial leukapheresis or treatments for CRS. The therapy is managed as part of the pharmacy benefit, Tran said.
A CAR T-cell therapy monitoring program carefully tracks how patients are doing and gets biweekly updates on outcomes. Under an agreement with Novartis, if the tisagenlecleucel therapy is not working at 30 days, MassHealth does not pay for the therapy carveout.
Future considerations for all stakeholders include:
- At the 1-year mark of therapy approval, there will be more data which could have an effect on prices
- New indications may change affordability of the treatments
- Payers will have to consider how to change the administrative process to improve work flow