Improving Access to CAR T Therapy: It’s Not Just Payment

Michael Kolodziej
ADVI Health
Ira Klein
Marc Samuels
ADVI Health

he development of chimeric antigen receptor (CAR) T therapy for patients with advanced hematological malignancies represents one of the greatest successes in bringing basic tumor immunology to the bedside. The ability to potentially cure patients with end-stage acute lymphocytic leukemia (ALL) and diffuse large B-cell non-Hodgkin lymphoma (DLBCL) with a personalized immunotherapy seems science fiction. And yet the rate of uptake of this therapy has been slow, particularly in US Medicare beneficiaries.

At least part of this is related to Medicare’s reimbursement methodology, antiquated for the advent of cell and gene therapies. But this isn’t the only factor holding back access; challenges inherent to the complexity of cell therapy will need to be solved in order to move this forward. Other cell therapies that are in the pipeline will likely be in the same boat, so now is as good a time as any to solve these issues. These solutions will require collaboration of all the stakeholders in the oncology ecosystem.

We have identified three major areas that should be addressed:

  1. Measuring what matters: recognizing real world value.
  2. Patient flow and practice management: enhancing the patient experience.
  3. Payment policies for cell therapies: acknowledging and fixing Medicare’s shortcomings.

CAR T Data Challenges: Measuring What Matters

The US Food and Drug Administration (FDA) has embraced the rapid evaluation and approval of novel cancer therapies. Although this has allowed accelerated access to these treatments, the price associated with this speed has been a lack of comprehensive data on safety and efficacy. Many approvals are based on surrogate endpoints, and evidence indicates that these surrogates may not in the end prove predictive of clinical endpoints that matter. Many trials have a single arm, meaning comparison to the standard of care is exclusively by extrapolation. In addition, the trials are often small, with the study population not always representative of the clinical population overall.

All these criticisms apply to CAR T. The lack of data puts physicians, patients, regulators, and payers in a tough spot. Post-marketing data collection either through mandatory registry reporting or real world evidence (RWE) collection would help answer questions about toxicity and efficacy. Should data submission be mandatory? Should the data be standardized across manufacturers and sites of service?

The proposed CMS National Coverage Decision (NCD), which codifies national Medicare coverage policy for cellular therapies, did require coverage with evidence development (CED) for treatment of Medicare beneficiaries, but alas this did not make the final proposal. The Center for International Blood and Marrow Transplant Research (CIBMTR) has been collecting data from many centers delivering CAR T, but this is incomplete, unfunded, and neither widely known nor accessible. For everyone in the cancer care ecosystem, true toxicity and efficacy profiles of these agents are needed to make informed decisions on their use.

Optimizing the Care Delivery Model for CAR T

Delivering CAR T therapy is complicated. At present, CAR T can only be delivered in a hospital or hospital-associated treatment center that can fulfill the FDA’s Risk Evaluation and Mitigation Strategies (REMS). Individual centers are accredited by the manufacturers via a process that is not transparent. In addition, these centers are often required to meet Foundation for the Accreditation of Cellular Therapy (FACT) accreditation, a proxy where there is no CAR T specific accreditation process.

Ideally, a way to measure quality of care is needed, benchmarking one treatment site to another, to facilitate care delivery improvement. However, treatment today does not represent treatment methodology forever. There is tremendous interest in moving CAR T to the outpatient setting. What monitoring is required? Finally, the needs of patients must be met. Issues regarding coordination of care, especially as care is transferred to centers of excellence, must be addressed. The patient journey should be optimized.

Paying for Cell Therapies

Without question, most stakeholders in the oncology ecosystem believe that paying for cell therapies is the major obstacle to patient access. But this may not in fact be the case. Evidence suggests that patients with commercial health insurance are receiving this therapy with the average degree of difficulty, i.e., with prior authorization and case rate negotiations between payer and provider. Medicaid and Medicaid health plans are paying for CAR T as covered outpatient drugs or through a value-based payment model.

The problem is that most patients who will be candidates for this treatment will be Medicare beneficiaries. And Medicare reimbursement has been an issue from the beginning because the payment offered by Medicare does not adequately cover the cost of treatment. With an acquisition cost for the cellular product of approximately $400,000, the payment offered by Medicare (which consisted of some combination of the diagnosis-related group [DRG], i.e., the traditional Medicare inpatient “bundled reimbursement,” for a complicated bone marrow transplant plus a New Technology Add-On Payment for the cellular product plus some outlier payment) puts treating institutions “in the red.” A new DRG has been proposed but may in many cases still not be enough. As a result, many institutions are not treating Medicare beneficiaries with CAR T. How should this be resolved?

Several proposals have been offered, but none have been widely adopted. The real challenge is that CAR T is the tip of the proverbial cell therapy iceberg, suggesting a need for policy changes like a new Medicare benefit category and best price and anti-kickback reform to facilitate outcomes-based contracting. One thing is clear: Given the price tag of CAR T, shifting financial responsibility to patients will not be a solution.

Why Now?

Scientific advances like CAR T, CRISPR, and gene therapy have the potential to revolutionize medicine. But these are not going to be inexpensive. For hereditary diseases that cause profound human suffering, which play out over many years, and ultimately lead to premature death, gene therapy will be the right thing to do for many reasons, including cost effectiveness (especially with payment over time). But the durability of this gene therapy is certain to be questioned, and the evidence will certainly not be available at market entry. Collecting the necessary data to understand the real value of these treatments will be critical. Defining the best way to deliver the therapies will be crucial. Finding a large sum of money at the time of care delivery with the expectation of benefit over time is not a trivial exercise. It is time for the regulatory and payment sciences to catch up to the basic science. If CAR T is a trial run that foretells a future of expensive innovations, we have a great deal of work to do to ensure affordable patient access to them.