The CAR T- cell therapy revolution
In 2017, the first therapy designed to genetically modify patients’ own T-cells to attack cancer was FDA-approved. These are known as CAR T-cell therapies. The therapies are an exceptional scientific advancement that have captured global attention due to their innovative nature. They have been hailed for their efficacy in late-line oncology patients, especially given their one-time administration.
There are currently two Food and Drug Administration (FDA) approved CAR T – cell therapies
With over 100 CAR T products in the pipeline, there are several that are expected to launch in the next few years. KYMRIAH™ and YESCARTA™ were approved in 2017-2018 with limited long-term evidence and high price tags (USD 475,000 and USD 373,000 in ALL and DLBCL respectively), which provoked the question: How will The Centers for Medicare and Medicaid Services (CMS) cover the costs of these groundbreaking and expensive products?
The Centers for Medicare and Medicaid Services (CMS) National Coverage Determination process
Initiated NCA – 2018: CMS initiated a National Coverage Analysis (NCA) in May 2018 to assess whether the evidence on CAR Ts is adequate to draw conclusions on the efficacy and generalizability of treatment outcomes in the Medicare population, which is required to support national, public reimbursement (i.e., Fee-for-Service (FFS) Medicare and Medicaid beneficiaries).
Initial Decision – 2019: An initial decision memo was posted in February 2019, proposing that CMS would cover the costs of CAR T treatment through coverage with evidence development (CED), whereby CMS would track outcomes for CAR T patients over time in a centralized database. This memo also required CAR Ts to be administered in a hospital that meets requirements such as having a Cellular Therapy Program, a designated care area, ICU transfer protocols, and adhering to outcomes reporting requirements.
While several of these comments included requests to remove the reference to inpatient hospital services, others included support for hospital administration and Foundations for the Accreditation of Cellular Therapy (FACT) requirements. Many comments also requested that the National Coverage Determination (NCD) recognize new emerging technologies and cover them to FDA label requirements, while others indicated that CAR T is still an evolving treatment paradigm, and thus an NCD remains inappropriate until more conclusive evidence is available.
Final Decision – 2019: Following the public comment period, CMS published the final NCD on August 7, 2019, which states that CMS covers CAR T therapy when administered at all healthcare facilities enrolled in the FDA risk evaluation and mitigation strategies (REMS) program, and when the CAR T therapy is used for an FDA-approved indication, or an indication supported for use in one or more CMS-approved compendia.
Implications for CAR T- cell therapies due to the lack of CED requirement
The original CED decision would have required hospitals who offer CAR T cell-therapy treatment to participate in a prospective, national, audited registry which would track CAR T patient outcomes on a national basis. By removing this requirement, CMS increases the flexibility in which types of sites are able to administer CAR T, thus improving patient access.
While removing the CED requirement may seem to limit the ability to develop a centralized tracking system for CAR T patient outcomes, several organizations, including the FDA (through their REMS program), and independent organizations such as The Center for International Blood & Marrow Transplant Research (CIBMTR), have developed databases which serve this purpose. Further, these databases extend to commercially insured patients, who make up the large majority of the current CAR T-treated population.
Removing the hospital-based requirements and only limiting CAR T treatment to REMS certified sites (FACT accreditation not required) will also increase access to CAR T for patients, allowing for treatment at specialized community oncology sites with greater geographical accessibility to patients.
Implications for CAR T- cell therapies due to the expansion of eligible treatment sites
This update to the NCD provides an opportunity for outpatient CAR T treatment where adverse event management allows, improving patient convenience factors and improving site capacity constraints. The NCD’s less-restrictive-than-expected criteria also means that commercial health plans may follow suit, despite some of the site of care restrictions that are currently in place within current CAR T policies.
Patient limitations for CAR T- cell therapies considering the NCD
While this NCD seems to offer increased access for CAR T patients, particularly when compared to what was proposed in the original memo, there remain several challenges associated with making CAR T accessible to all who are eligible.
Inpatient FFS Medicare reimbursement remains a significant challenge. Sites that administer CAR T to FFS Medicare patients in the inpatient setting are taking on significant financial losses given the insufficient reimbursement offered by the current blood and marrow transplantation (BMT) DRG designation. This insufficient reimbursement amount exists despite the new technology add-on payment (NTAP) increase to 65% in the proposed IPPS 2019 Rule. NTAP being an additional amount provided on top of the DRG specifically for innovative and first-to-market technologies. While more sites will now be able to offer CAR T treatment, it is unlikely that most will be willing to, given the financial losses incurred in the inpatient FFS Medicare population.
Another limiting factor to optimal patient access is that currently, CAR T manufacturers have a discrete number of manufacturing slots available per day, which results in patient waitlists nationally. While the NCD is certainly indicative of progress in the CAR T space, and a national acknowledgement that current CAR T therapies meet criteria for medical necessity, it remains to be seen whether outpatient oncology clinics will take on CAR T therapy, and how the landscape will evolve to meet the remaining needs of CAR T patients.
For further information on CAR Ts and other cell and gene therapies, read: CAR T Combos: Potentially the Next Big Thing in Solid Tumors, Yes Pricing ‘Sticker shock’ is not the Only Hurdle to be Overcome.