The Centers for Medicare & Medicaid Services (CMS) has proposed to cover chimeric antigen receptor (CAR) T-cell therapies approved by the FDA, under “Coverage with Evidence Development,” according to a recent press release issued by the agency.
The proposal has been issued in response to a formal request made by Efrem Castillo, MD, Medicare & Retirement chief medical officer, at UnitedHealthcare (UHC), who asked for clarification of the circumstances under which FDA-approved CAR T-cell therapies will be covered in order to create consistent patient access to this treatment nationwide as well as financial sustainability in the Medicare Advantage program.
“As UHC anticipates emerging clinical advances, including these CAR T[-cell] therapies, UHC has several concerns that while promising clinically, CAR T[-cell] therapies could create significant financial risks for CMS, both Original Medicare FFS and Medicare Advantage plans,” Castillo wrote in his letter.2
“As CMS is aware, Medicare Advantage plans cover services as they are covered under Original Medicare, including Part B drugs,” he added. “For that reason, we believe there is an industry-wide need for a National Coverage Determination (NCD) to ensure a level playing field across Medicare Advantage plans, so that providers and members are better equipped to make treatment decisions.”
In the proposed NCD, Medicare will cover the therapy on a national level when the treatment is offered through a CMS-approved registry or clinical study in which patients are monitored for at least 2 years after they complete treatment.
The data from these registries and trials will help the CMS determine the types of patients who will benefit most from the therapy, which, in turn, will help the agency eventually decide which kinds of cases Medicare will cover without a registry or trial requirement.
The CMS plans to leverage the FDA’s requirements for post-approval studies for these agents when reviewing studies for CMS approval.
“CAR T-cell therapy was the first FDA-approved gene therapy, marking the beginning of an entirely new approach to treating serious and even life-threatening diseases,” Seema Verma, CMS administrator, said in the press release. “The proposed coverage decision would improve access to this therapy while deepening CMS’s understanding of how patients in Medicare respond to it, so the agency can ensure that it is paying for CAR T-cell therapy for cases in which the benefits outweigh the risks.”
In August 2017, the FDA granted the first approval to a CAR T-cell product with tisagenlecleucel (Kymriah) for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
The approval was based on positive data from a single-arm trial comprised of 63 patients with relapsed/refractory pediatric precursor B-cell ALL. Results showed a confirmed overall remission rate of 82.5% (95% CI, 70.9%-91.0%) with the therapy; 63% of these patients achieved a complete remission (CR), while 19% had a complete remission with incomplete hematological recovery (CRi).3
All patients with a CR/CRi were found to be minimal residual disease (MRD) negative.
In May 2018, the agent also received FDA approval for the treatment of adult patients with relapsed/refractory large B-cell lymphoma after 2 or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma, and DLBCL from follicular lymphoma based on data from the phase II JULIET trial. Results from the study demonstrated an overall response rate (ORR) of 50% (95% CI, 37.6%-62.4%) in 68 patients, as assessed by an independent review committee.4
Another CAR T-cell agent, axicabtagene ciloleucel (Yescarta), received FDA approval in October 2017 for use in adult patients with relapsed/refractory large B-cell lymphoma after 2 lines or more of systemic therapy, including DLBCL not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.
This approval was based on positive data reported from a single-arm trial consisting of 108 adult patients with B-cell non-Hodgkin lymphoma, which showed an ORR of 72% as assessed by independent central review, with a CR rate of 51% (95% CI, 41%-62%) in patients treated with the therapy.5
The CMS will issue a final decision on the proposal no later than 60 days after the conclusion of a 30-day public comment period, which was initiated on February 15, 2019. The expected date for National Coverage Analysis completion is May 17, 2019.
- CMS Proposes Coverage With Evidence Development for Chimeric Antigen Receptor (CAR) T-Cell Therapy. Centers for Medicare & Medicaid Services. Published February 15, 2019. https://go.cms.gov/2SXbV52. Accessed February 18, 2019.
- Castillo E. Formal request for National Coverage Determination for chimeric antigen receptor T-cell therapies. UnitedHealthcare. Published February 22, 2018. https://go.cms.gov/2BHbCSi. Accessed February 18, 2019.
- FDA approves tisagenlecleucel for B-Cell ALL and tocilizumab for cytokine release syndrome. FDA. Published August 30, 2017. https://bit.ly/2GwlpOW. Updated September 7, 2017. Accessed February 18, 2019.
- FDA approves tisagenlecleucel for adults with relapsed or refractory large B-cell lymphoma. FDA. Published May 1, 2018. https://bit.ly/2GLUA8W. Updated May 3, 2018. Accessed February 18, 2019.
- FDA approves axicabtagene ciloleucel for large B-cell lymphoma. FDA. Published October 18, 2017. https://bit.ly/2U14Ca9. Updated October 25, 2017. Accessed February 18, 2019.