Providers, Industry Raise Concerns About CMS Plan for CAR T-Cell Reimbursement, Reporting on PROs

Academic medical centers and a group representing community oncology practices have both raised concerns about CMS’ proposed reimbursement plan for chimeric antigen receptor (CAR) T-cell therapy,^1,2 the individually manufactured gene treatments that are revolutionizing cancer care. The plan will be finalized next month, a year after the federal

government launched a national coverage analysis (NCA) to determine how to pay for these lifesaving yet expensive cancer treatments.

Since February 15, 2019, when CMS issued its proposal,³ cancer centers that have been losing money on CAR T-cell treatments have raised questions about the feasibility of the

plan’s centerpiece: a model called Coverage With Evidence With Development (CED), which critics fear could overburden providers, causing some to opt out of offering CAR T-cell treatment to Medicare patients. Others have asked about requirements to report patient-reported outcomes (PROs) and language they say could squeeze out community practices.

Finally, there are concerns that the plan’s language is tightly crafted based on the first 2 CAR T-cell therapies approved by the FDA, without enough flexibility to reimburse providers

for treatments in the research pipeline. CMS accepted comments on its proposed decision memo through March 17, 2019, and its final national coverage determination (NCD) is

due May 17, 2019.³

Those who support CMS’ approach say it addresses 2 knowledge gaps involving CAR T-cell therapy that are important to Medicare: the lack of data for treatment of those 65 years and older and the need to gather more information about PROs. This is the first year that the National Comprehensive Cancer Network (NCCN) has included a section on CAR T-cell—related toxicities in its guideline on Management of Immunotherapy-Related Toxicities,⁴ and clinicians continue to fine-tune methods for preventing and treating the significant adverse events associated with CAR T-cell therapy, including cytokine release syndrome.

But providers who spoke with Evidence-Based Oncology™ (EBO) and have filed comments with CMS and appeared in public forums said these good intentions could have consequences if CMS’ proposal isn’t modified. Data gathering requirements include evidence for the CED model and reporting with specified PRO tools. An expert panel that discussed CMS’ CAR T-cell reimbursement plan at the NCCN Annual Conference on March 21, 2019, said the losses cancer centers have already seen from CAR T-cell treatment are not sustainable, and some were unsure who would pay for data collection (see Sidebar). A comment letter sent to CMS from City of Hope in Duarte, California, which infused its first CAR T-cell trial patient in 2000, warned that the requirements “risk exacerbating patient access issues and compounding the financial losses that currently experience while serving Medicare beneficiaries.”¹ (A signer of the letter, Joseph Alvarnas, MD, vice president for Government Affairs, is editor-in-chief of EBO.)

Even NCCN, which supports the concept of enrolling patients in registries, urged CMS to be mindful of the current financial burdens on cancer centers. In his comment, NCCN Chief Executive Officer Robert W. Carlson, MD, wrote, “NCCN firmly agrees with the principles of the registry.…We recommend that implementation of registry and data-collection be enacted with considerable focus on reducing administrative burden and supporting patient access to innovation.

“While NCCN recognizes that coverage determinations are made separate and apart from reimbursement determinations, we feel it is important that CMS implement the CED with an appreciation of the current reimbursement environment. Given that most providers of CAR T-cell therapy are currently being undercompensated by several hundreds of thousands of dollars for each Medicare patient treated, and possibly more if complications arise, NCCN has concern that an overly onerous CED process could lead providers to not participate due to the additional administrative cost.”¹

The National Coverage Analysis Process

In August 2017, the FDA approved the first CAR T-cell therapy, tisagenlecleucel (Kymriah), and CMS simultaneously announced a value-based agreement with sponsor Novartis that oncologists described as “you’re only charged if you respond in 30 days.”⁵ The FDA approved the second therapy, axicabtagene ciloleucel (Yescarta), in October 2017. The treatments cost either $373,000 or $495,000, depending on indication.⁶

Early on, cancer centers focused on getting billing codes and finding out which state Medicaid programs would cover tisagenlecleucel when indicated for pediatric acute lymphoblastic leukemia.⁶

But UnitedHealthcare brought this to a halt with a request for an NCA; during the recent NCCN conference, UnitedHealthcare Senior Medical Director of Oncology and Genetics

Jennifer Malin, MD, PhD, said the company needed consistency nationwide. That process included an August 2018 meeting of the Medicare Evidence Development & Coverage

Advisory Commission to determine whether CMS would measure PROs as part of reimbursement;⁷ this led to the inclusion of 2 measurement tools in the February 2019 proposed decision memo despite industry objections.

In the meantime, cancer centers that administer CAR T-cell therapy have been in limbo. With no Medicare national coverage policy in place, they have been using an existing billing code plus an add-on technology payment that does not come close to covering the cost of the manufactured cells; because of the lack of consistency between Medicare Administrative Contractors, oncologists in some states say Medicare basically doesn’t pay for the engineered cells. In parts of the country, Medicaid patients who would benefit from treatment cannot gain access.

Features of the proposed reimbursement plan include³:

• The NCD would have highly specific criteria for what types of institutions can be reimbursed for CAR T-cell therapy; these criteria include staff they must employ. In comments filed with CMS, the Community Oncology Alliance (COA) said the use of the word hospital throughout the document could prevent member practices from receiving Medicare reimbursement.^2,3
• The plan outlines what information must be tracked, based on whether the person receives therapy as an inpatient or an outpatient.
• The plan specifies measurement tools that must be used to report PROs and at what intervals over 2 years. A comment from MD Anderson Cancer Center said that tying reporting PROs to reimbursement is questionable, as patients often “come to CAR T providers for treatment and then return to [the] referring facility immediately after treatment completion.”¹ In their letter, City of Hope leaders said that requiring either the National Institutes of Health Patient-Reported Outcomes Measurement Information System or the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events creates costs for institutions and “creates additional demands upon those patients who have undergone these treatments.”¹
• Repeat treatments will not be funded unless a new primary cancer is diagnosed.
• The “furnishing hospital” treating patients must accept “all manufactured products.”

Balancing Need for Evidence With Burdens

Ted Okon, MBA, executive director of COA, told EBO in an interview last month that it appears CMS created the proposal to address a lack of data on how CAR T-cell therapy works in the Medicare population. But who should pay to gather these data? During the NCCN panel, Malin suggested the cost should be borne by the pharmaceutical companies. In their letter, City of Hope officials expressed concern that without clarity, the costs will fall on cancer centers.¹

Representatives for the Biotechnology Innovation Organization (BIO), a trade group for the biotechnology industry, told EBO in an interview that its members are also concerned

that the proposal is written based on how CAR T-cell therapies are being used at the moment, not how they might be used in the future. City of Hope leaders concurred in their

letter: “There are numerous CAR T-cell products that are in development that are differentiated from the 2 currently FDA-approved products. These emerging therapeutics may employ a different method of action or represent effector cell populations that are targeted against cancer types other than those targeted by Kymriah and Yescarta.”¹

In an interview with EBO, Mallory O’Connor, BIO’s director of healthcare policy and federal programs, said the group shares COA’s concern about language that could exclude community practices from reimbursement and seeks additional changes:

• The February 15, 2019, proposal states that CMS will pay for CAR T-cell therapy only in relapsed/refractory cancer, which reflects current FDA approvals. However, in the future, CAR T-cell or similar therapies may be used as the initial treatment for certain cancers. Already, discussions during the 2018 American Society of Clinical Oncology annual meeting have suggested the therapy may be more successful (and cost-effective) if patients’ immune systems are not weakened by prior rounds of treatment.⁸
• There is a great need to clarify implementation dates and requirements and whether this NCD will serve as a model for treatments similar to CAR T-cell therapy.
• Other than many technical questions around the reporting requirements, BIO has asked about privacy issues: What if a patient needs treatment but does not want to share their data in a clinical trial or a registry?

Those who administer CAR T-cell therapy noted CMS’ goal is to increase access to treatment, which is what Administrator Seema Verma said in unveiling the February plan. “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,” she said in a statement. The proposed coverage decision “would improve access to this therapy while deepening CMS’ 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.”⁹REFERENCES:

1. View public comments for chimeric antigen receptor (CAR) T-cell therapy for cancers. CMS website. cms.gov/medicare-coverage-database/details/nca-view-public-comments.aspx?NCAId=291&bc=AAAAAAAAAQAA&#Results. Published February 19 to March 17, 2019. Accessed April 4, 2019.
2. Proposed decision memo for chimeric antigen receptor (CAR) T-cell therapy for cancers. Community Oncology Alliance website. communityoncology.org/proposed-decision-memo-for-chimeric-antigen-receptor-car-t-cell-therapy-for-cancers/. Published March 15, 2019. Accessed April 3, 2019.
3. National coverage analysis (NCA) tracking sheet for chimeric antigen receptor (CAR) T-cell therapy for cancers (CAG-00451N). CMS website. cms.gov/medicare-coverage-database/details/nca-tracking-sheet.aspx?NCAId=291. Published February 15, 2019. Accessed April 3, 2019.
4. NCCN Clinical Practice Guidelines in Oncology. Management of Immunotherapy-Related Toxicities, version 1.2019. National Comprehensive Cancer Network website. nccn.org/professionals/physician_gls/pdf/immunotherapy.pdf. Updated November 14, 2018. Accessed April 2, 2019.
5. Caffrey M. Paying for innovation in cancer care means no easy answers. The American Journal of Managed Care. website. ajmc.com/newsroom/paying-for-innovation-in-cancer-care-means-noeasy-answers?p=2. Published June 28, 2018. Accessed April 4, 2019.
6. Caffrey M. With approval of CAR T-cell therapy comes next challenge: payer coverage. Am J Manag Care. 2018;24(SP2):SP35-SP36.
7. Inserro A. MEDCAC panel mostly endorses PROs for CAR T therapies. The American Journal of Managed Care. website. ajmc.com/newsroom/medcac-panel-mostly-endorses-pros-for-car-ttherapies. Published August 22, 2018. Accessed April 3, 2019.
8. Cavallo J. Is CAR T-cell therapy setting new standard of care in lymphoma? ASCO Post website. ascopost.com/issues/april-10-2018/car-tcell-therapy-standard-of-care-in-lymphoma/. Published April 10, 2018. Accessed April 2, 2019.
9. CMS proposes coverage evidence with development for chimeric antigen receptor (CAR) T-cell therapy [news release]. Baltimore, MD: CMS; February 15, 2019. cms.gov/newsroom/press-releases/cms-proposes-coverage-evidence-development-chimeric-antigen-receptor-car-t-cell-therapy. Accessed April 4, 2019.