[1] Centers for Medicare and Medicaid Services, Proposed Decision Memo for Chimeric Antigen Receptor (CAR) T-cell Therapy for Cancers (CAG-00451N),

https://www.cms.gov/medicare-coverage-database/details/nca-proposed-decision-memo.aspx?NCAId=291.

[2] Targeted Oncology, Mesothelin-Targeted CAR T-Cell Therapy Shows Early Efficacy in Advanced Solid Cancers, April 1, 2019,

https://www.targetedonc.com/news/mesothelintargeted-car-tcell-therapy-shows-early-efficacy-in-advanced-solid-cancers.

April X, 2019

The Honorable Seema Verma

Administrator

Centers for Medicare & Medicaid Services

200 Independence Avenue, SW

Washington, DC 20201

Dear Administrator Verma:

We write regarding the proposed decision memo (PDM) for coverage of chimeric antigen receptor (CAR) T-cell therapies released on February 15, 2019.[1]  We appreciate and support CMS’ efforts to ensure that Medicare
coverage for these novel medicines reflects a firm understanding of the safety and efficacy of novel therapies through the Coverage with Evidence Development (CED) pathway.  Given our shared goal of patient access to this rapidly evolving form of transformative
cancer care, we ask for your consideration of changes that will accommodate the current and next wave of CAR T-cell therapies and ensure timely coverage for Medicare beneficiaries upon finalization of this coverage decision.

We support CMS’ desire to collect data on the clinical outcomes of Medicare patients who receive CAR T-cell therapy given that this population is not traditionally well represented in clinical trials. However, the proposed CED clinical data elements, other
than patient reported outcomes data, are already being collected as part of an existing registry.  The Center for International Blood and Marrow Transplant Research (CIBMTR) Cellular Therapy Registry is working collaboratively with participating CAR T-cell
patients in collecting long-term safety and efficacy data.  Specifically, the registry will capture data from 15-year post-marketing observational studies for both approved CAR T-cell therapies that assess long-term safety. The initiative will follow at least
4,000 patients for 15 years after CAR T-cell administration, to fulfill regulatory requirements required by the Food and Drug Administration (FDA).[2]  Cellular therapy is a rapidly advancing field that holds great
promise for many patients diagnosed with life-threatening disease, making appropriate data collection an important issue that must be carefully considered.  With this in mind, we encourage CMS to consider developing policy that will support the existing infrastructure,
exchange, and collection of important clinical data while avoiding the creation of barriers to new therapies and new methods of delivery.

As CMS considers appropriate patient eligibility and facility criteria in the final decision memo, we urge the agency to account for the significant promise for patients as next generation therapies are developed.  We believe it is important that the final
decision memo is sufficiently flexible to accommodate future FDA approved CAR T-cell therapies currently in development, given that the National Coverage Analysis (NCA) process takes significant time and agency resources.  This flexibility will help ensure
this coverage decision is prepared to cover patients that will benefit from future innovation.  Otherwise, the agency must remain committed to regularly updating the NCA. 

In working harmoniously with FDA, we also encourage CMS to consider establishing patient eligibility criteria that would provide coverage for new FDA approved indications. While currently approved therapies are only indicated for relapsed/refractory disease,
new products and ongoing clinical trials show promise for CAR T-cell as a front-line therapy in certain, aggressive cancers.  CMS should be able to provide coverage and collect data for cancer patients who receive CAR T-cell therapy for an FDA approved indication
or consistent with National Comprehensive Cancer Network (NCCN) guidelines already referenced in the PDM.

CMS has been consistent in how it covers products at sites, provided sites meet specific safety protocols and other regulatory requirements.  While CMS uses the term hospital throughout the PDM, we are mindful of the complexity of definitions surrounding
that term hospital, including recent changes enacted by Congress that reimburse certain hospital departments under the physician fee schedule.[3]  We understand the attributes of administering complex therapies
at hospitals due to the nature of their composition in comprehensive integrated care, but the term hospital may vary under the Medicare payment system in how the service is reimbursed as hospitals may have outpatient facilities as indicated above.  While not
licensed as a hospital, community oncology practices and cancer research centers are currently certified to administer CAR T-cell therapy, and do adhere to the same specific safety protocols as outlined by the PDM and FDA Risk Evaluation and Mitigation Strategy
requirements.  As you know, there are several sites that currently administer CAR T-cell therapies and that are currently engaged in CAR T-cell clinical trials.

Lastly, we want to highlight the importance of ensuring that there is no delay in patient access.  To that end, the CED pathway should not impede access and should not duplicate the collection of data efforts as the existing registry meets all of the data
elements, scope and collection frequencies.  We understand that in order to achieve this objective, CMS must approve at least one registry that can be operational by May 17, 2019 when this coverage decision is finalized; if not, an extension should be granted
until it is fully operationalized.  Patients currently eligible for CAR T-cell therapy have often exhausted every other available treatment option and are suffering greatly from the burden of their disease.  We urge CMS to work quickly and transparently with
stakeholders to this end in order to avoid any disruption in patient access.

Thank you for your consideration of these issues.  We appreciate CMS’ efforts to bring the best possible care to Medicare beneficiaries, and we look forward to continuing our partnership with you to ensure patient access to our healthcare system’s most exciting
advances.

Sincerely,