WASHINGTON (Feb. 15, 2019)
PMC Senior Vice President for Public Policy Cynthia A. Bens provided the following statement to reporters following the Centers for Medicare and Medicaid Services (CMS) proposal to cover chimeric antigen receptor (CAR) T-cell therapies "with evidence development" —
"CAR-T cell therapies have been approved by the Food and Drug Administration (FDA) based upon their demonstration of significant patient response rates. Sponsors of both FDA-approved CAR-T cell therapies have agreed to commitments for extensive post-market surveillance to better understand long-term benefits and toxicities of these therapies. These studies are of larger size and longer duration than studies of traditional cancer treatments. While CMS has pointed to limited data available on CAR-T therapies in the Medicare population, emerging data from post-market studies of currently-approved CAR-T products and those approved in the future will produce data to further demonstrate health outcomes for Medicare beneficiaries.
CMS’ stated position with regard to coverage with evidence development (CED) is that it should generally be limited to instances where it would expand coverage for Medicare beneficiaries. We are concerned that proposed CED requirement in this instance will not have its intended effect. The type of clinical data supporting FDA approval for CAR-T therapies will continue to vary by tumor type and indication. Therefore, if CMS determines that the evidence is insufficient to support coverage, the evidence gaps are likely to be very different for each CAR-T therapy and different types of evidence development with individualized data collection tools will be required for each therapy. Any such CED study must be reviewed and approved by CMS and there could be an extended period of non-coverage for the various CAR-T therapies until the studies are approved and begin enrolling patients.
While there have been a few successful CED cases, CED has proven challenging to implement. CMS has seldom used CED for drugs and biologics, and has never used CED for an FDA-approved anticancer therapy. Experience has highlighted that there are added costs and complexities to CED data collection and in some cases CED studies have not produced enough scientifically rigorous data needed to formulate sound coverage policy. PMC is further evaluating the full impact of CMS’ proposed decision memo but it is our hope that if CMS moves forward with a CED requirement, they will incorporate feedback received from our members who represent innovators, patients, providers and payors to ensure that Medicare beneficiaries facing poor prognoses and their health care providers will not suffer from delays in coverage of CAR-T therapies."