by Cynthia A. Bens, Senior Vice President, Public Policy, PMC

On November 30, 2017, the U.S. Food and Drug Administration (FDA) and the Centers for Medicare & Medicaid Services (CMS) announced the approval and preliminary coverage of Foundation Medicine’s FoundationOne CDx. As part of its announcement, CMS released a draft proposed decision memo for the FoundationOne CDx and similar next-generation sequencing (NGS) tests for use in the diagnosis of advanced cancer.

The draft coverage proposal was scrutinized by numerous stakeholders during an extended public comment period that resulted in CMS receiving nearly 400 pages of feedback. Many of these interest groups, including the Personalized Medicine Coalition (PMC), requested clarification from CMS on different aspects of the proposed decision memo. PMC’s comments highlighted the need to extend coverage for NGS testing to earlier stages of cancer and make allowances for retesting during the course of a patient’s life. In addition, PMC called on CMS to expand full coverage beyond FDA-approved NGS tests and consult with stakeholders to define a rigorous but less burdensome pathway to coverage with evidence development (CED) for NGS tests that do not meet CMS’ proposed criteria for full coverage.

Fortunately, CMS addressed many of the community’s concerns in the resulting national coverage determination (NCD) that was released on March 16, 2018. The following is a summary of what we view as major changes from the draft NCD.

• Coverage granted for FDA-approved and FDA-cleared NGS-based in vitro companion diagnostic tests. FDA-cleared NGS tests for advanced cancer would have fallen under a CED program in the original program. The final NCD expands the scope of coverage to NGS tests that are cleared as in vitro companion diagnostics through FDA pathways, such as 510(K), in addition to tests that are FDA-approved as in vitro companion diagnostics. Because the final determination does not specify sample type, FDA-cleared or -approved NGS liquid biopsy tests that are in vitro companion diagnostics will be covered as they become available, provided that all of the required patient and test indications are met.
• NGS tests indicated for use outside of advanced cancer not at risk of immediate non-coverage as previously thought. Under the proposed NCD, Medicare coverage was uncertain for tests that did not meet the narrow categories of FDA-approved or -cleared tests, and even these tests would have been subject to CED. Coverage decisions for both NGS-based in vitro diagnostics and laboratory-developed tests (LDTs) in areas such as hereditary testing, screening, and other non-cancer conditions will remain with local Medicare Administrative Contractors (MACs). Under the final NCD, coverage for NGS-based tests in patients with cancer without FDA-approved or -cleared companion diagnostic indications may be handled through the local coverage determination process, subject to the restrictions set forth in the final NCD.
• CED dropped. CED was an important but problematic feature of CMS’ draft. In the proposed coverage decision, CED programs would have been required for all other NGS-based cancer tests that were not FDA-approved NGS-based in vitro companion diagnostic tests for advanced cancer. Further, CED for LDTs was limited only to those participating in an NIH-NCI National Clinical Trial Network. CED was not included in the final CMS determination, and instead, non-FDA-approved NGS tests will need to be evaluated by the MACs to determine coverage status.
• Additional cancer indications included for coverage and one-time testing limitation removed. The proposed decision memo only provides pathways to coverage for NGS testing for Medicare beneficiaries with recurrent, metastatic, and stage IV cancer. In addition to coverage for these patients, the final NCD also covers NGS testing for patients with either relapsed, refractory, or stage III cancer when the NGS test meets the diagnostic assay requirements for coverage. The final NCD precludes coverage for patients with stage II or earlier cancer. In the final NCD, CMS also expanded the frequency of testing allowed, from using the same diagnostic laboratory test once to using the same diagnostic laboratory test once for each new primary diagnosis of cancer. Repeat testing for the purposes of treatment monitoring is not included in the final NCD.

The NCD is a net positive for NGS testing, as it now guarantees Medicare coverage at a national level for FDA-approved or -cleared NGS-based companion in vitro diagnostic tests for advanced cancer. PMC applauds CMS for recognizing that NGS testing is a breakthrough technology that is critical to advancing personalized medicine. We will continue to work with policymakers to advance diagnostics regulations and legislation that we believe will further improve patient care.

The featured speaker at PMC’s Policy Committee Meeting on April 24 will address the scope of CMS’ final coverage determination and its implementation. Ongoing federal activities related to diagnostics will also be the focus of a BIO 2018 session moderated by PMC. The panel, “Regulatory Oversight of Personalized Medicine: Today and Tomorrow,” will take place in Boston, Massachusetts, on June 6, 2018.