NEW YORK (GenomeWeb) – Ahead of the US Centers for Medicare & Medicaid Services finalizing a national coverage determination (NCD) on next-generation sequencing cancer panels, groups representing labs, pathologists, regional hospital systems, and patients are airing their concerns to Congress about the potential negative impact of this policy.
Industry insiders said that these lobbying efforts aim to shore up willingness among some legislators to step in and try to halt implementation of the NCD if certain provisions — such as the limited circumstances under which CMS proposes to cover lab-developed tests (LDTs) without US Food and Drug Administration's approval or clearance — remain in the final version slated for release Friday.
CMS issued a draft NCD last year after reviewing Foundation Medicine's FoundationOne CDx in parallel with the FDA. After evaluating the data submitted by Foundation and surveying the published literature on the use of NGS testing in cancer, the agency last year proposed a policy that would apply not just to Foundation's test, but to all NGS panels when they're used for recurrent, metastatic, or advanced Stage IV cancer patients.
"For this NCD analysis, we are proposing coverage for any next-generation sequencing diagnostic testing with the scope of this review limited to patients with advanced cancer," CMS said. "This is to ensure that similar claims for these tests will be covered in the same manner … This decision is in line with the vast majority of NCDs."
CMS has proposed three pathways through which NGS cancer panels may garner Medicare coverage. The agency said it will grant full coverage for genetic markers on the panel that have premarket approval (PMA) from the FDA as a companion diagnostic — where testing is required for the safe and effective use of a drug.
For markers without CDx status, but that the FDA has cleared or approved for use in a patient's treatment plan with other information (such as Memorial Sloan Kettering Cancer Center's 468-gene oncopanel MSK-IMPACT), CMS is willing to grant coverage with evidence development (CED), when the lab collects patient outcomes in a prospective registry.
Lastly, for LDTs without the FDA's blessing that are performed in a CLIA-certified lab — the majority of tests on the market — CMS is proposing CED when tests are included in a National Cancer Institute National Clinical Trial Network study. When tests don't meet these criteria, or when they are performed on patients who don't meet the agency's provisions, "CMS proposes non-coverage of NGS as a diagnostic laboratory test," the draft NCD states.
The American Clinical Laboratory Association has been active on Capitol Hill relaying its view that the draft NCD as written would restrict cancer patients' access to NGS panels and hinder diagnostics innovation. "In our discussions on the Hill, we have noted our concern about the scope of this proposed decision and the process that CMS followed in its development," said Paul Sheives, VP of reimbursement and regulatory policy at ACLA.
CMS extended the public comment period for the draft NCD from Dec. 31 to Jan. 17, due to stakeholder requests and the high volume of public comments. Among the more than 300 comments submitted to CMS, the feedback from labs, cancer centers, hospital systems, physicians, and pathologists are largely critical of the agency's non-coverage provisions for LDTs. The agency was originally expected to issue a final determination on Feb. 28, but said earlier this month that it was still reviewing comments and would issue a decision on Friday, March 16.
Many stakeholders believe that CMS should have done more to involve experts and the cancer community in crafting the draft NCD. ACLA recommended, for example, that before releasing a broad coverage policy that impacts a wide swathe of the lab industry, CMS should have engaged stakeholders through public meetings and consulted the Medicare Evidence Development & Coverage Advisory Committee, the Clinical Laboratory Improvement Advisory Committee, and those involved in the development and use of NGS testing.
"What we’ve communicated to Congress and to CMS is that to implement such sweeping non-coverage provisions with only a seven-week period to notify and engage stakeholders, really fails to provide adequate time to develop such an impactful policy," Sheives said.
The simplest way CMS can mitigate these concerns, according to ACLA and others, is by restricting the NCD to just FoundationOne CDx. "The coverage provisions of the NCD are really far outside the scope of the original request, and will take coverage away from Medicare beneficiaries who are benefiting from these innovative tests," Sheives said. "What [CMS is] bound by statute to do is to respond to the [coverage] request from Foundation Medicine."
In his comments to CMS about the draft NCD, John Brumsted, CEO of the University of Vermont Health Network, wrote that the proposed policy would reduce incentives to develop clinically validated NGS assays within CLIA-certified labs like the ones operated by the six-hospital system.
"The negative impacts on care to our patients, and on the UVM Health Network's academic and research missions, could be extensive," Brumsted told CMS. "It could create a monopoly for NGS testing, which could reduce or eliminate academic innovation. The profession of molecular pathology may move from the hospital setting to industry."
At the end of his comments, he indicated that he had sent copies of his statement to Senators Patrick Leahy (D-VT) and Bernie Sanders (I-VT), as well as to Representative Peter Welch (D-VT) in the House.
The College of American Pathologists has conveyed similar concerns to CMS and members of Congress about the "adverse and immediate consequences" the proposed NCD provisions would have on patient access to therapies and medically necessary tests. "The future development of test-based cancer therapies is now impacted by this NCD," said CAP President Bruce Williams.
Much of the objections to the draft NCD stem from the fact that CMS's proposal would essentially result in labs having to garner FDA's blessing if they hope to garner Medicare coverage. That's good news for the companies already intent on garnering FDA approval or clearance for their NGS cancer panels.
For example, Caris Life Sciences is taking its pan-cancer NGS assay through FDA review as a companion diagnostic to guide treatment with approved targeted therapies and immunotherapies. And Guardant has said that it will seek premarket approval through the FDA for its liquid biopsy assay, Guardant360, by year end.
The broader laboratory and pathology communities, however, have long fought to stave off FDA oversight of LDTs, which they say are not devices but lab procedures that are better regulated through CMS under CLIA. "ACLA is deeply concerned that the real-world effect of the NCD, were it to be finalized, would be a de facto requirement that each laboratory test using NGS technology would need to be approved or cleared as a medical device by the FDA before it is covered by Medicare," Sheives wrote to CMS. "The fact that the FDA has not reviewed and opined on the validity of a laboratory test using NGS technology does not mean that evidence of the test’s scientific validity is non-existent."
In ACLA's comments to CMS, it cited a recent study that found that for detecting mutations in certain cancer-linked genes, LDTs were just as good as FDA-approved tests and that a large proportion of study participants using FDA-approved assays modified the tests to broaden their use, rendering them LDTs.
ACLA and pathologist groups have previously lobbied Congress against the FDA's proposal to lift its long-standing practice of enforcement discretion and bring LDTs under its oversight. Those lobbying efforts, and a change of guard at the White House in the fall of 2016, likely pushed FDA to ultimately not finalize its controversial draft guidance on LDT regulation.
More recently, FDA Commissioner Scott Gottlieb has positioned his agency as a facilitator of lab test innovation, while looking to Congress to advance a legislative solution to this issue that involves the FDA, CMS's role administering CLIA, and other agencies.
Meanwhile, ACLA and CAP wouldn't share which legislators have lent their ears to the specific concerns about the proposed NCD. However, in mid-January, several patient advocacy organizations and cancer care groups — Association of Community Cancer Centers, International Myeloma Foundation, Myeloma Crowd, National Organization for Rare Disorders, and Susan G. Komen — sent a letter to CMS Administrator Seema Verma and a dozen senators and representatives on both sides of the aisle, expressing their concerns about the draft NCD.
The letter states that the organizations support FDA and CMS's efforts to streamline the approval and coverage processes under the Parallel Review program, and they back the positive coverage decision for FoundationOne CDx. However, they also requested that CMS not finalize the broader policy proposals in the NCD and instead convene discussion with stakeholders on coverage policies for NGS-based tests.
The legislators listed at the end of the letter include Senators Mitch McConnell (R-KY), Chuck Schumer (D-NY), Orrin Hatch (R-UT), Ron Wyden (D-OR), Lamar Alexander (R-TN), Patty Murray (D-WA), as well as House Speaker Paul Ryan (R-WI), Representatives Nancy Pelosi (D-CA), Kevin Brady (R-TX), Richard Neal (D-MA), Greg Walden (R-OR), and Frank Pallone (D-NJ).
The Association for Molecular Pathology said that since the draft NCD came out last November, it has been working to educate stakeholders, including the patient advocacy and cancer care organizations that signed this letter, about how the policy as written could limit access to NGS tests.
"To date, we have had over a dozen productive meetings with congressional offices on both sides of the aisle, including members of the Ways and Means, Finance, and Senate Health, Education, Labor and Pensions committees," said AMP Executive Director Mary Steele Williams. "We have also worked closely with several important patient advocacy groups and cancer care professionals to identify ways to improve the proposed policy."
It's unclear at the moment, however, which provisions in the final NCD might prompt ACLA, CAP, AMP, and cancer care groups to further lobby Congress or the US Department of Health and Human Services to step in and stop its implementation. "We’re waiting to see what the final [NCD] looks like before we make any inquiries into what the next steps would be," Sheives said.
However, industry insiders GenomeWeb spoke to believe that if CMS maintains the non-coverage language in the final NCD that could be the trigger to increase Congressional pressure.