NEW YORK (GenomeWeb) – Government and private payors should make greater use of databases and registries to determine if genomic testing helps personalize treatment for cancer patients and improves their outcomes, according to a commentary published today in Science by former National Cancer Institute Director Harold Varmus and the University of Michigan Law School's Rebecca Eisenberg.
Although academic centers and commercial firms are offering next-generation sequencing tests that can provide greater insights into a person's cancer, help them receive a targeted therapy, or guide them to a clinical trial, these tests are largely considered investigational and not paid for by most insurers, wrote Eisenberg and Varmus, who also runs a lab at Weill Cornell Medicine researching the molecular mechanisms of cancer.
"Reimbursement for these broader tests remains uncertain in the United States, leaving oncology caught in a 'Catch-22': Substantially more data are needed to evaluate clinical utility of such testing so that insurers can make rational decisions about coverage, but data collection on a large enough scale is impeded, in part, by uncertain reimbursement policies," they added.
Varmus and Eisenberg further noted that the US Food and Drug Administration is increasingly approving cancer drugs at an accelerated clip and are willing to wait for more definitive evidence from the post market setting, which in turn is shifting the line between clinical care and clinical investigation. However, when it comes to diagnostics, the agency has largely exercised enforcement discretion, leaving oversight responsibilities to the Centers for Medicare & Medicaid, which ensures analytical validity for lab tests, but not clinical validity.
While the FDA has approved genetic tests as companion diagnostics alongside drugs, which helps to garner evidence on clinical validity, this represents a minority of tests and large drug trials aren't possible to validate the use of the numerous rare genetic variants that occur in 1 percent of cancers.
"Accelerated approval inevitably comes at the cost of greater uncertainty about long-term safety and efficacy and a need for continued collection of relevant data. Meanwhile, as new products become available for use, caregivers begin to prescribe them, and patients are exposed to risks that have not been fully studied," the authors wrote. "The result is that insurers are paying for treatment that is, in effect, still under investigation, while withholding coverage of diagnostic tests that might show how to use these products more appropriately."
One solution, according to Varmus and Eisenberg, is that payors and test developers make greater use of a mechanism called coverage with evidence development (CED). CMS already makes use of CED as it sometimes agrees to pay for a new technology and approach within clinical trials or registries while it gathers evidence on whether it is "reasonable and necessary" for the Medicare population — lung volume reduction surgery for chronic obstructive lung disease, for example.
"CED could encourage patient participation in registries for studies designed to determine whether genomic testing is reasonable and necessary for the accurate diagnosis and appropriate treatment of cancer," they wrote.
To an extent, stakeholders in the genomics space have already launched registries for the purpose of gathering the kinds of outcomes data needed to sway payors.
CureOne (formerly called MED-C) is focused on building registries through which it aims to provide patients with access to quality NGS testing, collect data that can improve understanding of genetic markers in cancer, and track how genetic testing is being used in the real world and affecting patient outcomes.
Foundation Medicine, which markets a large NGS tumor profiling panel, is among the companies and academic cancer centers participating in CureOne's non-small cell lung cancer registry. This year, it partnered with the American Society of Clinical Oncology to help enroll patients into the Targeted Agent and Profiling Utilization Registry (TAPUR) study.
Medicare contractor Palmetto had shown interest in accepting outcomes data through registries in the context of covering genomic profiling tests for NSCLC, though it is not requiring that companies submit information to such databases at this time.
According to Varmus and Eisenberg, payors should be more willing to use the CED mechanism to gather clinical utility evidence on genomic testing in cancer because there is a compelling economic argument. While it's difficult to gauge the economic impact of NGS testing on patient outcomes or therapeutic benefit, the authors estimated that the cost of testing cancer patients would amount to around $1 billion per year, while the total cost of cancer care in the US in 2010 was $125 billion.
"Thus, the costs of more extensive testing would amount to roughly 1 percent of the total bill for cancer care," they wrote. "At those prices, the costs of testing seem justified if the tests bring about improvements in even a small percentage of patients."