Researchers: CMS Should Consider Becoming a Drug Evaluation Agency

Everyone in U.S. healthcare understands that a core function of the federal Centers for Medicare and Medicaid Services (CMS) has been, from its very inception in the 1960s as the Health Care Financing Administration (HCFA), the payment of medical claims from physician practices, hospitals, and other providers and patient care organizations. Over time, of course, CMS has inevitably become involved in such functions as evaluating care quality and the creation and development of alternative payment models (APMs), among other responsibilities.

But two healthcare researchers are suggesting that CMS should take on a broader role, including the approval of certain classes of drugs and of certain medical devices—even as the Food and Drug Administration (FDA) should maintain its fundamental role in approval drugs on the basis of their safety and efficacy.

Writing in The New England Journal of Medicine online on Aug. 19 in a Perspective article entitled “Turning CMS into a Health Technology Assessment Organization,” Peter J. Neumann, Sc.D., and Sean R. Tunis, M.D., MSc., write that “In 2001, HCFA’s name was changed to CMS, reflecting the fact that the agency did more than simply pay claims and was newly committed to improving services. As part of this change, CMS announced that in assessing new technologies, it would focus on whether adequate evidence existed to conclude that they improved net health outcomes for Medicare beneficiaries. Medicare also created a “coverage with evidence development” (CED) option to pay for promising technologies while requiring additional study to strengthen the available evidence,” the article’s authors write. Neumann is director of the Center for the Evaluation of Value and Risk in Health at the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center, and a professor of medicine in the Tufts University School of Medicine. Tunis is founder of the Center for Medical Technology Policy (CMTP) in Baltimore.

The researchers note that “Developments in recent years have compelled Medicare to further expand its role in conducting health technology assessment (HTA) — a systematic process to evaluate the evidence supporting technology deployment. In 2016, the FDA began granting breakthrough status to devices for serious illnesses (allowing for FDA authorization based on less-mature evidence), thereby expanding the need for Medicare to decide how to cover devices when faced with substantial uncertainty about their benefits and risks. In early 2021, the Trump administration issued final regulations that would extend automatic coverage for breakthrough devices approved by the FDA, granting national coverage for on-label uses for 4 years, after which CMS would determine future coverage status. The Biden administration, worried about paying for products despite insufficient evidence of their benefits and risks, rescinded the regulation, but committed to issuing a modified version of the rule (a proposed procedural notice on the new pathway was issued on June 22), which will further engage CMS in defining evidence requirements for medical device coverage,” they note.

The article’s authors recommend that CMS “develop a workforce that is commensurate with its growing responsibilities. Congress can appropriate resources for the agency to add staff with expertise in clinical research, trial design, epidemiology, and biostatistics.” At the same time, they recommend that CMS “improve coordination with the FDA, including by participating in earlier interagency discussions and interacting with product manufacturers about their clinical development programs to identify potential discrepancies between the agencies’ requirements related to end points and postmarketing requirements.”

And, they write, “CMS can improve coordination with the FDA, including by participating in earlier interagency discussions and interacting with product manufacturers about their clinical development programs to identify potential discrepancies between the agencies’ requirements related to end points and postmarketing commitments.”

In the end, the researchers write, “Assessing technologies has always made sense for a responsible payer seeking to ensure that new technologies work for the populations it serves. Recent developments are forcing Medicare to assume more formally the mantle of an HTA organization. We believe it should embrace the role and expand its capabilities to appropriately reflect the importance and magnitude of these responsibilities.”