Coverage Policy and Use Beyond the FDA-Approved Label
Authors: William T. McGivney
Journal: Journal of the National Comprehensive Cancer Network (2006)

Coverage Policy and Use Beyond the FDA-Approved LabelThe issue of insurance coverage for the use of drugs and biologics in cancer care for indications beyond Food and Drug Administration (FDA)-approved labeling is one that has been important to the oncology community since interferon was approved for use in hairy cell leukemia in the late 1980s.Today, the research pipelines of the pharmaceutical and biotech industries are replete with innovative and promising agents to treat solid tumors and hematologic malignancies and for supportive care.The approval and use of these important new therapies usually come with significant price tags in addition to improved therapeutic indices.Recent articles 1,2 focus on the added cost created by integrating new agents into existing regimens.Thus, introducing new agents into practice coincides with a resurgent and growing concern about the rate that health care expenditures rise in both the public and private sectors, with particular emphasis in the Medicare program.This commentary provides historical perspective on the issue of payment for uses beyond the FDA label, describes the issue within the context of today's environment, discusses related issues that may impact access to improved therapies, and describes the NCCN's response.The introduction of interferon in the 1980s focused the attention of payors on an already common practice in oncology.This biologic agent was approved for the aforementioned "orphan" indication but was quickly also studied and used in patients with a range of solid tumor types and hematologic malignancies.Insurers morphing into managed care companies began to assert their new orientation and managed to begin denying coverage for many of the new uses.Ultimately, this scenario resulted in the passage of legislation, now in 40 states, that requires insurance plans to cover uses beyond those approved by the FDA if such indications were recognized by 3 compendia: the USP-DI, The American Hospital Formulary Service Compendium, and AMA Drug Evaluations.In 1993 an amendment to the federal Omnibus Budget Reconciliation Act required the then Health Care Financing Administration to develop a regulation that was consistent with state laws regarding the use of drug compendia in both national and locoregional coverage policies and decisions.Notwithstanding these state and federal laws, use beyond FDA-approved labeling remained a major and contentious issue during much of the 1990s.The level of contention subsided as health care plan managers decided that the adverse public reaction to treatment denials, concern of major employercustomers, and large punitive legal judgments warranted a more hands-off approach and less intrusion into the decisions made by physicians and patients.However, the issue was raised anew in January 2004 in The New York Times 3 when a senior official at the Centers for Medicare and Medicaid Services (CMS) indicated that CMS was considering becoming more limiting in its coverage for use beyond FDA-approved labeling.Clearly, CMS, charged with the huge task of implementing the Medicare Drug Benefit, was facing intense pressure to manage the costs of that benefit.After negative reaction, the trial balloon floated in The New York Times article was purposefully deflated.However, although CMS as a national decision maker deferred, one or two Medicare in- Commentary