Off Label Use of Drugs
Betting that most of you have not given much thought to the off label use of drugs. This means the use of drugs in a way that has not been approved by the FDA, and is not so uncommon in cancer care. In adjuvant treatment, the standards of care are exactly that=standard. But, in the treatment of Stage IV disease, it becomes just as much the art as the science of medicine, and skilled oncologists not infrequently reach outside the usual box. This is an editorial from the Journal of Clinical Oncology:
Off-Label Use of Oncology Drugs: The Need for
More Data and Then Some
David G. Pfister,Memorial Sloan-Kettering Cancer Center, New York, NY
Off-label drug use refers to the prescription of licensed drugs for clinical indications or in a manner different from that approved by the US Food and Drug Administration (FDA) and thus not included in the official FDA-approved labeling for the agents. Off-label drug use takes many forms, and versions of it occur in countries outside of the United States as well.
Use of drugs for a clinical indication, in a patient
population, through a route of administration, or with a dose not
specified in the FDA-approved labeling can all be considered off
label. Off-label use does not necessarily mean a lack of evidence demonstrating the efficacy and safety of the used agent; but the supporting evidence for different off-label indications may vary considerably both in extent and quality.
Although estimates vary, there is general agreement that off-label use of drugs to treat cancer is particularly common. For example, in 2005 the National Comprehensive Cancer Network estimated that 50% to 75% of drug or biologic therapy used to treat cancer in the United States was off label. The reasons are multifactorial. There is a spectrum of cancer types that may have shared biology and sensitivity to therapeutic agents and yet are different diseases in terms of the drug
approval mechanism. FDA approval requires a lengthy and expensive process, even for a single-disease indication. Once a drug is off patent, there is little enthusiasm in pharmaceutical circles to invest the resources to expand the labeled indications for an agent.
Pediatric oncology and rare cancers pose special challenges to the traditional drug-approval paradigm. Given the worrisome prognosis and potentially life-threatening nature of a cancer diagnosis, particularly when recurrent or metastatic, time is critical, and discussions of risks versus benefits differ from many other disease settings.