Protecting Access to Off-Label Drug Use

In investigations, the Department of Justice (DOJ) advanced a legal theory that could greatly affect “off-label” use of Food and Drug Administration (FDA)-approved medications that entail criminal violations of the Food Drug and Cosmetic Act (FDCA). Off-label use occurs when an approved drug is prescribed in a way that it was not FDA-approved for, but can provide benefits to patients, and is commonly used to treat cancer.  Although the current DOJ investigations are directed at gender-affirming care, the theory could apply  much more broadly. The DOJ has argued that the FDCA's criminal misbranding and distribution prohibitions reach healthcare providers who purchase, store, or administer FDA-approved drugs prescribed for off-label uses.

ACS CAN joined the American Society of Clinical Oncology (ASCO) and other public health groups in filing amicus briefs in two parallel cases, Child Advocate for Rhode Island v. USA and Children's Hospital d/b/a Boston Children's Hospital v. DOJ.  The amicus briefs make three major points: 1) the FDCA does not regulate the practice of medicine, and off-label prescribing is protected by statute and court precedents; 2) off-label prescribing is critical to patient care where adequate alternatives do not exist and is often the standard of care, but the government's theory would criminalize routine treatment across medicine—from pediatric and geriatric oncology to multiple other conditions; and 3) the federal government itself reimburses off-label drug use through the Centers for Medicare & Medicaid Services (“CMS”) approved compendia as authoritative sources for Medicare coverage, so the government cannot criminalize practices it actively reimburses.