{ "id": "R45792", "type": "CRS Report", "typeId": "R", "number": "R45792", "active": true, "source": "CRSReports.Congress.gov, EveryCRSReport.com", "versions": [ { "summary": null, "sourceLink": "https://crsreports.congress.gov/product/details?prodcode=R45792", "source_dir": "crsreports.congress.gov", "type": "CRS Report", "formats": [ { "sha1": "f1727385c1e006f333cb47ddc434fbe18bafa9ce", "format": "PDF", "url": "https://crsreports.congress.gov/product/pdf/R/R45792/2", "filename": "files/2021-02-23_R45792_f1727385c1e006f333cb47ddc434fbe18bafa9ce.pdf" }, { "format": "HTML", "filename": "files/2021-02-23_R45792_f1727385c1e006f333cb47ddc434fbe18bafa9ce.html" } ], "title": "Off-Label Use of Prescription Drugs", "source": "CRSReports.Congress.gov", "retrieved": "2021-03-12T04:03:10.792340", "date": "2021-02-23", "typeId": "R", "id": "R45792_2_2021-02-23", "active": true }, { "source": "EveryCRSReport.com", "id": 601438, "date": "2019-07-01", "retrieved": "2019-12-20T17:37:59.928012", "title": "Off-Label Use of Prescription Drugs", "summary": "When the Food and Drug Administration (FDA) approves a drug for sale in the United States, the approval includes a section entitled \u201cIndications for Use.\u201d This section lists the one or more diseases, conditions, or symptoms for which the drug\u2019s sponsor (usually the manufacturer) has provided, to FDA\u2019s satisfaction, evidence in support of the drug\u2019s safety and effectiveness. FDA approval is also based on its review of the drug\u2019s dosage, packaging, manufacturing plan, and labeling. Before changing any of those elements, the sponsor must inform, and usually receive permission from, FDA.\nIn essence, FDA regulates all approval and post-approval aspects of a drug product. But FDA traditionally has not regulated the practice of medicine. Physicians, therefore, may prescribe an FDA-approved drug for indications that FDA has not reviewed for safety and effectiveness. Those uses, furthermore, are not addressed in the labeling information regarding, among other things, dosing, warnings about interactions with other drugs, and possible adverse events.\nHow Are Off-Label Prescription Drugs Used?\nPrescribing for so-called off-label uses can be accepted medical practice, often reflecting cutting-edge clinical expertise. For example, this is the case with oncology drug use, more than half of which is off-label. Off-label prescribing can be a reasonable choice when labeling overlooks certain populations\u2014for example, when a drug tested in adults is prescribed to children. A drug may be used off-label when it was tested for the treatment of one disease and prescribed in an attempt to prevent or treat another, when it was tested at one dose and used at higher or lower doses, or when it was tested in an eight-week trial and prescribed for long-term use. Estimates for how common off-label prescriptions are in the United States are hardly precise. Credible researchers have estimated they make up as little as 12% and as much as 38% of doctor-office prescriptions.\nWhat Are the Risks of Off-Label Prescriptions?\nPrescriptions for off-label uses of FDA-approved drugs are made without the benefit of an FDA-reviewed analysis of safety and effectiveness data. Physicians may resort to such prescribing to take advantage of new ideas and treatment approaches when available information to support them is inadequate. However, despite the potential risks associated with off-label uses, efforts to prohibit such uses might hurt the public. Some off-label prescribing may result because manufacturers have chosen not to invest the resources needed to have FDA add indications to the drug\u2019s approval and labeling.\nA worst-case scenario for the nation\u2019s health would be the widespread acceptance of a drug for an off-label use that sufficient research would have revealed to be ineffective, unsafe, or both. Aside from the drug\u2019s direct harm, the time spent waiting to see whether it worked would have been time not spent exploring other treatment options.\nUnchecked off-label prescribing may also threaten the FDA gold standard of drug approval. If clinicians had already accepted a new use into practice through off-label prescribing, a manufacturer may choose to not invest resources to go through clinical trials and the FDA process to win approval.\nAlthough manufacturers do share information on off-label uses, courts have sometimes found they had overstepped allowable bounds. Congress has given permission for limited sharing. Are there other ways to share clinical information that do not put the public\u2019s health or FDA\u2019s authority at risk?\nWhat Role Can Congress Play in the Use of Off-Label Prescriptions?\nHow might Congress, in its legislative or oversight roles, consider the use of off-label drugs to protect the public\u2019s health? Legislators and health analysts have suggested both restrictive and permissive actions regarding off-label use. Ideas\u2014some of which conflict with others\u2014include\ndisclosure to patients;\ndata collection, availability, and analysis;\ndissemination of clinical data;\nlinking reimbursement and coverage to evidence of safety and effectiveness;\nclinical research and research transparency;\nclinical guidance;\ncongressional oversight through the Government Accountability Office, the Federal Trade Commission, and the Department of Health and Human Services; and\nconsideration of other countries\u2019 approaches to off-label use.\nSome actions would require federal legislation. Other proposals would involve actions by other entities, such as state authorities and professional organizations, which Congress could urge.", "type": "CRS Report", "typeId": "REPORTS", "active": true, "formats": [ { "format": "HTML", "encoding": "utf-8", "url": "https://www.crs.gov/Reports/R45792", "sha1": "12dbf4d760f1bfc692ef69839239d648c93dcb4e", "filename": "files/20190701_R45792_12dbf4d760f1bfc692ef69839239d648c93dcb4e.html", "images": {} }, { "format": "PDF", "encoding": null, "url": "https://www.crs.gov/Reports/pdf/R45792", "sha1": "54ec8a460bad8e29325f8e7511c0c98ace860b1b", "filename": "files/20190701_R45792_54ec8a460bad8e29325f8e7511c0c98ace860b1b.pdf", "images": {} } ], "topics": [] } ], "topics": [ "Health Policy" ] }