Is off-label prescribing completely prohibited? Can a drug be prescribed by doctors for a disease or condition for which FDA has not approved it and hence this condition is not listed on the label? It is presumed that this disease or condition is not explicitly listed as a contra-indication, i.e. when the drug is clearly stated to be avoided since it may be harmful.

  • In which country? It is a legal question, and regulated differently. Also, are you asking about the general possibility, or also who pays for the off label use, patient or insurance?
    – rumtscho
    Commented Sep 16, 2015 at 10:02
  • I would like to know what is the status and practice in your country?
    – rnso
    Commented Sep 16, 2015 at 14:05
  • 1
    @rnso - Your comment makes this a very broad question, as anyone in a different country will weigh in on their laws, which may or may not even be a true representation. If you can narrow it to a specific country and medication, it will help it remain open.
    – JohnP
    Commented Sep 16, 2015 at 14:35
  • 1
    I would mainly like to know what is its status in the USA?
    – rnso
    Commented Sep 16, 2015 at 14:47

1 Answer 1


Off-label prescribing of medicines - that is, prescribing medicines for indications, age groups or other circumstances for which they are not approved, is not prohibited by the FDA. FDA regulates approval of medicines for marketing, but it does not regulate the practice of medicine. Hence, once the medicine is on the market for certain indications it is up to physicians how they will describe them. (1,2).

However, this is not an unregulated field. The FDA does state that off-label use must be distinguished from clinical trials - which means when physicians prescribe medicines under off-label circumstances they are not experimenting on their patients. Furthermore, FDA stresses that this practice must benefit the patient and be in line with Good medical practice. In their own words (3):

Good medical practice and the best interests of the patient require that physicians use legally available drugs, biologics and devices according to their best knowledge and judgement. If physicians use a product for an indication not in the approved labeling, they have the responsibility to be well informed about the product, to base its use on firm scientific rationale and on sound medical evidence, and to maintain records of the product's use and effects. Use of a marketed product in this manner when the intent is the "practice of medicine" does not require the submission of an Investigational New Drug Application (IND), Investigational Device Exemption (IDE) or review by an Institutional Review Board (IRB). However, the institution at which the product will be used may, under its own authority, require IRB review or other institutional oversight.

Even though such practice isn't prohibited and is in fact sometimes necessary (1, 4), it still opens physicians to potential liability. Legal complaints are usually phrased as medical malpractice due to negligence or due to lack of an informed consent, which is recommended, but not mandatory (1, 2).

What physicians can do to protect themselves from liability is take care that their off label prescribing is (2):

  1. made with the patient’s knowledge that a drug is being prescribed for an off-label use;

  2. principally motivated by a desire to diagnose, treat and directly benefit the patient for whom a drug is prescribed;

  3. based on the doctor’s own expert medical opinion;

  4. supported by reputable peer reviewed literature reflecting sound scientific evidence;

  5. generally supported by the opinions of the physician’s local colleagues

The situation is, however, different with the industry - promotion of off label use of medicines is prohibited. FDA has issued a draft document on it's viewpoint on industry providing information about off label use of their medicines. The request for information must be unsolicited (i.e. the company or its permanent or temporary employees should not prompt these requirements), and whether the request is public or not, the information should be provided in one-on-one communication (5). Furthermore, this document states:

FDA has long taken the position that firms can respond to unsolicited requests for information about FDA-regulated medical products by providing truthful, balanced, non-misleading, and non-promotional scientific or medical information that is responsive to the specific request, even if responding to the request requires a firm to provide information on unapproved or uncleared indications or conditions of use.


  1. Ten Common Questions (and Their Answers) About Off-label Drug Use

  2. Physicians’ liability for off-label prescriptions

  3. FDA: "Off-Label" and Investigational Use Of Marketed Drugs, Biologics, and Medical Devices - Information Sheet

  4. FDA: Understanding Investigational Drugs and Off Label Use of Approved Drugs

  5. FDA: Guidance for Industry Responding to Unsolicited Requests for Off-Label Information About Prescription Drugs and Medical Devices

  • 2
    Since we are speaking about the FDA which is a regulatory authority for the USA the answer applies to regulations and practices of that country.
    – Lucky
    Commented Sep 18, 2015 at 11:51

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