When authorized medicinal products are not available: Possible alternatives to meet legitimate expectations of patients

Objectives: Authorized medicinal products placed on the market do not cover all therapeutic needs. In order to meet the patient's legitimate right, a physician can prescribe other pharmacological treatments in other ways: off-label prescriptions, compounded medicinal products, compassionate use of medicinal products and medicinal products authorized in foreign countries. This is a critical review of the actual regulations which govern these alternatives, with particular attention to the Italian situation. Key findings and summary: The legality of prescribing and dispensing approved drugs or devices for uses not included in their official labelling is sometimes a cause for concern and confusion. The public administration should always safeguard patients' rights and health, evaluating the appropriateness of medical prescriptions. Off-label use should be based on sound scientific evidence, expert medical judgement and published literature. In its attempt to avoid misuse, the public administration should not limit prescribing activity by issuing rigid the rules to be followed. The physician and the pharmacist have to be trained to assess the risks associated with a single therapy and/or absence of therapy to evaluate the best practice case by case.