Coverage Guideline for Orphan Drugs and Off-Label and Labeled Use of FDA Approved Drugs

Triple S Salud has a procedure designed to ensure the evaluation of requests for coverage of covered medications recommended for orphan drugs, off- labeled and expanded access drugs indications that are prescribed for “medically accepted indications”. Off-label or “unlabeled” drug use is the use of a drug approved by the U.S. Food and Drug Administration (FDA) for other uses that are not included in approved labeling (may be for oncology or non oncology use). The FDA approves drugs for specific indications that are included in the drug’s labeling. When a drug is used for an indication other than those specifically included in the labeling, it is referred to as an off-label use. Many off-label uses are effective, well documented in the literature, and widely used. An “orphan drug” is a product that treats a rare disease (e.g., affecting fewer than 200,000 Americans). Products have FDA orphan drug approval when they meet the orphan drug criteria established by the FDA. The intent of the Orphan Drug Act (ODA) is to stimulate the research, development, and approval of products that treat rare diseases. Orphan designation can be obtained prior to submission of a marketing application. The safety and efficacy of the drug must be established through clinical studies. If the designated product meets the standard FDA regulatory requirements and process for obtaining marketing approval, it is given an FDA approved orphan drug designation status (i.e., “Designated/Approved”). Over 1,400 drugs and biologics have been designated as orphan drugs and over 250 have been approved for marketing. Expanded access refers to the use of an investigational new drug (IND) outside of a clinical trial by patients with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress. This type of access may be available, in accordance with United States Food and Drug Administration (FDA) regulations, when it is clear that patients may benefit from the treatment, the therapy can be given safely outside the clinical trial setting, no other alternative therapy is available, and the drug developer agrees to provide access to the drug. The FDA refers to such a program as an expanded access program (EAP). [1] EAPs can be used in a wide range of therapeutic areas including HIV/AIDS and other infectious diseases, cancer, rare diseases, and cardiovascular diseases. There are several types of EAPs allowed in the United States. Treatment protocols and treatment INDs provide large numbers of patients access to investigational drugs. A single-patient IND is a request from a physician to the FDA that an individual patient be allowed access to an investigational drug on an emergency or compassionate use basis. <a id="