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Report on the Evidence Regarding Off-Label Indications for Targeted Therapies used in Cancer Treatment

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The term "targeted therapies" refers to antineoplastic treatments designed not to kill cells but, more precisely, to attack growth factors, cell surface receptors, and intracellular proteins that mediate the cancer cell’s ability to proliferate, grow, or evade cell death. Examples of targeted therapies include small molecule inhibitors, monoclonal antibodies, and conjugated agents. Intended to damage or destroy cancer cells while minimizing the effects on normal cells, several targeted therapies have been successfully brought into routine clinical use, with approval from the U.S. Food and Drug Administration (FDA) for specific indications. The use of these FDA-approved agents has expanded to include multiple indications other than those for which they received FDA approval (i.e., "off-label indications"); in current clinical practice in oncology, off-label prescribing is common.

The primary purpose of this technology assessment is to evaluate the state of the evidence for/against the use of selected targeted therapies for off-label indications. Secondarily, the report also considers the practicality of the traditional systematic review approach, when applied to examine the evidence in rapidly evolving therapeutic areas such as targeted therapies for various cancers.