Off-label drug use in the treatment of polycystic ovary syndrome

Polycystic ovary syndrome (PCOS) is a complex lifelong disorder with an etiology and pathophysiology that is not yet entirely understood. Women with PCOS have clinical presentations that may vary from adolescence to menopause, including menstrual irregularity/anovulation and symptoms of hyperandrogenism, such as acne and hirsutism. Over a lifetime, treatment needs and requirements can change. Unfortunately, there are no Food and Drug Administration-approved medications that are approved solely for the purpose of PCOS, but the symptoms and presentation of PCOS are often amenable to several approved agents, such as oral contraceptives for the indication of acne and clomiphene citrate for the indication of induction of ovulation. However, to meet the needs of women with PCOS, off-label use of medications has flourished. This review explores the data for those agents that do not carry an indication for PCOS but have been used for treating the signs and symptoms of PCOS.