Oral chemotherapeutic drugs have been available for decades and include the familiar agents chlorambucil, cyclophosphamide, methotrexate, and 6-mercaptopurine (6-MP). However, the past 4 years has seen an accelerating expansion of the development of oral anticancer drugs, including oral cytotoxic agents, small molecule inhibitors directed at cell surface receptors and other proteins, and other agents targeted at the tumor microenvironment.
Capecitabine received FDA approval in April 1998, ushering in a new era of oral chemotherapy. Capecitabine approval was followed by FDA approval of a number of oral small molecule inhibitors of a variety of defined targets, including imatinib in 2001, gefitinib in 2003, and erlotinib in 2004. Five more new oral agents were then approved in the 7 months between December 2005 and July 2006. Lapitinib and nilotinib were approved in 2007. Experts anticipate that this trend will continue in the coming years. They further estimate that more than one quarter of the 400 antineoplastic agents now in the pipeline are planned as oral drugs.
Compared with the oral chemotherapy drugs available before 1996, these newer drugs, consistent with their parenteral contemporaries, are considered costly. For example, the estimated yearly cost of lenalidomide for a patient with multiple myeloma is $74,000, and, depending on dosage, the yearly cost of imatinib for patients with chronic myelogenous leukemia (CML) ranges from $29,000 to $57,000. Imatinib accounts for the largest percentage of spending on oral chemotherapy, ranging from 29% to 39%, depending on whether pharmacy benefits are provided by an insured health plan or self-insured employer. The availability of these new drugs has had an immediate impact on pharmacy budgets. Spending on oral chemotherapy drugs, while still a small proportion of total pharmacy benefit costs, has more than doubled between 2002 and 2006, from 0.3% to 0.7%.1
Anticancer agents, including oral drugs, can be broadly categorized as chemotherapy, which in the past generally referred to cytotoxic agents, and biologic therapy, which generally referred to therapy targeted specifically at cell surface proteins or pathways that are relatively specific to cancer biologic pathways. Biologic therapy is also often referred to as targeted therapy. However, chemotherapy has also been used as an inclusive term encompassing all antineoplastic therapies, and the distinctions between targeted and non-targeted and biologic therapy versus chemotherapy would, at this time, appear to be somewhat artificial. In this discussion, the term chemotherapy is used generally to describe both cytotoxic and biologic therapy.
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