ABSTRACT
This chapter discusses the complex areas of oncology and orphan drugs. Although subject to the same formal payer approval requirements, both categories typically benefit from more latitude in decision making. Payer willingness to intervene has gradually increased under cost and economic pressures. Oncology and orphan drugs are both often mentioned in the context of high drug prices and related concerns. Both categories of drugs concern high unmet needs for targeted patient populations, but are under continuous public scrutiny due to their high cost to healthcare systems and some individual patients. Payers in the US have been generally hesitant to take very restrictive coverage decisions for Food and Drug Administration (FDA) or compendia supported applications of drugs. The unmet need in oncology is still huge, with many cancer types still suffering from poor survival rates. It is unfortunate that oncologists have chosen to criticize drug companies rather than praise the truly remarkable benefits of life-saving drugs in Chronic Myeloid Leukemia (CML).
