ABSTRACT
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. There is a relatively wide discrepancy in payer management practices with respect to oncology, perhaps more so than in any other therapy area. A complicating factor in demonstrating value and cost-effectiveness in oncology is that it is often deemed unethical to experiment with new drug treatments in earlier lines of therapy, where the largest benefits are likely to be demonstrated. The US market has generally been tolerant with respect to coverage of oncology drugs. In the US, orphan designation can be obtained for drugs or biological products under the Orphan Drug Act.
