Despite a doubling in the number of candidates in the cancer drug pipeline from 1990 through 2006, the overall US approval rate for cancer drugs remains low (8%). As the cures for cancers remain elusive, cancer treatment approaches—especially for hematologic, prostate and breast cancers—have evolved into chronic therapies with long-term disease management. To support this evolving paradigm, drug developers are focusing on orally bioavailable therapies (alone or in combination with other therapies) with easier-to-use dosing regimens.

This article discusses selected approaches to reformulations, key US reimbursement issues, recently identified molecular targets, preclinical considerations, newer dosing schedules and combined therapy regimens, and selected US regulatory strategies to increase speed to market.

Regulatory Focus 14:37-44, 2009.