Defining and characterizing the late-stage biopharmaceutical pipeline

To identify and describe biopharmaceuticals in late-stage development in the United States and to understand their implications for third-party payers.

"Biopharmaceutical" and biopharmaceuticals in late-stage US development (had completed Phase 2 or higher) were identified through reviews of literature, 4 drug-development databases, a clinical trial database, and informal telephone conversations with representatives of the US Food and Drug Administration, faculty at academic institutions, and manufacturers.

"Biopharmaceutical" was defined as "any biology-based therapeutic that structurally mimics compounds found within the body." This includes recombinant proteins, monoclonal and polyclonal antibodies, peptides, antisense oligonucleotides, therapeutic genes, and certain therapeutic vaccines. As of April 2003, there were 102 unique biopharmaceuticals in late-stage development for 156 indications in 36 disease categories, affecting at least 22 distinct physician specialties. Cancer agents are most common, with 30 agents (29%) targeting 62 indications (40%). Nearly 90% of the biopharmaceuticals require administration via injection or infusion, nearly 70% will require administration by a healthcare provider, and more than 60% will be administered in ambulatory care settings. All 22 physician specialties are affected by healthcare provider-administered biopharmaceuticals. Excluding cancer-related biopharmaceuticals, 60% of biopharmaceuticals and 54% of indications require chronic administration. Up to 81 biopharmaceuticals for 123 indications may be approved within the next 4 years (including secondary approvals).

The broad range of the late-stage US biopharmaceutical pipeline has significant implications for third-party payers due to their likely premium price, once approved, as well as novel logistical considerations. Payers must prepare for a wide range of clinical, administrative, delivery, and economic issues.