Characteristics of and trends in the late-stage biopharmaceutical pipeline.

Objective: To quantify and characterize biopharmaceutical agents and new indications in late-stage development in the United States as of May 2006.

Study Design: Review of drug development databases and other secondary sources.

Methods: Biopharmaceutical was defined as "any biology-based therapeutic that structurally mimics compounds found within the body." Unique biopharmaceuticals, including new molecular entities or new indications in phase 2 or higher development, were identified and characterized through reviews of the literature, 5 drug development databases, a clinical trial database, and telephone inquiries with manufacturers.

Results: As of May 2006, there were 111 unique biopharmaceuticals in late-stage development for 190 indications. Of 111 unique agents in the pipeline, 87 are new molecular entities, and 24 are already approved for other indications. Overall, 38 disease categories were targeted, and at least 33 physician specialties are likely to be affected. The greatest proportion of agents (43 biopharmaceuticals and 83 indications) target cancer. More than 70% of agents in the pipeline will require administration by a healthcare provider. More than 50% of the indications in the pipeline will require long-term (chronic) treatment (defined as >1 year and excludes cancer).

Conclusions: The steady growth of the US biopharmaceutical pipeline and consequent anticipated near-term approvals will increasingly affect third-party portfolio decision making. Cost of therapy, identifying the right drug for the right patient, and outcomes-based value should drive that decision process.