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.

Defining and characterizing the late-stage biopharmaceutical pipeline.

OBJECTIVE: To identify and describe biopharmaceuticals in late-stage development in the United States and to understand their implications for third-party payers. STUDY DESIGN AND METHODS: "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. RESULTS: "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). CONCLUSIONS: 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.