Research on characteristics of the biopharmaceutical pipeline and on changes since 2003 includes a description of new features and recommendations for health policy decision makers.
: To quantify and characterize biopharmaceutical agents and new indications in late-stage development in the United States as of May 2006.
: Review of drug development databases and other secondary sources.
: Biopharmaceutical was defined as â€œany biology-based therapeutic that structurally mimics compounds found within the body.â€ 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.
: 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).
: 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.
(Am J Manag Care. 2008;14(4):226-229)
The growth of the biopharmaceutical pipeline and consequent approvals has increased each year from the first approval of human insulin in 1982. The growth of biopharmaceutical approvals is predicted to grow at a rate of 16% to 30%, compared with about a 4% growth of traditional small-molecule biopharmaceuticals.1-3 In 2003, a study4 was undertaken to quantify and characterize the biopharmaceutical pipeline as a means to prepare third-party payers for the unique challenges posed by availability of these agents, including appropriate use, per-patient and aggregate costs, utilization management, site of care issues, product delivery, and incorporation of these agents into existing benefit and information technology structures. The present study was undertaken to answer several questions. Like the development of traditional small-molecule pharmaceuticals, do we see a similar leveling off in the biopharmaceutical pipeline? If not, are there characteristics beyond those identified in 2003 that require the attention of third-party payers? Finally, is there a profile or a growing trend within the biopharmaceutical pipeline that we can identify early to proactively prepare for the landscape of the near-term future biopharmaceutical market?
In this study, biopharmaceutical is defined as “any biology-based therapeutic that structurally mimics compounds found within the body.”4(pS124) This includes recombinant proteins, monoclonal and polyclonal antibodies, peptides, antisense oligonucleotides, therapeutic genes, and certain therapeutic vaccines. The basis for the definition is previously described.4 Agents were defined as late-stage biopharmaceuticals if they had been tested in a completed phase 2 clinical trial, were in a phase 2/3 or 3 clinical trial, or had been submitted for regulatory approval by the US Food and Drug Administration (FDA) for an unapproved indication as of May 2006. The data variables for this study include generic and brand names, manufacturer, stage of development, class of agent, target indication and disease, probable method of administration (eg, subcutaneous, intravenous, etc), physician specialty likely to use the agent for the indication, expected setting of administration (eg, ambulatory care, hospital inpatient, etc), proposed frequency of administration (eg, acute, chronic, etc), and whether the agent was already FDA approved for 1 or more other indications. Because of the frequent incongruence of information in different sources used to collect these data, numerous sources were consulted.
As of May 2006, there were 111 unique biopharmaceuticals in late-stage development in the United States targeting 190 late-stage indications in 38 disease categories. Of 111 unique agents, 87 are new molecular entities, 24 are already approved by the FDA for other indications, and 25 have completed Phase 3 trials.
Cancer (including primary therapy and supportive care applications) is the most common disease category target for biopharmaceuticals (). Immune-mediated inflammatory disorders constitute the second largest disease target (by category); more than 20% of agents in the pipeline target a range of these inflammatory diseases. Examples include rheumatoid arthritis, Crohn’s disease, ulcerative colitis, psoriasis, type 1 diabetes mellitus, and multiple sclerosis. Like cancer, immune-mediated inflammatory disease represents a significant clinical and economic burden to the US healthcare system.5
Although the number and percentage of already FDAapproved biopharmaceuticals in the pipeline have not changed significantly, the number of indications they represent and the percentage of pipeline indications represented by these agents increased from 37 indications in 2003 (24% of pipeline indications) to 70 indications in 2006 (37% of pipeline indications), which is an 89% increase in indications and a 54% increase in the proportion of the pipeline. In addition, there is a greater percentage of “follow-on” indications in the pipeline in 2006 compared with 2003.
There is an increase in the number of agents and indications targeting cancer. In 2003, there were 30 cancer-related biopharmaceuticals (29% of pipeline agents) in late-stage development for 62 indications (40% of pipeline indications), while in 2006 there were 43 biopharmaceuticals (39% of pipeline agents) for 83 indications (44% of pipeline indications). In addition, the gap between cancer and other disease targets in the pipeline is widening. The second most common disease targets were infection in 2003 and blood disorders in 2006. There were 21 more cancer-related agents (21% of pipeline agents) than infection-related agents in 2003 versus 31 more cancer-related agents (28% of pipeline agents) than blood disorder agents in 2006 (an increase in the gap of 48%).
2. Simon F. Market access for biopharmaceuticals: new challenges. Health Aff (Millwood). 2006;25(5):1363-1370.
4. Nagle PC, Lugo TF, Nicita CA. Defining and characterizing the latestage biopharmaceutical pipeline. Am J Manag Care. 2003;9(6)(suppl):S124-S135.
6. Cowen and Company, LLC. Biotechnology: Industry Outlook: How to Survive a Biotech Bear Market. New York, NY: Cowen & Co LLC; July 2006.
8. Humphreys A. Med Ad News 200 - Worldâ€™s best selling medicines. Med Ad News. June 2006;25(6):1, 20-46.