As fine-chemical producers, custom manufacturers, and pharmaceutical companies gathered this week for Informex in Anaheim, California, one observation stands out: for all the inroads that biologic-based drugs have made, the pharmaceutical industry remains a small-molecule marketplace.
Current and market projections illustrate this reality. The global market for biologic-based prescription drugs was estimated at $157 billion in 2011, according to the IMS Institute for Healthcare Informatics. IMS defines “biologics” as single-identified components, whole cells, and some forms of polymers that may be purified from human, animal, plant, or microogranism sources and produced by recombinant DNA technology or chemically synthesized (i.e., for shorter-chain biologics). By 2016, the global biologics market is expected to reach between $200 billion and $210 billion, representing approximately 17% of the projected nearly $1.2-trillion pharmaceutical market. Although the share of biologic-based drugs is rising, the preponderance of drugs will still be small molecules.
These projections also are consistent with another metric, new drug approvals. In 2012, the industry experienced a 16-year high in new drug approvals (i.e., new molecule entities [NMEs] and biologic license applications [BLAs]) as approved by FDA’s Center for Drug Research and Evaluation (CDER). In 2012, FDA’s CDER approved 39 new drugs: 34 NMEs and only 5 BLAs. Biologic-based drugs, therefore, accounted for nearly 13% of new drug approvals in 2012.
Clinical efficacy, rather than modality (small or large molecule) is the determinant of which drug substance is most suited for clinical development and eventual commercialization. The targeted nature of biologic-based drugs, such as monoclonal antibodies, makes them attractive drug candidates, particularly for difficult-to-treat diseases, such as cancer and neurological disorders, for which there is unmet medical need. For other diseases, however, current therapies, including a slate of newly entered generic drugs, to treat more “conventional” diseases, such as cardiovascular disease and related conditions (i.e., hypertension and lipid modulation) may suffice. Other factors, such as cost and the method of drug administration (oral versus parenteral delivery), which influence patient compliance and preference, also are issues that cannot be overlooked in the small-molecule versus large-molecule debate.
Biologic-based drugs are certainly an important component of pharmaceutical development, but their small-molecule brethen will continue, at least in the near term, to dominate drug development and commercialization.