Enter at Your Own Risk: FDA Draft Guidance Highlights the Uncertainty in the Abbreviated Biosimilar Approval Pathway

By 2015, sales of biosimilars in the United States are expected to reach as high as $2.6 billion. Recognizing this market opportunity, biotech companies of all sizes are devoting significant resources to developing these biologic products, which are novel yet dependent on currently approved biologic drug products (at least in part). Following the passage of the Biologics Price Competition and Innovation Act (BPCIA) on March 23, 2010, an entity seeking to bring a biosimilar product to market now has two pathways to consider for securing Food and Drug Administration (FDA) approval. One pathway allows the biosimilar applicant (BA) to seek FDA approval of the product as a new biologic by filing a biologic license application (BLA) under § 351(a) of the Public Health Service Act (PHSA). Alternatively, the BA can follow the newly enacted abbreviated pathway under PHSA § 351(k), which created a new approval pathway for biologics that the FDA determines are “biosimilar” to a BLA-approved reference product (RP). This second option has been available since the passage of the BPCIA on March 23, 2010, yet the FDA still awaits the filing of the first § 351(k) application (referred to as abbreviated biologic license application – “ABLA”).

The statutory language of the BPCIA outlines the structure of the pathway, but contains minimal guidance on the standards used by the FDA to determine biosimilarity as required for ABLA approval. Faced with this uncertainty, it comes as no surprise that BAs have been hesitant to test the ABLA approval pathway. In a first step toward addressing this uncertainty, on February 9, 2012, the FDA released three draft guidance documents on the development of biosimilar products. These guidance documents were published in the Federal Register on February 15, 2012, with a 60-day period for comment ending April 16, 2012. Commentary and reaction to these long-awaited guidances have been mostly negative, with common criticisms being that these supposed “guidances” provide only broad conceptual ideas, lacking any sufficient detail to set BA expectations, and do not resolve any uncertainty over the FDA’s decision for awarding ABLA approval. Despite the lack of explicit guidance in the documents, the FDA does provide some clarity regarding the most pressing of issues facing BAs in the early stages of biosimilar development. Whether intentional or not, the FDA message that manifests from this clarity is that there is little to gain, but much to lose, by following the ABLA pathway.