The US biosimilars act: Challenges facing regulatory approval

Abstract

Ever since the signing of the US healthcare reform legislation, the Patient Protection and Affordable Care Act (PPAC), speculation as to what a US FDA biosimilar programme might look like has been rife. Now the FDA has published guidance documents on biosimilar product development, which has been described by Dr Janet Woodcock, MD, Director of the FDAs Center for Drug Evaluation and Research, as "an innovative approach to supporting the development of biosimilars at every step of the process." Nevertheless, these initial guidelines are rather general in nature, so the looming question remains as to what extent the US biosimilar pathway will represent a viable route to market.The pathway for resolution of inevitable patent disputes is long and torturous and it seems that it will inevitably result in delays and litigation; the impact and implications of this represents a key consideration in the choice of the regulatory pathway. Thus, if there are no benefits in terms of reduced data requirement, a more rapid route to market and benefits to market access with the biosimilar route, then one really would have to question the value of this route.As far as whether the biosimilar pathway will offer reduced data requirements, the jury is still out. Initial indications suggest that the FDA review divisions might initially take a more conservative stand, but one might expect that, as policy becomes more transparent and with the publication of the guidelines, a more pragmatic position might emerge.One of the most controversial concepts raised by the Biologics Price Competition and Innovation Act of 2009 (BPCI) is that of interchangeability between the innovator and the biosimilar product. This presents a very high bar and in many instances may simply not be achievable or financially viable. Yet, without interchangeable status, a biosimilar will be no different to any other biological approved through a standalone route and, inevitably, an extensive marketing effort would be required to achieve any meaningful market penetration. On the other hand, a standalone application may not offer a viable alternative either, as this would require a more conventional and graduated development programme with the need for extensive non-clinical and phase II data. Furthermore, the inability to extrapolate between indications would require large comparative trials for each indication.Biosimilars are an essential and inevitable part of enhancing patient access to life-saving medications, and if nations want to maximize the value of healthcare spend, bringing health and well-being to their citizens, biosimilars are a necessity and their exclusion an unaffordable luxury. Thus, one would anticipate that, inevitably, despite the debate, the BPCI will spawn an attractive US biosimilar approval pathway. Certainly, there are many sponsors of biosimilar products banking on just that and the recently published guidelines suggest that the FDA will ultimately apply sound scientific principles, which is, undoubtedly, the optimal way forward. © 2012 Springer International Publishing AG. All rights reserved.