Biosimilars Regulation in the US: The Challenges

Published on: 
BioPharm International, BioPharm International-11-01-2010, Volume 23, Issue 11
Pages: 40–45

The pathway for biosimilar approval in the US has been set. But are US patients too far behind Europe?

On March 21, 2010, the US House of Representatives passed the landmark Patient Protection and Affordable Care Act, which includes the Biologics Price Competition and Innovation Act. Finally, a path has been set for the approval of biosimilars through the US FDA by an abbreviated biologics license application that, in theory, should broaden patient access to these new drugs. As stated by Jim Greenwood, Biotechnology Industry Organization (BIO) president and CEO, "...patients living with debilitating diseases will have expanded access to safe and effective cutting-edge medical therapies at lower costs."

Philip Ridley-Smith

But how true will Greenwood's statement ring in the years to come?


Some might say it has taken too long and will probably take even longer for US patients to benefit. With recent announcements from across the pond such as the approval of Nivestim (filgrastim) in Europe, a biosimilar to Amgen's very successful Neupogen, US patients may question the delay. Nivestim is the fifth European approval of a biosimilar of Neupogen. The EMA is a step ahead, leading the way forward with a regulatory framework for the emerging biosimilars market that already is being put into practice. The results can clearly be seen with the approval of five biosimilars to Neupogen alone. Is the new act welcome? Of course, but it has fallen short of providing a streamlined approval pathway that broadens access to cutting-edge medical therapies.

Countless issues have been raised with the dawn of biosimilars, such as patient safety, comparable efficacy, and the substitution of originator biological products, to name a few. Patient safety should be paramount and should remain so. It is well known that variations in the quality and efficacy of biologics can be seen at different production facilities despite every effort being made in process transfer. With this in mind, biosimilar production is made even more complex when processes are not readily disclosed for originator biological products. Therefore, safety and efficacy testing become paramount through the costly and time-consuming route of clinical trials.


But where does this leave the US patient? In Europe, patients already can take advantage of the supply of biosimilars, as can patients in many developing countries around the world. In the near term, it would appear that US patients may have to access more advanced affordable therapies overseas, which could put US patients at risk.

The goal of the act must be to provide the impetus to make a competitive market, which encourages more affordable medicines in a dynamic, innovative, and fast growing sector of the healthcare industry. However, the competition will be muted because only a few companies will have the financial pockets deep enough to fund the regulatory requirements to deliver a biosimilar to market. In addition, any targeted originator product market will need to be of a size capable of providing acceptable returns on the time and investment. Consequently, smaller product markets may well not see accessibility increase. Of course, companies are already looking at solutions to the significant investment required. Joint ventures, partnerships, and alliances will no doubt proliferate as shown by the likes of Teva and Lonza, which have an alliance to exploit their relevant expertise to develop biosimilars.

Ultimately, regulators must remember that the aim is not only to deliver safe and efficacious medicines but also to provide a framework for affordable and equitable healthcare. However, what the biosimilars market has put into perspective is the decreasing return on R&D investment to provide new and innovative drugs. That is still the biggest challenge that we all face in healthcare from the innovator to the patient, and regulators have a huge and positive role to play in this.

Philip Ridley-Smith is the business development and marketing manager at RecipharmCobra Biologics, Keele, UK, +44 (0)208 246 5895,