Clinical Studies for Biosimilars

Gillian Woollett, MA, DPhil: Do you see a point ever where we need no clinical studies whatsoever? And this can be a very short answer—no clinical studies whatsoever for a biosimilar to be approved, safely available, and used in the United States. And I’ll just go in order.

Kashyap Patel, MD: Based on history, I told you that we don’t use an FDA-approved process for every use in clinical practice. I think having the biosimilar in the market for extrapolation designation would allow me to use that drug in spite of not having enough studies.

Juliana Reed: I think the science will continue to evolve. I think it’s very important that it’s not a one-size-fits-all concept. The regulatory agencies will evolve, but they won’t lower standards, which is extremely important for this space. But I think the education and the physician expectation that there’s clinical data, we need to evolve that as well.

Carlos Sattler, MD: I think we will get there. Again, let’s remember why clinical studies are required. They’re required to remove any residual uncertainty about the biosimilarity of a proposed biosimilar to the reference product. If that residual uncertainty has been taken care of, if you may, through other earlier steps in the biosimilar development process, whether it be through analytical comparability or pharmacokinetic studies, then there wouldn’t be a need.

And in fact, this month* a pegfilgrastim product was approved based on a large pharmacokinetic study. It was not based on a clinical study. So analytics, pharmacokinetics led to an FDA approval of a biosimilar, pegfilgrastim in this case. So it has happened. And as we evolve our techniques, as we understand better the relationship between structure and activity or function, then there may be less of a need for clinical studies.

Gillian Woollett, MA, DPhil: And to loop back right to the very beginning, Janet Woodcock, MD, testified in 2007 that when using comparability in support of manufacturing changes, less than 1 in 50, 1 in 100 times when comparability is used are any clinical studies required at all. So it would appear based on the regulatory science already in use at the FDA, that we may, maybe even in our lifetimes, reach that point.

*as of the date of filming.