Today's FDA publication
of the biosimilars user fee performance goals letter represents an important step forward in the years-long effort to establish a science-based regulatory pathway for these medicines. You can read our statement about it here
But besides that, it also is an example of how many puzzle pieces need to be assembled in order to turn a Congressional bill into a reality for patients.
For years - at least as long as I have worked here - PhRMA has advocated for a biosimilars pathway that ensures that these products will be proven to be both safe and effective. Unlike conventional small molecule medicines, biologics aren't chemically replicable, so it is essential that biosimilars be studied thoroughly, in a step-wise process that includes clinical trials, to ensure they are safe and efficacious. We've been consistent about this; back in 2009, we wrote the following in a statement: "Such a pathway must protect patient safety through sound science."
Through a provision in the Affordable Care Act, the establishment of a biosimilars pathway became law - but that didn't mean that the work was completely done.
Now, we're working with FDA and many other stakeholders to help ensure that implementation of that provision is done right, based on the best available science and in a way that serves patients well. And we think that the performance goals letter published today is a sign that we're heading in the right direction as we continue to move through the guidance and regulation phase. For example, the special protocol assessment tool will help to establish, in a scientific way, biosimilarity with innovator products.
But once this guidance and regulation step is complete, we will turn our attention to the next puzzle piece - the research needed for each company, some of which are PhRMA members, to establish the safety, purity and potency of biosimilar products before they make their way to patients.
I know that I write at length about the ecosystem that exists among academia, government, and industry researchers, but this is an example of another triad in which we play a role: policy, regulation and science. When each piece of that puzzle is dedicated toward the well-being of patients, we end up with a pretty good fit.