FDA Issues Industry Guidance on Application Pathway for Biosimilars

The Food and Drug Administration recently issued three draft guidance documents on biosimilar product development to assist industry in the United States.   The Patient Protection and Affordable Care Act (P.L. 111-148) (PPACA), signed into law by President Obama on March 23, 2010, amended the Public Health Service Act to create an abbreviated approval pathway — referenced as a section 351(k) approval — for biological products that are demonstrated to be highly similar (biosimilar) to or interchangeable with an FDA-licensed biological product.

Biological products are therapies used to treat diseases and health conditions and include a wide variety of products including vaccines, blood and blood components, gene therapies, tissues, and proteins. Unlike most prescription drugs made through chemical processes, biological products generally are made from human and/or animal materials.

A biosimilar is a biological product that is highly similar to an already approved biological product, notwithstanding minor differences, and for which there are no clinically meaningful differences between the biosimilar and the approved biological product in terms of the safety, purity, and potency.  The advent of these biosimilars is forecast to eventually become a multi-billion-dollar market.

Janet Woodcock, M.D., director of FDA’s Center for Drug Evaluation and Research stated in an FDA press release that the “[d]raft documents are designed to help industry develop biosimilar versions of currently approved biological products, which can enhance competition and may lead to better patient access and lower cost to consumers.”

As discussed in a previous post, PPACA set a 12 year limit on patent protection for these biologics; the provision was added because biologic drugs were not covered by previous law governing most pharmaceutical competition.  In turn, the draft guidance materials provide some insight on how the agency may address the immediate future of the biosimilar pathway.

The 351(k) draft guidance document, most anticipated by industry, discusses the FDA’s views on the new application pathway.  The draft guidance describes a risk-based “totality-of-the-evidence” approach that the FDA would use to evaluate the data and information submitted in support of a determination of biosimilarity of the proposed product to the reference product. As outlined in the draft guidance, the FDA recommends a stepwise approach in the development of biosimilar products.  Additionally, the FDA noted the importance of extensive analytical, physico-chemical and biological characterization in demonstrating that the proposed biosimilar product is highly similar to the reference product notwithstanding minor differences in clinically inactive components.