In February 2012, the FDA issued three draft guidance documents on biosimilar product development regarding key scientific and regulatory factors involved in submitting applications for biosimilar products to the agency:
Scientific Considerations in Demonstrating Biosimilarity to a Reference Product:
The draft guidance is intended to assist companies in demonstrating that a proposed therapeutic protein product is biosimilar to a reference product for the purpose of submitting a 351(k) application to the FDA. This draft guidance describes a risk-based “totality-of-the-evidence” approach that the FDA intends to use to evaluate the data and information submitted in support of a determination of biosimilarity of the proposed product to the reference product.
Quality Considerations in Demonstrating Biosimilarity to a Reference Protein Product:
This guidance provides an overview of analytical factors to consider when assessing biosimilarity between a proposed therapeutic protein product and a reference product for the purpose of submitting a 351(k) application. This includes the importance of extensive analytical, physicochemical, and biological characterization in demonstrating that the proposed biosimilar product is highly similar to the reference product notwithstanding minor differences in clinically inactive components.
Biosimilars: Questions and Answers Regarding Implementation of the Biologics Price Competition and Innovation Act of 2009:
The draft provides answers to common questions from companies interested in developing biosimilar products. The question and answer format addresses questions that may arise in the early stages of product development, such as how to request meetings with the FDA, addressing differences in formulation from the reference product, how to request exclusivity, and other topics.
The FDA provided a 60-day period for stakeholders to offer comments. The comment period has ended and the FDA will issue final regulations, respond to comments, or both, later this year, probably in the third quarter.
The FDA received submissions from an array of stakeholders, including the Biotechnology Industry Organization (BIO). BIO has advocated that the FDA expand the requirements to prove biosimilarity significantly beyond the minimum tests mandatory in the statute, and generally recommends policies that make it more difficult to obtain expedited approval created under the BPCI Act.
Congressional intervention is unlikely at this stage. The Supreme Court’s upholding of the BPCI Act as part of the Affordable Care Act now clears the way for final FDA rulemaking and the beginning of a new era of U.S. healthcare that will include affordable biosimilar medicines.