FDA Moves to Streamline Biosimilar Development to Improve Medicine Affordability

The U.S. Food and Drug Administration (FDA) has announced additional measures to streamline the development of biosimilar medicines, which are comparable to “generic” versions of biologic drugs. Through newly issued draft guidance, the agency has recommended reducing certain clinical pharmacokinetic (PK) testing requirements when there is sufficient scientific justification.

According to the FDA, the updated approach could allow biosimilar developers to reduce PK study costs by up to 50 percent, potentially saving around $20 million per development programme, while also helping bring more affordable treatment options to patients.

"Streamlining biosimilar development reflects our ongoing commitment to lowering drug costs for everyday Americans," said FDA Commissioner Marty Makary, M.D., M.P.H. "Using common sense, we are embracing more precise analytical testing approaches than have been used in the past."

Biologic medicines are widely used to treat conditions such as autoimmune disorders and cancer, but they are often associated with high costs. Although biologics account for only about 5 percent of prescriptions, they represent around 51 percent of overall drug spending and can cost hundreds of thousands of dollars annually.

Biosimilars, similar to generic medicines for conventional drugs, are designed to provide more affordable treatment alternatives, thereby improving access to important therapies that may otherwise remain financially out of reach for many patients.

The latest guidance builds on an earlier FDA initiative announced in October, when Commissioner Makary introduced another draft guidance aimed at reducing certain comparative efficacy studies that can take one to three years to complete and cost approximately $24 million.

The new draft guidance titled “New and Revised Draft Q&As on Biosimilar Development and the BPCI Act (Revision 4)” is intended to support companies developing biosimilar and interchangeable biosimilar products. It replaces an earlier draft version titled “New and Revised Draft Q&As on Biosimilar Development and the BPCI Act (Revision 3)”, which was issued in September 2021.

The updated guidance also revises certain questions and answers previously included in the final guidance document “Questions and Answers on Biosimilar Development and the BPCI Act.”

Under the revised recommendations, biosimilar developers may, in certain situations, rely on clinical data from comparator products approved outside the United States to demonstrate biosimilarity to a U.S.-licensed reference product.

In particular, the draft guidance outlines circumstances where applicants can use non-U.S.-licensed comparator products without conducting additional three-way pharmacokinetic studies, which typically compare the proposed biosimilar with both the U.S.-licensed reference product and a non-U.S. comparator.

The revisions also remove the earlier recommendation requiring at least one PK study directly comparing the proposed biosimilar with the U.S.-licensed reference product. Instead, a study may rely on a comparator approved outside the United States if it is scientifically justified.

As part of these updates, the FDA is also withdrawing its earlier guidance titled “Scientific Considerations in Demonstrating Biosimilarity to a Reference Product.” The agency stated that the document no longer reflects its current scientific approach, noting that significant regulatory experience has been gained since the guidance was first issued in April 2015, when only one biosimilar had been approved.

The Biologics Price Competition and Innovation Act (BPCI Act) of 2009 established an abbreviated regulatory pathway to help improve patient access to safe and effective biological medicines.

To date, the FDA has approved 82 biosimilars, expanding treatment options for patients with conditions such as cancer, rheumatoid arthritis, diabetes, Crohn’s disease and osteoporosis, while also supporting efforts to reduce healthcare costs.