FDA Proposes New Steps to Streamline Biosimilar Drug Development

The U.S. Food and Drug Administration has announced a new regulatory initiative aimed at accelerating the development of biosimilar medicines while reducing costs for drug manufacturers and patients. The agency released new draft guidance recommending that certain clinical pharmacokinetic (PK) studies used in biosimilar development could be streamlined or eliminated when scientific evidence supports the change.

According to the FDA, the updated approach could significantly reduce development expenses for companies working on biosimilar therapies. In some cases, developers could save up to 50 percent of the costs associated with PK studies—an estimated reduction of about $20 million per program. Officials say these changes are part of a broader effort to increase competition in the pharmaceutical market and make biologic medicines more affordable.

Biosimilars are medicines designed to be highly similar to existing biologic drugs, which are complex therapies derived from living organisms. These treatments are widely used to manage serious conditions including autoimmune disorders and cancer, but they often come with extremely high price tags. Although biologics account for only about five percent of prescriptions in the United States, they represent roughly 51 percent of total drug spending.

By simplifying some regulatory requirements, the FDA hopes to encourage greater biosimilar competition, which could lead to lower prices and expanded access to critical treatments. Marty Makary said the agency is adopting more modern scientific approaches to drug evaluation while maintaining strict safety and effectiveness standards.

The newly released document, titled “New and Revised Draft Q&As on Biosimilar Development and the BPCI Act (Revision 4),” updates earlier guidance issued in 2021. The document outlines revised recommendations for pharmaceutical companies seeking approval for biosimilar or interchangeable biosimilar products. One key change involves the use of comparator products approved outside the United States.

Under the updated guidance, biosimilar developers may be able to rely on clinical data from a non-U.S. comparator product without conducting an additional three-way pharmacokinetic study involving the proposed biosimilar, the U.S.-licensed reference product, and the non-U.S. version. If scientifically justified, developers could use international clinical data directly, reducing the need for additional costly trials.

The FDA also removed a previous recommendation that required at least one clinical PK study comparing the biosimilar directly with the U.S.-approved reference product. Instead, developers may use a comparator product approved outside the country as long as sufficient scientific evidence supports its relevance.

As part of the announcement, the agency said it is withdrawing earlier guidance titled “Scientific Considerations in Demonstrating Biosimilarity to a Reference Product,” which was published in 2015. At the time, the FDA had approved only one biosimilar therapy and had limited experience reviewing these products. Since then, the regulatory landscape has evolved considerably.

The pathway for biosimilar approvals was created under the Biologics Price Competition and Innovation Act of 2009, which established an abbreviated approval process intended to encourage competition in the biologics market.

To date, the FDA has approved 82 biosimilar products covering treatments for conditions such as cancer, rheumatoid arthritis, diabetes, Crohn’s disease and osteoporosis. Regulators believe the latest changes could further accelerate biosimilar development and help bring more affordable treatment options to patients across the United States.