Biosimilars Council Encourages FDA to Streamline the Development of Biosimilars


The Council recommends the elimination of unnecessary clinical efficacy trials and establishing global regulatory comparators.

Biosimilars Council Encourages FDA to Streamline the Development of Biosimilars

Credit: Adobe Stock/ILOVEART

The Biosimilars Council published a new position paper outlining their suggestions regarding streamlining regulatory requirements for biosimilar development.1 These changes aim to enhance access, affordability, and efficiency in bringing these critical treatments to market faster.

Currently, the US Food and Drug Administration (FDA) requires that a biosimilar application must include data demonstrating high similarity between the biosimilar and reference drug, a toxicity assessment, and ≥ 1 clinical study that proves the potency, purity, and safety of the biosimilar, unless otherwise stated by the FDA. They also usually require a comparative pharmacokinetic (PK) data—and sometimes comparative pharmacodynamic (PD) clinical data—as well as an immunogenicity assessment and comparative clinical efficacy study.2

Although several studies have been exempt from a comparative clinical efficacy study in the past, most of the 50 biosimilar approvals since 2015 have included this step. These trials often include a transition arm with a single cross-over step, with the reference cohort switching to the proposed biosimilar. These studies then compare patient responses for those in the biosimilars cohort and those who made the switch to the biosimilar. This is further complicated when using a European-sourced reference product, as the FDA requires a 3-way PK/PD comparison between the EU reference drug, US reference drug, and the biosimilar.1

“The absence of regulatory clarity about what, if any, clinical efficacy studies will be required, combined with this previous experience, means that sponsors generally plan to conduct comparative clinical efficacy studies as part of their development programs regardless of their scientific utility,” the authors wrote.1 “Some companies may decide to forego biosimilar development programs because of the projected expense, reducing patient access to these important, safe and effective, more affordable medications.”

Further, the Biosimilars Council argues comparative efficacy studies and 3-way PK comparisons are not usually necessary as analytical, functional, and PK methods are now sensitive enough to identify clinically meaningful differences between the reference drug and biosimilar. Therefore, additional clinical efficacy trials do not generally provide new or meaningful data for the regulatory decision-making process. They do, however, delay competition, increase critical resources, and discourage investment in biosimilars.1

“[The] FDA should clarify in regulations and/or guidance that requests for clinical efficacy studies should be the exception rather than a generally applied rule,” the Biosimilars Council stated.1

Establishing a global comparator, as opposed to a 3-way PK comparison, could eliminate the need for the redundant testing of multiple reference drugs. This would in turn give patients access to biosimilars more quickly and at a lower cost, while ensuring safety and efficacy.1

The authors noted streamlining the development of biosimilars in the US could also spur competition as the high costs associated with bringing a biosimilar to market—estimated at $100–300 million—reduces the number of biosimilars companies can develop. As the clinical efficacy studies generally account for half of this cost, eliminating this usually unnecessary step could help increase investment in biosimilars, particularly for those with smaller markets. The current approach, however, leaves many patients in both the US and internationally deprived of access to treatment.1

“The recommended streamlining will not lower safety, efficacy, or quality standards, nor will it involve an extensive revision of the regulatory framework for biosimilars,” the authors concluded.1 “It does not seek to eliminate clinical efficacy studies altogether. Rather, it preserves FDA’s authority to request additional evidence in exceptional circumstances when scientifically justified, unique risk-based considerations are identified.”


  1. Biosimilars Council. Streamlining the Development of Biosimilar Medicines. Biosimilars Council. May 30, 2024. Accessed June 5, 2024.
  2. U.S. Food and Drug Administration (2015). Scientific Considerations in Demonstrating Biosimilarity to a Reference Product Guidance for Industry (“Biosimilarity Guidance”) at 8. Accessed June 5, 2024.
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