WHO Revised Biosimilar Guidelines: Streamlined Regulation for Improved Global Access

Key Takeaways

• WHO's 2022 revised guidelines eliminate the need for mandatory clinical efficacy studies for biosimilars when strong analytical and pharmacokinetic data demonstrate similarity.
• The shift reduces development costs and accelerates regulatory approval, particularly benefiting low- and middle-income countries.
• Strong pharmacovigilance and post-marketing surveillance are critical to mitigate risks from streamlined approval pathways.
• Regulatory convergence with WHO guidance is essential for global biosimilar access and market growth.

What does this document cover?

This WHO Perspectives article translates the 2022 revised biosimilar evaluation guidelines into actionable policy messages. It explains the scientific rationale for reducing reliance on clinical efficacy studies, highlights opportunities for faster and cheaper biosimilar development, and outlines risks and mitigation strategies for regulators, especially those in resource-limited settings.

Why does this matter for pharma teams?

For commercial and regulatory teams, the revised guidelines open a faster, lower-cost path to market for biosimilars. Teams should prioritize advanced analytical characterization and pharmacokinetic studies over large clinical trials. The document also underscores the need for strong pharmacovigilance systems and stakeholder education to maintain public trust and ensure patient safety.

How do revised WHO guidelines streamline biosimilar approval?

The 2022 revised WHO guidelines prioritize analytical comparability and pharmacokinetics studies over mandatory large-scale clinical efficacy studies. This shift aligns with the evolving practices of regulatory authorities such as the U.S. Food and Drug Administration, European Medicines Agency, and Health Canada, which increasingly accept streamlined clinical trials when strong totality of evidence is demonstrated.

What are the risks of streamlined biosimilar regulation?

The approach introduces risks in settings with weak pharmacovigilance and may also challenge public trust and perception. These risks can be mitigated through strengthened post-marketing surveillance, including active pharmacovigilance systems, clear product identification and traceability, and targeted risk management plans. Transparent communication and stakeholder engagement are essential to building confidence in biosimilars.

Frequently Asked Questions

How do I open a.docx file in Word?

While this document is a web-based publication from the World Health Organization,.docx files can be opened in Microsoft Word, Google Docs, or LibreOffice. For the WHO Bulletin article BLT.25.294908, the full text is available online through the Bulletin of the World Health Organization.

What evidence supports eliminating clinical efficacy studies for biosimilars?

Data and experience over the last decade clearly show that comparative clinical efficacy and safety studies have not been critical for regulatory approval. Any differences between a candidate biosimilar and the reference product in clinical comparability were resolved through physicochemical and functional data together with comparative pharmacokinetics studies.

When are clinical efficacy studies still required under the revised guidelines?

While clinical efficacy studies may not be necessary in most cases, specific situations may arise where comparative clinical efficacy, safety, or immunogenicity data are required. For example, a product whose mechanism of action is unknown or poorly understood would fall into this category.