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Breaking the mould: challenging the status quo of clinical trial response definitions for invasive fungal diseases—a debate

Summary

Doctors and researchers use standard definitions to determine if antifungal treatments work in clinical trials. This debate examines whether the standards created in 2008 are still appropriate today. Key concerns include whether stable disease should always count as treatment failure, how to handle deaths from other causes, and whether newer testing methods should be incorporated. The expert panel concluded these definitions need updating to reflect modern treatment options and patient needs.

Background

Clinical trial response definitions for invasive fungal diseases were established by EORTC/MSG in 2008 based on consensus criteria. These definitions have guided efficacy assessments for antifungal therapies for 15 years, but advances in the field and new antifungal agents with novel mechanisms of action have raised questions about their continued applicability.

Objective

To critically evaluate whether the 2008 EORTC/MSG response definitions remain appropriate and applicable for assessing new antifungal therapies in clinical trials, with focus on invasive mould disease. The debate addressed whether current definitions should be maintained, modified, or redeveloped.

Results

Initially 45% of attendees were unsure about the appropriateness of current definitions. After the debate, 84% agreed that definitions should be updated. Key concerns included: stable disease being classified as failure despite being clinically desirable in salvage therapy; treatment of all deaths as failures despite non-IFD attribution; divergence among clinical, radiological, and mycological criteria; and limited assessment periods for different patient populations.

Conclusion

The symposium faculty concluded that the 2008 EORTC/MSG definitions are no longer optimal for assessing new antifungal therapies. Revised definitions from relevant societies and leading experts are needed to reflect the evolving clinical landscape, incorporate new assessment tools and biomarkers, and ensure effective antifungals reach clinical practice while maintaining regulatory compliance and global applicability.
  • Published in:Journal of Antimicrobial Chemotherapy,
  • Study Type:Review/Debate,
  • Source: PMID: 39084680, DOI: 10.1093/jac/dkae158
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