Evaluation of anticancer medicinal products - Scientific guideline

The purpose of this guideline is to provide guidance on all stages of clinical drug development for the treatment of malignancies, including identifying target population with optimised benefit risk in exploratory trials, design of confirmatory trials, choice of endpoints, the impact of adverse drug reactions on the benefit-risk. Supportive measures such as anti-emetics and haematopoietic growth factors are covered by separate guidelines. 

The guideline is complemented by a number of appendices:

• Appendix 1 provides methodological guidance on the use of progression-free survival (PFS) as endpoint in confirmatory studies.
• Appendix 2 focuses on the use of patient reported outcome (PRO) measures and health related quality of life (HRQoL) from a regulatory perspective.
• Appendix 3 is dedicated to specific guidance on the SmPC.
• Appendix 4 deals with condition-specific guidance. 

Definitions and abbreviations used in this guideline are summarised at the end of the document. The 6th revision addresses biomarker-guided medicinal product development, recent designs in oncology (such as designs of master protocol studies), as well as clarifying and structural changes throughout the text.

Keywords: Cancer, malignancy, biomarker, targeted drugs, pharmacogenomics

Current effective version - Revision 6 (under revision, see document history)

Guideline on the clinical evaluation of anticancer medicinal products - Revision 6

Adopted Reference Number: EMA/CHMP/205/95 Rev.6

Appendix 3 to the guideline on the clinical evaluation of anticancer medicinal products - Summary of Product Characteristics for an Anticancer medicinal product – mock-up of 4.8

Adopted Reference Number: EMA/631612/2021 Legal effective date: 23/05/2022

Document history

Concept paper on the revision of the guideline on the evaluation of anticancer medicinal products and appendices - Revision 7

Draft: consultation closed Consultation dates: 30/04/2025 to 31/07/2025 Reference Number: EMA/122980/2025

Draft guideline on the evaluation of anticancer medicinal products in man - Revision 6

Draft: consultation closed Consultation dates: 13/11/2020 to 15/02/2021 Reference Number: EMA/CHMP/205/95 Rev.6

Draft appendix 3 to the guideline on the clinical evaluation of anticancer medicinal products - Summary of Product Characteristics for an Anticancer medicinal product – mock-up of 4.8

Draft: consultation closed Consultation dates: 13/11/2020 to 15/02/2021 Reference Number: EMA/593364/2020

Concept paper on the revision of the guideline on the evaluation of anticancer medicinal products in man

Draft: consultation closed Consultation dates: 14/01/2019 to 14/04/2019 Reference Number: EMA/CHMP/755489/2018

Guideline on the evaluation of anticancer medicinal products in man - Revision 5

Adopted Reference Number: EMA/CHMP/205/95 Rev. 5 Legal effective date: 01/04/2018 Summary:

This guideline replaces guideline Guideline on the evaluation of anticancer medicinal products in man' EMA/CHMP/205/95 Rev. 4.

Draft guideline on evaluation of anticancer medicinal products in man - Revision 5

Draft: consultation closed Consultation dates: 15/03/2016 to 15/09/2016 Reference Number: EMA/CHMP/205/95 Rev. 5 Summary:

The purpose of this guideline is to provide guidance on all stages of clinical drug development for the treatment of malignancies, including drug resistance modifiers or normal tissue protective compounds. Supportive measures such as anti-emetics and haematopoietic growth factors, however, are covered by separate guidelines.

Draft concept paper on the need to revise the "Guideline on the evaluation of anticancer medicinal products in man" in order to provide guidance on the reporting of safety data from clinical trials - Revision 5

Draft: consultation closed Consultation dates: 01/05/2015 to 31/07/2015 Reference Number: EMA/130525/2015 Rev. 5 Summary:

The shift from conventional cytotoxic drugs to so called targeted drugs and immune modulators administered continuously and at maximum tolerated dose has changed the tolerability and toxicity profiles of anti-cancer drugs. The aim of this revision is to find ways on how to report adverse effects in order to improve the understanding of the toxicity and tolerability profiles of medicinal products.

Guideline on the evaluation of anticancer medicinal products in man - Revision 4

Adopted Reference Number: EMA/CHMP/205/95 Rev. 4 Legal effective date: 01/07/2013 Summary:

The purpose of this guideline is to provide guidance on all stages of clinical drug development for the treatment of malignancies, including drug resistance modifiers or normal tissue protective compounds. Supportive measures such as anti-emetics and haematopoietic growth factors, however, are covered by separate guidelines.

Draft guideline on the evaluation of anticancer medicinal products in man - Revision 4

Draft: consultation closed Consultation dates: 15/12/2011 to 31/05/2012 Reference Number: EMA/CHMP/205/95 Rev.4 Summary:

The purpose of this guideline is to provide guidance on all stages of clinical drug development for the treatment of malignancies, including drug resistance modifiers or normal tissue protective compounds. Supportive measures such as anti-emetics and haematopoietic growth factors, however, are covered by separate guidelines.

Concept paper on the need to revise the guideline on the evaluation of anticancer medicinal products in man - Revision 4

Draft: consultation closed Consultation dates: 22/07/2010 to 01/01/2011 Reference Number: EMA/CHMP/EWP/433478/2010 Rev. 4

Guideline on the evaluation of anticancer medicinal products in man - Revision 3

Adopted Reference Number: CPMP/EWP/205/95/Rev.3/Corr.2 Legal effective date: 01/06/2006