Appendix 1 to the guideline on the evaluation of anticancer medicinal products in man - methodological consideration for using progression-free survival (PFS) or disease-free survival (DFS) in confirmatory trials - Scientific guideline

This document is an appendix to the guideline on the evaluation of anticancer medicinal products in man. It provides a guidance on the use of progression-free survival or disease-free survival as endpoint in clinical efficacy trials.

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

Current effective version

Appendix 1 to the guideline on the evaluation of anticancer medicinal products in man - Methodological consideration for using progression-free survival or disease-free survival in confirmatory trials

AdoptedReference Number: EMA/CHMP/27994/2008Legal effective date: 01/07/2013Summary:

The use of progression-free survival (PFS) or disease-free survival (DFS) as endpoint in clinical efficacy trials presents several methodological issues which need to be addressed prospectively.

This appendix provides some general regulatory guidance on issues to consider relating to definitions, frequency and methods of assessment, ascertainment bias, handling of deviations and missing data, and radiology review. Guidance on the choice of primary endpoint, and appropriateness of using PFS/DFS as primary endpoint, is addressed in the main body of the guideline.

Draft Appendix 1 to the guideline on the evaluation of anticancer medicinal products in man

Document history

Appendix 1 to the guideline on the evaluation of anticancer medicinal products in man (CHMP/EWP/205/95 REV. 3), methodological considerations for using Progression-Free Survival (PFS) as primary endpoint in confirmatory ...

AdoptedReference Number: EMEA/CHMP/EWP/27994/2008Legal effective date: 01/08/2008

Draft appendix 1 to the guideline on the evaluation of anticancer medicinal products in man (CHMP/EWP/205/95 rev. 3), methodological considerations for using Progression-Free Survival (PFS) as primary endpoint in confirm...

Draft: consultation closedReference Number: EMEA/CHMP/EWP/267575/2006/Corr.