Evaluation of anticancer medicinal products in man - Scientific guideline
HumanScientific guidelines
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 and Appendix 4 deals with condition-specific guidance. Definitions and abbreviations used in this guideline are summarised at the end of the document. This 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