Alongside conventional aims such as defining the proper doses and schedule for a medicine, the guideline highlights the importance of identifying the target population during the development. The revised guideline explains how the use of biomarkers throughout non-clinical and clinical development can help identify patients with an increased likelihood to respond favourably to treatment.
Confirmatory trials should demonstrate that the investigational product provides clinical benefit. From both a clinical and a methodological perspective, the most persuasive outcome of a clinical trial is the demonstration of favourable effects on overall survival. Prolonged progression-free survival and disease-free survival are also considered to be of benefit to the patient. The guideline describes how to select the appropriate endpoints for confirmatory studies.
Two appendixes complement the guideline, to address methodological considerations for using progression-free survival in confirmatory trials, and to provide condition specific guidance for non-small cell lung cancer (NSCLC), prostate cancer, chronic myeloid leukaemia (CML), myelodysplastic syndromes, and haematopoietic stem cell transplantation.
The revised guideline comes into effect on 1 July 2013.