Dr. L. G. Hemkens
Status: completed Duration: Oct 2015 - Jan 2018
Timely access to innovative treatments is paramount for patients with cancer. However, by applying flexibility to the approval pathways, substantial uncertainty in clinical decision-making is created, especially directly following approval. In this meta-epidemiological study, the approval studies for all 70 cancer treatments that were approved since the year 2000 will be systematically reviewed and their methods, size, and treatment effects evaluated. We will also evaluate the post-approval generation of clinical evidence on effects on overall survival, patient-important outcomes, and the most important cancer specific surrogate outcomes progression-free survival and tumor response. nWe will consider eligible randomized controlled trials (RCTs), systematic reviews and meta-analyses of such RCTs, and CER-studies (i.e. studies using observational data collected from routine patient care after approval, such as patient or disease registries, administrative databases or electronic health records).nThe ultimate goal is to provide decision-makers with guidance to identify early indications which innovative drugs likely fulfill the promise of therapeutic success in the long run and which should be used cautiously until more evidence is generated.