Abstract
Cancer treatment has made significant strides towards the promise of personalized medicine. Recent scientific advances have shown that there are numerous genetic deregulations that are common in multiple cancer types, raising the possibility of developing drugs targeting those deregulations irrespective of the tumour type. Precision Cancer Medicine (PCM) was born out of accumulated evidence matching targeted agents with these tumour molecular deregulations. At the same time, the therapeutic armamentarium is rapidly increasing and the number of new drugs (including immune‐oncology agents) entering drug development continues to rise. These factors, added to strong collaboration with regulatory agencies, which have approved novel agents based on data obtained from phase 1/2 trials, have led to unprecedented evolution in the design of early‐stage clinical trials. Currently, we have seen rapid phase 1 dose‐escalation trials followed by remarkably large expansion cohorts, and are witnessing the emergence of new trials, such as adaptive studies with basket and umbrella designs aimed at optimizing the biomarker–drug co‐development process. Alongside the growing complexity of these clinical trials, new frameworks for stronger and faster collaboration between all stakeholders in drug development, including academic institutions and frameworks, clinicians, pharma companies and regulatory agencies, have been established. In this review article, we describe the main challenges and opportunities that these new trial designs may provide for a more efficient drug development process, which may ultimately help ensure that PCM becomes a reality for patients.
Highlights
Over recent years, much progress has been made in rendering cancer treatments more precise
The I-SPY1/2 studies and the National Cancer Institute (NCI)-MATCH have successfully implemented flexible designs allowing for the addition of new arms as new data become publicly available, while others have remained fixed to their initial treatment algorithms that could be outdated by the time they close
We present some of the complexities and recent advances of research in precision medicine
Summary
Much progress has been made in rendering cancer treatments more precise. At the same time, during these last years the arrival of immune therapy to the oncology therapeutic armamentarium has marked a groundbreaking milestone for the treatment of cancer patients, and the number of immune-oncology agents entering drug development continues to rise (Martin-Liberal et al, 2017) These factors, added to the strong collaboration with the regulatory agencies, approving novel agents based on data obtained from phase 1/2 trials, have led to an unprecedented evolution in the design of early-stage clinical trials (Bui and Kummar, 2018). In this regard, the US Food and Drug Administration (FDA) has approved 10 anticancer drugs matched to companion diagnostic biomarkers based on data obtained from nonrandomized trials in the last 2 years. With the growing complexity of clinical trials, new frameworks for stronger and faster collaboration between all stakeholders in drug development, including clinicians, pharma companies and regulatory agencies, have been established (Harrington et al, 2017; Weber et al, 2017)
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