Oops, you're using an old version of your browser so some of the features on this page may not be displaying properly.

MINIMAL Requirements: Google Chrome 24+Mozilla Firefox 20+Internet Explorer 11Opera 15–18Apple Safari 7SeaMonkey 2.15-2.23


8P - Clinical trial design in the era of precision oncology: an overview of the last 20 years


06 Oct 2021




Marco Filetti


Annals of Oncology (2021) 32 (suppl_6): S1345-S1371. 10.1016/annonc/annonc740


M. Filetti1, P. Lombardi2, R. Falcone2, F. Paroni Sterbini2, G. Daniele3

Author affiliations

  • 1 Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome/IT
  • 2 Fondazione Policlinico Universitario A. Gemelli IRCCS, 168 - Rome/IT
  • 3 Fondazione Policlinico Universitario Agostino Gemelli - IRCCS Rome, 168 - Rome/IT

Abstract 8P


The recent advent of the “precision oncology” model changed the face of modern drug development. The chance of quickly carrying out extensive molecular profiling and coupling driver mutations to specific selective inhibitors fostered the advent of new methodologies and trial designs. We systematically reviewed the precision oncology trials published in the last 20 years.


We included all the precision oncology trials published between January 2000 and June 2021. We collected data about screened patients, enrolled patients, overall response rate (ORR), progression-free survival (PFS), overall survival (OS), toxicities, and quality of life (QoL).


In the examined period, 34 papers were published for 27 different trials. Most of the studies (82%) had a basket design, while five studies used an umbrella design, and only one a platform design. Interestingly, most of the studies were non-randomized (81%). In total, 20,790 patients were screened, with an average of 990 (35 - 5,548) patients per study. The average duration of the enrollment phase was approximately 32 (9-88) months. Overall, 3,865 patients were enrolled (18% of screened patients), with an average of 114 (10 - 514) patients per study. An ORR was recorded in 426 patients (11% of enrolled patients, 2% of screened patients) with a mean of 12 patients per single study. Toxicity data were included in 26 publications (76%), while none of the publications had the patient-reported quality of life data. Finally, we found that 23 trials (67%) used ORR as the primary endpoint, 31 publications (91%) reported PFS data, while only 18 publications (52%) reported OS data.


In this analysis, we intended to offer a snapshot of the results produced by precision oncology studies over the past twenty years. In total, these studies enrolled a low percentage of patients, less than 20%. Moreover, we show that most of the trials evaluated ORR as a primary endpoint, and in about half of the publications, no data of OS was reported. In conclusion, despite the vast effort produced in the screening phase, precision oncology trials had modest results and often reported incomplete data regarding OS, toxicity, and QoL.

Legal entity responsible for the study

The authors.


Has not received any funding.


All authors have declared no conflicts of interest.

This site uses cookies. Some of these cookies are essential, while others help us improve your experience by providing insights into how the site is being used.

For more detailed information on the cookies we use, please check our Privacy Policy.

Customise settings
  • Necessary cookies enable core functionality. The website cannot function properly without these cookies, and you can only disable them by changing your browser preferences.