The Revolution of Trial Design

As we move deeper into the era of precision medicine, our researchers are constantly evolving our drug development process to identify the next generation of therapies – and to bring them to patients more quickly.

Clinical trial design is one of those areas we are looking to improve.

Clinical trials play an essential role in drug development, but in their traditional form require large numbers of participants and extensive time – only to sometimes deliver “one-drug-fits-most” treatment options.

The push towards precision medicine has necessitated a revolution in clinical trial design. A revolution that many, including our scientists within AstraZeneca, believe could hold the key to improved efficiencies and greater probabilities of success for delivering the future of precision therapies.

Understanding Conventional Multi-Drug Studies

The conventional multi-drug study approach was introduced during the mid-1940s. Within its design, two or more treatments are compared in a pre-defined population, for a set period or until a certain number of patient outcomes have been observed.

Once completed, the results from each intervention group are reviewed and performance relative to predefined endpoints is assessed for statistical significance. Sometimes the data also show other trends or “signals” that can be followed up with additional studies.



Conventional studies don’t allow researchers to adapt to the results they are seeing throughout a study. As a result, trials that take years to run with participation from thousands of patients can return inconclusive results because teams were not able to adapt to observed trends as they were occurring. And the investigators won’t even know about these inconclusive results until the study is over. These delays can necessitate additional studies leading to greater demands on patients and resources.

Platform Trials and the Promise of Responsive Adaptation

Platform trials may offer a solution.

These innovative trials are designed with an open master protocol, which allows for multiple treatments to enter or exit the trial over the course of the study, providing researchers the ability to adapt to results that are observed throughout a study.

This flexibility allows teams to drop treatments for futility, to declare one or more treatments superior, or even to add new treatments for assessment during a trial – allowing clinicians to better meet the unique needs of patient populations within a study.



As a result, platform trials offer the promise of more efficient clinical investigation, capable of finding the same beneficial treatments in less time, with fewer patient participants.

AstraZeneca is continuing to expand our use of platform and other innovative trial designs – all intended to improve our ability to turn science into medicines. Find out more about these different types of trials in the video below:


Veeva ID: Z4-20738    

Date of Prep: October 2019

Date of Expiry: October 2021