When researchers at Pfizer first began a Phase 2 trial of an acute stroke therapy in 2000, they decided to take a novel approach. The study—called the ASTIN trial—would determine the drug’s optimal dose not with three or four different dosing arms, as trials often have, but with 15. Data from the trial would be captured continuously and used to make changes in real time to how the trial was run. As new patients joined, they would be randomized to a particular arm based on those real-time results—a process which required an intensive level of relatively novel statistics.
Before the drug entered Phase 3, the data showed that it was ineffective, and the trial ended in 2001. But processing a massive amount of real-time data as the trial went on, rather than waiting to conduct the analysis after it ended, saved Pfizer about $3.5 million by the company’s estimates. “Pfizer ...
