For Novartis CEO Daniel Vasella, the rare opportunity came in April 1999, when he saw the results of a Phase I trial of the compound that would become Gleevec (imatinib), the company's breakthrough drug for chronic myeloid leukemia (CML). "At first," he later wrote, "I had trouble believing the data." The results were unprecedented: Of 31 patients in the trial, all had their white blood cell counts reduced to normal levels.
What happened next is a story that's been told many times: the outpouring of patient interest, a breathtakingly quick ramp-up of manufacturing, a New Drug Application filed just 32 months after the first dose in man, a record-breaking two-and-a-half-month review at FDA—and the blockbuster success of a drug whose original indication was for a disease diagnosed in just 6,500 patients a year in the United States.
To Vasella, there were several lessons in the experience. One was confirmation of a Novartis cornerstone belief: Innovation matters. "If we are growing dynamically today," he says, "it has to do with the fact that we were able to introduce a series of innovative products and have relatively little generic erosion ahead of us. I think the commitment has to stay on innovation."
Another lesson had to do with involvement with patients. "Collaboration with patients was earlier and deeper in Gleevec than in most cases," he says. This paid off in many ways—especially the speed with which Novartis' people brought the drug to market. "In Ringaskiddy, Ireland, where we produced Gleevec, when people learned what the drug was for and how patients would be impacted, they would work around the clock," Vasella says. "To be aware that you can contribute to a life-saving therapy is incredibly motivating." Since Gleevec, "patient-centricity has increased in the company. And that's only for the better."
The experience also provided a reminder of something every pharma company has experienced: the limits of prediction. "You can't project accurately in all cases what a drug will do," Vasella says. "That doesn't mean one should stop making projections, but one has to be modest about what's feasible.