Exmplr

The FDA just made one of the most significant shifts in its evidentiary standards in decades—and it feels like a lot of people have slept on the news! The FDA will now default to ONE pivotal trial for drug approval, not two. The science of evaluating whether a drug works has advanced enormously. Today, development programs build evidence across multiple dimensions—mechanism of action, biomarker effects, intermediate endpoints—that collectively tell a more complete story. That body of confirmatory evidence, from mechanistic data to real-world evidence, can replace a second pivotal trial. We have historically seen more regulatory flexibility in rare diseases and oncology where need is acute. But the biggest impact we expect to see with the shift from two to one pivotal trial is likely to be in common conditions like cardiovascular and respiratory where the two pivotal trial dogma has been more entrenched. Potentially up to $350M in savings per program and years off timelines. And this might just be the start—could continuous trial designs that collapse the rigid phase structure be the next paradigm shift in clinical development?

This monumental meeting has spurred an amazing collaboration between our countries to solve mankind’s biggest challenges together. So inspired now and the evolution of addressing infection and cancer and longevity is now unstoppable.