Small is the new Big.
It's fast becoming an n of 1 world, where every disease is an orphan disease and success is measured by individual outcomes rather than large population studies such as CATIE or ALLHAT or the multitude of programs being funded by PCORI.
Small is the new Big means we must also think differently about pharmacovigilance. While we must continue to capture adverse event data, we must also strive to capture Substandard Pharmaceutical Events (SPEs). SPEs occur when a product does not perform as expected—perhaps because of API or excipient issues. SPEs can arise because of an issue related to therapeutic interchangeability. When it comes to 21st-century pharmacovigilance, we have to both broaden and narrow our views about bioequivalence to the patient level. Small is the new Big.
When it comes to drug development, adaptive clinical trials and companion diagnostics further define the urgency of small-scale thinking. Demonstrating outcomes on an n of 1 level is crucial not just for 21st century healthcare technology assessment but also for physician pay-for-performance measures and the benefit of actual patients.
There's a lot of lip service paid to the comment that “the era of the blockbuster is over.” Now consider that statement from the perspective of another industry—in the 21st century would you rather be Blockbuster or Netflix?
Small is the new Big. That means a focus on individual patient outcomes, which means a focus on the individual patient rather than the general population and on long-term care rather than short-term cost.
And it's about time.
Peter J. Pitts, Ph.D.
Chief Regulatory Officer