This simple step could save thousands of lives and increase the number of people seeking to try a new medicine as early as possible.
All of which begs the question of why we need compassionate use exemptions at all. Why not turn Phase 2 and 3 into a study of people using medicines in the real world? Joseph Cooper notes in "Regulating New Drugs that "the ultimate test of safety and efficacy is how man responds in significant numbers under diverse conditions over relatively long periods of time. We have tended to substittue for that ultimate test one of the fads of the times -- the scientifically controlled double blind trial "
Cooper wrote that in 1972.
We have not come very far since then. In fact, the fad of that time has become Holy Writ. Peter and I wrote a publication about how to use molecular markers and digitized real world data to accelerate all drug development and to turn medicine into a true learning system. Very little of what has been written since then is more than a footnote to our original work. And in turn, our work was shaped by the thinking of Cooper and others such as the late Nobel Laureate Josh Lederberg who wondered aloud "if innovation was possible" under the current regulatory regime.
We can learn more in less time using real world data than in a decade of clinical trials. Kudos to the FDA. By removing the obstacles to using new medicines in the real world and by encouraging the sharing of data from these experiences, it has given us -- and policymakers -- a look at how the agency can serve the public health in the century ahead. And it has acknoweldged that patients should and will have more control over what medicines to use and has increased that control.