Curious Scenario
Wouldn’t this be a curiously favorable scenario.
Anavex2-73, as an orphan drug, attains FDA approval for treating Rett Syndrome.
But, then, some astute physicians recognize (as we here do) the drug’s safety and efficacy for Alzheimer’s treatment. A few Alzheimer’s patients are prescribed the drug, which they pay full cost for, as their health insurance companies won’t cover off-label drug uses.
But, lo and behold, each of the Alzheimer’s patients taking the Rett Syndrom drug — Anavex2-73 — finds that they are no longer experiencing ever-greater degrees of bad Alzheimer’s symptoms. In fact, a few who took the Rett Syndrome drug at the earliest stages of Alzheimer’s discover, along with their care-givers and physicians, that Anavex2-73 actually restored normal mental health; that they were now living normal lives. Before, they were beginning their descent into the hell of debilitating Alzheimer’s.
Word of this spreads (as can be imagined), and off-label prescriptions of the Rett Syndrome drug Anavex2-73 proliferate for Alzheimer’s patients.
The medical insurance companies discover that their Alzheimer’s patients on the Rett Syndrome drug will not be progressing to the costly last stages of Alzheimer’s. Medical insurance company bean-counters quickly figure out they can save tens of thousands of dollars per Alzheimer’s patient by getting each on the FDA-approved Rett Syndrome drug Anavex2-73 — which is soon added to the covered drugs of each medical insurance company.
Tens of thousands of Alzheimer’s patients then are covered for and treated — successfully — by Anavex2-73.
During all of this, FDA officials still press for a big double-blind Phase 3 Anavex2-73 clinical trial. They are perplexed and confounded that so many people are using Anavex2-73 off-label for Alzheimer’s. They give the usual and expected warnings that the drug has never been thoroughly tested for Alzheimer’s safety and efficacy, and recommend that its use be restricted solely to Rett Syndrome girls.
The end result? Anavex2-73 becomes, de facto, the standard of care for Alzheimer’s, sans any Phase 3 double-blind clinical trials.
