So the FDA has the good ol' drug approval process. And then there's Priority Review, and Fast Track, and Accelerated Approval, and now the Breakthrough designation. What, exactly, do all these things mean, and how are they different?
Matthew Herper has a good overview here at Forbes. In short, Priority Review is supposed to take a few months off the usual review period. Fast Track is for drugs that target some unmet medical need, and speeds up their review as well. Accelerated Approval is the process for approving important unmet-need drugs based on preliminary data, with a review once larger studies are completed. (That, for example, is what Avastin went through for its onetime breast cancer indication). Note that these categories aren't mutually exclusive; a drug can have more than one at a time.
And the new "Breakthrough" category is similar to Fast Track and Accelerated Approval, in that it's supposed to be for drugs whose early clinical evidence shows that they might be a real advance over existing treatments. According to Herper's interview with Richard Pazdur, of the FDA's Office of Oncology and Hematology Products, the big difference in this latest category is early and broad cooperation with the agency.
. . .(the designation) catalyzes communication between a company and the FDA. Traditionally, drug reviews take place through a series of scheduled meetings. A breakthrough designation means there are more times a company can expect to be able to pick up the phone and get an answer. The designation can lead to cleared calendars, and it also means that the senior management of the FDA division becomes involved, not just the reviewers who serve on the FDA’ s front lines.
“The true measure of success is going to be how active we are in working with the companies,” Pazdur says. “If someone gets a breakthrough therapy and it’s business as usual, than the breakthrough therapy is meaningless.”
And these communication isn't just about trial design, although that's one of the biggest issues. Manufacturing, naming, and all the other regulatory issues are treated as well. Pazdur says that the biggest reason that some companies haven't achieved breakthrough status for their new compounds, though, is that they come in when it's still too early to make a decision. It sounds like this is something companies have to time pretty well: too early, and you'll be told to come back later. But if you wait too long, the designation might not be as much help as it could be.
Here's an overview from the FDA, and here's ta more detailed guidance document. The FDA says that just over half the drugs approved in 2012 took advantage of one or more of these categories, and it looks like the trend will continue. If the majority of things get Special Expedited Priority Shipping, what does that say about the Regular Shipping option? An outside observer (or investor) should keep this in mind - you probably shouldn't celebrate when a drug gets a designation like this, as worry about when it doesn't.