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Derek Lowe The 2002 Model

Dbl%20new%20portrait%20B%26W.png After 10 years of blogging. . .

Derek Lowe, an Arkansan by birth, got his BA from Hendrix College and his PhD in organic chemistry from Duke before spending time in Germany on a Humboldt Fellowship on his post-doc. He's worked for several major pharmaceutical companies since 1989 on drug discovery projects against schizophrenia, Alzheimer's, diabetes, osteoporosis and other diseases. To contact Derek email him directly: Twitter: Dereklowe

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July 10, 2013

On the Priority Breakthrough Accelerated Fast Track

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Posted by Derek

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.

Comments (6) + TrackBacks (0) | Category: Regulatory Affairs


1. CMCguy on July 10, 2013 9:54 AM writes...

Early and frequent communication with FDA is always a good idea providing 1) FDA resources are adequate and appropriately experienced to support increased interaction and 2) when things don't quite as planned the FDA is willing to modify efforts with focus on how to advance forward with extensive shifting goal-posts. Both of these would seem to require a huge adjustment for the current FDA.

Further does this mean based on Pazdur's previous positions to be classified as a Breakthrough then "Survival" outcome is the clinical trial criteria?

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2. Hap on July 10, 2013 9:58 AM writes...

Perhaps it's a problem with the way the FDA or insurers/private payers look at new drugs, but if a potential drug doesn't fit into one of those categories, does it have a reasonable chance of either 1) being approved or 2) being paid for by insurance (and thus being used by its customers)?

It seems like the fast-track line-jumping fees at amusement parks (when the rides are up) - when half the people in the park (or drugs in the pipeline) use and pay for a fast-track, when do you think the people/drugs that aren't are going to get their turns? At whatever large fees (relatively) both are paying, being second-class (or lower) citizens seems someone galling - unfortunately, while I can stop the amusement park thing by not going (although, at that point, the line-jumping fee simply becomes a massive admission increase), we don't really want people to stop submitting drug applications, do we?

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3. barry on July 10, 2013 10:18 AM writes...

Although Pfizer filed piroxicam first, Lilly's benoxiprofen got approved earlier. I'm told that was because someone at Pfizer irked someone at FDA. Of course, benoxiprofen had to be yanked from the market for a $billion loss.
So was without a "fast-track" or "priority" designation, time-lines are still maleable.

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4. emjeff on July 10, 2013 9:15 PM writes...

Shakespeare said it best.;

"...full of sound and fury, signifying nothing."

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5. jsamick on July 11, 2013 1:01 AM writes...

I haven't seen anyone ask the real question yet:

If the FDA has to have so many special shortcuts to get around their system, doesn't that imply the system is broken?

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6. CMCguy on July 12, 2013 4:00 PM writes...

#5 jsamick although your statement reflects a definition I have heard for classification an ineffectual Bureaucracy (known by how many routine pathways that must be circumvented normally to make any progress at all) and would agree FDA largely fits in to a category of entrenched Bureaucracy(as does Big Pharma, Majority of US Govt, etc) in the cases of the different drug approval approaches available this is more about focusing priorities for both the companies and FDA. Except for maybe the use of "surrogate endpoints" there are not really any shortcuts to the drug development process that I know of as, in the end full expectations are mostly the the same with the hows and whens of submissions variable and then occasionally come across how much considerations (i.e. rare diseases).

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