Developers find that Phase II hurdle is getting harder to clear

Phase II has long represented the key crossroads for a biotech company. Getting a promising outcome is a good way to earn a lucrative partnering deal, possibly even a buyout. But a new analysis shows that achieving success in mid-stage development--never all that easy to begin with--is getting harder. Lots harder.

The Centre for Medicines Research analyzed the results of 16 companies which collectively controlled 60 percent of the global drug R&D budget and found that the 28 percent success rate they enjoyed in Phase II from 2006 to 2007 dropped to an alarming 18 percent in 2008 and 2009. According to a report in Nature Reviews Drug Discovery, Thomson Reuters Life Science Consulting did the math on the 108 mid-stage failures recorded from 2008 to 2010, narrowing it down to the 87 which reported outcomes.

Slightly over half, 51 percent, failed to achieve sufficient efficacy. A total of 29 percent failed due to strategic reasons (which evidently had a lot to do with an inability to demonstrate a significant advantage over existing treatments) and 19 percent flunked the safety standard. And even where the researchers didn't know the exact cause, a problem with demonstrating superiority over another drug appeared to play a big role.

The four biggest disease areas where drugs failed most frequently: alimentary/metabolism (primarily diabetes), cancer, cardiovascular, and neuroscience. Of course, if the study was weighted toward Big Pharma, which has lamented its poor pipeline record of recent years, it may be no surprise that failure rates are running higher. Somebody should do a study that compares the outcomes of biotechs and Big Pharma. 

In his blog, researcher Derek Lowe found it interesting that cardio, where developers have had so much success, is proving a thorny problem in Phase II. "Perhaps that one is also suffering from the standard of care being pretty good (and often generic, or soon to be). So the high-success-rate mechanisms of the old days are well covered, leaving you to try your luck in the riskier ideas, while still trying to beat some pretty good (and pretty cheap) drugs. . ."

- read the story from Nature Reviews Drug Discovery
- here's Derek Lowe's take from In The Pipeline

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