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Drug Fail

The failure rate for the approval of new drugs is bad, everyone knows that. But it may be worse than most everyone thought, writes Derek Lowe, who summarizes the latest data on drug success rates in the blog Corante.

The study, published in Nature Biotechnology, looked at success rates for 835 drug developers from 2003 to 2011 and analyzed their success rates by clinical phase, molecule type, disease area, and lead versus nonlead indication status. The goal of the study, "the largest and most recent of its kind," was to incorporate data from a wider range of organizations, drug modalities, and targets.

Using this approach, the investigators found that 10 percent of all indications in Phase I eventually make it through the FDA, and failure rates are roughly in the 30 percent range in Phase I, in the 60 percent range in Phase II, and 30 to 40 percent in Phase III.

The drug class with "the most brutal attrition" is small molecules, as just over 92 percent of these entering Phase I were not approved.

Oncology appears to be the most difficult therapeutic area. Over 93 percent of these drugs fail, and even in Phase III the failure rate is over 50 percent. At the other end of the scale are infectious disease drugs, which have a failure rate a bit over 83 percent.

The reason this study found lower success rates than previous ones, according to the paper's authors, may have to do with the more recent time frame they looked at, the larger number of small biotech firms (which tend to pursue riskier drugs and targets) they included, and the higher regulatory hurdles for new drugs.

"So there we have it - if anyone wants numbers, these are the numbers," Lowe writes.

"The questions are still out there for all of us, though: how sustainable is a business with these kinds of failure rates? How feasible are the pricing strategies that can accommodate them? And what will break out of this system, anyway?"