Between 2012 and 2014, more than 11 percent of clinical trials succeeded, which meant compounds being tested survived the arduous journey from the laboratory to the pharmacy counter. This reversed a downward trend seen over the past 20 years, according to executives at McKinsey & Co., the consulting firm that conducted the analysis and does consulting work for drug makers.
Between 1996 and 1999, the cumulative success rate was 16.4 percent, but gradually began declining in subsequent years. From 2000 to 2003, 10.8 percent of trials succeeded before falling to 10 percent between 2004 and 2007, and then bottoming out at just 7.5 percent between 2008 and 2011.
“In some ways, this is rather remarkable,” said Martin Moller, one of the coauthors, who heads McKinsey pharma R&D consulting in Europe, the Mideast, and Asia. “We’ve been used to seeing a decline. This is a turning of the trend. Whether that is sustainable is an open question.”
The analysis, which was published last month in Nature Reviews Drug Discovery, examined more than 9,200 novel compounds that were developed from 1996 through 2014.
The declines in past years likely reflected industry cutbacks, according to the McKinsey consultants.
From 2007 through 2010, many drug makers were cutting back on R&D amid large mergers and reorganizations that resulted in what was described as “pipeline pruning.” Basically, most of the largest companies turned their backs on researching medicines for combating certain diseases and became much more selective about how they invested their research dollars.
For this reason, the McKinsey team suggested the more judicious use of resources resulted in higher quality product pipelines which, in turn, could account for the improved showings in clinical trials. The larger proportion of failing compounds in Phase 1, or early-stage trials, may indicate that companies are running more thorough evaluations and doing so sooner.
“Basically, the industry is learning how to fail earlier,” said Moller. “And that’s a good thing.”
This could be seen by looking at data for both Phase 2 and Phase 3, or mid- and late-stage, studies. From 2012 through 2014, the likelihood that a Phase 3 study would advance to product registration was 64 percent, up from 60 percent during the previous three-year period. Similarly, the odds of a Phase 2 study moving to the next round was nearly 40 percent, up from about 30 percent during 2007 and 2010.
There was a caveat, though. The increasing success rates for midstage trials may be slightly inflated not only because there are more life-saving medicines being shepherded to later-stage trials thanks to new trial designs, but also because of breakthrough regulatory mechanisms. “Such an approach carries a risk of a later-stage failure,” the consultants wrote.
For instance, drugs that were developed for treating rare diseases, which can benefit from speedier development paths, had a higher overall success rate from Phase 2 to regulatory approval. During the past three years, this amounted to 29 percent compared with 10 percent for drugs used to treat other maladies. In Phase 3 trials, the success rates were 73 percent and 64 percent, respectively.
In any event, what remains unclear is whether the ongoing rebound in clinical trial successes can continue, given the variables and uncertainty surrounding drug development.
“It’s going to take a little time before we come back to [the] level of confidence around [what we saw in] this area in the late 1990s and early 2000s,” said Moller. “Everyone still sees pharma R&D as a precarious game … Even though the aggregate success rates now have turned, it’s still true that there will be enormous variations among individual companies.”