We now have an updated look at clinical success rates in the industry, and it’s a timely topic. Last year there were 59 approvals by the FDA (a new record), and the year before was good as well. So the question is always whether such numbers are artifacts, random noise, or part of a real trend.
This article compares the success rates at the beginning of the 2010s with the 2015-2017 period, so we’re missing last year’s record haul in this data set. But even so, there are differences. First off, though, the chances of success for a compound entering Phase I trials have not increased: it’s still slightly under 10%, same as other estimates over the last couple of decades. That (to me, although it’s not just me!) is probably the single more important figure in the pharmaceutical industry. No other major business type operates under such a high failure rate, and it’s the source of so many things that makes ours distinctive. And I mean “distinctive” in all its senses, ranging from impressive to infuriating. Greater than 90% failure in the central, crucial process of the whole industrial drug discovery business: fix that and everything changes.
But that said, this landscape of failure might be changing. If you can get a compound all the way to Phase III, the historical success rate at that point has been a bit under 50%. But recent cohorts have been showing increased success from Phase III, which is interesting. I have no idea if this is an artifact, but you can be sure that people will be running the numbers with the 2018 set (and this year’s eventual approvals, etc.) to see if it’s a trend that holds up or not. Phase II to Phase III success has been running at pretty much the same levels, though.
The way these numbers can add up is that there are far fewer compounds in that Phase III set, so increased success there doesn’t lift the much larger overall numbers all that much. The reasons for these failures appear to be unchanged: 75 to 80% are due to problems with efficacy and/or safety. And that, to me, is the second most important figure in the pharmaceutical industry, showing just how much we don’t know about what we’re doing. What targets to pick, what else they might do when we affect them, what other things our compounds might do when we try: find a way to understand and predict those things better and everything changes. As for breakdown by therapeutic area, CNS and cardiovascular drugs are at the bottom of the probability of success list at Phase III, followed by musculoskeletal and oncology drugs. Respiratory, metabolic, and anti-infective drugs are all notably higher (these are for the whole 2010-2017 period).
Meanwhile, and this might be surprising, the number of drugs in early-stage development has apparently been dropping steadily since the 2009-2011 period. That’ll be an interesting wave to watch as it works its way through the big python of drug development. Are we doing fewer but better projects? Wait and watch. We may already be seeing it; the number of compounds in late-stage development declined in the 2015-2018 period for the first time in years. So those Phase III-to-approval success rates do need to hold up.
Finally, the share of these new drugs that have been brought to market by the large pharma companies has been declining every year since 2013. I’m not quite ready to opine on that, though, because that still just takes the numbers back to 2010 levels, so it’s not like some unprecedented shakeup. But it’ll be interested to see if that trend continues, and at that point I’m sure we’ll all have opinions to share freely!