Clinical trials are the most important step of getting a new drug approved for marketing, as well as the most expensive, and it’s no secret that the odds of bringing a project from first-in-human testing through to market are pretty long.
Once a program reaches the pivotal trial stage, the odds are supposed to improve, of course. But every year, plenty of drugs fail at the last hurdle after millions or even billions of dollars have been invested in them.
This year’s top 15 list has been drawn up to include trials whose failure we judge to have had material consequences on their sponsors and the patients for whom the drugs are being developed.
For the sponsors, that can mean wasted investment as well as missed commercial opportunities, and in some cases raise questions about the future of a therapeutic platform or even the entire company. When it comes to patients, we’ve focused on trials that missed the mark in diseases with no approved therapies or that could have provided a clinically significant step forward in treatment.
The catalog includes multiple trials for aggressive brain cancer, Alzheimer’s disease and nonalcoholic steatohepatitis, all recognized as seriously tough diseases for drug development. Heart failure as a whole meanwhile has seen some strides forward in the last few years, but some forms remain resistant to treatment, and a pair of drugs on this year’s list fell short in that setting.
A range of large and smaller companies are represented, but some names are more prominent than others. Novartis sponsored three of 2019's biggest trial flops, while AbbVie and Biogen/Eisai sponsored two apiece. A range of technologies are also featured, from small molecules through biologics, vaccines, gene therapy and gene-editing drugs.
We’ve also included a plus one: a medicine that was written off earlier in the year only to stage a miraculous comeback later. You can probably guess that one, but read on for the full breakdown.