The new Report from Amplion, BIO, and BioMedTracker examined approximately 10,000 clinical trials by therapeutic area as well as three categories: rare diseases, chronic high prevalence diseases, and programs using selection biomarkers.
In order to quantify the benefit of using selection biomarkers in drug development, the researchers combined Amplion’s BiomarkerBase with BioMedTracker’s clinical transition records.
According to the report, the Likelihood of Approval (LOA) of a new drug increased from 1 in 10 to 1 in 4 when using biomarkers.
While the researchers were expecting biomarkers to improve LOA, John Audette, Amplion’s founder and president, and one of the report’s authors, told us the scale to which biomarkers had an impact was a surprise.
“I think we all knew intuitively that using biomarkers to select responsive patient populations would yield more successes, but the finding that biomarker-enabled drug programs are three times more likely to reach approval was definitely a surprise,” Audette said.
Improving trial success rates
In the study, researchers focused on biomarkers that are used to identify patients who are most likely to respond to a developmental drug, which in turn increases the chance that a therapeutic benefit will be demonstrated.
“It also means that you exclude patients who are unlikely to respond, and both of these dynamics increase success rates,” explained Audette. Biomarkers can also be used to indicate whether drugs have the desired effect, which can also increase success rates.
“Patient selection biomarkers are the key enabling factor to precision medicine, and they are fundamentally changing drug development and dosing strategies,” added Audette.
The focus on precision medicine is causing a paradigm shift in drug development in which companies that originally focused on developing blockbuster drugs are now embracing programs with narrower indications, shorter development timelines, and reduced costs.
Audette uses a baseball analogy to explain the benefits, describing the new dynamic as less home runs and more singles and doubles. He added, “and of course patients benefit because they aren't prescribed drugs that aren't going to provide them any benefit.”