Biomarkers and receptor targeted therapies reduce clinical trial risk in non-small-cell lung cancer

This study analyzed the risk of clinical trial failure during non-small-cell lung cancer (NSCLC) drug development between 1998 and January 2012. We also looked for factors that impacted clinical trial risk in NSCLC.

NSCLC drug development was investigated using trial disclosures from http://www.clinicaltrials.gov and other publically available resources. Compounds were excluded from the analysis if they had begun phase I clinical testing before 1998, did not use treatment-relevant endpoints, or if they did not have a completed phase I trial in NSCLC. Analysis was conducted in regard to treatment indication, compound classification, and mechanism of action.

Six hundred seventy-six clinical trials that included 199 unique compounds met our inclusion criteria. The likelihood, or cumulative clinical trial success rate, that a new drug would pass all phases of clinical testing and be approved was found to be 11%, which is less than industry aggregate rates. Over half of the biomarkers used in NSCLC have not yet been approved by the Food and Drug Administration in any indication. Biomarker targeted therapies (62%) and receptor targeted therapies (31%) were found to have the highest success rates. The risk-adjusted cost for NSCLC clinical drug development was calculated to be U.S. $1.89 billion.

Biomarker use alone in this indication resulted in a sixfold increase in clinical trial success whereas receptor targeted therapies did so by almost threefold. Physicians who enroll patients in NSCLC trials should prioritize their participation in clinical trial programs that use biomarkers and receptor targeted therapies.