The landscape of precision cancer medicine clinical trials in the United States

Published:February 28, 2015DOI:


      • We identified 684 precision cancer medicine trials from
      • 38 unique genomic alterations were identified for enrollment.
      • These trials were more likely to involve breast, colorectal and skin cancers.
      • The proportion of these trials increased from 3% in 2006 to 16% in 2013.



      Advances in tumor biology and multiplex genomic analysis have ushered in the era of precision cancer medicine. Little is currently known, however, about the landscape of prospective “precision cancer medicine” clinical trials in the U.S.


      We identified all adult interventional cancer trials registered on between September 2005 and May 2013. Trials were classified as “precision cancer medicine” if a genomic alteration in a predefined set of 88 genes was required for enrollment. Baseline characteristics were ascertained for each trial.


      Of the initial 18,797 trials identified, 9094 (48%) were eligible for inclusion: 684 (8%) were classified as precision cancer medicine trials and 8410 (92%) were non-precision cancer medicine trials. Compared with non-precision cancer medicine trials, precision cancer medicine trials were significantly more likely to be phase II [RR 1.19 (1.10–1.29), p < 0.001], multi-center [RR 1.18 (1.11–1.26), p < 0.001], open-label [RR 1.04 (1.02–1.07), p = 0.005] and involve breast [RR 4.03 (3.49–4.52), p < 0.001], colorectal [RR 1.62 (1.22–2.14), p = 0.002] and skin [RR 1.98 (1.55–2.54), p < 0.001] cancers. Precision medicine trials required 38 unique genomic alterations for enrollment. The proportion of precision cancer medicine trials compared to the total number of trials increased from 3% in 2006 to 16% in 2013.


      The proportion of adult cancer clinical trials in the U.S. requiring a genomic alteration for enrollment has increased substantially over the past several years. However, such trials still represent a small minority of studies performed within the cancer clinical trials enterprise and include a small subset of putatively “actionable” alterations.


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