Cancer genome sequence data combined with incidence statistics suggests large numbers of patients could benefit from off-label therapies.


Targeted molecular therapies offer the hope that they can be used in any disease that carries the specific molecular aberration being targeted. Combining data on the prevalence of specific molecular aberrations with cancer incidence statistics allows estimates to be made of the number of patients that might benefit from off-label treatment. In some cases the estimated population of potential patients is as large as the population already receiving the therapy for its approved indication. Whilst these estimates are easy to make, clinical trials required to verify new indications are costly and complex, and there are already examples where this off-label treatment has failed; however this estimation may help with the prioritization of therapies, molecular tests and patient groups to focus research efforts on. The promise of personalized medicine is being realized in oncology. The potential is that next-generation sequencing may offer a route to next-generation
CDx tests.

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