Codevelopment of genome-based therapeutics and companion diagnostics: Insights from an Institute of Medicine Roundtable

摘要

More than 100 therapeutics, mostly for oncology, have biomarker tests recommended in their US Food and Drug Administration (FDA) labels.1 Moreover, a strategy to codevelop and cosubmit to the FDA a diagnostic test (companion diagnostic [CoDx]) that specifically targets a drug to patients predicted to respond has had success recently with vemurafenib, targeting the BRAF V600 mutation in metastatic melanoma, and with crizo-tinib, targetingthe EML4-ALKmutation in non-small cell lung cancer.2 Companion diagnostic tests define the subset of patients who are most likely to benefit from a therapy or who should not receive the therapy because of ineffectiveness or predicted adverse effects.3 Genome-based, targeted therapeutics and codeveloped CoDx tests are the foundation of personalized medicine and have potential for contributing to high-value health care.