Introduction

The U.S. Food and Drug Administration (FDA) has approved both gene- and immune-targeted drugs for tissue-agnostic, genomic biomarker–based indications in patients with lethal solid tumors and blood cancers. High response rates were the basis for these approvals.[1]

Precision medicine has rapidly changed the oncology field. Next-generation sequencing can identify patients with specific gene alterations or immune markers for which there are targeted therapies. Biomarker-based treatments are increasingly emerging, and the FDA has approved the following tissue-agnostic therapies:[1,2]

Precision medicine is constantly evolving, and improved diagnostic tools allow for more comprehensive genomic definition of the tumor. Tissue-agnostic targeted therapies are a promising treatment strategy tailored to the specific tumor genomic profile, leading to improved patient survival outcomes.

The list of drugs approved for tumors with genomic alterations will likely expand as understanding of the science driving these cancers continues to advance. As oncology drug development continues to move rapidly, the location where the cancer originated will become less critical.

Companion tests can determine the presence of specific genomic alterations or immune markers. These tests may have been approved by the FDA as part of the agnostic drug approval. Most insurance companies have not required these specific tests before covering the prescribed medications.