There is a lack of survival data for many oncology drugs approved for genomic indications in recent years, according to research published in the European Journal of Cancer.

Researchers looked at drugs approved by the US Food and Drug Administration (FDA) for genomic indications from 2006 to 2020.

The team found that overall survival (OS) data are available for about half of these drugs, and about one-fifth of the drugs demonstrated improvements in OS.

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The analysis encompassed 53 drugs approved by the FDA for 92 genomic indications. This included 58 regular approvals and 34 accelerated approvals.

Indications with the most approvals included drugs targeting the Philadelphia chromosome (14 approvals), EGFR (12 approvals), HER2 (10 approvals), BRAF V600 (9 approvals), and ALK (8 approvals).

The most common cancers were non-small cell lung cancer (23 approvals), breast cancer (12 approvals), chronic myeloid leukemia (10 approvals), colorectal cancer (8 approvals), and melanoma (7 approvals).

There were 50 drugs (55%) that had been evaluated for OS in a randomized trial and 52 drugs (57%) that had been evaluated for progression-free survival (PFS).

Overall, 22 drugs/indications (24%) demonstrated an improvement in OS, and 51 (55%) showed an improvement in PFS.

There were 34 drugs/indications that showed no improvement in OS, PFS, or overall response rate (ORR). However, 15 drugs/indications demonstrated improvements in all 3 endpoints.

There were 18 drugs/indications that demonstrated an improvement in PFS alone, 2 showing an improvement in OS alone, and 3 showing an improvement in ORR alone.

The researchers acknowledged that this study is limited by the fact that they used only PubMed to search for randomized studies associated with each drug and indication.

Despite that limitation, the researchers concluded that these results suggest “further trials or post-marketing studies are needed to ascertain the impact on survival and quality of life, the magnitude of these gains, and the cost-effectiveness of these agents.”

Disclosures: This research was funded by Arnold Ventures. One study author disclosed affiliations with Arnold Ventures and other organizations. Please see the original reference for a full list of disclosures.


Haslam A, Kim MS, Prasad V. Overall survival for oncology drugs approved for genomic indications. Published online November 21, 2021. doi:10.1016/j.ejca.2021.10.028