UCSF

Aim

Drug approvals for genome-informed indications have been increasing in recent years, but it is unknown how many of them have demonstrated an improvement in overall survival (OS). We assessed the frequency of approved genome-informed drugs demonstrating improvements in OS and progression-free survival (PFS) and whether the frequencies differed by cancer type.

Materials and methods

We searched all Food and Drug Administration approvals from 2006 to 2020, and for each drug that was approved for a genomic indication, we then searched on PubMed for randomised studies examining OS or PFS.

Results

We found 53 drugs approved for 92 unique indications from 2006 to 2020. We found that 50 drugs (55%) approved for a genomic indication had a randomised study evaluating OS benefit, and of those, only 22 demonstrated an improvement in OS. Similarly, 52 drugs (57%) evaluated PFS benefit, and 51 of these studies demonstrated an improvement in PFS. Drugs approved for BRAF V600 melanoma demonstrated an improvement in OS more often than drugs approved for ALK non-small cell lung cancer. The median improvement in OS was 4.7 months (range 1.5 months-49.1 months).

Conclusion

Although there is widespread enthusiasm for this class of agents, and many demonstrate impressive response rates, 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.