Few investigational cancer drugs are approved by the US Food and Drug Administration (FDA) within 10 years of entering clinical trials, according to research published in the Journal of the National Cancer Institute.

Researchers found that the probability of FDA approval within 10 years was 10.4%, and the probability that development would stall within 10 years was 49.2%.

Researchers evaluated 572 investigational cancer drugs that were starting first-in-human trials in 2010-2015. The agents were most commonly small molecules (55.2%), antibodies (25.9%), and antibody-drug conjugates (7.7%). 

Continue Reading

These investigational cancer drugs were evaluated in more than 6000 trials through the end of 2020, with a mean of 95 drugs entering clinical development per year (range, 76-112). Over a mean follow-up of 8 years, 173 drugs were not tested beyond first-in-human trials. 

A total of 39 investigational cancer drugs (6.8%) were approved by the FDA during follow-up. The median time to approval was 6.1 years, and the drugs had been tested in a mean of 26 trials.  

The probability that an investigational cancer drug would receive FDA approval was 2.6% at 5 years and 10.4% at 10 years. The probability of a drug’s development stalling within 10 years of entering trials was 49.2%.

Nearly a third of the drugs (32.2%) had development stalled over a mean follow-up of 8 years. Most drugs (72.3%) stalled after a first-in-human phase 1 trial, 24.5% stalled after a phase 1 trial that was not first-in-human, and less than 0.1% had development stalled after a phase 2 trial. 

Pediatric testing was conducted for 67 of the investigational cancer drugs examined (11.7%). The median time from the start of clinical development to the first pediatric-eligible trial was 4.6 years. 

Of the 39 cancer drugs that were approved during follow-up, 5 were approved for use in pediatric cancers. The probability of an investigational cancer drug attaining pediatric approval within 10 years was 1.4%.

“More efficient clinical development strategies are needed to evaluate new cancer drugs, especially for children, and incorporate approaches to ensure knowledge gain from investigational products that stall in development,” the researchers concluded.

Disclosures: One study author declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.


Arfè A, Narang C, DuBois SG, Reaman G, Bourgeois FT. Clinical development of new drugs for adults and children with cancer, 2010-2020. J Natl Cancer Inst. Published online May 12, 2023. doi:10.1093/jnci/djad082

This article originally appeared on Cancer Therapy Advisor