Slim chance participants in phase 1 cancer trials will benefit

Only a tiny proportion of therapies are ultimately approved, researchers say
Reuters Health Staff writer
patient having somethign explained to her by doctor with clipboard

The chance that a therapy used on a patient in a phase 1 cancer trial will be later approved by the US Food and Drug Administration is almost as slim as one in a 100, a review suggests.

Only 1.2% of participants â€” in a total of 1000 phase 1 cancer drug trials â€” received a treatment that was later FDA-approved for the indication and dose they received.

Given published estimates of serious adverse event rates of 10%-19%, this represents low therapeutic value for phase 1 trial participation, the authors conclude in the Journal of the National Cancer Institute.

To assess what proportion of phase 1 trial participants may receive a therapeutic benefit, the researchers randomly selected 1000 phase 1 oncology trials initiated between 2005 and 2010 from a total of 3229 eligible trials on ClinicalTrials.gov.