A Faster Way to Try Many Drugs on Many Cancers

The biggest breakthrough here is the FDA's acceptance of basket studies-- claiming that they would approve drugs on it alone. That's a big win for patients, who based on current practices, would have found themselves in the control group.

This article describes "basket studies" in which patients with different rumors that share underlying mutations will receive the same targeted therapies. The name is inelegant but the logic of targeting shared mutations in a basket of genetic disease is rational. The hard part will be teasing out signal due to drug effect from signal due purely to enrollment in a trial, which as stated in the article will require large-magnitude effects, and probably heavy comparison to outside data.

One could also imagine a study protocol in which a single patient receives many drugs targeted at various mutations, and the results could make sense only at a large scale after decomposing the signal from many recipients of the cocktail. This, however, is not what is described in the article.

This startup (http://www.flatiron.com/) is attempting to create a database of cancer etc. At first I thought that the idea was a bit too ahead of its time but now its value is becoming clearer. Data will be a very powerful part of defeating cancer.

Paywall.