4 ms·
This article describes "basket studies" in which patients with different rumors that share underlying mutations will receive the same targeted therapies. The na
by carbocation 12y ago
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.