The organization "Kids vs Cancer" offers help to pediatric patients for access to experimental drugs that are in trials but not approved yet:
http://www.kidsvcancer.org/compassionate-use/
Sometimes a medication shows promise in a trial but the patient is not eligible for the trial and can only get access to the drug through compassionate use. This happened with yondelis (trabectedin) which was in trials for many years before it was finally approved...