Off-label use is the practice of prescribing pharmaceuticals for an unapproved indication or in an unapproved age group, unapproved dose or unapproved form of administration (Link). There is a new drive to run clinical trials, before FDA approval, and develop hypotheses and clinical trials based on patient results. This method is only currently being used for rare and life-threatening disease. It is proposed to be beneficial for testing drugs with a small affected population, for whom a generic clinical trial would be ineffective or un-feasible regardless of FDA approval.
For those without FDA approval, patient results could be a way to get that approval for a trial that is developed from the results. The conclusion, made by the developers of the portal that tracks the information and patient results, is that one day patients or clinicians themselves might run their own trials.
But there are some ethical questions that this brings up. Should patients and clinicians be developing their own clinical trials for drugs they had no role in developing? How are recommendations made for the dosage and administration of these drugs?
So what do you think?