Trying to get investigational medicines outside of a clinical trial is challenging. Compassionate use of a medicine happens when an investigational drug is used outside of a clinical trial to help someone with a serious or life-threatening condition or disease.
When does this happen? The FDA regulations do allow intermediate sized groups or even individual patients to have access to experimental medicines on a case-by-case basis...although certain characteristics may NOT allow them to be in a clinical trial AND no comparable or satisfactory alternative treatment options are available. Benefit to risk considerations are crucial and some manufacturers still may not allow or may not be willing to provide access to an investigational drug outside of a clinical trial.
For companies who do allow use of investigational drugs outside of a clinical trial, please remember that safety and efficacy has NOT been proven and that the medicine may be associated with uncovered drug costs. Your doctor must approach the maker of the medicine and must make the benefit to risk decision about the drug versus your condition. Additionally, an Investigational Review Board (IRB) must review the use of these medicines.
All in all, expanded access is a challenging and in some cases seductive road. Despite the uncertainty, those facing life-threatening and serious diseases might gain use of a medicine that could otherwise not be obtained.