This is something that I am passionate about. The "go it slow" approach to drug trials when millions of people are afflicted with a disease that will kill or cripple them.
My father had multiple sclerosis. Although he did not know it the military diagnosed the MS back in the late 50's and just referred to it as an unspecific condition. Later, in the early 70's he was involved in an accident at work (not caused by the MS or anything his fault) and the traumatic event caused the disease to become active again. It took a further five years for the doctors to identify that his ongoing medical problems were caused by MS and was verified when they finally got a hold of his military records that showed the diagnosis 20 years earlier.
As a teen and young adult I watched a healthy, active man gradually lose his ability to walk, participate in family activities and suffer terrible mental changes. I know that if he had been given any sort of opportunity for an experimental treatment, even if it had a chance of causing a premature death, he would have taken it. We, as his family would have stood behind him all the way.
The medical community wants to preserve this illusion of scientific principle by treating promising medical procedures as if they were in a laboratory setting. Double-blind testing, keeping folks in a study as you dispassionately watch them deteriorate and die and then compiling your report to show how 90% of the untreated folks died while only 85% of the treated folks died is a failure. I bet in that sample for those 5 out of 100 folks who "beat the odds" they did not consider the treatment a failure.
Medical advances are always made in small steps. 1-2% here or there off of the mortality of any condition and sooner or later you are talking about real lives.
There are many drugs that provided a positive benefit in treating an invariably fatal condition but had some much smaller incidence of causing a different type of fatal condition. To the folks who are facing a death sentence without treatment for something like MS, many would take what treatment they can get today.
This is not some acne medicine that may cause your liver to fail where the side effect is much worse than a bad looking school yearbook picture. Humans when put in extraordinary situations will to to extraordinary lengths to find a treatment. This is why folks to to Mexico or Thailand or the back-room doctor for an unapproved therapy. Do you really think they give a damn about the legality of it all?
Why in the hell do you need a control group for a study? You have 99.99% of the people who are not in the study as your control group. Doctors, hospitals, research centers, drug companies and regulatory agencies need to provide for whatever minimal beneficial effect to treating a condition once it is clear that the patient and patients family understands that there is no magic bullet for everything.