> Who tells dying patients they are not allowed them, and is there a black market or large amounts of money changing hands for experimental procedures?
The FDA regulates every aspect of clinical trial design. Dying cancer patients are always the first to receive untested drugs -- the FDA has set the bar that any new therapy must outperform what is currently used. There is no "black market" for experimental procedures, mostly because you must visit a hospital to receive them in the first place. Some drugs are intravenous; all are given with standard oncology examination. A doctor cannot write a prescription for a drug not on the market yet for you to pick up at the pharmacy!
> Surely with hundreds of thousands of desperate, dying, last chance sufferers, it is better to go to extreme measures and offer the most promising yet dangerous treatments to everyone. Is it simply a side effect of the way pharmaceutical companies have to do business?
In order for the testing of a Phase I trial to be complete, patients must meet certain criteria. The FDA (and, by extension, the pharmaceutical companies who want FDA approval) require enrolled patients in a clinical trial to have similar levels of disease. A patient that is clearly near end of life may not be able to physically swallow a pill...their outcome may bias the trial's results.
Yes, there are desperate and dying cancer patients, but a Phase I trial can (and should!) only accommodate dozens of them to achieve proper statistical power. Phase II may take hundreds, and Phase III will take even more -- but when a drug is approved, it has the possibility of being given to many thousands or millions of patients all at once.
As the trial population grows, medicine rears its stochastic side -- despite patients having pathologically similar disease, some will be helped and others will not. This is at the core of all FDA trial design: big populations are necessary to see just how effective your drug is. It should not be surprising, then, that the majority of failures happen at the bigger scales (PhII and PhIII).
The FDA regulates every aspect of clinical trial design. Dying cancer patients are always the first to receive untested drugs -- the FDA has set the bar that any new therapy must outperform what is currently used. There is no "black market" for experimental procedures, mostly because you must visit a hospital to receive them in the first place. Some drugs are intravenous; all are given with standard oncology examination. A doctor cannot write a prescription for a drug not on the market yet for you to pick up at the pharmacy!
> Surely with hundreds of thousands of desperate, dying, last chance sufferers, it is better to go to extreme measures and offer the most promising yet dangerous treatments to everyone. Is it simply a side effect of the way pharmaceutical companies have to do business?
In order for the testing of a Phase I trial to be complete, patients must meet certain criteria. The FDA (and, by extension, the pharmaceutical companies who want FDA approval) require enrolled patients in a clinical trial to have similar levels of disease. A patient that is clearly near end of life may not be able to physically swallow a pill...their outcome may bias the trial's results.
Yes, there are desperate and dying cancer patients, but a Phase I trial can (and should!) only accommodate dozens of them to achieve proper statistical power. Phase II may take hundreds, and Phase III will take even more -- but when a drug is approved, it has the possibility of being given to many thousands or millions of patients all at once.
As the trial population grows, medicine rears its stochastic side -- despite patients having pathologically similar disease, some will be helped and others will not. This is at the core of all FDA trial design: big populations are necessary to see just how effective your drug is. It should not be surprising, then, that the majority of failures happen at the bigger scales (PhII and PhIII).