We have all seen the horror stories in the news 'My child will die with out this medication' or join Susie's Army on facebook to lobby the company to allow her to get their new drug to save her life. They seem like a good idea at the outset but not always.
This is called compassionate use of medications which is often allowed by the FDA and provided for by drug companies as they bring new drugs to the market. A drug isn't quite ready but the patient might actually benefit from it. It would be wonderful if the drug companies and the FDA could okay every request but it doesn't quite work that way.
First of all the FDA approval apparently is not the issue in many of these cases. The problem often comes from the companies themselves. If a company is getting a drug ready for market, they make enough of what they need for the next step in their clinical trials and allow for a little extra for these cases or whatever reason.
So they simply might not have any more doses to give out without going back into production - which can be VERY expensive. Think of making cupcakes for 24 people. All of a sudden you need 25 so you have to start all over again for a single cup cake. A lot of effort for a single cup cake.
Second of all, social media may not be a big help at all. Social media has created 'patient's armys' full of well intentioned people who do things like threaten the lives of the pharmaceutical executives. That doesn't exact endear the patients to the pharmaceutical companies.
This is a growing problem. New drugs seem like they are the solution for the patient but they still are not completely tested. Could they lead to more problems for the patient down the road?
There is lots more to this. Go read this article on Drugs for the Dying and see a few stories up close.