osullivb

I agree with you completely about direct to consumer advertising being a problem. Especially when pharmaceutical companies push products for problems that are not necessarily real ("low T" may be an issue for a small number of men, but we don't all need to be on testosterone replacement therapy).

This is tough. As a physician I try to be realistic, but given the advances that have been made over the last 20 years and the hopes that we have for even better therapies soon, it is much easier to be hopeful now than it was in the 1980s. But that hope is coming at a huge monetary cost and this is the issue. The UK won't even approve Orkambi for their CF patients because of the cost,

There is a fair amount of debate about just how much it costs to get a drug to market. Some estimates are a billion dollars or more, others are much, much lower. Unfortunately, there is very little transparency in drug pricing. Many companies roll in non-research costs (such as the advertising you allude to) in the so-called "cost" of R&D. Without more information it is very hard to know what a fair price for a medication is.

Orphan diseases (that is rare diseases) have become a huge target for pharmaceutical companies due to tax breaks given to companies developing medications for them, more rapid FDA approval, a need for small (and thus less expensive) clinical trials, increased exclusivity, and of course the huge prices they can charge and the limited competition in these niche disease categories. One major issue is that this is not a free-market situation. There is no competition, there are no alternatives if you have one of these devastating illnesses, and Congress has outlawed Medicare from negotiating with companies regarding price. It is an unfair situation where the companies hold all the cards.

Major misconception = that CF is a fatal disease for children. In 2016 more than half of people with CF in the USA are over 18 years of age.