Maybe I should keep my trap shut about some things. I do know there are some studies out now where the participant is required to sign a confidentiality notice. I did not have to with this program, therefore the posts I make are my opinion and my opinion only.

Thank you for taking my comment seriously and for posting such a long and thoughtful reply.

I did the same google search you did, and read the same document from the CF Research Institute, which makes many of the same points you have been making. But what I still don’t understand is what validly scientific basis underlies the FDA’s decision. The FDA doesn’t win brownie points with anyone by withholding a new drug from the market; the manufacturer, obviously, wants to start earning a return on its investment in R&D and patients, as you emphatically note, want the benefit of the new treatment. Even the insurance companies shouldn’t object, assuming the drug helps keep people away from those expensive inpatient stays.

My day job is as a law professor, but prior to that I worked for nine years as a bureaucrat in a state agency, rising to the post of general counsel. Though our agency did not have anything to do with health issues, some of what we did aroused emphatic public opinion, and I noticed that cynicism about government is so rampant that when individual citizens disagreed with something we did they tended to leap immediately to the explanations that involved venality, laziness, corruption or general bad faith. Almost always, we were really just doing what we thought was right, given the law we were tasked with implementing, although in candor I have to say that once in a while there was a bit of laziness in there. My point is that citizens who are affected by the FDA’s decisions owe them the benefit of the doubt, if only to avoid squandering our credibility. For a look at the alternative — i.e., no federal agency to review drug therapies before they are generally available — look at what was going on before there was an FDA and anyone could peddle anything and make any claims about its efficacy that whim and desire for a fast buck prompted.

This coming semester I will be teaching Administrative Law for the first time. Though the course covers general topics, we try to illustrate them by keeping a current-events focus on one particular agency. I’ve chosen to have my class keep an eye on the FDA, one reason being the interest in the agency that I have as a CF dad. I will get to the bottom of this, if only to educate myself about what legal authority, and what exercise of judgment and discretion, would lead the FDA to defer approval of such a useful therapy.

As for the CF Foundation, I try to be especially circumspect about assigning ignoble motives to them. I don’t always agree with everything the Foundation does, and if you keep an eye on their work regularly you definitely can find examples of when they don’t walk the talk. But the fact is — please forgive me for being blunt — that without the CF Foundation you’d be gone by now, as would my daughter.

In any event, I don’t get it either. The efficacy of the drug appears to have been proven and I don’t understand what is preventing the FDA from approving it. But I will do my best to find out.