Here’s the comment:

Dear Salty:

I think you’re being a little unfair to the FDA. They made a decision the CF community didn’t like — but that doesn’t mean they’ve been unethical as you suggest. And “foot dragging” would have meant no decision at all.

Still, I share your irritation because nobody — not even the CF Foundation — seems inclined to to explain exactly why the FDA decided that further clinical study was necessary. It always gets my dander up when the experts won’t let the regular folks in on the secrets, especially when their work is of such importance to CF patients like yourself.

The decision about which you complain was actually made last September and, as best I can tell, did not result in all access to inhaled astreonam being cut off. From the CFF web site:

“Because the FDA did not raise any safety issues related to the drug in their ruling, Gilead [the manufacturer] will continue to provide early access to aztreonam lysine for inhalation for those CF patients with limited treatment options and a serious risk of disease progression. Individuals with CF should contact their care centers to determine their eligibility to participate in the EAP.”

Presumably you’ve done that . . .?

My reply:

Although I appreciate your comment, it appears you aren’t completely up to speed on the study I speak of. The FDA has made clear their reasoning… the new board is not up to speed (pardon the pun) with the needs of cystic fibrosis patients, nor understands the need of getting drugs to market quickly to help save people’s lives.  As far as “foot dragging” I don’t know exactly how to address that.  The drug was up for review, they asked for more study results, Gilead studied, got more results, appealed the decision, the FDA now asks for another study, which will be reviewed at yet a later date… despite ”the FDA did not raise any safety issues related to the drug in their ruling” as you quoted.  I don’t know if there are better words than to say they are dragging their feet …as I have stated before, numbers are my game, not words so I apologise for my lack of vocabulary. …Yet this entire time  If there are no safety issues are related to the drug, then why on earth are they not approving it? I just don’t get it, and I guess that is what I was trying to describe in my previous post.

The inability to receive proper treatment for one month, may shave YEARS off a cystic’s life, no exaggeration there my dear. Speed means lives being saved. An ounce of prevention is worth a pound of cure my mother always told me. A good solution now is better than the best solution ten years from now.

For example, the year previous to my start on inhaled aztreonam I suffered horribly from lung infections, three rounds of IV antibiotics, at least three weeks in each round. And guess what drug I was infusing in my veins????? – AZTREONAM! Now if I happened to be lucky enough to have enrolled in the expanded access program at that time, I could have saved my insurance company quite a bit of money for the hospitalization, picc line placement, home nursing… the list goes on and on. Plus my employer would have benefited, not only from my ability to work instead of being sick (considering I was sick for weeks prior to each IV treatment), but maybe it would have kept their insurance premiums from going up astronomically (but that’s just speculation).  Not to mention that since starting the study I have not had a single round of IVs, and my need for oral antibiotics (which are pretty much useless to me anymore) has almost deminished.  I have yet to miss a day of work since starting on this program due to lung issues.

An excellent description of why the FDA has not approved the use of inhaled aztreonam, and has denied appeals since September 2007, is spelled out here by the Cystic Fibrosis Research Institute.  I found it with a simple google search, yes, I do my homework. APPEAL TO FDA FOR APPROVAL OF INHALED AZTREONAM TALKING POINTS FOR ADVOCACY (pdf file)

Perhaps the CFF has been keeping the reasoning quiet as to not step on the FDA’s new board member’s toes? You really don’t want to go pissing off someone saying they aren’t knowledgeable of a topic when they have the authority of denying what you are desperately trying to get approved.  Especially when people’s lives are at stake. -makes sense to me.

Not just anyone can be on the expanded access program (link to details on ClinicalTrials.gov), unfortunately. There are 115 CFF approved care centers and only 65 are participating in the study. Also the study is limited to those who have a lung function with an FEV1 LESS than 50%.  Hence, the Expanded Access Program is not open to those with a lung function above 50%, which rules out most children, adolescents and young adults who definitely could use this drug as a preventative measure, allowing them to keep their highly valued high lung function until a cure comes along.  This program does not account for other people who may not have cf, but still culture pseudomonas, or other bugs that aztreonam could possibly treat.

Yes, I am on this expanded access program. I am getting my inhaled aztreonam for free.  If I didn’t think it could help others, I sure as hell wouldn’t be wishing the FDA would hurry up and approve this drug because then I’d have to open up my wallet.  BUT, out of the goodness in my heart, I wish others could get on this drug, the ones who do not qualify to be in the study, because I know for a fact it has extended my life in years, and improved my quality of life greatly.  I wish the same for my co-cystics, my transplanted buddies, and others who must deal with the nasty vicious Pseudomonas Aeruginosa maybe due to severe lung disease like COPD, PCD, and the list goes on.

I must say again, I just don’t “get it”.

Finding Brodie has turned out to be an absolutely priceless wonder for my health. He is always there to cheer me up and doesn’t shy away when I have to take my insulin shots. Now I only wish I could turn him into a diabetic service dog, but being so hyper-active (him not me! haha) I don’t see how I could accomplish that.

I also found out the hard way I have a new allergy to Bactrum. Strange to me since I could take it as a kid, but not anymore! Scary to be since I am already allergic to Penicillin, and have built up a resistance to a lot of other antibiotics… hopefully the researchers can find some more drugs to help soon!