Seriously?! – Dear FDA,… comment reply

I started to reply to this comment within my last post Seriously?! – Dear FDA,… but I after I got going I figured it would be best if I address the comments with a new post…

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”.

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This entry was written by Salty and posted on August 16, 2009 and filed under Challenges, Cystic Fibrosis, treatments.
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4 Comments so far (Add 1 more)

I couldn’t agree with you more. In the past the FDA has approved drugs that were harmful and or fatal (vioxx). So I would assume that they are hesitant to approve any drugs, as they approved less than 69 new drugs in 2007, 26% fewer than in 2006. I too have expanded access to Aztreonam, and during my last visit understood that Aztreonam Inhaled was already available in many, many other countries. Giliad was hoping to combine the evidence from the studies done in other countries with the new data from the US head to head, Aztreonam vs.Tobi six month study which will soon close enrollment by the end of the year. I would have done that study, but I have been off of Tobi for too long (as it no longer worked). So like in everything else, the USA is falling farther behind, not being any safer and becoming more and more a large ineffective bureaucracy. Look at how poorly they are in food safety these days. I think they are in need of a reorganization /overhaul more than ever.

1. Dan on September 9th, 2009 at 9:59 pm
Maybe this article (which was just passed to me minutes ago) can answer some of your questions Don – Gilead’s FDA Warning Letter Contains Lesson About the Internet

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.

2. Salty on August 18th, 2009 at 3:20 pm
p.s. Further research reveals that back in the fall, the FDA issued what’s known as a “Complete Response Letter” to Gilead Sciences, the company developing the inhaled version of Aztreonam. The letter presumably explains the basis of the FDA’s determination that further clinical trials are necessary prior to approving the drug. I think that if Gilead, or the CF Research Institute, are going to encourage CF patients and families to lobby Congress to pressure FDA to change its mind, Gilead should release the letter or at least offer some kind of explanation of what led the FDA to decide to apply the brakes here. Presumably that’s what “complete response” means in this context.

3. Don on August 17th, 2009 at 8:50 pm
Dear Salty:

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.

4. Don on August 17th, 2009 at 7:38 pm