Jeannie or someone set up petition for FDA comment?

[Aboveallislove]

I'll be doing a detailed comment to the FDA for the combo for approval, but thought it would be good to have one with as many folks signing as possible. I'm wondering if Jeannie could put together here or somewhere a "petition" type thing where folks can sign their real names connection to CF, etc. (while maintaining anon. they have here). (I.E. Joe SixPack, 40 ddf508, Chicago, Illinois). Or if someone else can do if Jeannie can't. I draft the following which is rough. My goal was to keep it both short and non-controversial so everyone would feel comfortable signing, but my language might not be the best. ...writers block this am!!

Needs to be in by end of April, but once we get "final" language and it posted somewhere, if we could get it published here and all the other support places folks use for signatures then we could really hit the FDA with the voice of the CF community.

Pulmonary-Allergy Drug Advisory Committee
Docket No. FDA-2015-N-0001
NDA 206038

We, the undersigned, are individuals suffering from Cystic Fibrosis and/or their caregivers and loved ones. We write in support of the NDA for lumacaftor/ivacaftor and strongly urge the Committee to recommend this drug to the FDA for approval.

We recognize that the average FEV improvement shown in clinical trials is modest when compared to ivacaftor, but even a modest improvement in FEV, when living with a degenerative disease, is a huge benefit. Further, this drug offers the chance for patients to remain stable until better drugs are available. Moreover, in reviewing the NDA, we urge the Committee to consider the very real benefit this drug offers to those with Cystic Fibrosis in the form of reduced exacerbations, improved weight, and overall improved quality of life.

Submitted by CysticFibrosis.com, on behalf of its members and the undersigned:

 

[Imogene]

Of course! Let me think about the best way to do this..Peter and Dank will have ideas too....this type of thing...comes through email all the time. We have members who may post ideas of the best software etc to use to get all the signatures together and to maintain anonymity while seeming to be authentic. I think it is a good Easter project as families gather we can tell more the story of why this matters, reaching the FDA and getting approval will save lives!
let's find out who is on this committee.
Salt and light,
jeannie

 

[Aboveallislove]

Wonderful! A couple of thoughts:
1) Individuals can also send in their own comments. My view is that longer more personal comments should be INDIVIDUAL ones (I'll be doing one), but that a short and sweet paragraph with 1000s of signatures would be very helpful as well. (Not that it has to be MINE short and sweet paragraph.
2) I think we need real names, but that if we can do it without logging in here, that would be better so that here is still anon. but their it is real.

Thanks so much!

 

[Imogene]

http://www.fda.gov/AdvisoryCommitte...y-AllergyDrugsAdvisoryCommittee/ucm107568.htm

this is the roster with their terms...it looks like they should understand this community! And know how important this request is!

 

[Dank]

Hm! I like the idea. End goal is to get them to allow this drug on the market for all, instead of just individual genetypes, is that correct?

Do petitions usually present good results in this sort of situation?
How do drugs usually get through the FDA for all users? Is that usually done by drug trials?
Do we know of any circumstance where a petition has approved a drug in the past, with other conditions maybe? This would be great leverage to use.. xyz petition followed through on abc date and this is our way of presenting the same data so you can approve us - in so many more articulate words of course..

Is there a better way to get to our end goal?

I want to brainstorm here and make sure we're spending time on the right things! Your head is in the right place!

 

[Aboveallislove]

Dank,
The NDA is only for ddf508. Unfortunately, the FDA will not approve for all mutations no matter how many signatures. The FDA has an advisory committee which reviews applications and it takes public comments on views and makes a recommendation to the FDA whether to approve or not. The goal is to have the FDA advisory committee see that the view of many many with CF and in the CF community is that it should approve the combo's NDA (New Drug Application). The FDA could reject the combo's NDA. I don't think that is likely but the more in the CF community who comment and express the view that it should approve the less likely rejection will be.

 

[Imogene]

Yes, it is important to tell our stories! They matter and numbers matter too. I think it is great that Above gave the sample letter. Personal stories along with the letter would be a good way to proceed. I always opt for email rather than snail mail, but both are warranted here.

I have reached out to the health programs coordinator of the FDA... to get her suggested directions.
For those of you who like good movies..."Dallas Buyers Guide" is a must to see how the HIV community circled around the FDA in the 1980's...not for those who object to X rated scenes.

The last time I heard the director speak, she made mention of the FDA response to allow more comments from patients and she even heads the Patient Representative program of the FDA, so we have the HIV AIDS community to thank for that!

 

[Aboveallislove]

I do agree the personal stories are great and I hope others do. What might help is if you post the info on how they can submit it to FDA. (I also called yesterday to get details). And I will be doing a personal letter.

BUT I also think that a simple "group" letter with thousands of signatures would help too and that would allow those who don't have time to do an individual one to sign their name. For that we need: 1) that letter (I drafted what I thought was a simple highlight that was non-controversial but it obviously doesn't need to be that); 2) a place for folks to "sign"; and 3) it to be submitted (and info on how to do that, which I had also intended to ask about when I hear back).

 

[ethan508]

Are FDA hearing motivated by public comment? I was hoping this would a process that would rest on the merits of the science and not on popular opinion. I guess I just don't trust the larger public as much as I do the experts and the results of well done (double blind placebo controlled) studies.

 

[Aboveallislove]

Ethan,
Totally agree re the science. My thought for the comment is to discuss how the science/results make a real difference in the life of those with CF. Take a look at what I threw together (which I'm sure someone can improve upon). My hope is to provide a logical analysis using the scientific DATA so the Committee, as well as to make it personal. What fewer exacerbations (scientifically proven) MEAN to us as people. Etc. The Committee is suppose to consider public comment. That's why they publish it in the Fed Reg. It won't overcome science and maybe they don't give a hoot, but it can't hurt for those with CF to say: Look, "quality of life" improving means DS doesn't wake 3 times a night with a tummy ache; spend the entire day rocking with stomach aches, etc. etc.

 

[static]

Eh I'd have to go with Ethan here on this one.

Its one of those things where you're telling the community to buy it before you try it. Only a small amount of people actually know what it's like to be in the trial, and an even smaller amount to be on active drug. How disappointing would it be to advocate for something that doesn't do as it was promised for you/your child?

Generally I believe people want to do the right thing, and as cynical as this sounds a lot was invested into this financially and people want to see a return on their investments.

 

[Aboveallislove]

I hold no misperceptions that this is our kalydeco, but it does provide positive benefits as shown in a double blind placebo study. While fev improvement is modest that is oppose to a decline that is the status quo now And the reduction in exacerbations is very significant. If it delivers the study results, that will be good enough for now. And for some it actually delivers much better results and I want the to have that option...to get the medicine...to have it available so they can try it....I have no issue advocating for apporval based on the study results and that is what im suggesting...not merely saying we want a drug. but the study results mean something to us.

 

[Aboveallislove]

Hey static in thinking more about your post, I'm wondering do you think it shouldn't be approved? Just wondering and if so why?

 

[regina65]

I'll sign it I'm DDf508. I am at a 35 on PFT's. All the antibiotics that the bugs are sensitive to I am allergic to. Any one else have this problem?

 

[Aboveallislove]

Oh Regina, I am so sorry. That is exactly what the fda has to hear. That an exacerbation might befor someone who has no safe antibiotics and thus he scientific evidence of reduced exacerbations is meaningful in the real world We're still working on logistics.

 

[Imogene]

Hi All,
We did hear back from the committee and they are ready to receive "thousands" of email requests...as I originally asked them! They responded within an hour.
This is the American way of government...lobbying for ourselves.
They are all doctors so they know the science. In looking at some of their CVs I noticed many are involved with asthma and the one in charge is an oceanographer.

They need our stories to complete the picture. They are willing to receive them.. This is their job.

We are writing the sample letter and will have it out shortly!
Salt and Light,

Jeanne

 

[Gammaw]

Great job! Is there a specified email address for them?

 

[ethan508]

This is the American way of government...lobbying for ourselves.

You are probably right. Sadly science loses without the backing of popular opinion.

Does anyone have a direct link to the address to which we should send our comments?

 

[JENNYC]

I haven't logged in a while and just saw this. Abby is not ddf508, so does my voice or experience with Abby help? I couldn't post pics on here of her last bronch that we just did but the results were amazing! Blew us away amazing! If I can help I want to! Let me know.

 

[Imogene]

I think it is wonderful to give testimony in any case. I think we have to remember that the approval of this NDA (New Drug Application) makes a difference to insurance companies. Some are fearful that the off label prescription will not be paid by insurance. This drug is expensive! The following is a sample letter Above wrote: A New Drug Application for the lumacaftor/ivacaftor combination is under consideration by the FDA. The Pulmonary-Allergy Drug Advisory Committee will be holding a public meeting on May 12, 2015 to discuss the application and to provide advice to the FDA. 1. You have the opportunity to submit written information and views directly to the Committee. Members of cysticfibrosis.com have created a sample letter you may want to copy and send directly to the committee at: PADAC@fda.hhs.org Of course, you may write your own email, and in both cases you may take the opportunity to add your personal stories. Pulmonary-Allergy Drug Advisory Committee Docket No. FDA-2015-N-0001 NDA 206038 Dear Committee Members, I have Cystic Fibrosis [or insert appropriate relationship]. I write in support of the NDA (New Drug Application) for lumacaftor/ivacaftor and strongly urge the Committee to recommend this drug to the FDA for approval. I recognize that the mean FEV improvement shown in clinical trials is modest when compared to ivacaftor, but even a modest improvement in FEV, when living with a degenerative disease, is a huge benefit. Further, this drug offers the chance for patients to remain stable until better drugs are available. Moreover, in reviewing the NDA, I urge the Committee to consider the very real benefit this drug offers to those with Cystic Fibrosis in the form of reduced exacerbations, improved weight, and overall improved quality of life. Signature Salt and Light, Jeanne Barnett CFTechnology.org 501 (c) (3)