Email address for the fda committee

I

Imogene

Administrator
PADAC@fda.hhs.gov

Dear Ms. Barnett,

Yes, this is the email address to send all written submissions to for the May 12th PADAC meeting.
Thank you,



From: Jeanne Barnett [mailto:jeanne@medrise.com]
Sent: Thursday, April 02, 2015 12:17 PM
To: PADAC
Subject: on line patient community at cysticfibrosis.com

Dear Dr. Hong,

I run an on line patient community at cysticfibrosis.com. Several thousand of our community are very eager to reach out to you concerning the upcoming deliberation for the NDA lumacaftor/ivacaftor.

We wonder if this is the best email to tell our stories and encourage you to vote in favor of this medication.

Please let me know the best way for the community to approach your committee and if this is a good email to which we may petition.

Thank you for any help or advice you can give us.

Sincerely,
Jeanne Barnett
 
A

Aboveallislove

Super Moderator
In case you're like me and don't often read the emails, I thought I'd post the text of the note Jeanne sent to our community. (See below). Also, I have an acquaintance in the bio-tech industry and asked him about the comment stage. He noted the FDA is trying to be more atune to patients and that they do read/listen to comments, but that of course it won't overcome the science. He also said that the more analytical comments the better and that if there is a huge response it might have an impact, but not be dispositive, but that it depends on the committee.

Greetings!

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.

You have the opportunity to submit written information and views directly to the Committee.
Email [email]PADAC@fda.hhs.gov[/EMAIL] Members of cysticfibrosis.com have created a sample letter you may want to copy and send directly to the committee at:

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


Some examples for your personal to add in your personal stories:
FEV Improvement: The drugs improve FEV between 2-4% over placebo. Has your FEV been continually declining? What would stabilizing FEV and having a slight increase in FEV mean to you?
Exacerbations: The drugs reduce exacerbations significantly. What does an exacerbation mean to you? Lost work? Lost school? Expenses? Did you come out of the hospital with another infection? MRSA? Are you resistant to antibiotics now because you've used them so much? Did you have negative reactions to antibiotics? Do you have invasive testing because of infections?
BMI: The drugs improve BMI. Do you struggle trying to gain weight? Do you have a feeding tube? Is eating enough always a battle?
Quality of Life: The drugs improve Quality of Life. What do you do to keep your lungs healthy? How much time a day? This is a pill. It could add to your health with a minor treatment burden. Do you have lots of GI issues that might be resolved?
These are just some thoughts and suggestions from our community.
The more personal the illustration, tied to the scientific data, the better.

Salt and Light,
Jeanne Barnett
CFTechnology.org 501 (c) (3)

​
 
I

Imogene

Administrator
Wondrful! Grammakaky,
Let's keep it moving!...if you have a story to tell you still have time to send it! We are fortunate in the USA to have this committee who will listen...Of course the science trumps all...but the anecdotal evidence matters...and pharma and the FDA are listening!
Go for it...
Salt and Light,
Jeanne
 
A

Aboveallislove

Super Moderator
Also, if someone has a personal little blurb they want me to include in my letter (which is VERY LONG, analytical, and focusing on the science, etc.), such as: "John, ddf508, 31 explains: Every exacerbation I lose 3 weeks of work." etc., PM me and give me what you want included (I have a couple), and your "permission" and what you want for your name (first name only, both names, etc.)
 
R

rmotion

New member
Curious to see how common this letter writing is to help get a drug approved, I dont remember this going on with Kalydeco. My concern is that VX-809 is only marginally helping and the cost is outrageous. If the drug worked excellently then that is enough to get the drug approved and not some passionate plea from a 14 yo with Cf. Trust me I want this drug to be approved [I have been in the clinical study for the last 1.5 years] and would be bummed if it does not get through. My plead to the CFF and the drug companies is to find something that really works. [Stepping off soap box]
 
A

Aboveallislove

Super Moderator
I agree that it is "marginal," but it does increase FEV from a range of 2.6-4.0 and 46% of Traffic participants and 39% of Transport participants had 5% or more increase. And that is absolute FEV, not relative. Also, it reduced exacerbations by 28 to 43 percent. Kalydeco is clearly MUCH better. (But this compares well to Pulmozyme which has a much higher treatment burden and I would argue is a great drug even with its own "modest" results.)

We definitely need something that works better. But that will take years (and there is lots going forward...this approval won't change that...if anything it will spur companies to invest more to get in on this $$$$ market). And this will help keep CFers healthier in the meantime (on average CFers lose 2% per year). It could mean no feeding tube (weight gain is another huge plus), it could mean fewer hospitalizations and hospital acquired infections, it could mean no lung transplant or less permanent damage until we get these drugs. So to me, we should push to get the drug approved. If it doesn't work for a specific individual, they and their doctor can decide not to use; but if it isn't approved, no one can use it.

Re the "letters." It is actually a "comment" in the form of a letter. The way the government works is they publish things asking for "comment" by those affected by governmental decisions. That is what happened and where I pulled the info (the federal register, where the government solicited comments) and Jeanne so gratiously got more detail. I have a classmate who runs a biotech and asked him "do they read?" "do they care" "does it even matter". As he explained, the FDA is attempting to become more open to patient's input (it is something they are working on but not yet there), that every committee differs, but yes, they do read. And if they get enough support it might sway, but it won't overcome science. He said the "I'm dying I need this drug" doesn't help the committee, but the more analytical the better. As I noted in another thread, having worked on various agency (other agencies, not FDA), things I've learned that the decisionmakers don't always "catch" everything, they don't always understand the ramifications of their decisions, and it helps for those affected to lay it out for the decisionmaker. That's what I'm suggesting: Lay it out. Why are these results good enough for approval, using the personal experience to show why this science matters to us.

To reiterate: I agree this isn't "enough"; we need something that really works. But it isn't a choice between this and that. It is a choice between this now or nothing, until the later drug that works better comes.
 
A

Aboveallislove

Super Moderator
rmotion,
Re: "I dont remember this going on with Kalydeco"

I forgot to add that Kalydeco never went to an Advisory Committee for its initial approval so there was no request for public comment. But it did go to a committee for R117H for approval for those 6 and older. You might recall that it didn't show improvement for those under 18, and the Advisory Committee was split on whether to recommend it for those 6 and older....but it did by a majority vote. I'm sure there were lots of comments for that expansion and I'd think they helped--to understand age, severity, progressive nature etc. That Committee hearing transcript actually is part of what prompted me to think of the comments: One of the Advisory Committee members actually expressed concern that the statistics of it benefiting those over 18 might not be valid because it was pulled after the study from a sub-set and not part of the initial study design. Others on the panel explained, no that doesn't matter. The stats still are good. But that illustrates that they don't all have perfect knowledge, ...so let's share ours.
 
R

rmotion

New member
Ok good, well put.
It is hard for me to be wildly enthusiastic, I guess it is just dampend by my own heightened expectations. But last week at clinic we talked about if the drug does not go through. [I have been on VX-809 roll over study for 1 year], and I was like oh crap we need this to get approved, and hopefully that leads to VX-661 going forward and then the next.
My results are about lock step with the usual results. But I had high hopes like all of us. We wanted a homerun and we got a bunt! It took me a year of pleading and finding where to get on this study, got on it and had a hard time initially adjusting to the regiment. But now I need it, we all do.
I will send my "good analytical data!
 
S

stephen

Guest
Just sent my two cents to the PADAC.

I tried to stress how Kalydeco has/can improve the quality of life and prevent future respiratory damage.
 
I

Imogene

Administrator
Great Stephen...I thought of you throughout this process! So glad you are feeling sooo much better..
Salt and Light,
Imogene
 
R

rmotion

New member
Any suggestions before I send, tried to keep it brief

Dear Committee Members,

I am a 45 year old cystic fibrosis patient , in relatively stable health, happily married and working on starting my own business. Even though these last few years has been challenging I am feeling optimistic for the next few years. It seems that finally new drugs are coming out that can help us live better with Cystic Fibrosis.

For over the last 30 years I have participated in many clinical trials and have been a first hand witness of the hope, optimism and inevitable disappointment of the incredibly slow progress of drug discovery. Every few years it seemed like a new drug was right around the corner and never panned out. My life has always lived precariously in the balance. Now I have chance to witness a win for myself and the cystic fibrosis community.

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 have been on the Vertex 770/809 study over the last year and do not want to think about the possibility of this drug not passing through the approval process.

Over the last year my PFT’s has stabilized, I was in pulmonary decline over the last few years. Every 3-4 months I would need to be hospitalized for acute exacerbations.

Since being on the drug my overall health has improved and I am able to stay out of the hospital I have had a 50% reduction of hospitalizations.

This drug gives me hope.


Sincerely,
Robert
 
I

Imogene

Administrator
Hope matters! hopefully the science and the anecdotal evidence will string together into a successful launch. It is important to imagine this is just a beginning...more and more drugs will be developed in the realm of personalized medications and our patient community is on the forefront!
Thanks rmotion! Nicely stated.
Imogene
 
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