FDA Comments due by April 27 for lumacaftor/ivacaftor combo

Aboveallislove

Super Moderator
Just a quick reminder that the deadline for submitting comments to the FDA for the lumacaftor/ivacaftor (i.e., Kalydeco combo for ddf508), is April 27, 2015. Below is Jeanne's email to our community re the FDA meeting and how to make a comment.

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 PADAC@fda.hhs.gov
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)
 

Imogene

Administrator
Above: Thanks for reminding us...April 27th is MONDAY already...it is exciting to know/believe that a committee wants to hear the anecdotal data that goes with the quantitative data...but then with 3 million messages since 2003 held on this database...just under a million messages searchable...I am a fan of the qualitative end.

I am working on the survey comments...so I understand how hard it is to put this type of data in perspective.
Go for it!
Salt and Light,
Jeanne
 

Aboveallislove

Super Moderator
Four-page treatise comment just submitted to FDA with appropriate bolding of key points, scientific data points, etc. for a quick read.
 
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