Aboveallislove
Super Moderator
Thanks Jeanne!
A quick
clarification: If the NDA doesn’t get approved the combo drug cannot be sold. It is not an issue of insurance coverage now. This drug isn’t approved for sale AT ALL, unlike Kalydeco. That’s why I think it is so important the FDA hears from us and approves it. (Even if it has minor or no benefit to my son; it will have benefits to others and without approval it can’t used by anyone).
I also wanted to add a couple thoughts to, with a quick backdrop: I'm a lawyer and have professionally worked on many cases involving regulations, agencies, reviews of regulations, the notice process etc. While in the ideal world those charged with making regulations and issuing decision would have perfect knowledge, I/they never do. What was always helpful to me in any role was for an advocate to lay out logically the facts, the law, and then apply it, showing the consequences of the decision. While I have no specific experience with the FDA review process, I have read minutes of past proceedings and gleaned what it seemed concerned with, which has been "modest" FEV improvement. (You can google the Pulmozyme hearing and read that; they did recommend it for approval but there was great concern at the meeting about its "modest" FEV improvement.)
So given my experience, I would think it would be extremely helpful for folks affected by CF to point out with personal stories why this drug should be approved given the scientific results from the study, highlighting facts the Committee might not think of on its own. (This link provides a summary of the study results: [url]http://investors.vrtx.com/releasedetail.cfm?ReleaseID=856185).[/URL]
Regina’s recent post above is a perfect example. The Advisory Committee might be thinking 2-4% that’s not much that’s too modest. But for Regina who struggles with her FEV AND is allergic to antibiotics, this drug means the world. Here are some more examples/types of information that if I were on the Committee it would be helpful for me to think of in reviewing the information:
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. The more personal the illustration, tied to the scientific data, I think the better.
Finally, for those who are not inclined to write on their own, Jeanne is also working on a "group letter" which folks can easily sign.
A quick
clarification: If the NDA doesn’t get approved the combo drug cannot be sold. It is not an issue of insurance coverage now. This drug isn’t approved for sale AT ALL, unlike Kalydeco. That’s why I think it is so important the FDA hears from us and approves it. (Even if it has minor or no benefit to my son; it will have benefits to others and without approval it can’t used by anyone).
I also wanted to add a couple thoughts to, with a quick backdrop: I'm a lawyer and have professionally worked on many cases involving regulations, agencies, reviews of regulations, the notice process etc. While in the ideal world those charged with making regulations and issuing decision would have perfect knowledge, I/they never do. What was always helpful to me in any role was for an advocate to lay out logically the facts, the law, and then apply it, showing the consequences of the decision. While I have no specific experience with the FDA review process, I have read minutes of past proceedings and gleaned what it seemed concerned with, which has been "modest" FEV improvement. (You can google the Pulmozyme hearing and read that; they did recommend it for approval but there was great concern at the meeting about its "modest" FEV improvement.)
So given my experience, I would think it would be extremely helpful for folks affected by CF to point out with personal stories why this drug should be approved given the scientific results from the study, highlighting facts the Committee might not think of on its own. (This link provides a summary of the study results: [url]http://investors.vrtx.com/releasedetail.cfm?ReleaseID=856185).[/URL]
Regina’s recent post above is a perfect example. The Advisory Committee might be thinking 2-4% that’s not much that’s too modest. But for Regina who struggles with her FEV AND is allergic to antibiotics, this drug means the world. Here are some more examples/types of information that if I were on the Committee it would be helpful for me to think of in reviewing the information:
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. The more personal the illustration, tied to the scientific data, I think the better.
Finally, for those who are not inclined to write on their own, Jeanne is also working on a "group letter" which folks can easily sign.