Good thoughts Jonathan- I completely respect the process and understand the necessities of trials, etc. However, if a drug is available that might provide some improvement why wait? I am blessed to have good pft's that I have worked hard for... but a big part of that has been doing anything/everything I can to preserve lung function. However, I also struggle w/ hemoptysis. I recently had an embolization which I hope is like hitting the "reset" button on my crazy bronchial arteries. If I can take something to treat the underlying cause - the reason I develop these erratic, tortuous arteries - maybe I can prolong the time until my next bout of bleeds... I don't want to seem ungrateful for the state of my health - but I want to continue to do all I can.
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DF508ers who participated in VX-770 trials
- Thread starter jmiller
- Start date
Good thoughts Jonathan- I completely respect the process and understand the necessities of trials, etc. However, if a drug is available that might provide some improvement why wait? I am blessed to have good pft's that I have worked hard for... but a big part of that has been doing anything/everything I can to preserve lung function. However, I also struggle w/ hemoptysis. I recently had an embolization which I hope is like hitting the "reset" button on my crazy bronchial arteries. If I can take something to treat the underlying cause - the reason I develop these erratic, tortuous arteries - maybe I can prolong the time until my next bout of bleeds... I don't want to seem ungrateful for the state of my health - but I want to continue to do all I can.
musclemania70
New member
I agree with jmiller. Any improvement and preservation of lung health is worth it to me. Its not a matter of life or death- it can simply be that each person who improves even SLIGHTLY would like the opportunity!
MINIMAL improvement is STILL improvement. It could mean less bleeds for her. She should be allowed to take the chance.
MINIMAL improvement is STILL improvement. It could mean less bleeds for her. She should be allowed to take the chance.
musclemania70
New member
I agree with jmiller. Any improvement and preservation of lung health is worth it to me. Its not a matter of life or death- it can simply be that each person who improves even SLIGHTLY would like the opportunity!
MINIMAL improvement is STILL improvement. It could mean less bleeds for her. She should be allowed to take the chance.
MINIMAL improvement is STILL improvement. It could mean less bleeds for her. She should be allowed to take the chance.
Yup, I can definitely understand your situation. I suppose my disinterest is 3 fold; (i) I am very lucky that I do not have any major health issues with CF (ii) we aren't sure the efficacy that 770 will have (if any) on varying mutations (iii) there could be people who would benefit so much more by having the correct mutations, especially if there is a supply issue.
Of course, this is my reasoning and I am not trying to impose it on you. Speak with your research team and see if they think it will be worth a try and if so then go for it. I wish you luck!
Of course, this is my reasoning and I am not trying to impose it on you. Speak with your research team and see if they think it will be worth a try and if so then go for it. I wish you luck!
Yup, I can definitely understand your situation. I suppose my disinterest is 3 fold; (i) I am very lucky that I do not have any major health issues with CF (ii) we aren't sure the efficacy that 770 will have (if any) on varying mutations (iii) there could be people who would benefit so much more by having the correct mutations, especially if there is a supply issue.
Of course, this is my reasoning and I am not trying to impose it on you. Speak with your research team and see if they think it will be worth a try and if so then go for it. I wish you luck!
Of course, this is my reasoning and I am not trying to impose it on you. Speak with your research team and see if they think it will be worth a try and if so then go for it. I wish you luck!
But wouldn't vertex adjust the supply if demand was higher than anticipated?? I certainly won't elbow my way in for the chance at a pill that might provide a little improvement when there is someone with G551D in dire need of it. But if Vertex would adjust their manufacturing of Kalydeco to meet the increased demand, all the better right? Maybe it's not this simple.
But wouldn't vertex adjust the supply if demand was higher than anticipated?? I certainly won't elbow my way in for the chance at a pill that might provide a little improvement when there is someone with G551D in dire need of it. But if Vertex would adjust their manufacturing of Kalydeco to meet the increased demand, all the better right? Maybe it's not this simple.
musclemania70
New member
Maybe Vertex could initiate something that was need-based. That would make more sense- For those of other mutations that are struggling to survive they could be given the Kalydeco to see if their condition improves. I think that would be fair and give those who need it most, the chance for an improvement if it they were on the cusp of transplant.
If anyone who is interested in trying Kalydeco, talk to your doctor. Contact Vertex too and tell them other mutations are requesting the opportunity to try the new drug.
I will post as soon as I have some feedback as well.
If anyone who is interested in trying Kalydeco, talk to your doctor. Contact Vertex too and tell them other mutations are requesting the opportunity to try the new drug.
I will post as soon as I have some feedback as well.
musclemania70
New member
Maybe Vertex could initiate something that was need-based. That would make more sense- For those of other mutations that are struggling to survive they could be given the Kalydeco to see if their condition improves. I think that would be fair and give those who need it most, the chance for an improvement if it they were on the cusp of transplant.
If anyone who is interested in trying Kalydeco, talk to your doctor. Contact Vertex too and tell them other mutations are requesting the opportunity to try the new drug.
I will post as soon as I have some feedback as well.
If anyone who is interested in trying Kalydeco, talk to your doctor. Contact Vertex too and tell them other mutations are requesting the opportunity to try the new drug.
I will post as soon as I have some feedback as well.
musclemania70
New member
some people who are trying to quit smoking try prescription anti-depressants even though it wasn't made for that purpose. Doctors are willing to give the patient theopportunity because they want to give their patient the best possiblechance to quit.
Even thoughKaleydeco wasn't initially intended for other mutations, it doesn't mean it won't work. (as stated earlier) It just means the improvement wasn't 'significant enough' on other mutations to use for the application to the FDA.
Even thoughKaleydeco wasn't initially intended for other mutations, it doesn't mean it won't work. (as stated earlier) It just means the improvement wasn't 'significant enough' on other mutations to use for the application to the FDA.
musclemania70
New member
some people who are trying to quit smoking try prescription anti-depressants even though it wasn't made for that purpose. Doctors are willing to give the patient theopportunity because they want to give their patient the best possiblechance to quit.
Even thoughKaleydeco wasn't initially intended for other mutations, it doesn't mean it won't work. (as stated earlier) It just means the improvement wasn't 'significant enough' on other mutations to use for the application to the FDA.
Even thoughKaleydeco wasn't initially intended for other mutations, it doesn't mean it won't work. (as stated earlier) It just means the improvement wasn't 'significant enough' on other mutations to use for the application to the FDA.
saveferris2009
New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>jmiller</b></i> I, like Amy, am interested in trying Kalydeco even though I am DF508/2184delA. I called my doctor last week to see about that process. She said that it would likely require some kind of petition to Vertex. Because it is an orphan drug (and approved for use with G551D), Vertex is not making enough of the drug to serve the entire CF population. She said it might be possible to petition them and see about getting the drug provided that I did some sort of sweat test analysis pre and post to determine if it was in fact providing improvement. Originally I thought this would me more of an insurance issue - that I would need some sort of pre-auth from my doctor... I wasn't even thinking in terms of supply/demand. What do you all think of this?</end quote>
That just sounds bizarre.
I realize they have certain manufacturing capacity, certainly. But an extra CFer here or there isn't going to impact them. We're not going from 1400 CFers to 30,000. We're thinking maybe 1400 to 1450. Like you said, most won't be able to get their insurance to cover it if they don't have G551D.
Vertex is a publically traded company and they are in the business to make money. So if your insurance is willing to pay for it, I have a feeling they'll be happy to take the money.
That just sounds bizarre.
I realize they have certain manufacturing capacity, certainly. But an extra CFer here or there isn't going to impact them. We're not going from 1400 CFers to 30,000. We're thinking maybe 1400 to 1450. Like you said, most won't be able to get their insurance to cover it if they don't have G551D.
Vertex is a publically traded company and they are in the business to make money. So if your insurance is willing to pay for it, I have a feeling they'll be happy to take the money.
saveferris2009
New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>jmiller</b></i> I, like Amy, am interested in trying Kalydeco even though I am DF508/2184delA. I called my doctor last week to see about that process. She said that it would likely require some kind of petition to Vertex. Because it is an orphan drug (and approved for use with G551D), Vertex is not making enough of the drug to serve the entire CF population. She said it might be possible to petition them and see about getting the drug provided that I did some sort of sweat test analysis pre and post to determine if it was in fact providing improvement. Originally I thought this would me more of an insurance issue - that I would need some sort of pre-auth from my doctor... I wasn't even thinking in terms of supply/demand. What do you all think of this?</end quote>
That just sounds bizarre.
I realize they have certain manufacturing capacity, certainly. But an extra CFer here or there isn't going to impact them. We're not going from 1400 CFers to 30,000. We're thinking maybe 1400 to 1450. Like you said, most won't be able to get their insurance to cover it if they don't have G551D.
Vertex is a publically traded company and they are in the business to make money. So if your insurance is willing to pay for it, I have a feeling they'll be happy to take the money.
That just sounds bizarre.
I realize they have certain manufacturing capacity, certainly. But an extra CFer here or there isn't going to impact them. We're not going from 1400 CFers to 30,000. We're thinking maybe 1400 to 1450. Like you said, most won't be able to get their insurance to cover it if they don't have G551D.
Vertex is a publically traded company and they are in the business to make money. So if your insurance is willing to pay for it, I have a feeling they'll be happy to take the money.
Amy, you may have missed my question in amidst the many other posts here- are you going to have any baseline testing at clinic (sweat test, pft's etc) before starting the new med and again after a month or two? Esp given so few people with other mutations will be able to get approval for it, it will be great to have that info documented for the cf registry as well as for your own purposes.
Amy, you may have missed my question in amidst the many other posts here- are you going to have any baseline testing at clinic (sweat test, pft's etc) before starting the new med and again after a month or two? Esp given so few people with other mutations will be able to get approval for it, it will be great to have that info documented for the cf registry as well as for your own purposes.
saveferris2009
New member
yes <img src="i/expressions/face-icon-small-smile.gif" border="0">
saveferris2009
New member
yes <img src="i/expressions/face-icon-small-smile.gif" border="0">
C
cfsucks
Guest
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> yes <img src=""></end quote>
i'm also curious as to your mutations- are either of them in the gating mutation class?
i'm also curious as to your mutations- are either of them in the gating mutation class?
C
cfsucks
Guest
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> yes <img src=""></end quote>
i'm also curious as to your mutations- are either of them in the gating mutation class?
i'm also curious as to your mutations- are either of them in the gating mutation class?