DF508ers who participated in VX-770 trials

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saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>cfsucks</b></i> <div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> I have insurance that doesn't require approval for any drug. Any drug that a doc prescribes, I can get. Yes, I am extremely blessed - this is one of the reasons I chose the job that I have. I thank God for this blessing, absolutely. So I'm going to try Kalydeco for myself, even though I don't have a gating mutation. Even a tiny improvement, however small, is worth it to me. You have to remember that when doing a clinical trial, the goal for a drug company is to maximize the chances of FDA approval. I don't work for Vertex nor do I speak for them. But it's important to understand that just because Vertex didn't pursue FDA approval for DF508 doesn't mean it didn't work. D551G showed incredible results, therefore this presented the best chance of getting FDA approval. Once a drug is on the market, docs can Rx for any reason they please (doesn't mean insurance will pay for it, of course, but the point is that the drug is available). So it's obvious that Kalydeco doesn't have the same impact on DF508 that it has on G551D or any gating mutation for that matter. BUT that doesn't mean it won't have any impact. Some of the phase II trials show otherwise. To me this doesn't represent false hope - it represents trying something out. If I see some improvement, great. If not, at least I tried. I'll never wonder "hmmmmm..... what impact would this drug have had on me?" I try stuff. Like I tried NAC to see if it would work. Like I tried allergy shots. Like I tried cromolyn sodium. Like I tried denufosol. Like I tried Magnesium supplements. Some of it fails, some if it works. But much of what I try has little downside, so for me, it's worth a shot. As Dr. Warwick told me when I visited him in 2006 - every CFer is a scientist, doing experiments to understand how this disease works. And yes, I don't blow smoke. Well, I blow pulmozyme smoke. But that's about it.</end quote> this is very interesting- i'm in canada which is unfortunate so we won't see kalydeco for a whilebut i have good insurance coverage so i will see if i can possibly try to get it as well. keep us updated on your own trial- are you going to measure your improvement with clinical results, or just how you feel? will you be attempting to get sweat chloride tests and so forth done?</end quote>
i'll get as objective as I can. for those that know me, that's kind of my style <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
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saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>cfsucks</b></i> <div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> I have insurance that doesn't require approval for any drug. Any drug that a doc prescribes, I can get. Yes, I am extremely blessed - this is one of the reasons I chose the job that I have. I thank God for this blessing, absolutely. So I'm going to try Kalydeco for myself, even though I don't have a gating mutation. Even a tiny improvement, however small, is worth it to me. You have to remember that when doing a clinical trial, the goal for a drug company is to maximize the chances of FDA approval. I don't work for Vertex nor do I speak for them. But it's important to understand that just because Vertex didn't pursue FDA approval for DF508 doesn't mean it didn't work. D551G showed incredible results, therefore this presented the best chance of getting FDA approval. Once a drug is on the market, docs can Rx for any reason they please (doesn't mean insurance will pay for it, of course, but the point is that the drug is available). So it's obvious that Kalydeco doesn't have the same impact on DF508 that it has on G551D or any gating mutation for that matter. BUT that doesn't mean it won't have any impact. Some of the phase II trials show otherwise. To me this doesn't represent false hope - it represents trying something out. If I see some improvement, great. If not, at least I tried. I'll never wonder "hmmmmm..... what impact would this drug have had on me?" I try stuff. Like I tried NAC to see if it would work. Like I tried allergy shots. Like I tried cromolyn sodium. Like I tried denufosol. Like I tried Magnesium supplements. Some of it fails, some if it works. But much of what I try has little downside, so for me, it's worth a shot. As Dr. Warwick told me when I visited him in 2006 - every CFer is a scientist, doing experiments to understand how this disease works. And yes, I don't blow smoke. Well, I blow pulmozyme smoke. But that's about it.</end quote> this is very interesting- i'm in canada which is unfortunate so we won't see kalydeco for a whilebut i have good insurance coverage so i will see if i can possibly try to get it as well. keep us updated on your own trial- are you going to measure your improvement with clinical results, or just how you feel? will you be attempting to get sweat chloride tests and so forth done?</end quote>
i'll get as objective as I can. for those that know me, that's kind of my style <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
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Kristen

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> <div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Kristen</b></i> but he was concerned about it not being approved for my mutation (yet) </end quote> Concerned for safety? Or efficacy? If it's efficacy, how is that a concern? if you use it, and it doesn't work, nothing lost.... <div class="FTQUOTE"><begin quote>and it being so expensive.</end quote> He's not paying for it. Why does he care? I hate when docs comment on cost - if you're not paying for it, don't mention cost to me! <div class="FTQUOTE"><begin quote>He said he will at least call Vertex to see if he can get info on the in vitro results for my mutation.)</end quote> Let me know if you get a hold of that. I would love to learn</end quote>
I'm sure he was concerned about efficacy. I will definitely share the in vitro info with you if he sends it to me!
 
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Kristen

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> <div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Kristen</b></i> but he was concerned about it not being approved for my mutation (yet) </end quote> Concerned for safety? Or efficacy? If it's efficacy, how is that a concern? if you use it, and it doesn't work, nothing lost.... <div class="FTQUOTE"><begin quote>and it being so expensive.</end quote> He's not paying for it. Why does he care? I hate when docs comment on cost - if you're not paying for it, don't mention cost to me! <div class="FTQUOTE"><begin quote>He said he will at least call Vertex to see if he can get info on the in vitro results for my mutation.)</end quote> Let me know if you get a hold of that. I would love to learn</end quote>
I'm sure he was concerned about efficacy. I will definitely share the in vitro info with you if he sends it to me!
 
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musclemania70

New member
So why wouldn't ANY cf patient go forward and ask their doc for an RX of the Kalydeco just to try it? If one person's doctor will give the script, then there should be other doctors that do the same.

Who wouldn't want to try it? Except for those whose insurance wouldn't approve it, we should all be on the bandwagon of trying this drug no matter what mutation we have. If only one person had minimal results, wouldn't that still be an improvement? Would the Foundation limit the drug to the 5% simply because 'maximum results' are better than 'minimal results'?

I'd take minimal results any day over NOT TRYING IT AT ALL just like Amy said. WHO WOULD NOT BE WILLIING TO TRY IT?
 
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musclemania70

New member
So why wouldn't ANY cf patient go forward and ask their doc for an RX of the Kalydeco just to try it? If one person's doctor will give the script, then there should be other doctors that do the same.

Who wouldn't want to try it? Except for those whose insurance wouldn't approve it, we should all be on the bandwagon of trying this drug no matter what mutation we have. If only one person had minimal results, wouldn't that still be an improvement? Would the Foundation limit the drug to the 5% simply because 'maximum results' are better than 'minimal results'?

I'd take minimal results any day over NOT TRYING IT AT ALL just like Amy said. WHO WOULD NOT BE WILLIING TO TRY IT?
 
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Anomie

New member
Wasn't clinically beneficial which means its yours if you can cover the full 300k price tag and get a script from your doc. I think if the foundation had to buy this drug for every delta out there they would go broke and not be able to fund anymore research. Besides the results DF508 had with 770 are similar to those who took the placebo so I really think the drug fails to correct the underlying issue with this mutation. I mean do you think someone going from a sweat test of 120 to 115 is going to make much of a difference in their health or quality of life? I think your best bet is to try to get on the 770/809 trials which are currently recruiting if you are 18 years old and live near a participating center.
 
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Anomie

New member
Wasn't clinically beneficial which means its yours if you can cover the full 300k price tag and get a script from your doc. I think if the foundation had to buy this drug for every delta out there they would go broke and not be able to fund anymore research. Besides the results DF508 had with 770 are similar to those who took the placebo so I really think the drug fails to correct the underlying issue with this mutation. I mean do you think someone going from a sweat test of 120 to 115 is going to make much of a difference in their health or quality of life? I think your best bet is to try to get on the 770/809 trials which are currently recruiting if you are 18 years old and live near a participating center.
 
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hmw

New member
Amy, I'm curious to know as well if you will be getting a sweat test first as well to assess yourself on some kind of clinical level, get baseline pft's, weight etc at clinic.
 
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hmw

New member
Amy, I'm curious to know as well if you will be getting a sweat test first as well to assess yourself on some kind of clinical level, get baseline pft's, weight etc at clinic.
 
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saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>musclemania70</b></i> So why wouldn't ANY cf patient go forward and ask their doc for an RX of the Kalydeco just to try it? If one person's doctor will give the script, then there should be other doctors that do the same. Who wouldn't want to try it? Except for those whose insurance wouldn't approve it, we should all be on the bandwagon of trying this drug no matter what mutation we have. If only one person had minimal results, wouldn't that still be an improvement? Would the Foundation limit the drug to the 5% simply because 'maximum results' are better than 'minimal results'? I'd take minimal results any day over NOT TRYING IT AT ALL just like Amy said. WHO WOULD NOT BE WILLIING TO TRY IT?</end quote>
It all comes down to insurance coverage. Most insurances won't cover the drug off label.
 
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saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>musclemania70</b></i> So why wouldn't ANY cf patient go forward and ask their doc for an RX of the Kalydeco just to try it? If one person's doctor will give the script, then there should be other doctors that do the same. Who wouldn't want to try it? Except for those whose insurance wouldn't approve it, we should all be on the bandwagon of trying this drug no matter what mutation we have. If only one person had minimal results, wouldn't that still be an improvement? Would the Foundation limit the drug to the 5% simply because 'maximum results' are better than 'minimal results'? I'd take minimal results any day over NOT TRYING IT AT ALL just like Amy said. WHO WOULD NOT BE WILLIING TO TRY IT?</end quote>
It all comes down to insurance coverage. Most insurances won't cover the drug off label.
 
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Anomie

New member
You won't be able to get the drug until its approved in Canada and I have no idea when that will be. Hopefully really soon!!
 
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Anomie

New member
You won't be able to get the drug until its approved in Canada and I have no idea when that will be. Hopefully really soon!!
 
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jmiller

New member
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?
 
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jmiller

New member
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?
 
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Havoc

New member
Frankly, if you are holding your own as far as PFT's and your particular pathologies, I would wait and let vertex do their thing. If you find yourself in rapid decline and are grasping for straws, then that is a different situation and I would probably try for it.
 
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Havoc

New member
Frankly, if you are holding your own as far as PFT's and your particular pathologies, I would wait and let vertex do their thing. If you find yourself in rapid decline and are grasping for straws, then that is a different situation and I would probably try for it.
 
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