If you don't have a gating mutation and want Kalydeco....

[albino15]

You know, if people with different mutations want to take this drug, then go ahead and take it. In my opinion it's a LITTLE ridiculous to be taking a drug that wasn't proven to be significantly affective for your mutation.

And having a bunch of people just get this drug, try it and then post about how well it works or doesn't work on a forum isn't a scientific study, so the controlled environment of the clinical trial is probably best.

Also, if they thought this drug would work for people with Df508, than of course they would jump on it, it would mean tons of money.

If you want to blame anyone for the amount of time and money it takes to do these trials, blame the FDA, That's who these drug companies are trying to please. I mean think about it, if you spent all this time and resources jumping through the hoops of our bureaucracy you'd want to do it just right so you can get that big YES from the FDA.

 

[albino15]

You know, if people with different mutations want to take this drug, then go ahead and take it. In my opinion it's a LITTLE ridiculous to be taking a drug that wasn't proven to be significantly affective for your mutation.

And having a bunch of people just get this drug, try it and then post about how well it works or doesn't work on a forum isn't a scientific study, so the controlled environment of the clinical trial is probably best.

Also, if they thought this drug would work for people with Df508, than of course they would jump on it, it would mean tons of money.

If you want to blame anyone for the amount of time and money it takes to do these trials, blame the FDA, That's who these drug companies are trying to please. I mean think about it, if you spent all this time and resources jumping through the hoops of our bureaucracy you'd want to do it just right so you can get that big YES from the FDA.

 

[Incomudrox]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>albino15</b></i> You know, if people with different mutations want to take this drug, then go ahead and take it. In my opinion it's a LITTLE ridiculous to be taking a drug that wasn't proven to be significantly affective for your mutation. And having a bunch of people just get this drug, try it and then post about how well it works or doesn't work on a forum isn't a scientific study, so the controlled environment of the clinical trial is probably best. Also, if they thought this drug would work for people with Df508, than of course they would jump on it, it would mean tons of money. If you want to blame anyone for the amount of time and money it takes to do these trials, blame the FDA, That's who these drug companies are trying to please. I mean think about it, if you spent all this time and resources jumping through the hoops of our bureaucracy you'd want to do it just right so you can get that big YES from the FDA.</end quote>
The problem that you and a lot of other people seem to not realize is, while the % of Chloride change maybe less, VERTEX THEMSELVES SAY THAT THERE IS NO DIRECT CORRELATION BETWEEN CHLORIDE CHANGE AND FEV1. So why <strong>NOT</strong> try? <strong>They even tested it on normal people and they saw an increase in sweat chloride change. The FDA isn't there to tell people if it works for this mutation or not, they're there to make sure it's</strong> <strong>SAFE IN HUMANS.</strong>
However, your opinion is well your opinion and you are intilted to it.

 

[Incomudrox]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>albino15</b></i> You know, if people with different mutations want to take this drug, then go ahead and take it. In my opinion it's a LITTLE ridiculous to be taking a drug that wasn't proven to be significantly affective for your mutation. And having a bunch of people just get this drug, try it and then post about how well it works or doesn't work on a forum isn't a scientific study, so the controlled environment of the clinical trial is probably best. Also, if they thought this drug would work for people with Df508, than of course they would jump on it, it would mean tons of money. If you want to blame anyone for the amount of time and money it takes to do these trials, blame the FDA, That's who these drug companies are trying to please. I mean think about it, if you spent all this time and resources jumping through the hoops of our bureaucracy you'd want to do it just right so you can get that big YES from the FDA.</end quote>
The problem that you and a lot of other people seem to not realize is, while the % of Chloride change maybe less, VERTEX THEMSELVES SAY THAT THERE IS NO DIRECT CORRELATION BETWEEN CHLORIDE CHANGE AND FEV1. So why <strong>NOT</strong> try? <strong>They even tested it on normal people and they saw an increase in sweat chloride change. The FDA isn't there to tell people if it works for this mutation or not, they're there to make sure it's</strong> <strong>SAFE IN HUMANS.</strong>
However, your opinion is well your opinion and you are intilted to it.

 

[saveferris2009]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>albino15</b></i> You know, if people with different mutations want to take this drug, then go ahead and take it. In my opinion it's a LITTLE ridiculous to be taking a drug that wasn't proven to be significantly affective for your mutation.</end quote>
But what if your mutation wasn't tested? Is it ridiculous to try?
Or what if your mutation is DF508, and you know that there was a small subset of people with DF508 in the study that had significant FEV1 increase on the drug, but no one yet understands why. Is it ridiculous to try?
Exercise can impact chloride transport. Diet can impact chloride transport. Modifier genes can impact chloride transport.
I think it's a LITTLE ridiculous not to try a drug that probably won't hurt you, and it may help.

 

[saveferris2009]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>albino15</b></i> You know, if people with different mutations want to take this drug, then go ahead and take it. In my opinion it's a LITTLE ridiculous to be taking a drug that wasn't proven to be significantly affective for your mutation.</end quote>
But what if your mutation wasn't tested? Is it ridiculous to try?
Or what if your mutation is DF508, and you know that there was a small subset of people with DF508 in the study that had significant FEV1 increase on the drug, but no one yet understands why. Is it ridiculous to try?
Exercise can impact chloride transport. Diet can impact chloride transport. Modifier genes can impact chloride transport.
I think it's a LITTLE ridiculous not to try a drug that probably won't hurt you, and it may help.

 

[saveferris2009]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>albino15</b></i> Also, if they thought this drug would work for people with Df508, than of course they would jump on it, it would mean tons of money. </end quote>

Sorry one last comment. This isn't necessarily true either.
Plenty of drug companies will pick the lowest hanging fruit for their clinical trials, in this case G551D, to make the way for easy FDA approval. These companies know that docs can Rx for any reason they want, once a drug is on the market.
I'm not saying Vertex is taking this approach, but I think it's improtant for everyone to get a clear picture of how drug development and drug commercialization work in the US.
So just because a drug doesn't have FDA approval for a certain indication, doesn't mean the drug won't work for that purpose. We are seeing great evidence of this now - docs are Rx-ing Kaly off label and patients have it in their bodies, without having G551D. This happens every day across many disease states - again, not saying that is Vertex's intent but your statement isn't accurate.

 

[saveferris2009]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>albino15</b></i> Also, if they thought this drug would work for people with Df508, than of course they would jump on it, it would mean tons of money. </end quote>

Sorry one last comment. This isn't necessarily true either.
Plenty of drug companies will pick the lowest hanging fruit for their clinical trials, in this case G551D, to make the way for easy FDA approval. These companies know that docs can Rx for any reason they want, once a drug is on the market.
I'm not saying Vertex is taking this approach, but I think it's improtant for everyone to get a clear picture of how drug development and drug commercialization work in the US.
So just because a drug doesn't have FDA approval for a certain indication, doesn't mean the drug won't work for that purpose. We are seeing great evidence of this now - docs are Rx-ing Kaly off label and patients have it in their bodies, without having G551D. This happens every day across many disease states - again, not saying that is Vertex's intent but your statement isn't accurate.

 

[Anomie]

What if you're mutations are so rare that not even enough people in the world have them to conduct a proper clinical trial? What do we tell these people? Sorry but the CF foundation is strongly opposed to you possibly enjoying the benefit of this drug. What, do they speak for all people with CF now? Maybe they should just stick to the fundraising and let doctors do their job.

 

[Anomie]

What if you're mutations are so rare that not even enough people in the world have them to conduct a proper clinical trial? What do we tell these people? Sorry but the CF foundation is strongly opposed to you possibly enjoying the benefit of this drug. What, do they speak for all people with CF now? Maybe they should just stick to the fundraising and let doctors do their job.

 

[Incomudrox]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Anomie</b></i> What if you're mutations are so rare that not even enough people in the world have them to conduct a proper clinical trial? What do we tell these people? Sorry but the CF foundation is strongly opposed to you possibly enjoying the benefit of this drug. What, do they speak for all people with CF now? Maybe they should just stick to the fundraising and let doctors do their job.</end quote>

This is why mutations belong to classes. Most likely we will see approval for entire classes with potentiators as they come along once a majority of the mutations in the class have been test in-vivo or in-vitro.

 

[Incomudrox]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Anomie</b></i> What if you're mutations are so rare that not even enough people in the world have them to conduct a proper clinical trial? What do we tell these people? Sorry but the CF foundation is strongly opposed to you possibly enjoying the benefit of this drug. What, do they speak for all people with CF now? Maybe they should just stick to the fundraising and let doctors do their job.</end quote>

This is why mutations belong to classes. Most likely we will see approval for entire classes with potentiators as they come along once a majority of the mutations in the class have been test in-vivo or in-vitro.

 

[SarahProcter]

What about mutations rare enough that no one knows what class they fall into? My current plan is to bluff, but it does give one pause.

 

[SarahProcter]

What about mutations rare enough that no one knows what class they fall into? My current plan is to bluff, but it does give one pause.

 

[Incomudrox]

I would guess the exon deletions or duplicated exon are hard to place. I don't have a straight answer... maybe someone else does.

 

[Incomudrox]

I would guess the exon deletions or duplicated exon are hard to place. I don't have a straight answer... maybe someone else does.

 

[saveferris2009]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SarahProcter</b></i> What about mutations rare enough that no one knows what class they fall into? My current plan is to bluff, but it does give one pause.</end quote>
Try the drug and you'll find out. What do you have to lose?

 

[saveferris2009]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SarahProcter</b></i> What about mutations rare enough that no one knows what class they fall into? My current plan is to bluff, but it does give one pause.</end quote>
Try the drug and you'll find out. What do you have to lose?

 

[SarahProcter]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> <div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SarahProcter</b></i> What about mutations rare enough that no one knows what class they fall into? My current plan is to bluff, but it does give one pause.</end quote> Try the drug and you'll find out. What do you have to lose?</end quote>

Sorry, I didn't quote enough context. I entirely endorse the idea that they can't possibly figure out for everyone with a rare mutation whether the drug will be helpful, and so the obvious way to proceed is to try it and see if it helps. I was speaking in reference to someone who was saying that insurance companies and/or medical providers might start to restrict prescriptions to people with specific mutation classes.
In the event that some mythical powerful "they" decides to say that only people with "gating mutations" can use kalydeco, I will cheerfully inform them that my daughter's extremely rare mutation is indeed a "gating mutation". It's not like anyone knows that it isn't!

 

[SarahProcter]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> <div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SarahProcter</b></i> What about mutations rare enough that no one knows what class they fall into? My current plan is to bluff, but it does give one pause.</end quote> Try the drug and you'll find out. What do you have to lose?</end quote>

Sorry, I didn't quote enough context. I entirely endorse the idea that they can't possibly figure out for everyone with a rare mutation whether the drug will be helpful, and so the obvious way to proceed is to try it and see if it helps. I was speaking in reference to someone who was saying that insurance companies and/or medical providers might start to restrict prescriptions to people with specific mutation classes.
In the event that some mythical powerful "they" decides to say that only people with "gating mutations" can use kalydeco, I will cheerfully inform them that my daughter's extremely rare mutation is indeed a "gating mutation". It's not like anyone knows that it isn't!