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View Full Version : If you don't have a gating mutation and want Kalydeco....



Incomudrox
03-23-2012, 08:23 PM
If you don't have a gating mutation and want Kalydeco you better push your doctors NOW before 2nd quarter of 2012. I just spoke with a pharmasist at one of the pharmacies that fills Kalydeco, they are FULLY expecting after Q2 meetings a large amount of insurance companies will require proof of G551D and or other gating mutations (Class 3).

Point being push as hard as possible to get it now that was if you will see an improvement from it you know now and have ammo to fight requirements that may need to be satisfied later down the road. Document EVERYTHING with your clinic.

They also told me that not many insurance companies (including medicaid plans) are not currently denying it. They said the only medicaid plan off the top of her head with issues was in Kansas.

For for thought people.

Incomudrox
03-23-2012, 08:23 PM
If you don't have a gating mutation and want Kalydeco you better push your doctors NOW before 2nd quarter of 2012. I just spoke with a pharmasist at one of the pharmacies that fills Kalydeco, they are FULLY expecting after Q2 meetings a large amount of insurance companies will require proof of G551D and or other gating mutations (Class 3).

Point being push as hard as possible to get it now that was if you will see an improvement from it you know now and have ammo to fight requirements that may need to be satisfied later down the road. Document EVERYTHING with your clinic.

They also told me that not many insurance companies (including medicaid plans) are not currently denying it. They said the only medicaid plan off the top of her head with issues was in Kansas.

For for thought people.

tumbleon
03-23-2012, 08:39 PM
I have read a lot of talk about trying to get Kalydeco if you do not have the G551D mutation. I have not seen or heard a lot if any responses or results of people who actually have tried it? Can those people who do not have G551D and have gone on Kalydeco tell us if they have seen any improvement or decline or what has been their experience results? Thank You

tumbleon
03-23-2012, 08:39 PM
I have read a lot of talk about trying to get Kalydeco if you do not have the G551D mutation. I have not seen or heard a lot if any responses or results of people who actually have tried it? Can those people who do not have G551D and have gone on Kalydeco tell us if they have seen any improvement or decline or what has been their experience results? Thank You

dramamama
03-23-2012, 09:46 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Incomudrox</b></i> If you don't have a gating mutation and want Kalydeco you better push your doctors NOW before 2nd quarter of 2012. I just spoke with a pharmasist at one of the pharmacies that fills Kalydeco, they are FULLY expecting after Q2 meetings a large amount of insurance companies will require proof of G551D and or other gating mutations (Class 3). Point being push as hard as possible to get it now that was if you will see an improvement from it you know now and have ammo to fight requirements that may need to be satisfied later down the road. Document EVERYTHING with your clinic. They also told me that not many insurance companies (including medicaid plans) are not currently denying it. They said the only medicaid plan off the top of her head with issues was in Kansas. For for thought people.</end quote>

HUGE INFO!! You should repost this in other groups you are a part of... wink, wink.
I'll post ot on the praying for success on facebook.

dramamama
03-23-2012, 09:46 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Incomudrox</b></i> If you don't have a gating mutation and want Kalydeco you better push your doctors NOW before 2nd quarter of 2012. I just spoke with a pharmasist at one of the pharmacies that fills Kalydeco, they are FULLY expecting after Q2 meetings a large amount of insurance companies will require proof of G551D and or other gating mutations (Class 3). Point being push as hard as possible to get it now that was if you will see an improvement from it you know now and have ammo to fight requirements that may need to be satisfied later down the road. Document EVERYTHING with your clinic. They also told me that not many insurance companies (including medicaid plans) are not currently denying it. They said the only medicaid plan off the top of her head with issues was in Kansas. For for thought people.</end quote>

HUGE INFO!! You should repost this in other groups you are a part of... wink, wink.
I'll post ot on the praying for success on facebook.

Keepercjr
03-24-2012, 01:44 PM
Thank you! I just got it wednesday and am waiting to see if I see any improvement. I just came down w/ a cold last night so obviously I can't tell anything yet. I asked my doctor to prescribe me a home FEV1 meter (I tried to order it on drugstore.com but it was backordered and then they cancelled it) so that I can track any changes to have ammo in case I start getting denied.

tumbleon - I think many who are on it are waiting to post any results until they have been on it for at least a few weeks. I know I am. Plus I have no way to say whether it is raising my PFTs or not since I live 3.5 hours from my clinic and don't have a home FEV1 monitor yet.

Keepercjr
03-24-2012, 01:44 PM
Thank you! I just got it wednesday and am waiting to see if I see any improvement. I just came down w/ a cold last night so obviously I can't tell anything yet. I asked my doctor to prescribe me a home FEV1 meter (I tried to order it on drugstore.com but it was backordered and then they cancelled it) so that I can track any changes to have ammo in case I start getting denied.

tumbleon - I think many who are on it are waiting to post any results until they have been on it for at least a few weeks. I know I am. Plus I have no way to say whether it is raising my PFTs or not since I live 3.5 hours from my clinic and don't have a home FEV1 monitor yet.

jmiller
03-24-2012, 06:40 PM
Another FYI-- my doctor spoke w the cf foundation after I asked to try it. They are going to formally take a stance of strongly discouraging off-label prescriptions for it. So this reinforces the time sensitive nature of trying it. If I get my hands on it we are going to do a pre and post sweat chloride test with the assumption that pre and post pft's arent necessarily enough to demonstrate concretely that it is or isn't working.

jmiller
03-24-2012, 06:40 PM
Another FYI-- my doctor spoke w the cf foundation after I asked to try it. They are going to formally take a stance of strongly discouraging off-label prescriptions for it. So this reinforces the time sensitive nature of trying it. If I get my hands on it we are going to do a pre and post sweat chloride test with the assumption that pre and post pft's arent necessarily enough to demonstrate concretely that it is or isn't working.

saveferris2009
03-24-2012, 07:26 PM
FYI the Kaly Phase III study showed that change in sweat chloride didn't necessarily correlate with magnitude of FEV1 difference. So a sweat test is a nice to know, but it's not your sweat chloride # that predicts your health with CF - it's your FEV1.

Sad that the CF Foundation might take a stance - it's amazing what's out there for how widely this drug can work for people. We'll see what they say! Won't change what I'm doing, though.

But then again, I'm a bit more of an independent type - using the eFlow back in 2006 way before Cayston was approved; using NAC; using the InCourage Vest instead of the heavily promoted HillRom vest, pushing to do a blood sugar diary even though my OGTT came back normal and of course then being diagnosed with CFRD, using Magnesium to help MDRPA, etc. (influenced heavily by dramamama of course!)

So that's just me <img src="i/expressions/face-icon-small-smile.gif" border="0">

saveferris2009
03-24-2012, 07:26 PM
FYI the Kaly Phase III study showed that change in sweat chloride didn't necessarily correlate with magnitude of FEV1 difference. So a sweat test is a nice to know, but it's not your sweat chloride # that predicts your health with CF - it's your FEV1.

Sad that the CF Foundation might take a stance - it's amazing what's out there for how widely this drug can work for people. We'll see what they say! Won't change what I'm doing, though.

But then again, I'm a bit more of an independent type - using the eFlow back in 2006 way before Cayston was approved; using NAC; using the InCourage Vest instead of the heavily promoted HillRom vest, pushing to do a blood sugar diary even though my OGTT came back normal and of course then being diagnosed with CFRD, using Magnesium to help MDRPA, etc. (influenced heavily by dramamama of course!)

So that's just me <img src="i/expressions/face-icon-small-smile.gif" border="0">

jmiller
03-24-2012, 07:37 PM
Amen sister!!

jmiller
03-24-2012, 07:37 PM
Amen sister!!

dramamama
03-24-2012, 07:42 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>jmiller</b></i> Another FYI-- my doctor spoke w the cf foundation after I asked to try it. They are going to formally take a stance of strongly discouraging off-label prescriptions for it. So this reinforces the time sensitive nature of trying it. If I get my hands on it we are going to do a pre and post sweat chloride test with the assumption that pre and post pft's arent necessarily enough to demonstrate concretely that it is or isn't working.</end quote>

This devastates me... I cannot believe they would take this stance. so sad

dramamama
03-24-2012, 07:42 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>jmiller</b></i> Another FYI-- my doctor spoke w the cf foundation after I asked to try it. They are going to formally take a stance of strongly discouraging off-label prescriptions for it. So this reinforces the time sensitive nature of trying it. If I get my hands on it we are going to do a pre and post sweat chloride test with the assumption that pre and post pft's arent necessarily enough to demonstrate concretely that it is or isn't working.</end quote>

This devastates me... I cannot believe they would take this stance. so sad

Anomie
03-24-2012, 11:22 PM
How will they know which mutations it is effective against if they don't want to let people try it?

Anomie
03-24-2012, 11:22 PM
How will they know which mutations it is effective against if they don't want to let people try it?

Incomudrox
03-25-2012, 12:51 AM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Anomie</b></i> How will they know which mutations it is effective against if they don't want to let people try it?</end quote>

Through more long and gruling clinic trails that are expensive and drive the need for more fund raising so the CFF can keep exsisting! Also cell line in vitro testing will be a large part. CFF is a crooked as the rest of them. We live in a capitolist system you will never see a "once and done cure" they don't want it.

Incomudrox
03-25-2012, 12:51 AM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Anomie</b></i> How will they know which mutations it is effective against if they don't want to let people try it?</end quote>

Through more long and gruling clinic trails that are expensive and drive the need for more fund raising so the CFF can keep exsisting! Also cell line in vitro testing will be a large part. CFF is a crooked as the rest of them. We live in a capitolist system you will never see a "once and done cure" they don't want it.

albino15
03-25-2012, 02:36 AM
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
03-25-2012, 02:36 AM
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
03-25-2012, 03:00 AM
<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
03-25-2012, 03:00 AM
<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
03-25-2012, 03:16 AM
<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
03-25-2012, 03:16 AM
<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
03-25-2012, 03:24 AM
<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
03-25-2012, 03:24 AM
<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
03-25-2012, 07:56 AM
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
03-25-2012, 07:56 AM
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
03-25-2012, 08:06 AM
<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
03-25-2012, 08:06 AM
<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
03-25-2012, 02:46 PM
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
03-25-2012, 02:46 PM
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
03-25-2012, 02:53 PM
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
03-25-2012, 02:53 PM
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
03-25-2012, 04:18 PM
<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
03-25-2012, 04:18 PM
<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
03-26-2012, 03:51 AM
<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
03-26-2012, 03:51 AM
<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!