Bob Beall, CEO of CF Foundation

J

just1more

New member
Sorry, I normally quote sources, see link to actual press release from Vertex below.
<br />
<br />Compared to the placebo group, the improvement was 10.6% at 24wks, and 10.5% at 48wks. It also mentions a 16.7% improvement but does not clarify the difference.
<br />
<br />In addition FEV1 for the study group was ~63.5 (so ~50 pre-treatment??)
<br />
<br />
<br />Vertex Pharmaceutical Press Release: <a target=_blank class=ftalternatingbarlinklarge href="http://investors.vrtx.com/releasedetail.cfm?ReleaseID=551869">http://investors.vrtx.com/rele...l.cfm?ReleaseID=551869</a>
 
J

juliesdreamteam

New member
No biggie. I appreciate the link for the data. I can only verify that which was stated in the video. I'm certain that it would be better for the hard data to be used. I think we can both agree....a step in the right direction it seems. We'll keep our fingers crossed.

Thanks,

Roy Ice
Julie's Dream Team
 
J

juliesdreamteam

New member
No biggie. I appreciate the link for the data. I can only verify that which was stated in the video. I'm certain that it would be better for the hard data to be used. I think we can both agree....a step in the right direction it seems. We'll keep our fingers crossed.

Thanks,

Roy Ice
Julie's Dream Team
 
J

juliesdreamteam

New member
No biggie. I appreciate the link for the data. I can only verify that which was stated in the video. I'm certain that it would be better for the hard data to be used. I think we can both agree....a step in the right direction it seems. We'll keep our fingers crossed.
<br />
<br />Thanks,
<br />
<br />Roy Ice
<br />Julie's Dream Team
 
J

jmiller

New member
Some more encouraging info: A friend of mine is a parent of a child with G551D. During the trial she put on lots of weight (and had to practice portion control rather than "eat like a grown man"), was no longer salty tasting, and (during pre and post bronchoscopy) saw her scarred tubes heal up to a pink and healthy appearance. Very encouraging stuff.

While this drug will likely not affect many of us... it is evidence that scientists are understanding how to correct the protein production/transportation issues. I feel that this is the tip of the ice berg.
 
J

jmiller

New member
Some more encouraging info: A friend of mine is a parent of a child with G551D. During the trial she put on lots of weight (and had to practice portion control rather than "eat like a grown man"), was no longer salty tasting, and (during pre and post bronchoscopy) saw her scarred tubes heal up to a pink and healthy appearance. Very encouraging stuff.

While this drug will likely not affect many of us... it is evidence that scientists are understanding how to correct the protein production/transportation issues. I feel that this is the tip of the ice berg.
 
J

jmiller

New member
Some more encouraging info: A friend of mine is a parent of a child with G551D. During the trial she put on lots of weight (and had to practice portion control rather than "eat like a grown man"), was no longer salty tasting, and (during pre and post bronchoscopy) saw her scarred tubes heal up to a pink and healthy appearance. Very encouraging stuff.
<br />
<br />While this drug will likely not affect many of us... it is evidence that scientists are understanding how to correct the protein production/transportation issues. I feel that this is the tip of the ice berg.
 
J

juliesdreamteam

New member
HOW BEAUTIFUL IS THAT JMILLER!!!!!!!!!!? I can't raise enough money to end this disease fast enough. Very happy to hear that for the child mentioned in your post. We'll keep pushing towards our end goal of a cure for ALL people cystic fibrosis. Beautiful news indeed.

God Bless,

Roy E. Ice
Julie's Dream Team
 
J

juliesdreamteam

New member
HOW BEAUTIFUL IS THAT JMILLER!!!!!!!!!!? I can't raise enough money to end this disease fast enough. Very happy to hear that for the child mentioned in your post. We'll keep pushing towards our end goal of a cure for ALL people cystic fibrosis. Beautiful news indeed.

God Bless,

Roy E. Ice
Julie's Dream Team
 
J

juliesdreamteam

New member
HOW BEAUTIFUL IS THAT JMILLER!!!!!!!!!!? I can't raise enough money to end this disease fast enough. Very happy to hear that for the child mentioned in your post. We'll keep pushing towards our end goal of a cure for ALL people cystic fibrosis. Beautiful news indeed.
<br />
<br />God Bless,
<br />
<br />Roy E. Ice
<br />Julie's Dream Team
 
H

hmw

New member
<div class="FTQUOTE"><begin quote>In addition FEV1 for the study group was ~63.5 (so ~50 pre-treatment??)</end quote></div>
No... it looks like the average fev1 of both trial groups was in that range... the article states this:
"Baseline lung function in STRIVE was 63.5 percent predicted for patients in the VX-770 treatment group and 63.7 percent predicted among those in the placebo control group." So an increase of 10.6% would bump the treatment group up to about 70.2%.

This utterly confuses me though... we see this quote in many press releases: Results of the STRIVE study showed that people treated with VX-770 achieved a <i>mean absolute improvement from baseline compared to placebo</i> of 10.6%.

What does THIS mean, in comparison? "In addition, people treated with VX-770 experienced a 16.7 percent <i>relative mean improvement in lung function from baseline compared to placebo</i>."

Statistically speaking, what does that 2nd one even mean? Other lung functions aside from fev1? Other ways of spinning the numbers entirely?
 
H

hmw

New member
<div class="FTQUOTE"><begin quote>In addition FEV1 for the study group was ~63.5 (so ~50 pre-treatment??)</end quote>
No... it looks like the average fev1 of both trial groups was in that range... the article states this:
"Baseline lung function in STRIVE was 63.5 percent predicted for patients in the VX-770 treatment group and 63.7 percent predicted among those in the placebo control group." So an increase of 10.6% would bump the treatment group up to about 70.2%.

This utterly confuses me though... we see this quote in many press releases: Results of the STRIVE study showed that people treated with VX-770 achieved a <i>mean absolute improvement from baseline compared to placebo</i> of 10.6%.

What does THIS mean, in comparison? "In addition, people treated with VX-770 experienced a 16.7 percent <i>relative mean improvement in lung function from baseline compared to placebo</i>."

Statistically speaking, what does that 2nd one even mean? Other lung functions aside from fev1? Other ways of spinning the numbers entirely?
 
H

hmw

New member
<div class="FTQUOTE"><begin quote>In addition FEV1 for the study group was ~63.5 (so ~50 pre-treatment??)</end quote>
<br />No... it looks like the average fev1 of both trial groups was in that range... the article states this:
<br />"Baseline lung function in STRIVE was 63.5 percent predicted for patients in the VX-770 treatment group and 63.7 percent predicted among those in the placebo control group." So an increase of 10.6% would bump the treatment group up to about 70.2%.
<br />
<br />This utterly confuses me though... we see this quote in many press releases: Results of the STRIVE study showed that people treated with VX-770 achieved a <i>mean absolute improvement from baseline compared to placebo</i> of 10.6%.
<br />
<br />What does THIS mean, in comparison? "In addition, people treated with VX-770 experienced a 16.7 percent <i>relative mean improvement in lung function from baseline compared to placebo</i>."
<br />
<br />Statistically speaking, what does that 2nd one even mean? Other lung functions aside from fev1? Other ways of spinning the numbers entirely?
 
K

ktsmom

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>hmw</b></i>

What does THIS mean, in comparison? "In addition, people treated with VX-770 experienced a 16.7 percent <i>relative mean improvement in lung function from baseline compared to placebo</i>."



Statistically speaking, what does that 2nd one even mean? Other lung functions aside from fev1? Other ways of spinning the numbers entirely?</end quote></div>

Relavtive mean is an actual statistic all of its own, and quantifies the mean difference in comparison to the size of the mean. Wikipedia has an example - can't quote it because their website is down right now. But in my mind this stat indicates that the increase was even better than it appears in absolute numbers.
 
K

ktsmom

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>hmw</b></i>

What does THIS mean, in comparison? "In addition, people treated with VX-770 experienced a 16.7 percent <i>relative mean improvement in lung function from baseline compared to placebo</i>."



Statistically speaking, what does that 2nd one even mean? Other lung functions aside from fev1? Other ways of spinning the numbers entirely?</end quote>

Relavtive mean is an actual statistic all of its own, and quantifies the mean difference in comparison to the size of the mean. Wikipedia has an example - can't quote it because their website is down right now. But in my mind this stat indicates that the increase was even better than it appears in absolute numbers.
 
K

ktsmom

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>hmw</b></i>
<br />
<br />What does THIS mean, in comparison? "In addition, people treated with VX-770 experienced a 16.7 percent <i>relative mean improvement in lung function from baseline compared to placebo</i>."
<br />
<br />
<br />
<br />Statistically speaking, what does that 2nd one even mean? Other lung functions aside from fev1? Other ways of spinning the numbers entirely?</end quote>
<br />
<br />Relavtive mean is an actual statistic all of its own, and quantifies the mean difference in comparison to the size of the mean. Wikipedia has an example - can't quote it because their website is down right now. But in my mind this stat indicates that the increase was even better than it appears in absolute numbers.
 
H

hmw

New member
I tried looking it up and could not understand the math at all- none of the examples I could find involved 'real' numbers.

The 16% would be more exciting to me if they could tell us exactly what was seen- the 10.6% increase in fev1 that I can mathematically apply to their starting baseline is something tangible and gives me hope for what to look forward to with the 770/809 trials. I don't know how to interpret that 16% with nothing solid to apply it to and I wish they told us more, because that is such a *bigger* number, ya know?
 
H

hmw

New member
I tried looking it up and could not understand the math at all- none of the examples I could find involved 'real' numbers.

The 16% would be more exciting to me if they could tell us exactly what was seen- the 10.6% increase in fev1 that I can mathematically apply to their starting baseline is something tangible and gives me hope for what to look forward to with the 770/809 trials. I don't know how to interpret that 16% with nothing solid to apply it to and I wish they told us more, because that is such a *bigger* number, ya know?
 
H

hmw

New member
I tried looking it up and could not understand the math at all- none of the examples I could find involved 'real' numbers.
<br />
<br />The 16% would be more exciting to me if they could tell us exactly what was seen- the 10.6% increase in fev1 that I can mathematically apply to their starting baseline is something tangible and gives me hope for what to look forward to with the 770/809 trials. I don't know how to interpret that 16% with nothing solid to apply it to and I wish they told us more, because that is such a *bigger* number, ya know?
 
J

just1more

New member
Ok, putting on my math teacher hat:

absolute mean = good old average = ie, if you add everyone's improvement/decreases and divide by the # of participants. This came out at 10.6%. NOTE: This can and is tweaked by odd results, such as someone(s) with a decrease in FEV1, or maybe someone with a great improvement.

relative mean = calculus to try and discount the impact of results the farther they are from the average. IE if the absolute mean was 100; then in relative mean results between 80-120 would have more impact on the reported # than a result of 38. The math is pretty nasty; though I will explain via PM if anyone really has a burning desire to know.

So ultimately, if you take everyone they saw at 10.6% increase. If you only counted the 'normal' patients results then it was closer to 17%.
 
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