FDA drugs

[dyza]

Has anyone any insight into how long it takes FDA approved drugs to reach the shores of the UK, or other countries for that matter?
Obvoiusly I have the new drug in mind, and subsequently the next one ( 890?), Oh and on that note, fantastic news on the vX770,
its close, its coming
One would assume that the UK would do their own tests on outside drugs before approval, I'm sure other drugs, tobi, whatever, went troo the same scenario.
Only problem I can see in the UK is the cost, only today the NHS (national health service), knocked back the use of a drug for use by men suffering from prostate cancer, their reason being that it onlygave an average 4 months extra life, and that it costs £3000 per month.

craig

 

[dyza]

Has anyone any insight into how long it takes FDA approved drugs to reach the shores of the UK, or other countries for that matter?
Obvoiusly I have the new drug in mind, and subsequently the next one ( 890?), Oh and on that note, fantastic news on the vX770,
its close, its coming
One would assume that the UK would do their own tests on outside drugs before approval, I'm sure other drugs, tobi, whatever, went troo the same scenario.
Only problem I can see in the UK is the cost, only today the NHS (national health service), knocked back the use of a drug for use by men suffering from prostate cancer, their reason being that it onlygave an average 4 months extra life, and that it costs £3000 per month.

craig

 

[LouLou]

Hi Craig, You'll want to contact the Scottish Medicines Consortium. Express in writing and voice (IMHO) how important and life saving this drug is for your family. I would send it certified mail to show you want this attended to ASAP! It seems that once the EU medication review board approves the drug, the SMC will need to also approve it and decide on the methodology that the drug will be released. The process for marketing this drug will be different than others because of its ULTRA ORPHAN DRUG STATUS. As you research on google, make sure to use those search terms. Some EU counties release the UOD with a stipulation that they have to get it from a certain care center and be followed by that care center for it's effectiveness. It sounds sort of like a mini trial without the placebo being given out. Definitely some data collection going on though and reporting back to SMC and possibly even the EU.

I wasn't aware that your family has G551D. But gathering from your post I assume you do. If you do not have G551D and are hoping for approval for the drug for another mutation, i think it's still worth a shot as the positive results in epithelial cells of other mutations might be enough for the EU to think it's a good idea.

Your efforts might be all for not as it might be like trying to push a river. 97% of Americans with cf are genotyped. Do you think there is as widespreadd genotyping...allowing the Scot officials accurate data of exactly how many people this will effect?

As for how long it takes...often times the EU follows suit immediately after the FDA because it is possible that the EU has simultaneously been revieiwng this medication. I think if you talk to clinic you will get some great info too. For example, I bet they know if they are likely to be one of these qualified clinics for participation or not if Scotland is one of the countries in the EU that requires this extra tracking. Personally I'd set an appt even if he isn't due for one just so you can get your foot in the door for a face-to-face to find out late breaking news.

 

[LouLou]

Hi Craig, You'll want to contact the Scottish Medicines Consortium. Express in writing and voice (IMHO) how important and life saving this drug is for your family. I would send it certified mail to show you want this attended to ASAP! It seems that once the EU medication review board approves the drug, the SMC will need to also approve it and decide on the methodology that the drug will be released. The process for marketing this drug will be different than others because of its ULTRA ORPHAN DRUG STATUS. As you research on google, make sure to use those search terms. Some EU counties release the UOD with a stipulation that they have to get it from a certain care center and be followed by that care center for it's effectiveness. It sounds sort of like a mini trial without the placebo being given out. Definitely some data collection going on though and reporting back to SMC and possibly even the EU.

I wasn't aware that your family has G551D. But gathering from your post I assume you do. If you do not have G551D and are hoping for approval for the drug for another mutation, i think it's still worth a shot as the positive results in epithelial cells of other mutations might be enough for the EU to think it's a good idea.

Your efforts might be all for not as it might be like trying to push a river. 97% of Americans with cf are genotyped. Do you think there is as widespreadd genotyping...allowing the Scot officials accurate data of exactly how many people this will effect?

As for how long it takes...often times the EU follows suit immediately after the FDA because it is possible that the EU has simultaneously been revieiwng this medication. I think if you talk to clinic you will get some great info too. For example, I bet they know if they are likely to be one of these qualified clinics for participation or not if Scotland is one of the countries in the EU that requires this extra tracking. Personally I'd set an appt even if he isn't due for one just so you can get your foot in the door for a face-to-face to find out late breaking news.

 

[saveferris2009]

Sorry to hijack the thread, but Lauren could you post the info you refer to about how Kaly impacted other mutations?

I have the copy of the NEJM article published rearding G551D, but I am having a tough time finding info on other mutations (although I know it's out there) - particularly, as you say, the positive results in epithelial cells of other mutations.

Thanks!

 

[saveferris2009]

Sorry to hijack the thread, but Lauren could you post the info you refer to about how Kaly impacted other mutations?

I have the copy of the NEJM article published rearding G551D, but I am having a tough time finding info on other mutations (although I know it's out there) - particularly, as you say, the positive results in epithelial cells of other mutations.

Thanks!

 

[hmw]

Amy: Have you watched the plenary sessions from the CF conferences (available to watch on the cff website)? There were slides on the presentation discussing the drug trials indicating positive results with other gating mutations. I cannot remember clear details right now, but if you haven't watched it I would- lots of good info.

 

[hmw]

Amy: Have you watched the plenary sessions from the CF conferences (available to watch on the cff website)? There were slides on the presentation discussing the drug trials indicating positive results with other gating mutations. I cannot remember clear details right now, but if you haven't watched it I would- lots of good info.

 

[saveferris2009]

Candidly, I am looking for info on how Kaly impacted DF508. I know some patients saw some statistically significant results both in sweat chloride and in FEV1 - I just can't recall where I found the info.

But I will absolutely watch the plenary sessions from the CF conferences. Thanks so much for the tip! <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[saveferris2009]

Candidly, I am looking for info on how Kaly impacted DF508. I know some patients saw some statistically significant results both in sweat chloride and in FEV1 - I just can't recall where I found the info.

But I will absolutely watch the plenary sessions from the CF conferences. Thanks so much for the tip! <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[LouLou]

Not a lot of info here but here's one link...
http://www.cendigital.org/cendigital/20110425_sub?sub_id=ELwo55awjyWD&folio=33#pg26

 

[LouLou]

Not a lot of info here but here's one link...
http://www.cendigital.org/cendigital/20110425_sub?sub_id=ELwo55awjyWD&folio=33#pg26

 

[LouLou]

here are a few more. I reviewed the Phase III results (believe it was a pdf) at one time. I'm not finding it again...everything requires a subscription. Here are some good links though and you can probably access from work if your workplace as a subscription to the journal.
http://www.ncbi.nlm.nih.gov/pubmed/19846789
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2773991/
http://www.businesswire.com/news/home/20111102007044/en/England-Journal-Medicine-Publishes-Data-Phase-3
http://www.nejm.org/doi/full/10.1056/NEJMoa1105185

 

[LouLou]

here are a few more. I reviewed the Phase III results (believe it was a pdf) at one time. I'm not finding it again...everything requires a subscription. Here are some good links though and you can probably access from work if your workplace as a subscription to the journal.
http://www.ncbi.nlm.nih.gov/pubmed/19846789
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2773991/
http://www.businesswire.com/news/home/20111102007044/en/England-Journal-Medicine-Publishes-Data-Phase-3
http://www.nejm.org/doi/full/10.1056/NEJMoa1105185

 

[saveferris2009]

ah-ha! http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2773991/ has EXACTLY what I was looking for.

Thank you thank you thank you, Lauren! <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[saveferris2009]

ah-ha! http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2773991/ has EXACTLY what I was looking for.

Thank you thank you thank you, Lauren! <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[cfsucks]

so is it worth it to try to get this drug if you don't carry the g551d mutation?

 

[cfsucks]

so is it worth it to try to get this drug if you don't carry the g551d mutation?

 

[Anomie]

If you have a gating mutation then its worth asking about. I don't see how vx 770 alone would be of much benefit to the double deltas. Because barely any of the salt makes it to the surface the benefit would be very minimal and probably not worth 300k a year.

 

[Anomie]

If you have a gating mutation then its worth asking about. I don't see how vx 770 alone would be of much benefit to the double deltas. Because barely any of the salt makes it to the surface the benefit would be very minimal and probably not worth 300k a year.