Aboveallislove
Super Moderator
Live blogging by an analyst here:
http://www.thestreet.com/story/1314...ic-fibrosis-fda-advisory-panel-live-blog.html
http://www.thestreet.com/story/1314...ic-fibrosis-fda-advisory-panel-live-blog.html
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FDA Advisory Committee on Combo in Process
- Thread starter Aboveallislove
- Start date
D
Dank
Guest
Just wow. The amount of emotion in the speakers is crazy! Thanks for sharing this.
Aboveallislove
Super Moderator
The committee is discussing now and will vote shortly...discussion is showing smooth sailing for recommendation for approval..then FDA will have to approve to get it to market
Aboveallislove
Super Moderator
The committee voted 12-1 to recommend approval. The only "no" vote was from the patient advocate!!!
Aboveallislove
Super Moderator
One member admitted being swayed by patient testimony: It is modest but these folks are desparate and need this. Thanks to all who submitted comments!
grammakaky
New member
Thank you for having the link for the live blogging. I followed it all the way and right now I so full of Emotion. Happiness, crying. Thank you, Thank you.
the FDA advisory panel votes to recommend the approval of Orkambi in CF patients with the f508del mutation.
the FDA advisory panel votes to recommend the approval of Orkambi in CF patients with the f508del mutation.
One member admitted being swayed by patient testimony: It is modest but these folks are desparate and need this. Thanks to all who submitted comments!
Yes, this: "Morrato admits to being swayed by patient testimony, that stuff matters"
And WHAT on the patient advocate being the only "no" vote?? Is she serious??
grammakaky
New member
What was her job there. She didn't do anything to help our cause. The ones they were worried about voted it through.
Aboveallislove
Super Moderator
I'm hoping the fda comes through earlier....well be waiting though until October when 6-11 trial results....Godspeed to all.
imported_Momto2
New member
Maybe the patient advocate knows something that was not brought to light. I know my doc was not exactly over the moon about this new combo.
The live blogger at The Street seemed to not think much of the patient advocate. But my Dr wasn't super excited about the combo when I asked about it after the Phase 2 studies a couple years back. I think my Doc was disappointed in the pricing of Kalydeco and expressed that if the combo was priced similar that I wasn't sick enough for the cost of it. I'm still excited about it, but like everyone I'm still hoping for that golden treatment someday.
Aboveallislove
Super Moderator
The patient advocate said they voted no because they had voted earlier that 809 didn't have improvement over kalydeco alone. But if you want the real reason it's this...the person has no clue what cf is or means. Bio http://www.fda.gov/downloads/Adviso...y-AllergyDrugsAdvisoryCommittee/UCM392028.pdf. The individual is an oceanographer! I agree though it is modest...and I see why the doctors aren't supper excited about modest. But I am.
S
stephen
Guest
It's great to hear that Kalydeco with 809 is closer to approval and will be available to many CFers soon.
I'm sure that like me, many with a residual function mutation not currently on the "approved list " can really benefit from it. Evidence shows this ONLY applies to pulmonary issues.
I've written to the FDA more than once about my experience with Kalydeco. For me, except for reversing previous lung damage, it has been the Golden Treatment thank G-d.
While the cost of Kalydeco is extremely high, reading that Vertex has accumulated over $4 billion in losses puts the cost in some perspective.
Recent articles indicate that progress is being made in techniques where defection genes can be fixed or replaced. This should be a real cure, except for maybe male reproductive issues.
(Please excuse any typos since I'm fortunate to be traveling - in Israel- and posted this with my iPhone)
I'm sure that like me, many with a residual function mutation not currently on the "approved list " can really benefit from it. Evidence shows this ONLY applies to pulmonary issues.
I've written to the FDA more than once about my experience with Kalydeco. For me, except for reversing previous lung damage, it has been the Golden Treatment thank G-d.
While the cost of Kalydeco is extremely high, reading that Vertex has accumulated over $4 billion in losses puts the cost in some perspective.
Recent articles indicate that progress is being made in techniques where defection genes can be fixed or replaced. This should be a real cure, except for maybe male reproductive issues.
(Please excuse any typos since I'm fortunate to be traveling - in Israel- and posted this with my iPhone)
Aboveallislove
Super Moderator
So true Stephen. Safe travels. And hoping that 661 gets the residual functions "on label" soon and that more will have doctors willing to try 809 combo off label if they can't get into studies in the meantime. Are you going to try to move to 809 combo or too concerned re insurance deciding to deny both when it gets on their radar?
Aboveallislove
Super Moderator
Hey Bill,
Nice to see you back. So sorry re your hospital stay.
I think the patient advocate on a FDA AdComm should be educated and trained/experienced in the disease for which the drug is under consideration and have day-to-day experience with the disease. My "ideal" would be a CFer who is also a doctor or has a master/doctor in a science field. But I'm sorry, someone who has a Ph.D. in Physical Oceanography and starts a consumer advocacy group doesn't cut it for a patient "ADVOCATE". And this individual voted "no," based on something the FDA said was not required: The FDA said you can vote "no," it doesn't provide added benefit or Kalydeco and still vote "yes," it should be approved. And yet this individual stated that they felt they had to vote no because of their earlier vote. And this individual was the only no vote even though the entire day included very strenuous questioning by others who are educated and trained in the area. I don't think "just" a mom would be a good advocate for the advisory committee. That's what the patient/public comments are for. Moms (or dads if they are doing the care 24/7) will "know" their kids better and know when something is wrong or when something isn't working, but won't (absent the education, science, training, etc.), be able to analyze the data the way the advisory committee is suppose too, although they'd be able to know when something isn't logical. But I know another CF mom who has the masters in science and reads and understands all the research. She'd be another ideal.
Best
Love
Nice to see you back. So sorry re your hospital stay.
I think the patient advocate on a FDA AdComm should be educated and trained/experienced in the disease for which the drug is under consideration and have day-to-day experience with the disease. My "ideal" would be a CFer who is also a doctor or has a master/doctor in a science field. But I'm sorry, someone who has a Ph.D. in Physical Oceanography and starts a consumer advocacy group doesn't cut it for a patient "ADVOCATE". And this individual voted "no," based on something the FDA said was not required: The FDA said you can vote "no," it doesn't provide added benefit or Kalydeco and still vote "yes," it should be approved. And yet this individual stated that they felt they had to vote no because of their earlier vote. And this individual was the only no vote even though the entire day included very strenuous questioning by others who are educated and trained in the area. I don't think "just" a mom would be a good advocate for the advisory committee. That's what the patient/public comments are for. Moms (or dads if they are doing the care 24/7) will "know" their kids better and know when something is wrong or when something isn't working, but won't (absent the education, science, training, etc.), be able to analyze the data the way the advisory committee is suppose too, although they'd be able to know when something isn't logical. But I know another CF mom who has the masters in science and reads and understands all the research. She'd be another ideal.
Best
Love
S
stephen
Guest
Aboveallislove,
I truly hope that more patients can how get a chance to at least try Kalydeco or the combo.
As as for me, I wouldn't want to rock the boat - but I can't see doing any better than I'm doing now. I just wish there were more like me. I do know that there are others.
I truly hope that more patients can how get a chance to at least try Kalydeco or the combo.
As as for me, I wouldn't want to rock the boat - but I can't see doing any better than I'm doing now. I just wish there were more like me. I do know that there are others.
How would she do, for example, if three drugs were reviewed the same day, one for CF, one for Huntington's and a third for Sharko, Marie, Tooth. Would she have expertise to vote on all these drugs? Or do we need a different advocate for everything including right hand vs left hand surgery?