FDA Advisory Committee on Combo in Process

[Aboveallislove]

Yeah, I would probably do the same...not rock the boat. And I know it must feel like you can't possible get more but those with 551 and df508 got another 4.6% improvement when the 809 was added to Kalydeco. I just realized though that you don't have df508, so I guess it would depend on how your two mutations work if the second would help. I am so very thrilled how well you are doing and pray more will succeed in getting soon off-label or in the 661 studies.

 

[Aboveallislove]

Hey bill, If it is suppose to be a patient advocate it should be someone who is familiar with the type of patient...just like you say see a cf specialist and rightly so,...have a cf Advocate. And if most doctors can't even spell CF, how do you think an oceanographer spells it? H2O? Left right or ambidextrous wouldnt matter for surgery (although there might well be some studies that say otherwise), but surely oceanography versus cf specilaist isnt analogous! And they wouldn't have all three reviewed the same day before the same committee because even the fda understands that there are different expertises...they use over 50 different committees and panels to get expert advise. So huntingtons wouldnt be a pulmonary committe and neither would charcot marie tooth. The fda put someone on the committee to be an advocate for the patient and I think the choice was a poor one. i Followed the entire hearing and all the questions and i think that is why the advocate voted no...not some other meritorious reason, especially in light of the reason the person themselves gave for voting no.

 

[Printer]

OK, so you want a specialized advocate for each disease or type thereof. Got it.

 

[Aboveallislove]

Yup. I think that's what patients deserve.

 

[jricci]

Aboveallislove
Speaking of advocates, I just wanted to thank you for being such an advocate for the CF community on this forum. You do a wonderful job not just keeping us informed, but also offering support. You started the thread alerting us about this advisory committee and encouraging us to write letters. As you said, committee members were swayed by patient testimony. You had a direct impact in the making of this landmark decision. Thank you!

 

[triples15]

Aboveallislove
Speaking of advocates, I just wanted to thank you for being such an advocate for the CF community on this forum. You do a wonderful job not just keeping us informed, but also offering support. You started the thread alerting us about this advisory committee and encouraging us to write letters. As you said, committee members were swayed by patient testimony. You had a direct impact in the making of this landmark decision. Thank you!

Amen Jricci! Thanks Aboveall and Jeanne for leading the effort! The CF community should be proud. Here's an article I came across on FB newsfeed (thanks Liza!)a bit ago:

http://www.bizjournals.com/boston/b...nalyst-patients-were-key-to-vertex-panel.html

An excerpt from the article:

But it wasn’t specifically that argument which seems to have won 12 of the 13 panel members over on Tuesday. Rather, it was the 17 patient advocates who spoke passionately in favor of approval, at least according to Leerink Partners analyst Howard Liang, who called it “perhaps the most effective and best organized plea for the need of the drug, the differences it made in some patients and for approval of the drug that we have ever seen at an FDA advisory panel.”

I'd like to thank those 17 individuals who testified! They must have done an amazing job.

Autumn

 

[Aboveallislove]

thank you jricci and Autumn. And thank you Jeanne! Honestly, to me, this is my coping. Information. This forum. Jeanne and her crew make it possible!
And re the testimony: The liveblogger had some excerpts and it did sound awesome. They all sounded powerful and emotional, but what might have "clicked" most was the teenager who said something to the effect that instead of losing lung function every year, 2-4 improvement is amazing. I wish I remembered the words but it was that I think they needed hammer home! It sounds like the dad who testified too had an even greater benefit "it saved my life" he said.

 

[jricci]

Agreed. Statements were very powerful, accurate, and effective. Here is an example:
Masters, who testified before the advisory panel, said the drug has dramatically changed his life, allowing him to gain weight and improving his lung function enough so he can run 5 kilometer road races.“For the first time in my life, I feel healthy,” Masters said. “This drug has given me hope for the future. I’m here to tell you in no uncertain terms that this drug has saved my life.”Standing before the committee with his 6-year-old daughter Ariana, he said:“I ask you to vote yes today so I can watch her graduate from high school and graduate from college. I ask you to vote yes so I can walk her down the aisle when her time comes.”

 

[static]

Agreed. Statements were very powerful, accurate, and effective. Here is an example:
Masters, who testified before the advisory panel, said the drug has dramatically changed his life, allowing him to gain weight and improving his lung function enough so he can run 5 kilometer road races.“For the first time in my life, I feel healthy,” Masters said. “This drug has given me hope for the future. I’m here to tell you in no uncertain terms that this drug has saved my life.”Standing before the committee with his 6-year-old daughter Ariana, he said:“I ask you to vote yes today so I can watch her graduate from high school and graduate from college. I ask you to vote yes so I can walk her down the aisle when her time comes.”

That is very moving.

I'd be curios to find out the patients advocates reasoning for voting no. I'm under the belief that everything comes with risks, but I agree people who want it should have a chance to try it. Ultimately I want what we all want, minimize risk (health wise and financially) maximize reward and I don't think we are there yet, but it's progress nonetheless

 

[triples15]

That is very moving.

I'd be curios to find out the patients advocates reasoning for voting no. I'm under the belief that everything comes with risks, but I agree people who want it should have a chance to try it. Ultimately I want what we all want, minimize risk (health wise and financially) maximize reward and I don't think we are there yet, but it's progress nonetheless

As far as the patient advocate's reasoning.. Like Ethan mentioned, the live blogger reporting on the hearing said that her questions seemed to demonstrate that she did not understand how the drug works. In addition, he said something to the effect that it appeared she just "didn't get" the data.

 

[Aboveallislove]

I'd also add the advocate voted the drug was safe. And I think it was the advocate who said the risk was minimal and could be managed. The rational the advocate gave was that having voted "no" on the earlier question (about whether it was Kalydeco or the combo alone that worked), that the advocate felt they had to vote note. When the transcript comes out that will have the exact language.