Re: Re: Clinical Trial - Il-1, Rilonacpet Update - Unbelievable

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You ROCK Jo!! and Rob 19 JAS Re: Clinical Trial - Il-1, Rilonacpet Update - Unbelievable > PS: Here are the links to their Wiki web pages-> > http://en.wikipedia.org/wiki/Rilonacept> > http://en.wikipedia.org/wiki/Regeneron> > > So, you know how people can log on and add to the info? Perhaps > an > addition mentioning the sudden discontinuation of the Rilonacept > trial, "for reasons as yet not made clear", might help....> > Sorry, it's the devil in me.> > Jo> > > > > > >> > Hi all-> > > > As you may know, Caitlin has been participating in > a clinical > trial for a new Il-1 targeting biologic for almost two > years. The > drug is being developed by Regeneron. It's similar to > kineret, so > it's supposed to be great for systemic kids - and it's a once a > week > shot as opposed to daily.> > > > I haven't been 100 percent sold on the drug's > benefits - mainly > because we have had some ups and downs, and because Caitlin has > experienced some drug free remission prior to starting the > study. So > it's hard to be certain whether the drug has turned things > around, or > whether they were already in motion. It wasn't the > overnight change > you sometimes hear about with systemic kids and kineret. > For the > first six months of the drug, she was still in a flare and still > had > to take pred. Then we had six stable months, no pred > etc. Then, a > flare last May which required pred. SInce September of > 2007 though, > she has been quite stable. She's only been on MTX and the > study > drug. Her labs have been normal, and all of her joints > have been > great!! We have been so happy, that I certainly would have > NEVER > chosen to take the risk of stopping the drug, when we have so > few > options left to try.> > > > Unfortunately, we were given no choice. The > company halted the > study suddenly - not for reasons of bad side effects - but > primarily > for financial reasons. I guess we are not considered a big > enough > market. And worse news - I am told that they will not be > filing for > FDA approval for RA or JRA. It is already approved for a > periodic > fever syndrome and should be available on the market ... but > getting > the drug would mean that insurance companies would have to > approve an > off-label prescription. We were called in this week, and > asked to > turn over all of the remaining drug.> > > > And now for the best part - My doc has been trying > to work with > my insurance company to prime them for approving the drug when > they > finally have it available (he went all the way up to the medical > director before he found somebody who had even heard of the > drug). > He's been working with the drug company to find out when they > will be > mass manufacturing etc..... and then the drug company dropped > the > bomb. They plan on marketing this drug at $200,000 A > YEAR!!!!!!!!!! > I am not kidding. Even IF our insurance miraculously > approved it, we > couldn't do it because of Caitlin's lifetime insurance cap of > $1million. With that stupid drug, we'd hit it in a few years!!!!> > > > I hope this turns out to be a BIG > misunderstanding... and that > somehow the drug company will actually pull through and do the > right > thing. As for us, we participated in the study in the > hopes of > helping find something that would help my daughter and other > systemic > kids. I know there are plenty of parents who go through a > daily > trauma trying to give the very painful kineret shots... and I > was so > hopeful that this alternative would really improve lives.> > > > I'm not sorry we did it. But now we are off > the drug cold turkey > at the very month that Caitlin has flared the last two > years. I'm > praying we make it through the month (and the summer!) with the > same > great health she has been blessed with since September.> > > > Even though I am resigned - I'm still pretty > ticked. And as soon > as our walk is over, I'm throwing some energy at getting all of > this > incredible BS confirmed in writing - and then I'll be knocking > on the > news people's doors. With those kind of crazy numbers, in > this bad > economy, with the health care crisis we are in - I am hoping > somebody > will pick up the story!!!!!> > > > Best hopes to all-> > > > Colleen - mom to Caitlin, 10, systemic> > > > > > ---------------------------------> > Be a better friend, newshound, and know-it-all with > Mobile. > Try it now.> >> > >

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Colleen, I am so sorry to hear that news about your drug company and the Il-1. I hope some development comes through and you can get it for Caitlin cheaper. Good to hear that she is doing well lately and hope that continues. We heard about that drug becoming available now for purchase and have considered it for our son Louis,but at that price?....Our best to your family and great work on the walk. You inspire us all. Jan and Louis, age 9, systemicOn May 1, 2008, at 2:51 PM, Jo & Grant wrote:Colleen, I'm with everyone else on this one- take it to your electedrepresentative and get in touch with your friendly media contact too.And if the media gets in touch with your elected rep, they can oftenconvince them to do more about it! LOLBut I agree, what the drug company did is WRONG- fancy getting hopes upwith a drug that actually seems to work, then rip the rug out from underyou. And how unrealistic is that price?? OMG!Sending you and Caitlin gentle genuine Aussie hugs.JoBayly, 6, extended oligo>> Hi all->> As you may know, Caitlin has been participating in a clinical trialfor a new Il-1 targeting biologic for almost two years. The drug isbeing developed by Regeneron. It's similar to kineret, so it's supposedto be great for systemic kids - and it's a once a week shot as opposedto daily.>> I haven't been 100 percent sold on the drug's benefits - mainlybecause we have had some ups and downs, and because Caitlin hasexperienced some drug free remission prior to starting the study. Soit's hard to be certain whether the drug has turned things around, orwhether they were already in motion. It wasn't the overnight change yousometimes hear about with systemic kids and kineret. For the first sixmonths of the drug, she was still in a flare and still had to take pred.Then we had six stable months, no pred etc. Then, a flare last May whichrequired pred. SInce September of 2007 though, she has been quitestable. She's only been on MTX and the study drug. Her labs have beennormal, and all of her joints have been great!! We have been so happy,that I certainly would have NEVER chosen to take the risk of stoppingthe drug, when we have so few options left to try.>> Unfortunately, we were given no choice. The company halted the studysuddenly - not for reasons of bad side effects - but primarily forfinancial reasons. I guess we are not considered a big enough market.And worse news - I am told that they will not be filing for FDA approvalfor RA or JRA. It is already approved for a periodic fever syndrome andshould be available on the market ... but getting the drug would meanthat insurance companies would have to approve an off-labelprescription. We were called in this week, and asked to turn over all ofthe remaining drug.>> And now for the best part - My doc has been trying to work with myinsurance company to prime them for approving the drug when they finallyhave it available (he went all the way up to the medical director beforehe found somebody who had even heard of the drug). He's been workingwith the drug company to find out when they will be mass manufacturingetc..... and then the drug company dropped the bomb. They plan onmarketing this drug at $200,000 A YEAR!!!!!!!!!! I am not kidding. EvenIF our insurance miraculously approved it, we couldn't do it because ofCaitlin's lifetime insurance cap of $1million. With that stupid drug,we'd hit it in a few years!!!!>> I hope this turns out to be a BIG misunderstanding... and that somehowthe drug company will actually pull through and do the right thing. Asfor us, we participated in the study in the hopes of helping findsomething that would help my daughter and other systemic kids. I knowthere are plenty of parents who go through a daily trauma trying to givethe very painful kineret shots... and I was so hopeful that thisalternative would really improve lives.>> I'm not sorry we did it. But now we are off the drug cold turkey atthe very month that Caitlin has flared the last two years. I'm prayingwe make it through the month (and the summer!) with the same greathealth she has been blessed with since September.>> Even though I am resigned - I'm still pretty ticked. And as soon asour walk is over, I'm throwing some energy at getting all of thisincredible BS confirmed in writing - and then I'll be knocking on thenews people's doors. With those kind of crazy numbers, in this badeconomy, with the health care crisis we are in - I am hoping somebodywill pick up the story!!!!!>> Best hopes to all->> Colleen - mom to Caitlin, 10, systemic=