Are their any open clinical trials for deletion 22-41?

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Hi Ofelia,

 I guess I wasn't very clear with my thought process, sorry.  I wasn't thinking organizations should front money to treat individual boys.  I was thinking money should be spent to start communications with GSK and the National Health/Health Ministry in the UK on the subject of helping duchenne boys from the US obtain treatments until FDA comes through for us. 

Allied countries spend billions working together to kill enemies, maintain cultural elements, rebuild after wars or provide relief in a natural disaster.  I'd rather not automatically assume disease remain ignored simply because it's never been done before.   Our world has become more and more connected via new technology and medicine.  I think someone should make room for this issue to be addressed on a global scale so that those living too many miles from treatment don't die waiting.     

 

Ofelia Marin said:

Cheryl, I am talking about US approval o/w the majority of us do not afford to treat our sons. If exon skipping is approved in Europe one would need hundreds of thousands of dollars for one year of treatment and a doctor in the US willing to dose thier son weekly or every other week. Finding a doctor to inject the drug would probably be much easier than finding the money to pay for the drug. I do not think the DMD foundations would be able to cover the price of the drug, if they do for one boy they would need to do it for all which means millions/year.

cheryl cliff said:

Of course to me it appears as though you all are referring to regulatory approval here in the states only (?).  I do believe approvals are going to take place in other parts of the world well before it happens here, cause it's going that way right now.  Therefore, it becomes necessary to formulate plans to take boys from the US to places where meds are available.  I realize many think this approach is nuts, but it has always been my opinion that nuts is still better than dead. 

 

It would be good that organizations such as PPMD and all the others begin formulating plans now on how they can assist US families with this and soon.  If organizations can't/won't help make this happen, then it is left to us as parents. 

 

Any thoughts?

I dont know how that works Cheryl...I never heard about something like this being done. Until FDA approves it, the only options I know are compasionate use or buying the drug from Europe or other countries where it's approved. In both cases we would need to pay for treatment.

Perhaps somebody needs to pay for treatment, yes, but maybe contributions from several sources such as organizations, governments and pharma's could handle this in a coordinated way.  If contributions and some initial profit cutting can be made available to families for at least the first couple of years, it would make a huge difference between who lives and who dies.   Not to mention make international PR a breeze for big pharma who will most likely spend tons on advertisments anyway.  How bad can saving children be for a GSK no matter the cost?

 

We've already seen worldwide organizations work with governmental agencies and private industries to bring relief to people when bad things happen.  That's what the Red Cross does to mention one.  And on a much larger (more expensive) scale than what our small population requires.   We don't need to re-invent the wheel but perhaps take other organizational examples, ditto that and point it at DMD.  Also, PPMD is in a prime spot to spear-head this effort since they are already internationally connected and very involved with US government, due to Pat's relentless and hard work. 

Exon skipping is new to the world of duchenne.  Getting it to our children requires thinking outside the box.   

 

 

 

 

 

 

 

Hi Cheryl and Ofelia, 

 

Back to Leeandra's original question - will Exon skipping help her boy - or mine?  We all know that skipping Exon 51 or 44 will only help a relatively small percentage of boys, so how can we come together as a community and move ALL of the science forward to help ALL of the boys?  

I like your thinking, Cheryl, but I don't quite understand what you mean - in the world of HMO's how does international cooperation play a part - or how do we MAKE it play a part (I must admit, after seeing Sicko by Michael Moore, I wanted to move to France, but...)?  

Ofelia's right, drug approval takes too long (and costs too much) when you are dealing with a disease that gives you NO time.  So what do we do?    I think this discussion has come to the crux of every parents pain/quandary - I want treatment NOW, what do I have to do to get it?

im not focused on exon skipping so much for my son, i understand it may be years....what about ACE-031? any news on US trial for our boys?

Leeandra, I would also like to know what happens with the announced "soon" to take place ACE-031 trial...last year everyone was so excited about it coming to the US Q1 2011, then we did not hear much at all. 

 

Steve, your questions are great. I do not see us being able to get a "treatment" soon unless our sons take part in a successful trial and at the end of the trial that company keeps the boys on the drug until market approval. The boys can be in the placebo group during the trial, so we are still looking at a few years until they have access to the drug. To me, this is the only way to have access to some treatment in the near future. For example, in the AVI exon 51 dosing trial planned to start this summer in Columbus, they will have 8 boys on the drug for 24 weeks, then they promissed to continue to have these boys in a pivotal trial after the 24 weeks. Not sure how exactly this will work out. They will also have a pivotal placebo controlled trial including more boys planned to start in 2012. So if we add time for enrollment, 48-week trial, analysis of the data, this will get us to 2013-2014 for exon 51 trial completion...


Hi Steve,

To be more concise and hopefully answer your question my thinking, such as it is, relates to not using anybody's insurance company, PPO or HMO.  International cooperation with the governments who will approve exon skipping well and before the US, combined with non-profits in the DMD world and perhaps others, and some modification by GSK on their pricing could work together to solve the problem of cost and access to meds.  We have to wait a couple of years minimally for skipping on 51.  While we wait we should be doing something to bring organizations and elements together to make this happen.  It should be starting from PPMD, in my opinion, since they are already poised for this. 

 

MDA makes loads of money from putting our sons in front of a camera annually.  They could pony up some funds to make this happen.  Other non-profits should be approached to contribute as well, from the DMD world and outside - Red Cross, Salvation Army, MDA organizations in other countries....  

 

While contributions are being collected and earmarked for treatment - PPMD should begin communicating with our government as well as UK's NHS, France's  Ministry of Health and so on...  And someone appropriate should be selected to speak with GSK.  Negotiations to lower initial costs of skipping could begin now, with the promise of this all ending at a certain point when the FDA approves skipping so GSK can then change their profit margins with the inclusion of insurance from USA patients.  Really, it might not be that difficult to convince GSK since their profit only relates to how many patients they can sell treatments to.  If we increase their target population a few years earlier they might not object to being generous.  Plus, there is the option to use this generosity in ads internationally. 

 

If we don't think outside the box, as they say, more will die while waiting for FDA approval while we watch other families, god bless them, treat their sons.  Just because we don't have socialized medicine here in the states (yet?) shouldn't be a reason to allow this to happen. 

Cheryl, I think we need to look at other diseases, if this did not happen for any other terminal disease before will not happen for DMD. DMD is not going to be treated any differently. 

 

My hope is that FDA approves start of GSK trials in the US as soon as possible. Same with ACE-031. Does anyone know the status of the Canadian ACE-031 trial? Were they able to enroll boys in all cohorts? What is the estimated completion time for that?

I agree on the timing, Ofelia.

 

I worry about a scenario where we have an approved treatment available overseas - but we in the US can't access it because the cost is too high to pay for it yourself and insurance won't cover it because it's not FDA approved.  Compound that with you'll have some boys in the US will have access as part of the trial or continuation.

 

Think about psychologically what that'll do to parents and patients.  A treatment is out there - but YOU can't get it.  

 

No different than the 100,000 people a year that die of cholera, I guess.  But not something I think we've had to deal with in the US.  

 

I would not underestimate the power of money in all of this.  I think it works in our favor in this case (at least for the common skips)  These companies want access to this market.  Badly.  Our badly broken health care system makes this the most profitable market in the world for them.  I believe that they will be moving heaven and earth to get in here as soon as they can.  Especially when there are competing products on the horizon.  They need this market.  

 

Ofelia Marin said:

Keith, I think that 2 years+2 years is very optimistic unfortunately. I do not even see skipping 51 approved in 2 years. It takes 1-2 years AFTER the clinical trial including analysis of data is completed to obtain FDA approval (IF the results of the analysis look good). I would estimate exon 51 hits the market in the US in ~3-4 years if not more, assuming the results from the pivotal trials are good.

Best scenario probably for the boys participating in the trials, after the trials are completed, if the pharma companies continue to provide the drug at no cost before market approval... But it's going to be a LONG way until then

 

 

 

And so if GSK will be moving heaven and earth to get into the US market, why not help them with the caveat that US boys obtain a discounted treatment until FDA approval? 

 

Usually I agree with what you think Ofelia because I admire your intelligence.  But with respect, I disagree on your concept that because it hasn't been done before - it can't be done.  I believe the contrary.  We live in a world of ever changing research, medicine, science, technology.  These changes come about faster and faster than ever before.  New ideas, new politics and laws must become part of those changes.   

 

The world of DMD has never before seen the upcoming opportunities about to take place, ever.  As part of that community it is up to us to contribute to making those changes work to the benefit of our sons.   Otherwise we are left powerless while watching American children die.  We all should refuse to remain powerless.   



Keith Van Houten said:

I agree on the timing, Ofelia.

 

I worry about a scenario where we have an approved treatment available overseas - but we in the US can't access it because the cost is too high and insurance won't cover it because it's not FDA approved.  Compound that with you'll have some boys that will have access as part of the trial or continuation.

 

Think about psychologically what that'll do to parents and patients.  A treatment is out there - but YOU can't get it.  

 

No different than the 100,000 people a year that die of cholera, I guess.  But not something I think we've had to deal with in the US.  

 

I would not underestimate the power of money in all of this.  I think it works in our favor in this case (at least for the common skips)  These companies want access to this market.  Badly.  Our badly broken health care system makes this the most profitable market in the world for them.  I believe that they will be moving heaven and earth to get in here as soon as they can.  Especially when there are competing products on the horizon.  They need this market.  

 

Ofelia Marin said:

Keith, I think that 2 years+2 years is very optimistic unfortunately. I do not even see skipping 51 approved in 2 years. It takes 1-2 years AFTER the clinical trial including analysis of data is completed to obtain FDA approval (IF the results of the analysis look good). I would estimate exon 51 hits the market in the US in ~3-4 years if not more, assuming the results from the pivotal trials are good.

Best scenario probably for the boys participating in the trials, after the trials are completed, if the pharma companies continue to provide the drug at no cost before market approval... But it's going to be a LONG way until then

 

 

 

Completely agree Keith. I think that's a probable scenario unfortunately.

Keith Van Houten said:

I agree on the timing, Ofelia.

 

I worry about a scenario where we have an approved treatment available overseas - but we in the US can't access it because the cost is too high to pay for it yourself and insurance won't cover it because it's not FDA approved.  Compound that with you'll have some boys in the US will have access as part of the trial or continuation.

 

Think about psychologically what that'll do to parents and patients.  A treatment is out there - but YOU can't get it.  

 

No different than the 100,000 people a year that die of cholera, I guess.  But not something I think we've had to deal with in the US.  

 

I would not underestimate the power of money in all of this.  I think it works in our favor in this case (at least for the common skips)  These companies want access to this market.  Badly.  Our badly broken health care system makes this the most profitable market in the world for them.  I believe that they will be moving heaven and earth to get in here as soon as they can.  Especially when there are competing products on the horizon.  They need this market.  

 

Ofelia Marin said:

Keith, I think that 2 years+2 years is very optimistic unfortunately. I do not even see skipping 51 approved in 2 years. It takes 1-2 years AFTER the clinical trial including analysis of data is completed to obtain FDA approval (IF the results of the analysis look good). I would estimate exon 51 hits the market in the US in ~3-4 years if not more, assuming the results from the pivotal trials are good.

Best scenario probably for the boys participating in the trials, after the trials are completed, if the pharma companies continue to provide the drug at no cost before market approval... But it's going to be a LONG way until then

 

 

 

I do not say that it cannot be done. I say that we should look at other terminal diseases where/if this situation took place and learn from what they were able to do or not so we do not waste precious time. Was it done before? I believe all parents/patients would think about this and fight to have access to treatments approved outside US...were they successful? If yes, we should approach it similarly. If not we should think at other options. Are companies like GSK allowed to sell the drug to US patients if only approved in EU? Not sure how this works but we need to find out.

 

I believe that if something will be approved in EU it will be a matter of time until FDA approves it, 1-2 year difference I do not know. It is not a huge time difference b/w AVI and GSK's exon 51. AVI will probably move to the next exon soon and then try to discus platform approval. I am not sure how this will all work out but we first need to see if this treatment works. The results obtained so far are positive however these are small trials, we need to see what hapens when 80+ boys are included in a placebo controlled trial. I think that there is a lot more work to be done until people will be talking about approvals unfortunately. At the moment, we should focus our energy on bringing these trials (GSK, Shire/Acceleron) in the US. Just my two cents.


cheryl cliff said:

And so if GSK will be moving heaven and earth to get into the US market, why not help them with the caveat that US boys obtain a discounted treatment until FDA approval? 

 

Usually I agree with what you think Ofelia because I admire your intelligence.  But with respect, I disagree on your concept that because it hasn't been done before - it can't be done.  I believe the contrary.  We live in a world of ever changing research, medicine, science, technology.  These changes come about faster and faster than ever before.  New ideas, new politics and laws must become part of those changes.   

 

The world of DMD has never before seen the upcoming opportunities about to take place, ever.  As part of that community it is up to us to contribute to making those changes work to the benefit of our sons.   Otherwise we are left powerless while watching American children die.  We all should refuse to remain powerless.   



Keith Van Houten said:

I agree on the timing, Ofelia.

 

I worry about a scenario where we have an approved treatment available overseas - but we in the US can't access it because the cost is too high and insurance won't cover it because it's not FDA approved.  Compound that with you'll have some boys that will have access as part of the trial or continuation.

 

Think about psychologically what that'll do to parents and patients.  A treatment is out there - but YOU can't get it.  

 

No different than the 100,000 people a year that die of cholera, I guess.  But not something I think we've had to deal with in the US.  

 

I would not underestimate the power of money in all of this.  I think it works in our favor in this case (at least for the common skips)  These companies want access to this market.  Badly.  Our badly broken health care system makes this the most profitable market in the world for them.  I believe that they will be moving heaven and earth to get in here as soon as they can.  Especially when there are competing products on the horizon.  They need this market.  

 

Ofelia Marin said:

Keith, I think that 2 years+2 years is very optimistic unfortunately. I do not even see skipping 51 approved in 2 years. It takes 1-2 years AFTER the clinical trial including analysis of data is completed to obtain FDA approval (IF the results of the analysis look good). I would estimate exon 51 hits the market in the US in ~3-4 years if not more, assuming the results from the pivotal trials are good.

Best scenario probably for the boys participating in the trials, after the trials are completed, if the pharma companies continue to provide the drug at no cost before market approval... But it's going to be a LONG way until then

 

 

 

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