So, to be quite honest, the uncertainty of how this would ever work in our current drug model is one of those haunting fears that keeps me up nights. Correct me if I am wrong, but the current trial on exon 51 is because the 15% of boys that can be helped from that is by far the highest percentage of any mutation (and that in a rare disease). The only way I could see this working is if a company developed a menu of all the compounds and was able to use them to address individual mutations. These can't be considered drugs in the conventional sense because each child presents a different need.
I go back to my original question, what advocacy is being done currently, and what groundwork can we lay to help the process move forward once the 51 trial is complete? It doesn't feel right to just wait and see.
Keith, you're probably right that going to Europe to get expensive treatments is not a realistic option...I was thinking of it as a band-aid until the FDA approved the therapies. Isn't it outrageous, though, that we are having this discussion in the allegedly most medically advanced country in the world?
Ofelia, I am not terribly concerned about insurance covering a proven therapy - they really would have little choice. My wife Tonya recently completed a pretty customized 18 month treatment for breast cancer, and it was covered - I don't even want to think about how much it cost. The challenge, I think, is twofold. First, proving the therapy (which I hope they are doing) and then advocating in such a way to make it clear that it is a moral imperative that we treat all the boys that we can regardless how rare their mutations are.
The thing that scares me is wondering how this can happen quickly enough that the individuals we are talking about (our sons) will benefit, and all of this doesn't take another 20 years to fall into place.
Ofelia Marin said:I'm not sure about moving to EU either. I assume it might be possible to buy it from there (Prosensa's requires sub-cut injections) if you have a prescription from a neuro from here and there(?). Of course, the high price needs to be paid unless you are citizens of one of those countries where a high % of the price is paid by state (?).
I do however have a related question. If this becomes an FDA approved therapy, what are the chances that insurance covers something as expensive? Keep in mind that, hopefully, at some point in the future, more drugs can be approved for DMD and a cocktail of drugs can be the solution for slowing the disease progression. Will the insurance cover 100K+/year?
Keith Van Houten said:Couple folks mentioning in this thread their willingness to get an exon skipping therapy overseas if need be.
A tidbit I picked up in the web conference with a Leiden researcher last month that I hadn't heard before you may be interested in...
If exon skipping ever became a therapy - which is still a significant "if" at this point - the work done thus far has shown that the effect only lasts about 3 months. You'd need repeated treatments for a lifetime.
So, if it's not FDA approved, you're talking about scenarios like frequent travel, or permanent relocation, until and if it becomes available in an oral form. You're also probably talking about paying for it yourself. The cost for this is going to be astronomical. I've read estimates of over $100k a year. For life. Keep in mind that GSK just agreed to pay $650 million if this work results in a therapy for only 4 exons. It's going to be very expensive in order to recoup that. If you travel to get a non-FDA approved therapy, your insurance in the US is not going to cover it - right? If you emigrate to a Western European country with socialized health care - are they allow an immigrant family to enter with a catastrophic illness, and cover the cost? I haven't researched it, but I'd doubt it. If so, why wouldn't every critically ill person with no insurance just move?
So - why do I bring this up?
First off, I've put a lot of thought into this subject of potentially moving overseas myself, and I wanted to see if anybody can pick any holes in my thought process.
Second, quick FDA approval is critical. Once and if it's been established through the trials that exon skipping is a viable therapy - which again, it has not been proven, a fact no one should lose sight of - the laser-like focus of advocacy should be to gain FDA approval as quickly as possible. For the reasons I cited above, I don't think that getting this therapy in another country is a viable alternative for most of us. We need it approved for use in the US. FDA approval means not only that it's available here, but that this very expensive potential therapy could potentially be covered by insurance.
As far as advocacy with FDA, I know that PPMD has an ongoing relationship with FDA. Tim Cote spoke at the the Advocacy Conference two years ago. I agree that the relationship needs to be nurtured, and FDA needs to know that there's a large group of people standing behind the effort, ready to make a lot of noise if the program doesn't move at the pace it deserves. I thought that Mr. Cote was very sincere, and he certainly seemed like he would be an ally.
I think GSK's involvement is going to help with the approval also. Big pharma is awfully connected, and they work the system on a daily basis. I believe GSK doesn't pony up the money until after a treatment is approved, but I'd assume they want it approved as soon as possible, since they think they can make money on it.
I believe that the insurance coverage of any therapy that's developed is an important issue to us as the development of the treatment itself. How cruel a world would it be if a treatment is found, but few can afford it?
These are the kinds of things that keep me up at night, especially as they relate to the health care reform debate happening in Congress. It's obviously a controversial subject, but I hope that if nothing else, we wind up eliminating the consideration of pre-existing conditions, and the elimination of lifetime maximums. Some insurers already have lifetime maximums, and as costs continue to escalate, I expect them to become more and more common. MD families could run into this, even if exon skipping never becomes a reality.
To address Steve's point about the development of therapies beyond the first 4 exons. I asked specifically about that in the web conference. It totally depends on the degree of clinical trials that are required, but the business case beyond those 4 starts to deteriorate quickly, because the rate of occurrence drops off so rapidly. That's going to substantially impact a private company's interest in funding it. We talked about options like advocacy groups funding development and trials beyond the first 4. Again, though, it totally depends on the clinical trial requirements for additional exons. If limited trials are required, it increases the likelihood that we'd get more exons covered. Keep in mind also that not all mutations are even potentials for exon skipping - mutations in the regions near the beginning and end of the gene cannot be skipped, and it's a question as to what the effectiveness of skipping will be for all exons. We already know that not all in-frame deletions produce BMD. So, even in a best case scenario, this won't work for everyone. Oh - and while it'd be a terrific therapy and welcomed by all, it's not a cure for anyone, so the fight isn't over...