It's been almost a year since Gus was diagnosed with DMD. On the drive into work this morning, I was thinking about what I've learned about DMD since that November evening in 2009 when the doctor from Dartmouth called.
It comes down to four things:
That's where John Crowley, comes in. In 2003, Crowley, whose kids had Pompe disease (an even more rare genetic disorder than DMD - about 1/400,000 births - or 10 cases each year in the US ) created a treatment at Genzyme. He also created an economic model that makes sense to big pharma. The answer to the economics question? Charge A LOT - about $200,000 annually per patient to treat Pompe.
I'm no mathematician, but, based on those numbers, I think we're looking at about $20,000/year to treat Gus when a treatment comes along. When that time comes, we'll happily pay $20,000. At the same time, we can't fund a drug through trial.. How much does it cost to get a drug through all three phases of an FDA trial? I've posed that question to doctors and execs over the months, and the consensus seems to be about $20,000,000. Only Bill Gates or drug companies have $20 mil to spend on trials (are you listening Bill Gates?).
So what's our role?
We fund the research and the treatments. We find the doctors who are figuring out ways to improve kids lives and fund them. We find researchers who have research that's so promising that big pharma can't ignore it and fund them. We stay up later and get up earlier in order to make the single dollar that will break through it all.
In the end, we ignore the economics and we believe that this is the generation of boys who, with our help, will beat DMD. There are 38,000 kids each year who are counting on us. We can't let them down.
Comment
Hi Keith -
I have no doubt that you're right - my number was based solely on John Crowley's number divided by 10 - far too simplistic, I know.
In the end, I believe that even at the $200,000 level, we would fund it - in the pantheon of patients, our young sons, with all their potential, are a pretty compelling bunch. As you imply, much of the rest of the world would (or could) not fund that level, however...so. what does that mean?
I was unaware of the billion dollar price-tag on drug approval - but I believe you. I think it's a number that's hard to pin down.
Thanks for reading and I hope you and your family are having a terrific and blessed holiday season!
I've heard numbers for drug development and approval more in the range of a billion dollars.
And I think a treatment is likely to cost a lot more than $20k a year. More like $200k a year. GSK has an agreement to pay $660 million for the rights to market 4 exon skipping drugs that cover about 1/3 of the DMD population. Most of world is not going to be able to afford a drug of that cost - leaving the cost to be borne by those with private insurance or national health care. You could do a little financial analysis using the number of patients, the $660 million, and a reasonable payback period (like before the drug patent expires) - I think you'll find the number to be astronomical. This is one of the reasons I was so in favor of health care reform in the US - no annual or lifetime maximums.
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