Here it is: The mining of rare diseases. It's lengthy but intriguing, especially for those of us who've participated in drug trials and who have to pin the hopes for our kids' futures on the work these drug companies are doing.
The article says that "... over the years, its (the Orphan Drug Act's) good intentions have been subverted by the pharmaceutical industry, which has increasingly found ways to exploit this once-obscure health-care niche, transforming it into a multibillion-dollar enterprise."
The article talks about a 10 year old girl named Violet who was diagnosed with a rare, inoperable brain tumor. The only sliver of possibility for her was a $50,000 drug that might, might, help her live a month or so longer. No guarantees.
That's more money than her parents made in a year.
What to do? They had to try it. Whether or not Violet lived a month longer than she would have without the drug, no one will ever know. At any rate, they had to take the gamble and try it.
As it turned out, miracles came through and a couple of different charity groups helped them fund the treatment.
The article tells stories of several people with different rare ailments whose "only hope" appeared to be an Orphan Drug sometimes still in trials. These people and their families and friends spent years and raised millions of dollars to fund drug testing, based on the olive branches of hope handed out by the pharmaceutical companies.
And after years of clinical testing and initial (and by initial, I mean after a decade or more of testing) showed promise, lots of these drugs were ultimately not worth a dime and were even potentially harmful.
But we, the parents of children with rare diseases, have no other option but to trust the drug companies, the doctors working for them, and the trial results we are presented with. We are constantly shown how far medical research has come in the past couple of decades, how much more we know about the body and how it works, and how it gives us real hope for our kids' futures. We grab that dangling vine of hope and scramble to ascend it, to save our kids.
I'm starting to feel a little duped. They don't really know that much about these drugs before they start advertising how miraculous they are going to be. It sounds like they are just telling us what we need to hear, so that we'll get excited and desperate and raise funds and pay them to do the research.
And that gets us parents working on fundraising, doesn't it? The drug company says the only thing standing between our children and their good health is money. If only we can make it.
So we work our butts off holding fundraiser after fundraiser. So they can perform clinical tests and work toward FDA approval, as fast as possible, because our children are running out of time.
Nobody tells the drug companies how much they can charge us for the medications we raised funds for them to develop.
"Britain does it. So do France, Germany and Canada. The only major industrialized country that doesn’t regulate the cost of prescription drugs is America.
Here, prices reflect what the market will bear. That’s dictated largely by insurance carriers, which negotiate reimbursements, and Medicare, which pays for essential treatment no matter the cost."
Sounds like those drug companies need to have a little more regulation.
Violet's parents moved mountains to get her the medication that had been studied for 16 years in clinical trials, that would at best only prolong her life a month, but they did it. Violet passed away in her sleep in 2012. Then her parents found out this:
"But it wasn’t until this year that a comprehensive trial with more than 600 patients — the drug’s first double-blind study for newly diagnosed brain cancer — found no difference in survival between those who got the drug and those given a placebo."
O. M. G.
I haven't decided yet, whether or how this changes how I feel about the termination of the STX209 drug trial. I'd love to know what some others think. Comments welcome.