Sunday, November 17, 2013

The Orphan Drug Act and Expliotation by Drug Companies

This morning I noticed an article in our Sunday paper about how drug companies are profiting from the Orphan Drug Act.  It caught my eye, having put my own children through a drug study for what would have been an orphan drug, had it survived the testing process.  I was curious.

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.

No comments:

Related Posts with Thumbnails

Labels

#youmightbeanautismparentif 2012 in review 9/11 memories ABA therapy Acceptance acronyms advocacy affection aggression AJ Aliza Aliza the playwright All I really need to know... Alphabitty Moments American Girl Ann Coulter antibullying anxiety anxiety in parents of children with special needs apple orchard apps for autism AppSmitten Arbaclofen Arbitrary Thoughts ARC autism autism brushing autism portrayed in TV shows Autism Shines awareness backyard band baseball bath toys beds behavior problems being tall Birthday Boys biting blog change blog hop blogging books bottles brushing bubbles Burnsville Fire Muster bus Cabin Fever in Minnesota candy Carly Fleischmann Carly's Voice cats cats and dogs chewys Chicago childcare for special needs children childhood Children's Museum chocolate Christmas Church circumin clinical trials Clonidine CNN Hero of 2011 coffee communication comparisons computer Conference cost of special education Courage Center Curcumin daddy dance dance competition dance moms Dental surgery dentist developmental milestones diagnosis diapers Diego Disability Day dogs Dolphin Tale Doomsday Preparation Dora Doritos drug trials DVD player early intervention earrings Easter ECSE Parent Retreat electronic gadgets electronics Everything I need to know... Evil Overlord fall falling asleep at school families family fashion fear Featured Feel Good Friday field trip fireworks first day of school Flash Gordon Food Chronicles food issues in Fragile X and autistic children forms forts Fragile Face of God Fragile X Fragile X advocate Fragile X and autism Fragile X Awareness Day Fragile X carriers Fragile X in the news Fragile X presentation Fragile X statistics Fragile X Writers friends fundraiser for Fragile X funniest Funny Gabrielle Giffords Galveston games getting carsick Girls' Night Out Giving Spirit glasses global warming going home Good Morning Great Quotes guest blogs guest post haircuts Halloween hearing test Heaven is for Real hippotherapy holidays Holland Holly home life homework hotel hugging human behavior hyperactivity IEP Meeting IEPs in the news inclusion inspiration integration iPad iPad apps iPad apps for autism IQ testing Jack Jablonski January First Joke journal entry kids with Fragile X and animals Kindergarten Kindle kisses language study learning to talk leaves lemonade stand Lily Little Einsteins losing teeth Mad Gab makeup mall Mall of America marcia braden McDonalds media sensationalization medications Melatonin Miami MIND Institute Minnesota Bloggers Conference minocycline Miracle League monkeys mosquito bites Mother's Day movies MVMOM Used Clothing and Equipment Sale nail trimming names naughtiness neighbors nicknames nightmares normal off topic one thing leads to another online dating Operation Beautiful oral sensory orphan drug act other bloggers Our Wedding outside overstimulation panic attacks parade parental stress Parenthood park Partners in Policymaking penicillin people with disabilities pets pharmacy fun photography Photoshop picnic Pictures pinching pink shirt Pinterest playing outside playing with toys poem politics poop potty training Presents protecting autistic children rash reading to kids research Retreat riding a bike Robin Williams Roger Ebert routine RSS feed RUSH University San Diego Sandy Hook Elementary Santa schedules school school bus school notes school pictures screaming self image self-checkouts sensory Seroquel siblings with developmental delays sick kids sippy cups sleep smile snow pictures Snowstorm social situations speaking of the unspeakable special education special education evaluation special needs kids special needs parents Special Needs Ryan Gosling Special Olympics spelling spoon feeding spring break staying positive stimming Strep STX209 Stylish Blog Award suicide summer Sunday School Sundays sunshine survival mode swimming talking talking to kindergarteners Target teacher's aides Teeth brushing Tegretol Temple Grandin Ten Commandments textbook case of Fragile X thankful thanksgiving that window/mirror thing The Autism Store The R Word the rapid passage of time The Right Things to say to parents of special needs children The Santa Experience the Shedd Aquarium The Twin Thing The Wiggles therapeutic horseback riding therapy This is Autism topless trampoline traveling with special needs children TV twins with special needs Twitter typical Fragile X characteristics typical kids typing vacation Vacation Bible School video games videos volunteering Waisman Center water play way-back-Wednesday What I've Learned What's your song? when a special needs parent dies Wiggles Wii games Winner Winner Chicken Dinner winter wonder Wonder Pets Wordful Wednesday Wordless Wednesday Words of Wisdom World Autism Awareness Day YMCA You Tube Zack Zoloft zoo animals

Fragile X Blogs