You all know how hard our rare disease community has been working on getting this language added to PDUFA:
"Considerations. – In developing the guidance . . . . the Secretary shall consider . . . . for drugs designated for a rare disease or condition under section 526 of the Federal, Food, Drug, and Cosmetic Act; and (2)how to incorporate novel approaches to the review of surrogate endpoints based on pathophysiologic and pharmacologic evidence in such guidance, especially in instances where the low prevalence of a disease renders the existence or collection of other types of data unlikely or impractical"
PDUFA- Prescription Drug User Free Act. I don’t expect the vast majority of our supporters to know what PDFUA is, I had no clue 6mo ago. I’m venting today and at the risk of loosing our supporters interest, I won’t bore you with the details. In short every five years congress can add legislation to PDUFA. We’re trying to add the above language so that we can save this generation of children.
Surrogate endpoint...It’s just another way to measure an outcome: “A measure of effect of a certain treatment that may correlate with a real clinical endpoint."
In most cases of ultra-rare diseases we’re not able to collect the clinical data. To put it bluntly we don’t have a large enough patient population and our kids don’t live long enough. In summary, grass-root organizations like JJB are driving the science to save our kids all on our own.
The moment that one of our treatments comes out of the lab and is ready to be approved by the FDA... BAM we hit a brick wall. I’d like to spray paint that wall: “The FDA and big pharma are actively preventing us from saving Jonah.”
Big Pharma is nervous! They don’t want any more competition. Pharma is pressuring the Energy and Commerce committee to not add our language. They’re afraid that once the the FDA starts working for us (the people) that small biotech's like “Phoinix Nest” will be able to research and develop drugs without their help. Cutting them out of huge profits. For us it’s not about the profit it’s about saving our kids. The whole thing just makes me sick to my stomach.
Anyhow, despite the rare disease communities best efforts, we still might not win the battle this time. David vs Goliath.... The Davids will never give up.
So we thank our friends and families and our representatives: Ed Towns and Cliff Stearns who stood up for us and made our voices heard to the Energy & Commerce committee. If you all could take a moment and give Sterns and Towns props for us we'd greatly appreciate it. Our country needs to know that there are some good politicians doing the right thing for their constituents.
https://www.facebook.com/EdTowns and https://www.facebook.com/RepCliffStearns
For those of you with twitter accounts please give them props and be sure to use the hashtags: #FAST, #PhRMA #PDUFA, #Ed Towns, #Cliff Stearns.
I say insult to energy because... In a few minutes we're going to wake up Jonah and take him to the hospital. We have a 6:30am appointment for minor surgery, we’re having the tubes replaced in his ears (they fall out every two years.)
Jonah has had a sinus infection for over a month, our doctors realized that Jonah’s body is resistant to the antibiotics. The solution for getting rid of the sinus infection... 2 direct shots of a more powerful antibiotic in his butt and thighs, everyday for 5 days.
Yesterday was day 4. When Jonah realized that we were going to see the doctor. He furrowed his brow and wagged his finger at me: “No Mom, No Hurt, No doctor.”
The tears streamed down my face. The nurse and I had to hold him down to get his shots. We all cried. It was so horrible. Today is day five, first we take him to have the surgery. Then we will take him to get his last round of shots. Cruel and unusual punishment.
When I asked my MPS community if their kids were resistant to the antibiotics, I got a resounding yes. One parent told me her horror story. They replaced the ear tubes three times, two tonsillectomies and shots every time that her child got sick.
The snot and fluid that ran out of her kids ear every morning, was so bad it took her 20min to wash it all off.
They had the allergy scratch test done and found that their child was allergic to 48 out of the 55 tests. Her child then had to get 6 shots a week for a year. Now they're finally down to two shots a week. There are hundreds of thousands of kids suffering from a rare fatal disease.
Please don’t feel sorry for us. Instead get mad and take actions tell our government aka the Energy and Commerce committee to choose our children over big Pharma's greed for more profits. How did we ever let it get this bad?
Ok time to go, please honor Jonah and take action.
