FDA

FDA Delays Threaten Kid's Life; Parents Plead With White House

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Austin Leclaire, 15, and his brother Max, 12, of Vermont, both suffer from a form of muscular dystrophy that leaves them with a life expectancy of 25 years. In 2012, Max began showing signs of improvement while participating in a clinical study with a drug called eteplirsen. Austin didn't qualify for the study, because he is wheelchair-bound. His condition has continually deteriorated: He can barely get himself out of bed or unlock a door. Making matters worse, the Food and Drug Administration (FDA) is now effectively blocking Austin's ability to access the drug by delaying its release. So, the Leclaire boys' parents are petitioning the White House.

The Boston Herald reports:

Cambridge-based Sarepta Therapeutics has been testing its [Duchenne Muscular Dystrophy] drug in 12 boys for more than two years and says the results are good, but the FDA has stalled approval until it sees results from a larger trial.

[…]

Sarepta spokesman Jim Baker said the company plans to launch a new, larger drug study later this year, with input from the FDA.

The process could take years.

But, "There is no reason in the world for this drug to not be approved. It's 100 percent safe," Jenn McNary, mother of the Leclaire boys, told the Herald. She isn't alone:

Dr. Jerry Mendell of Nationwide Children's Hospital in Columbus, Ohio, who designed the Sarepta drug trial, said eteplirsen is a first-of-its-kind therapy and has shown "superb" results so far.

"I think the product is very good, and I think it deserves to be approved. … It has absolutely no side effects," he said.

Frustrated by the bureaucratic delays, the Leclaire family teamed up with a campaign called "The Race To Say Yes." They filed a "We the People" petition at WhiteHouse.gov late in February, hoping to get at least 100,000 signatures in favor of accelerating the approval process for the drug. They met that mark this past Saturday.

Unfortunately, as the Herald notes, the White House has not yet replied.

For more Reason coverage of the FDA stifling progress in medicine, click here or watch the video below:

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  1. Unfortunately, as the Herald notes, the White House has not yet replied.

    They will get right on that right after they get done answering those IRS subpoenas.

    My advice is the family should take up a collection to send money to Valerie Jerrett and Obama For America. It will take a few million to get their attention, but it is the only way I am aware of to get Obama interested.

    1. So Obama’s security can shut down their town for a campaign stop? Haven’t these people suffered enough?

    2. Funny how Obama had time for the beer summit, but no time to save a kid’s life

  2. There is a special set aside pit in Hell for FDA-crats like this. Better someone die than they not mindlessly “follow procedures”.

    1. Not their fault at all, it’s standard procedure as specified in statute; the article does a bad job by not explaining that. From what I could gather, testing has gone thru only 2 of the required 3 phases. Blame Congress.

  3. but it is the only way I am aware of to get Obama interested.

    Get every radio, tv, news station you can think of, whether in the US or outside and repeat their story over and over. Make him look bad, that is the only way to get him interested. Throw in the head of the FDA as well. Make sure people know who is killing your kids.

    1. That is a great idea and it would work too. There is only one problem, those radio and TV stations you speak of are run by DNC operatives who will do or say anything to keep Obama from being embarrassed.

      1. of Vermont,

        Get the Canadian stations on it.

        1. Get the Canadian stations on it.

          Which one?

  4. but it is the only way I am aware of to get Obama interested.

    Get every radio, tv, news station you can think of, whether in the US or outside and repeat their story over and over. Make him look bad, that is the only way to get him interested. Throw in the head of the FDA as well. Make sure people know who is killing your kids.

  5. There is a special set aside pit in Hell for FDA-crats like this. Better someone die than they not mindlessly “follow procedures”.

  6. 12 subjects over 2 years is kinda light support for “absolutely no side effects.”

    That being said, signing a waiver saying you wont sue if complications result should be the only obstacle barring these parents from obtaining this treatment. They already reviewed the informed consent documentation to get the one kid in the study, so it should be a pretty short appointment.

    But no, better that one kid suffer and die rather than obtain potentially life saving treatment as long as it saves just one moron from the responsibility of having to make informed decisions about their life or the life of their child.

    1. The kids are going to die anyway. I’d think that’s worse than most any side effect.

      1. That depends on soomething we have no data on: the quality of experience of the non-living. We don’t know whether being dead is interesting, boring, awful, or no experience at all. We don’t remember experiences from before we were alive. We don’t even know what experience is like for other things that are alive!

      2. That depends on soomething we have no data on: the quality of experience of the non-living. We don’t know whether being dead is interesting, boring, awful, or no experience at all. We don’t remember experiences from before we were alive. We don’t even know what experience is like for other things that are alive!

  7. Today is a bad day to ask me to hate the FDA

    because this =
    http://alturl.com/csja3

    1. I liked him better when he was Cactus Jack.

      How about all the furor over HFT and Michael Lewis’ Flash Boys?

      1. I think its kind of ridiculous.

        Lewis writes well, but I think his stories are mostly about hyping details that seem ‘bizarre’ to outsiders but are fairly prosaic when you get closer to the daily reality.

        This businessweek comment puts in nicely:

        “…’ Lewis’s zeal to simplify complex subjects into catchy bites and to consign players to clearly heroic or villainous roles.””

        I am fairly neutral about HFT because despite making up a large chunk of the actual trading that goes on, they are 1) mostly price-neutral, 2) aren’t particularly ‘profitable’ 3) and don’t in any way interfere with any retail investor activity. They are mainly executed at the wholesaler level before they even hit exchanges. its just trying to make technical details of market-functionality out to seem like a kind of nefarious scheme, when you could pretty much do that with *any old component* of the markets already, sans HFT. Like market-makers themselves.

        I think you could make a far more “reality based” critique of financial ‘ripoffs’ by doing a book about things like *Annuity Pricing* or mutual fund fees.

        But that shit would be so boring that you couldn’t sell it.

        I once explained to someone that the most nefarious things on earth tend to be the most boring and banal, which is exactly how they succeed. See: municipal union pension scams, et al.

    2. MannKind? Do they sell people? What about dwarves? Dwarves are so hot right now.

      1. It’s true. Robert Reich is all over the TV.

  8. Unfortunately, as the Herald notes, the White House has not yet replied.

    Given the high they’ve got going today I wouldn’t be surprised if their response ends up pimping Obamacare to the family.

    1. “…Of course, your new obamacare coverage won’t cover the cost of study drugs.”

      1. “…Of course, your new obamacare coverage won’t cover the cost of study drugs much of anything because Fuck You, That’s Why.”

        FTFY

  9. I could be worse, the EPA could be poisoning their kid.

    The Environmental Protection Agency has been conducting dangerous experiments on humans over the past few years in order to justify more onerous clean air regulations.

    The agency conducted tests on people with health issues and the elderly, exposing them to high levels of potentially lethal pollutants, without disclosing the risks of cancer and death, according to a newly released government report.

    These experiments exposed people, including those with asthma and heart problems, to dangerously high levels of toxic pollutants, including diesel fumes, reads a EPA inspector general report obtained by The Daily Caller News Foundation. The EPA also exposed people with health issues to levels of pollutants up to 50 times greater than the agency says is safe for humans.

    http://dailycaller.com/2014/04…..ns-agenda/

    Of course this will be a big deal on a few right wing news sources and completely ignored by the major media and nothing will result from it. The Obama administration could be putting people in gas chambers and the media wouldn’t report it.

    1. I wonder if they did those studies in Tuskeegee for good measure.

      1. It would have been Progs coming home to their roots I guess.

      2. You beat me to that, darn you Susan!

    2. This report addresses the following EPA theme: ? Addressing climate change and improving air quality

      Testing for diesel fuel combustion tolerance makes sense. It will be one of the few affordable fuels after the AGW adherents ban natgas production methods.

  10. I’m surprised Reason hasn’t been on the Justina Pelletier story. (Even Slate had a story). Recently, Alan Dershowitz joined her legal team.

    I sent this to JD Tuccile and then to Scott Shackford, but… crickets. Apparently, I’ve made some powerful enemies.

    1. That case is terrifying.

    2. The parents were on Dr. Phil.

      This shit is so evil. Just pure, unnecessary evil.

      I don’t know how you people aren’t scared to death to bring children into this world.

      1. I am. You just have to hope it doesn’t happen to you. Luck and statistical improbability are your only defenses.

      2. How do you think I feel? My daughter also goes to Boston Children’s regularly.

      3. My case is in the next county over. The probate system in MA is scary as shit.

        Parents who have to walk the fine line between protecting their kids and keeping the court happy have to have an steely nerve.

        My ex and I are back in court, and I am obeying some court orders that make me fear for my kids’ safety. But defiance would probably cause the judge to give my ex custody, thereby putting the kids in even worse danger for a much longer term.

        So I am quietly and meekly submitting, and keeping my fingers crossed that my ex-wife’s next explosion of rage won’t arrive for another few months or that when it does come, it’s not too destructive.

        And the important quality is meekness. If you are defiant or too spirited, they are far more likely to make the snap judgement that you are the problem.

        Additionally, many judges seem to be transferring their role as finders of fact to the shoulders of the weirdos who become DCF social workers. It’s easy to just take a DCF analysis as being authoritative, just like taking a police report as being god’s honest truth. And I’m still waiting to participate in a DCF investigation where the outcome wasn’t ass-backward.

        In my experience, many DCF workers and social workers are actually dealing with demons from their childhood, and if they see their own childhood in the situation they are examining, their objectivity goes out the window. At one point I had a social worker who clearly was working through her daddy issues by attacking me.

        1. In most states you don’t have to even be a lawyer to be a family court judge. And when you consider the money to be made doing private child and family therapy you understand very quickly why the DCF is only able to hire congenital idiots and nuts. Who with any qualifications or expertise would agree to work there?

          I really think CPS should be eliminated in all states and all child welfare laws repealed. If someone is abusing a child, let the cops investigate it like any other crime. All CPS does is torture innocent people who are easy marks.

          1. Well, in their defense the judges have a thankless job:

            1) MA is a two-party consent state for recording conversations.

            2) MA has marital privilege – ie. no private conversation between married people can be submitted into evidence.

            So the judges often have very little to go on… The DCF people are less constrained and often have access to more info.

            In my case, if I had recorded any week’s phone conversations between my ex and me or the kids, and played it in court, there is little doubt in my mind that the judge would be worried for the safety of any kid in my ex’s custody.

            But I can’t do that, and she won’t communicate in writing, so I am up the creek without a paddle, having to make allegations that are solely supported by my testimony or that of my kids.

            In essence, we have recreated the trial by ordeal.

            1. Have someone with you whenever you talk to her on the phone and when she wigs out, put her on speaker phone. The other person can testify to what she is saying and since there is a third party there, no marital privilege attaches.

              1. Marital privilege no longer is an issue – divorce became final over a year ago :).

                However, it’s kind of hard to find an unbiased party to listen in… I can’t just grab people from my work or from the street and force them to listen to the crazy.

                And the people in my household are hardly unbiased…

                1. Set her up. Get someone who is unbiased and have them there. Then push her buttons a bit to set her off. I am sure you know how to do that. Don’t wait for her to freak out, use a little bait to get her to do it for you. Then put her on speaker and let her go.

    3. Holy shit. That is completely rage-inducing. The state is going to kill that girl.

    4. Powerful, evil, and wrong. That’s a bad combination.

    5. Johnston said the family, which vented its anger in various media interviews, hampered efforts to have her placed as near them as possible.

      Just be quite little compliant slaves and you’ll get to see your daughter again (or what’s left of her)!

      1. “We’re punishing you for exercising your first amendment right to petition publicly for a redress of grievances.”

    6. Given that Boston Children’s Hospital is publically accusing them of incompetence, you’d think Tufts would be suing over this.

      1. Children’s hospital is owned by the same guys who own the Partners Healthcare Network.

        I can’t reveal how I know this, but I have it from a good source that it’s pretty hard to find a judge in the MA courts that handle commercial litigation who doesn’t have a conflict of interest due to a relationship with Partners.

        Tufts is not part of that keiretsu.

  11. The point of power is to wield and expand it. If it means having the power to deny life saving medication to a child, then it’s all good. Force has been wielded. Boner has been stroked. Someone can go home satisfied because they imposed their will upon someone.

    1. the children must die…for the children!

      1. We had to destroy the child to save him!

        1. If we let this kid have the medicine, we will have to let every sick kid have unproven medicine as well. Some of those kids may needlessly die. So, this kid is just going to have to die so we can save those other kids.

          THIS is what they actually think.

          1. There is no such thing as informed consent (unless you’re negotiating sex with a feminist, then it’s required in triplicate).

      2. The children must die to satisfy the bureaucrat’s authority boner.

        Same with the case in the post above mine. That girl is going to die, simply to satisfy someone’s power boner.

  12. OT:

    Probably already been posted, but just in case, a follow up to the link I posted yesterday about Idaho cops hassling a 70 yr old guy for having CO plates.

    What does having CO plates mean? Obviously, that you are a dangerous drug fiend!

    1. Too bad we don’t have an actual DOJ. One state’s police picking on another state’s drivers is the kind of thing that we have a federal government to remedy. DOJ should be up the Idaho state police’s ass so far they can give them a complimentary colonoscopy as part of the investigation.

  13. I saw this story on Stossel and at one point I just had to fast forward past it because it was literally pissing me off. I rarely get emotional about something on TV.

  14. FDA Delays Threaten Kid’s Life; Parents Plead With White House

    This is confusing to me. I was told, can’t remember who, by T*ny or Joe, that federal agencies were completely democratic, because when you wanted change, you merely petitioned your representatives who would then confirm or deny a president’s appointment who would in turn implement the policies that would be beneficial to the people, every 4 to 6 years.

    Are these parents just too fucking lazy to go through the normal democratic process to save their kid’s life?

  15. http://www.dailymail.co.uk/tvs…..-Inch.html

    Doogie Houser has gone full tranny. NTTAWWT

  16. *It’s 100 percent safe,” Jenn McNary, mother of the Leclaire boys, told the Herald. She isn’t alone*

    Yes, the mother–who is completely unbiased and probably has an MD or D Pharm the story forgot to mention–and a physician who certainly has financial interest in the drug being approved, say it’s safe. I’m sure the mothers who had morning sickness said the same of thalidamide. What could go wrong?

    1. How about this, we let the kid’s doctors decide if it is a good course of treatment? If it is not safe or not worth the risk, the doctors won’t administer it. That sounds a little better than letting a bunch of bureaucrats at the FDA, who don’t have MDs or PHDs either deciding it.

      And it has been 60 years since thalidamide. There is a stack of dead bodies and a river of blood from the last 60 years with the FDA stamp of approval and that is all you got? That same tired example?

      1. That sounds a little better than letting a bunch of bureaucrats at the FDA, who don’t have MDs or PHDs either

        One would hope that a basic job requirement for working in that branch of the FDA would be having a MPH, if not a DPH or MD.

        However, I wouldn’t be surprised if the ranks of the FDA’s drug approvers are filled with Poli Sci majors.

        1. I am sure that the FDA is filled with MDs and PHDs. But those people are not the ones making the final call, I guarantee it.

        2. One would hope that a basic job requirement for working in that branch of the FDA would be having a MPH,

          Having people with degrees in “public health” would probably be worse than having miles per hour working in that branch of the FDA.

    2. Yeah cause it’s better the poor kid DEFINITELY DIE, rather than take the risk that there may be some potential side effects somewhere down the road.

      I can only hope that fascists (uh I mean “liberals”) like yourself come down with a fatal disease and are denied lifesaving medicine.

    3. 12-year-old boys rarely get pregnant.

      1. Stop othering us with your gender assumptions Sugar Free. Take your gender oppression elsewhere.

        1. Their bodies have ways of shutting it down, I hear.

    4. What could go wrong?

      Whereas, “going right” in this case means just letting the kid die after enduring a terrible quality of life for a few more years, at best.

    5. Government-funded science concludes we need more government, and that’s not biased, no sirree.

      As I said above, her kids are going to die anyway. That’s worse than any of the side effects.

  17. OT, but this is just as good a place as any to ask this question.

    The 7 million “goal” was never a goal. It was a CBO projection. If anyone should be celebrating anything it’s the CBO. They made an accurate prediction. Ok. But, 7 million sign-ups (not actual paid for policies) was never a goal of the law. It was just a CBO projection that was accurate. Why is this cause to for Obama and crew to celebrate?

  18. Unfortunately, as the Herald notes, the White House has not yet replied.

    Silly proles, those petitions weren’t actually intended to get a response, they were just there to give the impression that the God-King gives a shit about us mere peasants and our piddly ass problems.

  19. Hey Sandra Fluke, THIS is what “denying access” is.

  20. Ordinarily I find the FDA-is-killing-people argument to be extremely overblown and hysterical. But this is a case in which, while the FDA isn’t killing anyone, its delay in approving this drug is a shame. When you are confronted with a disease or condition that leaves people with an average lifespan of 25 years and a progressively worse quality of life for many years leading up to their ultimate death, it makes little sense to require the same kind of investigation into a drug for the treatment of that disease or condition that you would for a drug meant to treat acid reflux or even something serious, like breast cancer, for which there are proven effective treatments that already exist.

    If it hasn’t done so already, Congress should create an expedited approval process for such drugs. And if it has already done so, then Congress needs to start knocking heads together at the FDA.

    1. There is a process to allow them access, but it requires the permission of the patent holder and an application by the patient’s doctor. There is no indication from the author that such process is even going on.

  21. Duchenne’s (the correct spelling) is actually the most common form of muscular dystrophy, not a rare form as stated here.

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