The fda needs to recongize that these children and adolescents who have duschene muscular dystrophy do not have the luxury of another 2 years for sarepta to run a p3 trial. During that time hundreds if not thousands of md children will lose more lung function and eventually will die. The fda is supposed to
work for the children and parents. Sarepta should offer to provide their drug treatment to all parents of children who have dmd for free and allow the parents to decide whether their children should be treated. The hypocratic oath of a physician is first to do no harm. Their were children who were treated the p2 trial who showed some improvement in the
walking aspect of the trial. Even if some didnt show improvement none were harmed to
my knowledge by being treated. So if there is no harm then most every parent would want to give their children a chance versus the certainty that they will get worse or die if untreated. If 500 parents made the choice to have their children treated for free or if every parent whose child had dmd decided that their child should be treated and whatever percentage came away with some improvement or stabilization of the condition before their lungs became affected then the fda will have the results of the p3 trial in real time and these childrens lives will have been improved
for whatever length of time. Sarepta should demand an adcom meeting of experts and every parent who wants to give their child a chance the opportunity to improve should attend that meeting to implore the committee to allow serepta to treat their children under their doctors care the fda mission should be to help children who
have very little quality of life and survivability from this disease if it progresses beyond a certain finite point. If it takes the president of the united states to make this happpen so be it and the fda be damned. I implore everyone who believes that these children deserve a chance to communicate with the fda
About 100x more people will die of brain cancer in the next two years than this childhood form of MD.
Regrardless of pending and promising treatments for gioblastoma, they still must run the full phases of
clinical trials to get their treatments approved by the FDA. Fastrack designation, orphan drug, and novel drug designations were all designed to move the process along for very promising treatments like SRPT has.
If someone died because the FDA approved a drug where there were only 12 participants in early trials,
every lawyer and consumer rights advocate in the nation would be all over the FDA and the doctors who allowed the situation to happen. May not be "fair" but it is what it is unfortunately. It's the world we live in
I'm sorry you feel that way. But I would be willing toi bet that if your child had DMD you would want them to have a fighting chance. Yes the trial was very small but to my knowledge there were no adverse health complications among those children who did not respond. But just as in glioblastoma which has many other complications associated with it,,patients do die while trials for these diseases slowly wind down their neverending paths. With DMD the medication will work or won't work, and if it doesnt my understanding is that these children are no worse off except perhaps for the hope that they were given. If their parents are willing to assume that risk who are we to question their decision.
Sorry to hear that but really, you cannot put thousands of other kids at risk with the experimental drug.
Do t with valid numbers that FDA wanted.
SRPT must work harder to find the number of patients. There are tons of them out there.
SRPT endpoints studies are NOT enough to convince them.
The FDA has approved many other drugs with a much less arduous process. What we are talking about here is compassionate exemption which may arrest this disease before it reaches the point of no return. This disease is a death sentence and some cancer treatments have received breakthrough designation and fast track approval. So even though Sarepta's trial was small, it did meet certain important endpoints and the treatment did not cause children to die sooner.It just didnt work for some but the children who showed some improvement it helped.
Good idea, but appealing to the FDA now would be futile. IMO, appealing to President Obama to take over this drug and let the NIH run the program would be better. When the drug is deemed ready for market, the drug should be returned to Sarepta.
That really is the only way to elevate this outside the standard process. Get Mr. and Mrs. Obama on board to push and to try and find ways around the rules.. I said that earlier and got the same thumbs down... :-)
That is a brilliant idea as long as these children receive the treatment now and not 2 years from now. This disease has a very prounounced fatality rate once lung function is affected and waiting until that happens is the point of no return. Many of these children have incredible wills to live and
are very smart and attend public schools with assistance until they are no longer able to. They deserve every opportunity to live some measure of quality of life in spite of their prognosis. Without treatment there is no hope or peace for the families.
IF parents of dmd children have any type of medical insurance, even under the affordable care act doctors bills are covered.Furthermore there is also the Muscular Dystropgy Foundation which I guaranty would underwrite a solution to this hidious deady disease which usually affects boys predominantly. I do not have exact numbers on how many children of all ages have this specific type
of Duschene MD but I can tell you that my cousin's son had it and lived for 20 years but he had to endure countless surgeries for the problems that paralysis causes, and whether mu cousins private insurance or medicaid or medicare pays for these childrens' treatments it is fine by me. No child should have to suffer this illness when there is no harm in trying to treat it. As for the cost of the treatment Sarepta would have to pay for a P3 trial anyway so if that cost is not underwitten by MD Foundation money then the stockholders of Sarepta would pay initiallly and once the treatment was approved the stockholders would be paid back and then some. The truth is that for these MD children doctors,knowing the torment these children and their parents suffer normally donate their services or just accept what medicare or medicaid pays.