Off-Label Prescription of Afrezza to 15 & 17 Year Olds

[mnholdem]

Yesterday I was informed of a dad who is planning to request the family physician prescribe Afrezza for his T-1 teenagers. My understanding is that he reads ProBoards regularly, but doesn't post, and he may want the story of the impact diabetes has had on his family to be conveyed in a manner that protects his kids/family. Apparently he considers me trustworthy enough for the task.

I'll keep you informed (he's seeing his doctor today) but he got me to wondering how many other parents are considering the same. It's not illegal for a physician to prescribe off-label. Afrezza may truly be a God-send for parents who worry themselves sick every time their diabetic child goes to sleep.

[liane]

There are very few pediatric studies on most drugs. Off-label is the way to go.

[savzak]

I didn't know older teens would be considered off label. Is the cutoff 18? Seems pretty arbitrary if it is.

[rockyp]

I plan to ask my son’s endo about getting on Afrezza at his next appointment. He just turned 13. I don’t know if it is puberty hormones kicking in or what, but that boy’s blood sugar hops around with the frequency of a cheap ham radio. He will go sky high and we can’t seem to bring him down for hours and when … whoosh! … he drops like a rock and we are then fighting a low. He is already on a pump, so it is not the inhalable aspect of Afrezza that is appealing to us; it is the fast(er) acting aspect and the lack of a tail that would help him stay in range more of the time (and hopefully reduce hypos as a result).
Because he is only 13 I am not too optimistic that our endo will write a prescription for him, but I plan to ask anyway.

[esstan2001]

good luck... keep us posted; I hope it works out for your son.

[dreamboatcruise]

rockyp... It certainly seems worth being a bit insistent with the endo to at least give it a try, so that a decision whether to use it long term is informed by effectiveness in addition to theoretical risks. Hope things go well, regardless of whether it involves Afrezza.

[rockyp]

DBC - Thanks.  This endo does tend to be a bit more open to new technology than our last endo, so maybe there is some hope.  We've got our fingers crossed.

[hammer]

Mar 6, 2015 8:35:53 GMT -5 liane said:

There are very few pediatric studies on most drugs. Off-label is the way to go.

I would completely occur. If patients results are consistent with what we have been seeing, there will be no way a parent of a 15 or 17 y/o patient will allow this life saving advancement to be withheld from their child. Although compassionate use programs may be more associated with non approve drugs, this may be one in which the FDA feels label expansion may be indicated.

I sense a rumbling similar to what was experienced in the mid eighties when a very vocal political group demanded early access to un approved drugs for the treatment of a devastating disease.

If the early adopters experience becomes the experience of the norm, the FDA just may suffer the public embarrassment of why this drug was not approved last time around, why they appear to have egg on their face.

[joeypotsandpans]

Mar 7, 2015 6:06:40 GMT -5 hammer said:

Mar 6, 2015 8:35:53 GMT -5 liane said:

There are very few pediatric studies on most drugs. Off-label is the way to go.

I would completely occur. If patients results are consistent with what we have been seeing, there will be no way a parent of a 15 or 17 y/o patient will allow this life saving advancement to be withheld from their child. Although compassionate use programs may be more associated with non approve drugs, this may be one in which the FDA feels label expansion may be indicated.

I sense a rumbling similar to what was experienced in the mid eighties when a very vocal political group demanded early access to un approved drugs for the treatment of a devastating disease.

If the early adopters experience becomes the experience of the norm, the FDA just may suffer the public embarrassment of why this drug was not approved last time around, why they appear to have egg on their face.

In my opinion only it is not a coincidence that Hamburg is stepping down at the end of this month after having given her resignation just after the launch of Afrezza...the theme will be consistent regarding these parents, and others who will feel that the constant delays by the FDA in getting this life saving therapy for diabetics approved could be considered manslaughter (see my old posts on YMB regarding same and FDA has "blood on their hands"...). The following excerpts are from an article regarding Hamburg's stepping down from her illustrious post as head of the FDA...see the NY Times: www.nytimes.com/2015/02/06/health/margaret-hamburg-fda-commissioner-stepping-down.html?_r=0


{In a blog post earlier this week, Dr. Hamburg said the F.D.A. had approved 51 drugs in 2014, the most in almost 20 years. She called that a testament to its “innovative approaches to help expedite development and review of medical products that target unmet medical needs.” }

Anyone who witnessed the Adcom after years of multiple delays could see the ineptitude of the horses she had put in charge of setting up the poor trial standards/parameters to cause the issues both with the delays and the initial labeling of the product. Lets go on....

{Some public interest groups were critical. Dr. Michael Carome, the director of the health research group at Public Citizen, based in Washington, called her tenure a period “of weak and ineffective leadership.”

”Too often, the F.D.A. has succumbed to industry and political pressures, implementing policies and taking actions that tilt too far toward the bottom-line interests of pharmaceutical and medical-device companies, and away from protecting public health,” he said in a statement.}

Anyone that knows her history with "conflicts of interest" see following regarding the Henry Schein fiasco: www.mercuryexposure.info/scandals/food-drug-administration/item/763-fda-commissioner-margaret-hamburg-and-conflicts-of-interest-with-mercury-filling-rule

not to mention her husband's role regarding Renaissance Technologies see following from WSJ:  www.wsj.com/articles/SB124328188115551961

which it was widely known about their huge interests and holdings of NVO which as stated above their "bottom line interests" are threatened as witnessed by those that are about to request for their childs sake...off label use/prescriptions for a SUPERIOR (not non inferior) therapy in treating their previously unmet needs.

So again, mere coincidence in the timing of her stepping down? ....in her own words according to the NYT article:

Dr. Hamburg added, “I’m leaving because I feel like it is time.”

Hmmm SNY soft launch monday Jan. 26th 2015....Margaret feels like it is time thursday Feb. 5th 2015....

oh yeah btw, I believe Renaissance is now a holder of MNKD if I'm not mistaken....wonder what their peak holdings of NVO were and where they are now...haven't looked (so I could be wrong on this one and right now too lazy to check it out)  but would imagine not quite the same...

[notamnkdmillionaire]

If Public Interest had a clue they'd realize the FDA does more to kill humans with its inaction and red tape of getting drugs approved. There is obviously a balance but the power of our Government has swung way to far in it's direction.

[tripoley]

Mar 6, 2015 9:34:09 GMT -5 rockyp said:

I plan to ask my son’s endo about getting on Afrezza at his next appointment. He just turned 13. I don’t know if it is puberty hormones kicking in or what, but that boy’s blood sugar hops around with the frequency of a cheap ham radio. He will go sky high and we can’t seem to bring him down for hours and when … whoosh! … he drops like a rock and we are then fighting a low. He is already on a pump, so it is not the inhalable aspect of Afrezza that is appealing to us; it is the fast(er) acting aspect and the lack of a tail that would help him stay in range more of the time (and hopefully reduce hypos as a result).
Because he is only 13 I am not too optimistic that our endo will write a prescription for him, but I plan to ask anyway.

A lot of doctors start viewing pediatrics differently from about the age of 12 on.  I think Afrezza would be fine in this age group.

[mnholdem]

I am delighted to inform everyone that I was notified today that the family physician filled the prescriptions and dad picked up the Afrezza for his two kids this afternoon. I'm not certain whether "Dad" wants to track the kids' results and have me post them or whether he simply wanted everyone to know he was able to get Afrezza for them. I'll let you know after I speak with him about it.

Remember that his original concern was that nobody would believe him or, since he has never posted on this board before, folks might think he was a lying pumper. However, I have been able to have another person independently confirm that his information is indeed factual.

I suspect you will have a much happier family by the time Father's Day arrives.

Way to go, Dad!