They are interested in all shortages in drugs which they approve. They would likely enter a discussion with the company to understand the supply plans.
Specifically for Aus the TGA also have a “medicine shortages information initiative” www.tga.gov.au which (although dinutuximab is not licenced by them) might be a way to escalate the issue.
Cheers Alan - that’s very useful to know, I will get on to it!
Mark
Hi Mark
We are in this group of patients who have just been told that there will no immunotherapy for Summer, she has just completed her 2nd round of Mega Chemotherapy and a stemcell rescue, she will start radiaton this coming Monday followed by scans, MRI etc to see where the cancer is at right now.
She was diagnosed last May with Stage 4 Neuro Blastoma high risk.
I would be interested in joining forces to see what we can do, maybe media, govt departments etc…
Bev
Thanks for reaching out.
I know you have read the latest FB thread so I probably don’t have too much more to add at the moment, other than to say I’m sorry you are faced with this situation (as if you haven’t got enough on your plates) and I know time is of the essence if Summer is about to commence radiotherapy.
I don’t think we can do too much more until we’ve heard back from Sydney and ANZCHOG - which we hope will happen early next week.
But perhaps in the meantime you can inform your doctors in Perth about the Gold Coast child mentioned in the news article. The paeds oncology world is a small one, particularly in Australia and so they mostly know each other. I would be very surprised if they haven’t already been speaking to each other about this issue.
Keep positive, we will do whatever it takes to get Summer this antibody (including going down all the avenues you suggested), but I’m hopeful wont need to and the doctors will sort it out for us!!
Take care and I will speak to you next week,
Mark
Hi Bev,
Perhaps you can reach out to Dr. Trahir in Sydney Children’s Hospital. He heads the SIOPEN group for Australia and perhaps some discussion can be opened between SIOPEN and COG (the body you are treated under) to provide you with antibody via the SIOPEN study.
Looking at this huge, and unfair situation there are a few angles I think we can take. Any angle is going to require some fundraising and media.
First and foremost SIOPEN and COG need to discuss this situation and update us all on the plan for Australian / NZ COG patients. Best people to discuss this are Dr. Trahir and Dr. Park. We need to get a public statement out there from both ASAP.
Ideal outcome would be for SIOPEN to provide the drug to COG for all the Australian and NZ diagnosed children, this would be simplest and involve the least amount of disruption for families.
If you are able to raise funds to be treated in the US there are quite a few options. You could go to CHOP or some other COG hospital and continue your protocol there. You could go to Sloan and get the humanized 3F8 antibody under their trial. Unfortunately this option is extremely costly and would require you relocate to the US for an extended period of time (at least 6 months)
Consider Europe (Germany) for antibody. I think healthcare is much simpler there and mostly covered by the government. I don’t know exact details but it is worth investigating.
It is getting a bit late you, but other newly diagnosed children could possibly benefit if 2M USD was raised for Dr. Alice Yu, she could mount a trial for her vaccine in less than a year. It is possible Dr. Lode could also do something like this as well. If there was a vaccine trial available it could become an alternative longer term. And may, or may not be a substitute for antibody. (it would be a phase 1 trial so this is a long shot)
I would love to do whatever I can to help, feel free to message me or update this topic with any updates you have.
Unfortunately the FDA aren’t being helpful - looks like they are treating this as a marketing decision rather than a drug shortage:
“We understand your concern and appreciate you bringing this to our attention. FDA does not have authority over where companies sell their drug products. Please discuss this situation with both United Therapeutics Corporation, and with the Drug Regulatory authorities in Australasia.”
Will update this forum when we have a response from TGA - the Aussie equivalent of FDA.
Sam
Part of the problem is that all of the patients treated on COG upfront protocols are off-trial at the moment so it’s sort of not COG’s ‘problem’. Not that I’m saying they won’t take an interest and have something to say because it’s obviously blatantly in their interest, but there’s only a certain amount of influence they have over what is essentially a commercial decision.
The reassuring thing is that Lady Cilento Children’s Hospital in Queensland have secured the European antibody ch14.18CHO for at least one child, so I’m hoping that means the rest of the states and NZ will get it too. I’m hoping Chris Fraser as Chair of ANZCHOG (which is the official body of paediatric oncologists for Australasia and therefore oversees both COG and SIOPEN treatments here) will be able to confirm if there is a national plan to deal with this short-term supply issue. It would be crazy to think Queensland had secured the European antibody when other states haven’t because that would mean families having to move interstate to finish their treatment.
But we also still don’t know if this patient is a one off for Queensland or whether they have guaranteed a supply for all future patients. We obviously need to confirm this. Chris Fraser is based at Lady Cilento so will have a good understanding of this.
Mark
I’ve heard back from the TGA (Therapeutic Goods Administration) who are the regulators of medications here in Australia. Unfortunately they can’t help with the shortage because dinutuximab does not appear on the ARTG (Australian Register Therapeutic Goods) and therefore is not available for commercial supply.
I was thinking one thing families can look into is the “The Medical Treatment Overseas Program”. I think it may fit the bill here.
That’s not a bad suggestion Sam and definitely worth investigating if it comes to it. However, ANZCHOG have confirmed that it is up to individual states to fund the European antibody (ch14.18CHO marketed by EUSA as Isqette). If the states decline to fund it, I can’t believe the federal government will then fund patients to get it overseas, given how much more that will cost. We may well need to start a campaign to make sure the individual hospitals/states are going to fund it.
ANZCHOG have given Neuroblastoma Australia a short statement:
Due to manufacturing limitations United Therapeutics has made a decision to discontinue supply of Dinutuximab to Australia. An alternative product, Dinutuximab Beta, marketed by EUSA Pharmaceuticals, is currently available in Australia through a Managed Access Program but is very expensive. Applications to fund the supply of Dinutuximab beta can be made by individual oncologists with the support of their Hospital executive. Applications need to be made on an individual basis. ANZCHOG is committed to working with the two Pharmaceutical companies and clinical trials groups to try to ensure ongoing access to Dinutuximab for children with neuroblastoma.
@markshirran how was Unituxin being supplied before UTC they pulled the plug on it? Unless they were giving it away because they needed to gain some safety data for approval over there then I can’t see how any state/hospital could reasonably deny funding for Isquette. The cost won’t be significantly greater (if indeed it’s greater at all).
@birdni - UTC were giving it away for free as part of an expanded access program. There’s obviously concern that authorities here will look at NICE’s initial appraisal when deciding about funding. What’s the timeline for them to do their review?
Why were UTC giving it away for free rather than trying to get reimbursed for it? They weren’t giving it away free anywhere else, aside from their ongoing obligation to provide it free for NCI studies. Now that they cannot supply the drug we are waiting to see what it means in terms of the EMA marketing authorisation and NICE appraisal process. Isquette won’t be approved in Europe (if it is approved at all) until late Q2 at the earliest.
Sorry I couldn’t be of more help mark
That’s not entirely clear @birdni. The process of registration with the TGA is lengthy (probably as in any other country) so the assumption is that Australia, given its population size, would be low down on their priority list. And presumably they’ve known for some time that they wouldn’t be able to keep up with demand, pushing us even further down that list. But then the COG trial closed in early 2012 I think, so that’s quite a long time for them to have continued giving the drug away for free!
No need to apologise Alan - they were good suggestions of yours and well worth a shot! Thanks for your help and advice.
Good morning,
Eurordis, the European Organisation for Rare Diseases (patient organisation: www.eurordis.org), is investigating on shortages of medicines. In particular, we would like to obtain information on the current use and supply situation for Unituxin in the EU member states.
In this respect please could you provide us with the following information:
Do you know in which countries was Unituxin used (including named patient basis and compassionate use) in Europe?
Did patients have to stop treatment with Unituxin because it was not available?
Has treatment been delayed because of supply issues/unavailability?
Many thanks in advance,
Best regards,
François
hello,
my name is Marc and a friend’s son also required Unituxin (dinutuximab).
My friend was first very disappointed that Unituxin is not available in his country, especially as it is for small children (still not registered!).
He found over the internet a company called RCC Pharma, and they helped him getting the medication for his son.
I do not know if there is now a shortage, but this would be very disappointing.
Wishing everyone only the best!
Take care,