Lbazin22

We are looking for advice in respect of our sons Heathcare and the possibility of UK citizens travelling/relocating abroad in order to obtain treatment.

Our 22 month old youngest son Avery, was diagnosed with a rare genetic condition called Spinal Muscular Atrophy Type 2 in November last year. This is a degenerative muscle wasting condition, Avery never crawled or walked and although he can currently sit independently, his muscles will continue to weaken. The biggest threat is a cold developing into a chest infection whereby his body simply doesn’t have the strength to fight it.

A drug called Spinraza is available in numerous countries including Italy but not available in the UK.

The drug is one of the most expensive available ever, we have been quoted 1.5 million USD from Boston Children's hospital in USA for the first years treatment and 750,000 USD for each subsequent year for life (even with crowdfunding or a millionaire bank balance, this just is not sustainable.

SMA doesn't standstill and we know time is crucial to give Avery the best opportunity of some quality of life, so we are trying to exhaust every Avenue.

We have been advised that an S1 form is required but we don't seem to be getting any help from the UK government contacts. I have been passed around the departments and in the latest email, told we would have to be living abroad first and then let them know of the change in benefit before we could see if it was exportable and if we qualify for an S1. However other forums suggest that the S1 form can be organised and put in place before travel (which would make sense) . Avery receives DLA care component at higher rate and I receive carers allowance for him. Does anyone know if these are exportable benefits? And if our son would qualify for full healthcare in Italy? Being so expensive, would this treatment cost be covered 100%?

We are feeling desperately helpless knowing there is a drug to help our son, but we just can't access it. Avery's full story is at www.facebook.com/SMAType2

If anyone can offer any information to help, we would be extremely grateful.

Many thanks,

Lorna at Vicenza

Oh God help you. I'll find out what I can.

Lbazin22

Thankyou Lorna x

Thairetired2016

I can only comment to S1 form.
We were not able to get this form prior to moving and having a fixed address in the chosen EU country.
We did eventually achieve it after more than a year despite a promise "next week, next week". In the end we never signed up with the local health service as it was too complicated (Germany). Now we left EU area.
Hope all goes well for you.

Lbazin22

Oh Ok, Thankyou, that's disappointing but I didn't expect it would be easy. Thx again.Lisa

Thairetired2016

https://smanewstoday.com/2017/12/20/spinal-muscular-atrophy-researchers-shine-spotlight-on-spinraza-ethical-challenges/amp/

Cost for Spinraza is still considerable but a lot less than the price you were quoted. At least acc. to above link.

Geordieborn

Really sorry to read about this. It is no doubt not something you want at this time to have to deal with government bodies and be passed from one to another. However this .GOV Link I think give the body responsible. To be honest, at first glance I suspect you would need to be able to be claiming one of the other benifits mentioned, which sounds wrong to me. Not too sure if you have already thought/done this, but it might be worth trying to get your MP on board. If you do I would mention this in any discussion with the departments involved. Best wishes to you and your family.

37100

Would you be able to work here? i.e teaching English in a language school. This would entitle you to sign up for healthcare. Alternatively, you could chose a region that allows EU citizens to buy in to the NHS.

Lbazin22

Thank for the messages, we have contacted our MP to try to push for the treatment to be available in the UK but that's a good idea to get him on board with assistance in this option too. We will speak with him and try to get some advice.
With regards to working in Italy, unfortunately we don't speak Italian but we have a few Italian friends that we can stay with out there who were the ones that had suggested it might be an option (they add just unsure on the legal side of uk citizens entitlement). We have also written to the hospitals that are providing the drug, so we shall wait a reply.
I think we would struggle working not knowing the language but when you say find a region where you can buy into the NHS, what exactly do you mean? Thanks, Lisa

modicasa

Im afraid the likelihood of getting such a drug in Italy is remote. Only 6000 ish drugs are licenced in Italy, unlike the 60000 in the UK, its a protectionist market run to protect the italian drug manufacturers.

philat98

According to Wikipedia the drug is available in Italy. The challenge might be finding a specialist to prescribe the drug. Probably a specialist would have their own ideas about appropriate therapies for the condition.

Lorna at Vicenza

I haven't found out much more than what you've already been told except that the drug spinraza seems to have become available here just last year and can only be given in hospitals and similar type places as it has to be injected.

Perhaps you should first see if you can find an Italian specialist for atrofia muscolare spinale.

Thairetired2016

I have read your appeal on gofundme where you express your disappointment with the NHS. I understand you are desperate to get hold of this drug asap as it is more effective at an early stage. Be aware that in all those EU countries where Spinraza is available, doctors will not just hand it out for free either. In two wealthy EU countries where I lived, much cheaper and more common everyday drugs require special approval by the medical insurer. Often it gets turned down. Or the patient shoulders the cost privately.
Italian gov. provided health care as I once experienced is soso, not great. They are also short of funds. You may struggle to get free treatment and a very expensive drug as a non-resident/visitor. Plus Britain leaves EU a year from now.

Red_Wine_Fairy

I hope you don't mind me butting in. Like everyone here, I am heartbroken to hear of your situation, and unlike most, have had a child with a rare disease around the same age, albeit many years ago now.

I thought I would let you know the situation in my country, The Netherlands. (Google translation, so apologies)


The drug Spinraza against the muscle disease SMA is more widely available. Minister Bruins for Medical Care has reached an agreement on this with the supplier Biogen.

Spinal Muscular Atrophy (SMA) is a rare progressive muscle disease. Spinraza is already available for the treatment of babies with SMA type 1, the most severe form. The costs are reimbursed by the supplier. By the agreement, it will now also be reimbursed for children with type 2 and 3A who need urgent treatment.

In practice it concerns a group of about 30 patients, especially children younger than five years. The UMC in Utrecht determines which children are eligible for treatment. Because it is an intensive and complicated method, the UMC also decides on the order in which the patients are treated. The seriousness and duration of the disease are examined.

At this moment there is capacity for the treatment of one patient per week. A lot of hard work is being done to expand this capacity, according to the UMC.

Not in basic insurance
Bruins writes in a letter to the House of Representatives that the medicine is expensive. He negotiates with Biogen about an "acceptable price". The drug now costs 80,000 euros per injection. Patients need six in the first year and then three injections per year for life.

The Minister emphasizes that the drug is not reimbursed under the basic insurance, but that it is a temporary solution until a final decision has been taken.

The temporary arrangement applies until 1 May. Zorginstituut Nederland will provide the Minister with advice on final reimbursement early next year.

Muscle diseases The Netherlands, the association for patients and their relatives, is pleased with the news that more patients can be treated with Spinraza. "But we are not there yet", writes the association, which points out that a large group of young people and adults "with pain" are waiting for the medicine.

https://nos.nl/artikel/2207078-duur-...g-vergoed.html


Clarification:
Utrecht - as beautiful if not more so, city about 30 mins away from Amsterdam - the home of UMC (Utrecht Medical Centre, huge research hospital).
Basic insurance - everyone here needs at least a basic health insurance policy, at around 100 euros per month per adult. Low waged and unpaid get around 85% paid by the taxman, and they pay the difference from any benefit, but you must be resident/registered with tax authority. Top up policies are available for extra cost. Nobody can be refused insurance by Dutch law (it's not America) and all children are covered free of charge on their parent's health insurance policy - so if it was a possibility, the Dutch won't get nervous if you ask about getting this for your child, because the government will pay them directly for any long-term chronic illness - they (insurers) lose nothing