Spinal Muscular Atrophy (SMA) – A perspective

If you google ‘child abuse definition NZ’, an official government site comes up and clearly defines it.

The top three examples listed are emotional abuse, physical harm and neglect – where a child’s basic needs are not provided. The site states that if you are worried a child is being abused, you should report your suspicions so authorities can investigate.

How would you feel if you knew that right now, in New Zealand, there are children who have been suffering unnecessarily since 2018?

What would you think if you knew there were children who were suicidal because life was so challenging, and their health was declining?

How would you feel if that was your child and the only people who could help you were looking the other way? What if the damage being inflicted led to the need for spinal rods being inserted to stop their spine from collapsing? What if the child needed a feeding tube because they were losing the ability to safely chew and swallow food?

How would you feel knowing that there was a child so physically damaged they had lost the ability to hold their own head up?

How about a child who once ran and played with their friends, but was now permanently confined to a power chair and could only watch the others play? What would you do then? Would you be moved by learning of their death? Of course you would. Surely anyone with a heart would be sickened or disturbed by this?

What would you think if the authorities knew? Or if you knew these children and their families had been on the steps of parliament begging for the suffering to stop, and crying out for help? And what would you think if I told you that several children were suffering this level of harm and the authorities who could save them, won’t?

Who do you go to when the very people who pride themselves on making New Zealand ‘the best place in the world to be a child’ are not stepping in to save these kids?

Why would we not step in to ultimately save them from an outcome no different – or in some cases far worse – than what happens to children who have been harmed by someone who was meant to love, care, and protect them? This is the reality of Spinal Muscular Atrophy (SMA) in New Zealand.

The cruel and heartless fact is that New Zealand is in the minority, and is inflicting needless, irreversible damage, and it does not need to be this way.

There is a small group of around 35 children living with Spinal Muscular Atrophy in New Zealand. Like many other conditions, there are varying degrees of severity. It is a progressive disease where time is not on their side. Every new day has the potential for another motor neuron to be lost, or for another basic function to permanently disappear. That might be the ability to sit, to stand, to roll over in the middle of the night, to independently go to the toilet or even to breathe.

As a cruel twist of fate, the most common type of SMA (type 1), is also the most severe type, where Kiwi children die on average at 13 months old.

In 2016, a miracle arrived in the midst of this tragic tale, when the first treatment for SMA was fast tracked by America’s FDA (Food and Drug Administration). The SMA world rejoiced in the fact that there was finally a treatment and a beacon of hope. New Zealand and Australia were slightly slower in getting this hope to their shores, but in 2018 an application was made to the regulatory agencies, and medicine-buying decision makers.

Australia saw the value and their children had access to treatment that same year. New Zealand is still waiting. In that time, Kiwi children have died.

For years I have tried to do everything in my power to get access to treatment for those living with SMA. I have campaigned, taken a case to the Human Rights Commission, presented to the United
Nations, spoken at neurological events and met with countless politicians, but I have ultimately failed – I have failed to get our leaders to understand that to deny New Zealanders access to these modern medicines is wrong, and although I have support in every other corner, those with the power to help Kiwi families have done nothing. How is it okay to pick these kids to die?

You can screen new-born infants for SMA and you can treat this disease. We do neither!

There are now three treatments available to treat SMA and our neighbours across the Tasman have funded all three. New Zealand doesn’t have access to one.

We are not talking about medicines that might work – we are talking about medicines that do work. In fact, they work so well that overseas, there are cases of parents being taken to court if they deny access to them. Here, our kids do not stand a chance.

The Government holds all the power. It can change this situation. Heck, they rolled out the red carpet when covid arrived on our shores, but they have denied life to these precious children – but not before seeing them suffer through significant pain and harm. SMA is a cruel disease. Even though you lose function, carriers of this genetic condition still feel everything.

There is no excuse. There was a review into Pharmac that was damning, and although we could have said ‘I told you so’, this is no game. It is evident that people have died prematurely and there are serious issues within the organisation, but we are yet to see any action, any plan or hear an apology.

The Government could ease some of the pressure on our health system by funding treatment. It would reduce the need for surgeries, breathing machines, mobility supports, and there would be fewer ICU stays… if we saw the true value of funding access to treatment, we would not only be taking pressure off the health system, we would be allowing these kids to grow up and contribute to what should be a beautiful place for all.

If we return to the very definition of child abuse which is defined as neglect and emotional and physical harm, I think it is clear – right now we have a government that has campaigned on kindness and wellbeing, but is sanctioning the terrible harm this disease inflicts on children by its inaction and apathy.

One in 40 New Zealanders are carriers of the SMA gene. It is inevitable that more children will continue to be born with this condition this year, and in the years that follow. Without access to treatment, every one of them will face a lifetime of physical and emotional suffering or death.

We can fix this. The solution lies in three easy steps:

1. Fund Pharmac to an appropriate level so that we do not sit at the bottom of the developed world (like we do). Right now, we only spend about 5% of our entire health budget on medicines. It is no wonder our health system is buckling! Lets treat people so we can keep them out of hospital.
   
2. Have Pharmac follow Treasury Guidelines. Right now, they don’t. Medicines should be evaluated based on the full societal perspective. Let’s ask ourselves what is the cost of not funding these medicines? For example, we should consider the costs to other government departments when a carer or parent can no longer work, or a sufferer cannot contribute to society because of their illness. Our officials need to look at the overall value of treatment, as opposed to the initial, upfront price tag.
   
3. Courageous leaders need to take a stand and admit that what we have is no longer fit for purpose. The Pharmac Review has given us a once-in-a-generation opportunity for change; this Crown Entity and its leaders have failed many New Zealanders. Let’s clear our long medicines waiting list now. Let’s not accept any more human collateral damage. Good intentions created Pharmac, but bad policy and leaders who have been asleep at the wheel have created a monster that is far scarier than any child with SMA can imagine is hiding in the closet. This is very real, and right now these children (and others) are scared and need help.

Please never assume that because you can access over-the-counter medicines or Pamol when your child is sick, that you will also be able to access the best-in-class health system and medicines. The reality is something very different.

Children with SMA are just one example of a system that tells parents to go home, take photos and make memories. Their child has been picked to die.

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