Medication out of reach
Nelson man wants to halt progression of rare genetic condition
‘‘This is the way that it is for so many people with rare disorders in so many different ways.’’
Lisa Foster,
Rare Disorders New Zealand chief executive
Time is of the essence for a Nelson man with a rare genetic condition that causes his tissue to turn to bone.
Dylan Barker was aged four when he was diagnosed with the extremely rare condition fibrodysplasia ossificans progressiva (FOP).
As he ages, his muscles, tendons and ligaments calcify into bone, which constrains his movement.
Hope lies with a drug that can halt the bone growth, but despite being eligible for it in Australia, for the past year the 29-year-old and his family have been battling to access the drug in New Zealand.
There are only 800 people in the world with FOP, and only two in New Zealand. In a twist of fate, the other Kiwi with the condition, 12-year-old Kaitlyn McAlpine, also lives in Nelson.
For the last five or so years, Barker has had a chair to help him get around. He can still walk, although by his own admission ‘‘it’s not fast or very pretty’’.
But maintaining a range of motion is important, because when it goes, he knows it won’t return.
‘‘Once things lock up, that’s it. I don’t get the movement back,’’ he says.
‘‘I’m doing pretty good to be walking for this long, as slow and painful as it is.’’
His ankles are fused, he does not have movement in his spine, and his jaw is locked.
Barker misses being able take a bite of an apple or feel the texture of a Crunchie bar. Watching others yawn or sneeze is frustrating – he can remember what that felt like.
Dr Frederick Kaplan, the leading expert on FOP, who diagnosed Barker as a child, said that as at January 2018, there were 37 universities researching the condition and 12 pharmaceutical companies developing drugs for FOP.
Barker was granted compassionate access to one of those drugs, palovarotene, in Australia last March. Since then, he and his family have been jumping through hoops to try to get the
drug into New Zealand.
Carer Miyuki Duggan said she had been working for more than a year to help Barker access the drug, and it felt like everything she tried to do for him involved a fight.
‘‘It will be life-changing. They said it stops 75 per cent of flareups and bone growth, which is massive for him,’’ Duggan said.
‘‘However, trying to get it into the country has been very problematic. This drug he has been offered for free, but trying to go through the bureaucracy of it all has been awful.’’
It had been one thing after another. At first, the family couldn’t afford the GST on the value of the drug, which they were told meant they couldn’t bring it into the country.
‘‘You name it, it was an obstacle, but we are not giving up,’’ Duggan said.
Throughout that time, Barker had been in a near-constant state of his condition flaring up.
‘‘It’s frustrating. We just seem to be going around in circles, ever-decreasing circles,’’ he said.
They had received support from Rare Disorders New Zealand chief executive Lisa Foster, and remained hopeful that they would be able to gain access to the medication.
Foster has been dealing with the drug manufacturer, doctors, pharmacists, the Customs Service and the Inland Revenue Department to work out what the holdup is.
She said the doctor had been reluctant to prescribe it, as he was concerned about the cost of GST on his practice. It was agreed that the pharmacist would receive the medication once Barker had a prescription, but this had not happened either.
Foster said the situation was ‘‘deplorable’’.
‘‘This is the way that it is for so many people with rare disorders in so many different ways. Everything is overly complicated – there is no clear pathway, there is no advice or navigation, help or support.
‘‘Dylan really needed quick access to that medication on a compassionate basis, and it certainly hasn’t happened.’’
Foster said there were 300,000 New Zealanders with a rare disorder, and in a recent survey, the resounding feedback was that they felt invisible.
Medsafe group manager Chris James said palovarotene had not been scheduled as a prescription medicine in New Zealand, but it would not be stopped at the border if it was imported by a patient or a medical practitioner for a patient’s personal use.
A New Zealand Customs Service spokesman said people bringing prescription medicines or controlled drugs into New Zealand needed a prescription or letter from their doctor, and whoever was importing the medicine was required to pay the GST.
GST was not levied on drugs provided free as part of a clinical trial, the spokesman said. But medicines provided free of charge beyond the clinical trial stage had a ‘‘customs value’’ associated with them and were subject to duty and GST like other imported goods, as defined by the Customs and Excise Act.