Access to costly but life-saving treatment for rare diseases comes down to looking cute on TV or knowing a politician, sufferers say.
Allyson Lock is one of only eight Kiwis diagnosed with Pompe disease, an incurable degenerative disorder. Untreated, it can be fatal, but Mrs Lock of Masterton and other sufferers have been refused treatment because of its cost.
A medication called Myozyme is available, but it is expensive and gets no funding from government drug-buying agency Pharmac. Costs vary according to a patient’s age, weight and severity of illness, but in Mrs Lock’s case have been estimated at $575,000 a year.
She said patients with similarly unusual illnesses had obtained funding for expensive treatment, which was unfair. “They say it’s too expensive, but there are other treatments that cost more. If your family can get you on TV, if you’re a little kid, if the public gets into an uproar, you get funded. That’s what it comes down to.”
It’s probably true that public sympathy plays a role in decisions. But regardless, these are difficult decisions. There is obviously a limit at which the Government can fund treatment, as every dollar spent on one treatment is a dollar not spent elsewhere. $575,000 a year is hugely expensive. I’m not saying what the limit should be, as fortunately not my decision. And you can only have empathy for those with such conditions that need hugely expensive treatments.
New Zealand Organisation for Rare Disorders executive director John Forman raised the prospect of legal action against Pharmac’s funding refusal, and said it was unacceptable every other developed country was treating its Pompe patients, except New Zealand.
I’m not sure that is quite correct. In some countries I believe it is funded for juveniles only, which is a different variation of the disease.
It would be interesting to know what are the most expensive treatments that are funded by Pharmac. Is there a hard limit, or is it a combination of price and effectiveness?