Allyson Lock

I was diagnosed with Pompe Disease at the age of 46, in July 2010. I had symptoms for many, many years and had been sent from one specialist to another on a frustrating and tiring quest for diagnosis.

After my diagnosis I found that our government refused to fund the only medication available for Pompe. Myozyme is now available in nearly 80 countries, including Australia, but in New Zealand it is only funded for infantile patients - although we don't have any. Very generous, thank you PHARMAC.

Due to not being able to get treatment in my own country I decided that the only way to save my life was to seek out a medical trial. Fortunately, there was one recruiting in Florida, USA.

After six months I returned to NZ and began my fortnightly trips to Brisbane, Australia. This went on for three years until the trial site moved to Auckland. Then I travelled fortnightly to Auckland for 18 months until the trial was cancelled abruptly. The reason given was that the drug company wouldn't make enough money from the drug, and they needed to be responsible to their investors and shareholders. Yes, I know, it was terrible! Not only had this drug kept me alive and stable for five years, it was also my ONLY option for treatment.

Nine months later, Genzyme Sanofi gave me compassionate access to Myozyme, the drug that PHARMAC won't fund. I was so grateful!

I would like to say that our government and its entity called PHARMAC has since seen fit to treat New Zealand patients for Pompe disease. But no. Despite our best advocacy efforts, successive governments have failed us. Despite overwhelming evidence that treatment helps us, they still refuse to fund us. Despite so many countries, including poor ones, funding treatment for their citizens, our government refuses to see our lives as worth saving.

They tell us the medicine doesn’t work as well as people say it does. Well, I can tell you that it does. I’ve been on Myozyme for 9 and a half years, and I’m still alive! I wouldn’t be if not for that treatment. What more proof do they need?

The Government has promised us that they are working on making processes better. Remarkably, these processes are worked through then the decision is made that they were right all along and the medicines aren’t worth it.

From a patient’s perspective, we don’t care about processes. We care about getting treatment to save our lives. That’s it! We don’t care how you get there, as long as you get there in a timely manner. When I say timely manner, I mean when we are diagnosed, we instantly have access to the medicine we desperately need.

One good thing that we have achieved is to have medicine funded for infants with Pompe disease. BUT, the bad thing is, the Newborn Screening Unit which is part of Health NZ refuses to add Pompe to the newborn screening panel. Babies born with the infantile version of Pompe rapidly decline in health, then die. This has happened in New Zealand and will continue to happen. Because Pompe is such a rare and obscure disease, no one hears about this, but I can assure you this is true. How would you feel if it was your baby that was allowed to die because a small committee of gatekeepers are playing God?

When patients overseas are diagnosed, many instantly have a selection of 3 different Pompe medicines to choose from. If you’re an adult in New Zealand, you have ZERO. NIL. NOTHING. This has never changed in the 16 years since I was diagnosed.

Interestingly, I was diagnosed with uterine cancer a year ago. The moment I received my diagnosis I was showered with help and treatment options. If only the same could be said for people with Pompe disease, and all of the other rare disorders.