Rare Disease Day Award goes to Pompe Fighter
Rare Disease Day Award goes to Pompe Fighter for Patient Advocacy and Support
Allyson Lock will be awarded the Rare Disease Day Patient Advocacy and Support Award on Rare Disease Day, Thursday 28 February. The award will be presented at Government House by Her Excellency the Rt. Hon Dame Patsy Reddy, Governor General of New Zealand.
Rare Disease Day aims to raise awareness amongst the general public, medical people and decision-makers, about rare diseases and their impact on patients' lives.
Allyson is being recognised for the extraordinary difference she has made through advocacy and patient support in genetic and rare diseases in New Zealand. Allyson was diagnosed with Pompe disease in July 2010. Pompe disease is a fatal disease which causes muscle wasting and respiratory failure as it attacks the diaphragm - the muscle used to drive breathing. Since being diagnosed she has fought tirelessly to get funded treatment in New Zealand, not just for herself, but for the whole Pompe community and all those who may come after her.
The treatment that is funded in over 80 other countries is Myozyme, a proven enzyme replacement therapy which slows down and halts the progression of the disease. It is proven to reduce mortality by 59%. But in New Zealand, PHARMAC has said the cost outweighs the benefits of treatment, in their view longevity of life is not worth the expense!
Allyson has campaigned for over eight years to raise awareness about the lack of funding for rare diseases in New Zealand. She founded the charity the New Zealand Pompe Network to raise awareness, attend international rare disease conferences and support the other ten patients and families in New Zealand. She was instrumental in getting New Zealand patients on the BioMarin trial, the Amicus trial and liaised with Genzyme to get patients on their International Compassionate Access Program. Currently these are the only avenues for patients in New Zealand to access life saving treatment.
Allyson was shocked and humbled when she heard she had been nominated and had won the award. She said,” I am very surprised and grateful. Although I really don’t feel I deserve it until our government does the right thing and funds the medicine we need to live”.
Sanofi/Genzyme, the company who make Myozyme, entered a tender for Myozyme to PHARMAC late last year at the invitation of PHARMAC. However, this was declined by the Rare Disease Subcommittee yet again this February. Allyson says, “While it is extremely disappointing, frustrating, and upsetting to get that news, I will continue to fight PHARMAC to see Myozyme approved in New Zealand. ”
Allyson also calls for this current government to look at the way PHARMAC is allowed to operate. “PHARMAC is a government funded agency, funded with taxpayer money, it should be accountable to the government and to taxpayers. Yet day after day, there are stories of how patients with various diseases, particularly rare diseases and cancers, are left to die by not having access to modern medicines that most overseas countries fund for their citizens. No government funded agency should be allowed to operate this way and this government owes the citizens of our country better than that!”