NEWSLETTER
Supporting fair access to medicines for rare diseases
The Muscular Dystrophy Association, Lysosomal Diseases NZ and the NZ Organisation for Rare Disorders, are gathering at
Civic Square in Wellington at noon on Wednesday 16th November to march to Parliament seeking fair access to specialised medicines for rare diseases. You are invited to join them to
support their cause.
Leading the campaign for an orphan drug access policy are patients with Pompe disease, a serious muscle wasting disease
that without treatment will result in respiratory and cardiac failure. There are just a handful of Pompe patients in NZ.
Four have had their funding applications for Myozyme declined because Pharmac’s decision criteria assess this new
treatment against treatments for common diseases. The disease rarity puts this treatment at considerable disadvantage
when cost comparisons are considered. Fairness and equity have not been addressed by Pharmac in these decisions.
There is good evidence that Myozyme can improve muscle function in patients with Pompe disease and halt the decline of
this degenerative and life-threatening disease. This case offers a test for future access to innovative treatments that
may be developed for other diseases that are currently untreatable.
The patient groups are seeking commitments from all political parties to an orphan drug access policy that will give
fair consideration for very rare diseases. Most of the major parties have agreed to meet the march at Parliament and
state their policy relating to treatment of Pompe disease and the development of an orphan drug access policy.
ENDS