Allyson Lock's life is the stuff of Hollywood films.
She has a devastating, rare and terminal condition which has led to her travelling the world looking for a cure.
Pompe is a disease where muscles slowly deteriorate and it's terminal.
She has travelled the equivalent of five-and-a-half times around the world in the last six years as it's the only way she can get the miracle drug which helps her improve the quality of her life.
But now the trial has ended and she will have her final treatment in July.
So why is Ms Lock's fairy tale-ending now on the cutting room floor?
Pharmac has extensively examined the evidence for funding treatments for enzyme deficiency disorders, including Pompe Disease. Evidence has been reviewed by PTAC which has recommended funding be declined. To date our view has been that the evidence of benefit for these treatments is not sufficient to justify the high level of investment that would be required.
Pharmac has a policy for assessing individual patients' requests for funding for medicines not listed on the Pharmaceutical Schedule. This is the Named Patient Pharmaceutical Assessment policy.
NPPA is guided by a set of principles, which can be viewed on the Pharmac website. One of the principles is that NPPA complements and does not undermine the Schedule listing process. In practice, this means that applications for medicines that have already been considered for groups of patients through the Schedule listing process, would not meet the NPPA core principles.
As all individual cases are different, it is difficult to comment on the outcome of an individual case without it being assessed by Pharmac.
Watch the video for the full Story report.
