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Appeal over decision to deny funding for rare disease?

Pharmac to consider appeal over decision to deny funding for life-threatening disease.

Three patient advocacy groups have won a concession from Pharmac who will consider at their 27 September board meeting an appeal against their earlier decision to decline funding for Myozyme, a specialised enzyme replacement therapy. Myozyme treats Pompe disease, a rare Lysosomal disease that has similar symptoms to some forms of muscular dystrophy. Without treatment Pompe disease is fatal, leading over several years to increased muscle weakness and respiratory failure.

Lysosomal Diseases NZ, the Muscular Dystrophy Association of NZ, and the NZ Organisation for Rare Disorders filed an appeal in June to challenge the facts and the process Pharmac relied on when it declined an exceptional circumstances application for funding of Myozyme for Masterton mother Allyson Lock.

Mrs Lock is now in Florida on a clinical trial for a second-generation form of the enzyme replacement therapy and has just had the second infusion in a six-month course of treatment. Her family fundraised to get her to Florida and she was assisted by the three support groups and public donations.

LDNZ, MDANZ and NZORD consider the appeal very important because back in New Zealand there are three other patients with Pompe disease who were unable to travel to Florida for the trial, and whose exceptional circumstances applications will also be considered by Pharmac at the same board meeting. The matters in Mrs Lock's appeal are directly relevant to their applications too.

The appeal focuses on three key points:

1. a significant error by Pharmac staff who estimated up to 100 patients with the disease in New Zealand. Research by the three support groups shows that Pharmac misread the literature on incidence and prevalence and overstated the likely number by more than 10 times.

2. an apparent failure by Pharmac staff to alert the board to an offer by the pharmaceutical company to discount all the therapies in the Lysosomal diseases group, thus showing a more negative budget impact than would be likely.

3. a failure by Pharmac to factor into its decision the social and moral issues which are specified in the government's legislation and health strategies.

Auckland patient Freda Evans, New Plymouth's Laurie Hill, and Invercargill's Dean Jones are the three new cases up for a funding decision at Tuesday 27th board meeting of Pharmac. They have similar symptoms to Mrs Lock, and a pressing need for treatment with Myozyme. They are anxiously waiting on the outcome of the meeting. Myozyme is provided for patients in just about every OECD country. New Zealand stands out in its failure to see its patients treated, despite a consensus among treating doctors here that treatment should be provided.

LDNZ, MDANZ and NZORD have also noted the significance of Pharmac's decision to consider their appeal. They say getting Pharmac to accept an appeal process direct to them has been a breakthrough in dealing with the organisation. They add that the Herceptin case showed the limitations of court action against Pharmac's administrative and funding decisions, and the web of protection it has been given by the legislation. Even Health Minister's have very little influence over Pharmac's operations, the groups say. But protecting Pharmac from interference in its financial and medicine evaluation decisions, has left it relatively free to follow its own course on other matters, and the groups say it has not recognised important moral duties it has to patients.

The groups say that in direct discussions with Pharmac, the staff have often been dismissive of any notion that community values should be considered. This reinforces the concern of the groups that Pharmac's considerable competence in delivering medicine for the bulk of the population within its budget, overshadows its inability to effectively deal with rare and complex cases when moral issues can readily be brought to the fore by the circumstances. This leaves New Zealand patients at risk of seriously deficient decision-making, say the groups.

Since the 1990s enzyme replacement therapies have been developed for 6 of the 45 Lysosomal diseases but currently only two are funded by Pharmac. The groups say that each funding decision has been a major battle. They would like a change to the process so specialised medicines for very rare diseases did not have to go through such difficulties each time.

The current problems also highlight the failure of Pharmac to deliver the Health Ministers' expectations of a suitable process for dealing with specialised medicines for rare diseases. Three years of reviews to work towards the National party's commitment to finding a solution to this problem, came to nothing when Pharmac's recent review of the exceptional circumstances scheme, by their own admission, failed to solve the problem, despite the expressed wish of Tony Ryall and Peter Dunne that the review should achieve this.

LDNZ, MDANZ and NZORD say that the appeal to Pharmac is a last ditch effort to solve this case. They say that if the board of Pharmac cannot adequately deal with the issue the government should step in and shift the decision-making about highly specialised medicines to the Ministry of Health, who already manage a number of complex funding applications on behalf of District Health Boards. Similar systems are in place to decide funding issues for highly specialised medicines in several other comparable countries.

The groups are determined that these patients should not be abandoned to the ravages of a fatal disease when an effective therapy is available. That would be a death sentence that would be unacceptable to fair-minded New Zealanders.


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