2nd Asia-Pacifiic Lysosomal Conference 2019

Another call to action for all Lysosomal families – June 2011

The saga continues. Last year we called on you all to visit National Party MPs as part of our campaign to get a better Exceptional Circumstances Scheme in place so our therapies could get fair consideration for funding.

As reported in our May 2011 newsletter this process has faltered as Pharmac’s consultation document indicates a much tougher regime that would make it even more difficult to get our therapies funded. And in parallel with that, they have recently declined funding for one of our adults with Pompe disease, in what could be seen as a test case for whether things would change or not, as far as access to these highly specialised medicines is concerned.

We also reported that Jack’s treatment for Hunter disease was at risk because of Pharmac’s game playing about supply and cost issues. Fortunately his continued therapy seems secured for now, so part of our battle is won. However we have major challenges in front of us and we are using access to enzyme replacement therapy for adults with Pompe disease as the test case. Our arguments will be about all of the treatments available for Lysosomal diseases, and the need for a better way of deciding about funding for rare orphan diseases.

See the media briefing notes and a series of 20 questions about treatment for medicines for rare diseases, prepared for our press conference to launch this campaign in Wellington on Tuesday 7 June. National Radio “Nine to Noon” and TV “Close Up” coverage is already confirmed for that day and there is likely to be other media coverage about this.

The essential message in this campaign is that there needs to be a suitable system for determining access to medicines for rare “orphan” diseases because they simply do not fit the standard assessment criteria used for medicines for large populations. In addition, an orphan drugs access programme needs to be put in place by government, because Pharmac does not have the broad perspective needed to develop such a policy framework. Also, and quite importantly, it was an election manifesto promise of the National Party to find a solution for access to highly specialised medicines, and they are the ones who need to deliver on that promise.

We’d like you all to help in any way you can, by ringing talkback radio, writing letters to the editor, or to the National Party MP who covers your area. Writing directly to the Minister of Health would also be very helpful to our cause. Here is a brief outline of the policy issues as they have unfolded over recent years:

  • The National Party’s 2008 election manifesto promise on highly specialised medicines said “access to such medicines needs to be improved” and said “National will work with stakeholders to investigate ways to improve access.........”
  • Ministers appointed a special panel to advise on this issue and the panel (McCormack, Hansen and Quigley) advised in 2009 that the exceptional circumstances scheme should be used as the mechanism to consider applications for such medicines.
  • Health Minister Tony Ryall on 2 August 2010 announced a review of the exceptional circumstances scheme “with a view to providing better access to medicines not funded through the pharmaceutical Schedule”. He added that “The government is determined to see New Zealanders get better access to medicines”. Associate Minister Peter Dunne said in the same release “This review is the key recommendation of the ..... Review Panel, and is part of the New Zealand Medicines Strategy”.
  • Although that review is not yet completed (due in June 2011) all indications from their consultation document and their recent EC decisions are that medicines for orphan diseases will have even higher hurdles to overcome.

Another important part of this debate is the New Zealand Medicines Strategy which specifically mentioned factors such as fairness, equity and community values as important overarching principles for medicine decision making. Despite these provisions in the strategy, Pharmac does not specifically address these issues in its decision criteria. In fact, since the strategy was released in 2007, Pharmac has strongly resisted any explicit acknowledgment of fairness in its processes and decisions. Its 2007 updated version of its policy document  “A Prescription for Pharmacoeconomic Analysis” does not address equity issues in decision criteria at all, while its earlier version of the PFPA did in fact provide useful commentary on how such matters could be included in decision-making.

We have frequently proposed a solution to Ministers and Pharmac that would put a process in place similar to the special programme operating in Australia. Though not a perfect solution, the Life-saving Drugs Programme does provide access for most of these medicines, and thus achieves equitable access for Australians with these diseases. This programme provides access to all of the medicines we are pursuing for New Zealand patients, except for treatment of adults with Pompe disease, which is the subject of current negotiations between the supplier and the Pharmaceutical Benefits scheme.

Thank you all in anticipation of your help.

Jenny Noble
Field Officer | Administrator