Pharmac’s Decision Criteria- Proposal for change

8 February 2014

Last year Pharmac held consultation meetings around the country and also asked for submissions on their 9 Decision criteria. They have now provided a summary of the submissions and have developed a proposal for change.

LDNZ, NZORD and other rare disease groups will be doing an analysis about what all this means for our groups. Once we are clear about what Pharmac intend to do we will be asking families to again make submissions on their proposal for change.

Funding system for costly drugs unfair

21 January 2014

Another story on the inequity of access to medicines. Late last year NZORD and LDNZ submitted an appeal for our two untreated Pompe patients. We are still waiting to hear the outcome. If Pharmac decline the appeal as we expect they will. We will be looking towards court action. read more

LDNZ, NZORD, MDA request Pharmac to reconsider declined applications for Freda and Yakuta

4 October 2013

Finally after many months of work around the Ombudsman’s report and the two public consultations put out by Pharmac, we were able to submit an appeal for Freda and Yakuta to have their declined application reconsidered by Pharmac.

The key points in the appeal are:

  • That Pharmac must address the Human Rights considerations relevant to these cases. In both cases there was failure to address mandatory human rights factors.
  • That Pharmac is the agent for the DHB’s and must consider consistency with the requirement of the District Health Boards.
  • That Pharmac should address the recommendation of a trial period with clear entry and exit criteria
  • That Pharmac must consider community values along with moral and ethical issues.

We expect we wont get a decision on this submission until early 2014 by which time we should hear what Pharmac plans are for the decision criteria and the funding or non funding of eculizumab for PNH.

If the ruling is not in their favour we will be taking this issue to court.

LDNZ joins NZORD in seeking legal advice to Pharmac on Mandatory considerations

18 September 2013

One of the difficulties in dealing with Pharmac has been their preference for a narrow range of considerations, focused almost exclusively on Pharmacoeconomic analysis, measures of cost effectiveness, and budget management. We have encountered strong resistance on their part to take into account broader legal, social, and moral factors, despite there being a multitude of guidance in law, in public policy and in moral philosophy, that they should include such considerations.

NZORD and LDNZ assessed the recent round of consultation on their decision criteria, as posing a significant risk that Pharmac would treat all matters raised as discretionary matters, for them to include or exclude into future decision criteria as they saw fit.

In response to this risk and with legal advice from a team of experienced Barristers, NZORD provided Pharmac with a letter which spelt out matters that theyconsider to be mandatory considerations for them right now, as well as when any future changes are made to their decision criteria.

In the letter they highlighted human rights and health legislation matters, and NZORD maintain they must consider these in all future decision, including those decisions pending on eculizumab, on the appeals we will make following the Ombudsman’s report, and on the future shape of their decision criteria.

Read more at this link (link no longer active) and NZORD’s letter to Pharmac at this link (link no longer active).

Submission on Pharmac’s decision criteria

8 September 2013

Pharmac held public meetings around New Zealand seeking public input into their 9 decision criteria. Almost all the meetings had people from the rare disease community attending.

Did we challenge Pharmac – We think we did, and this was seen at the Tauranga meeting where LDNZ had two people attend and there was someone from the PNH group. It was very clear that the Public thought that the young lady with PNH should have her treatment funded. LDNZ and PNH stood united and told Pharmac what we thought about their decision not to fund treatments for rare disease people and how we thought they could solve the issues of access to treatments.

NZORD on behalf of many rare disease groups made their submission on the decision criteria. Read here. [Link no longer active.]

Submission on Pharmac’s propsal to decline funding for eculizumab

18 August 2013

LDNZ made a submission to Pharmac about their intent to decline eculizuma. Stating that they had failed in their duty to provide the public with the correct information on patient numbers and the cost of the drug. We also stated that patients with rare diseases have the right to life. and that our health system has a duty to address their needs in a far and equitable way.

Read the submission here

NZORD also submitted on this consultation read it here (link no longer active).

Ombudsman criticises Pharmac’s approach to rare diseases

18 July 2013

The Ombudsman has criticised a Pharmac decision from September 2012 on an exceptional circumstances application for a specialised medicine to treat Freda Evans who has Pompe disease. He has suggested Pharmac consider any further comments that Ms Evans wishes to make and give further specific consideration to items they have glossed over in their decision.

Read the Ombudsman’s opinion and a Q&A sheet on the opinion prepared by NZORD.

In the opinion released to the patient on 31 May 2013 and published 12 July 2013 Ombudsman Dr David McGee, who has since retired, raised a number of concerns about Pharmac’s policies and procedures. Specifically in the opinion:

  • He mentioned Pharmac’s documentation describing the decision criteria was “unduly complicated”.
  • He noted that Pharmac has a general discretion to consider applications despite the tight eligibility criteria in the Named Patient Pharmaceutical Assessment (NPPA) policy, but noted that the information on the policy did not explain this.
  • Dr McGee was concerned that Pharmac’s NPPA policy does not record the decision criteria to be used under that policy. He also considered the decision criteria under the NPPA policy ought to be clearly differentiated from those under the Pharmaceutical Schedule.
  • He was concerned that Pharmac didn’t specifically consider the patient’s suggestion for a trial period of the medicine. The Ombudsman said they should revisit this and consider any further comments on this suggestion the patient wishes to make.
  • He noted the lack of evidence in regard to rare diseases and treatments inevitably puts their assessment at a potential disadvantage.
  • Dr McGee specifically noted that the principle of states not abandoning their citizens is of obvious relevance where there is no known alternative method of treatment.
  • He noted a contestable legal argument about whether Pharmac appropriately excludes social and ethical factors from their consideration, though he said it was not his role to make a definitive decision on that legal dispute.
  • He considered that Pharmac should allow submissions to be made by Ms Evans and her supporters about questions of fairness, equity and community values, and consider these alongside submissions made by her clinician when the application is reconsidered.
  • He identified “matters of high policy to be addressed by the Government and Parliament”.

Ms Evans’ complaint to the Ombudsman has been supported by the New Zealand Organisation for Rare disorders (NZORD), Lysosomal Diseases New Zealand (LDNZ), and the Muscular Dystrophy Association of New Zealand (MDANZ). “Our groups are delighted that our concerns have been heard in the system and responses provided that recognise the weakness of individuals against the enormous power of the bureaucracy,” said John Forman, NZORD’s executive director.

The groups have been seeking better accountability and governance oversight of Pharmac for many years. “We were frustrated that approaches to Ministers, the Health select committee, and other officials over the past two years since the development of the NPPA policy, had not resulted in any external scrutiny of Pharmac or response to our concerns,”, says Forman. “At last we have found a part of the system willing to investigate Pharmac and hold them to account, and Pharmac has been found seriously wanting.”

The Ombudsman’s report includes criticism of aspects of the relationship between Pharmac and District Health Boards, and concern was expressed by Dr McGee at Pharmac’s apparent power to bind DHB’s medicine purchasing decisions through a provision it has put in the schedule. Dr McGee said, “I have seen no statutory authority which allows Pharmac to give directions to DHBs, whether through the schedule or otherwise.”

Forman concluded by saying that the report vindicates many years of detailed work the groups have invested in scrutinising Pharmac’s operations. “Pharmac should be commended for the significant achievements it has made with a Pharmaceutical Schedule that provide good savings and good pharmaceutical outcomes for a great number of New Zealanders. But it is time for Pharmac to acknowledge that a strict health economics focus without a moral compass is abandoning patients at the margins. We hope that this opinion will cause Pharmac, government, Ministers, the Health select committee and other officials, to respond with serious scrutiny and review of Pharmac’s policies regarding specialised medicines for rare diseases.”

NZORD will assist with a further application for funding of the treatment (Myozyme) that Ms Evans needs, as soon as discussions with clinicians and other advisors are completed.

John Forman
Executive Director

Freda Evans medical nightmare

12 July 2013

Campbell Live did a follow up story on Freda highlighting her stays in hospital and the continued lack of access to Myozyme. Freda asks the question wouldn’t it be cheaper to treat her now rather than paying for the many hospital stays.

Campbell Live says “Why don’t we just fund the drug Freda needs”? See the interview here (link no longer active).

Pharmac plays fast and loose with facts and figures for vital life-saving treatments

5 July 2013

More problems with medicines decision making – July 2013

NZORD, the New Zealand Organisation for Rare Disorders, and a group of patient advocacy organisations are calling on Pharmac to withdraw one of its current consultations because of significant errors and omissions in the document. They are also calling on the government to remove such decisions about orphan medicines from Pharmac’s responsibility, as they have shown they cannot effectively and fairly manage such decisions for very small patient groups, under their current framework. Full press release here. (link no longer active)

Big implications for funding of treatments for rare diseases

1 July 2013

Here we go again!!! – A call to Action for the Lysosomal Community.

Pharmac are consulting on their decision criteria. In parallel with this they are also consulting on a proposal to decline funding for a new medicine for treating the rare disease Paroxysmal Nocturnal Haemoglobinuria.

The battle that the PNH group are facing now is the same battle we have for our Lysosomal enzymes. If Pharmac get their way and they get good public support to decline this treatment the door will be effectively closed to the funding of rare disease medicines. We cannot allow this to happen!!

We encourage you to participate in these consultations to ensure the voice of rare disease patients is clearly heard by Pharmac.

To find out how you can make submissions read more here (link no longer active).