2nd Asia-Pacifiic Lysosomal Conference 2019

Access to Medicines Campaign

3rd August 2018 - A summary of recent developments

After a lot of activity on this issue over the past 6 months, a phone conference was held with a number of other support groups that are interested in these difficulties with medicine access, to brief them on the work done. This resulted in support for the approach taken and thanks to LDNZ for the leadership taken on the issue. The groups involved endorsed a joint approach from LDNZ and NZORD to pursue these issues on behalf of the groups interested.

Here is:

3 April 2018 - Meeting the Minister of Health Today

For the first time in many years, we got to speak directly to the Health Minister. LDNZ chair John Forman was pleased to meet with Dr David Clark after years of being blocked out by Health ministers in the previous government. The discussion centred on exactly where things are going with policy regarding funding for medicines for rare diseases. It was pleasing to hear the Minister commit to making progress in getting equity into the decision process. He clearly understood the disadvantage our diseases face and he is asking Pharmac for more information about how they will include equity in their next $5 million funding round.

John had the opportunity at this meeting to put a proposal to the minister on how a fairer system could be developed for funding of orphan drugs, and has subsequently had discussions with Ministry of Health officials about the practicalities of how such a system could work. We hold some optimism that there are "live" possibilities for us, and that all is not lost.

21 February 2018 - The Health minister to the rescue?

In a radio interview on Nine to Noon, NZORD Chief Executive Collette Bromhead and Health Minister David Clark discussed the policy shifts regarding funding of treatments for rare diseases. Despite the lack of policy in the coalition agreement, the Health Minister confirmed that he is committed to progress in funding treatments for rare diseases and is seeking advice on how this can be achieved within existing structures. Specifically the Minister emphasised he is wanting to see equity at the core of decisions for this group as a new round of funding of $5 million per year for 4 years, kicks off this year. A glimmer of hope remains.

Read the interview here. Listen to the interview here.

November 2017 to February 2018 - Two steps forward and three back?

We held great optimism for progress on funding of medicines for rare disorders, given the formation of a new government with the three parties (Labour, NZ First and Greens) who each made specific promises in line with our policy, of a separate fund, managed away from Pharmac, and with patient representation in the decision process. But slowly the wheels seem to fall off. The coalition and confidence and supply agreements did not mention this topic. The select committee's December report on Samantha's petition failed to nail the key issues and "kicked for touch" while waiting for Pharmac to review its first $5 million pilot fund for rare diseases. A meeting early in February 2018 with three Labour members of the Health select committee confirmed that the plan was shelved in the coalition negotiations to form the new government. We have sought an urgent meeting with the Minister of Health about this.

19 September 2017 - Pompe patients feature on Seven Sharp

Samantha Lenik and Freda Evans featured on TV current affairs clip about funding of treatments for rare diseases, and in the process doing a wonderful job of showing the human side of this issue, plus some heartwarming responses from members of the public to their plight. Watch the programme at here.

4 September 2017 - Political party election promises on medicine funding

A special edition of the Medicines New Zealand newsletter published details of responses form political parties about the medicine funding policies for the upcoming general election. Very pleasing to note that a combination of statements from Labour, NZ First and the Greens all confirm a new direction for funding of orphan drugs for rare diseases, with a separate fund managed away from Pharmac, and with patient involvement in the decision process.Similar promises are contained in messages sent to the NZ Pompe Network and published on their website. http://nzpompe.network Many years of hard work are starting to show positive results. The commitments we won from these parties in 2014 remain solid as we enter the general election period.

19 August 2017 - Labour politician confirms plans for orphan drugs fund

Labour politician confirms plans for orphan drugs fund for NZ At a campaign meeting in the Hutt Valley, Labour politician and former health minister, Annette King, confirmed Labour's policy of a separate fund for rare diseases, managed away from Pharmac, and with consumer involvement in the decision-making process.

19 July 2017 - Writing again to the Select Committee

Writing again to the select committee in response to Pharmac's submission Following the invitation from the select committee chair, we submitted further information in rebuttal of Pharmac's statements to the committee earlier in the month, and gave some more information about the weaknesses in their approach to funding medicines for rare diseases. Read our submission here.

5 July 2017 - Listening to Pharmac's response to Samantha's petition

A very interesting experience as Pharmac's chief executive appeared before the select committee to give their response to Samantha's petition. Labour's Health spokesperson David Clark grilled him on their approach to equity and it was informative to see the CEO twist the discussion to equality. This is the nub of the issue, as treating rare disease equally will most often see them disadvantaged, while an equity approach is needed to balance the inherent disadvantages of rarity and give them a fair go in the system.

The discussion confirmed that David Clark really "got it" while Pharmac either doesn't understand the distinction, or deliberately blurs it to retain its narrow interpretation of its mission. LDNZ Chair, John Forman, asked the committee if he could have a right of reply to comments made by Pharmac's CEO, and was invited to put additional comments in writing to the committee.

Interestingly, on leaving the committee room, John was confronted by Stefan Crausaz who expressed his "shock" the we'd be so bold as to ask for a chance to rebut their statements. In a rather tense discussion in the corridor, Stefan made it abundantly clear they were sticking to their narrow approach and not persuaded to give consideration to equity for rare disease patients. He and Pharmac clearly understand the difference between equity and equality, but choose to insist on the equality approach and remain out of step with our entire health system, and all of the guidance in our health legislation and health strategies. It is distressing to see them act so arrogantly and single-mindedly, while also spending so much time with clever PR and spin, to disguise what they are actually doing.

28 June 2017 - Attending the Health Select Committee

Samantha fronted up to the committee to speak to her petition and her written submission, and was supported by John Forman, Chair of LDNZ. Samantha's powerful but dignified presentation was an inspiration to see. We hope the messages will be taken on board and a report produced that deals with the great disadvantage faced by rare disease patients.

10 March 2017 - Samantha's submission to the Health Select Committee

Following a request from the select committee that Samantha provide a written submission about her petition to the committee, Samantha was assisted by LDNZ to provide them with a submission that was submitted in March 2017. We were inspired by Samantha's clear messages and personal courage in doing this, and very pleased to have supported her with the submission. Read the submission here.

28th February 2017 - Pharma Company gives up on Pharmac

Sanofi Genzyme developed Myozyme, a complex biological treatment for Pompe disease, a lysosomal disease, over ten years ago. Since that time funded treatment of affected patients has spread to 76 countries. Over that decade, NZ's drug purchasing agency Pharmac has consistently refused to fund it, leaving the 10 patients here abandoned and facing relentless decline in their health and quality of life.

After substantial renewed efforts to negotiate with Pharmac, and with a recent recommendation from Pharmac's PTAC committee, released early February, that funding should be declined, the company has decided to provide the treatment free to four NZ patients. Read more here.

13th December 2016 - Petition delivered to Government

Samantha Lenik and patients with Pompe and Fabry disease and their supporters delivered the petition to the government yesterday requesting that the government look under urgency to ensure Pharmac has enough funds to properly deliver on the rare disease drug policy.

Here the the media interviews:

The reading of the petition in the House

Radio NZPatients Call for Funding of Rare Disease Medicines

NZ Herald: Children of women with rare disease ask Santa for mummy's medicine

Rodney Times: Petition for rare disease funding delivered to Parliament

CheckOrphan: The presentation of the petition made it to CheckOrphan

Samantha and the affected patients along with LDNZ thank everyone who participated in the signing of the petition and left some truly amazing comments that we will be forwarding onto Pharmac. Thank you everyone.

30th November 2016 - Enzyme Replacement therapies - 3 new decisions and one more review

There is some very positive news to report this month, and some disappointment as well. LDNZ is delighted that Pharmac has approved the listing of Myozyme for infantile Pompe disease on the schedule. This is the first approval for this treatment and putting it on the schedule means that any baby diagnosed with this disease, and who fits the treatment guidelines, will be automatically given treatment, avoiding delays and anxious waits for special consideration under the exceptional circumstances scheme. Read more.

8th November 2016 - Rare Disease Mum Abandoned by her local MP

For Samantha Lenik’s potential life-changing Petition to make it to Parliament, Samantha needs an MP to personally present the Petition to the House of Representatives. Unfortunately, Samantha’s local MP Mark Mitchell (National MP for Rodney) declined to receive the Petition, and with one week to go she is scrambling to find an MP who is willing to help her and other rare disease patients. Read more here.

1 November 2016 - LDNZ submission on proposed funding of 3 enzyme replacement therapies

We are making progress and it is very satisfying to see some results. Just 2 weeks ago Pharmac issued for consultation a proposal to list 3 additional ERTs on the Pharmaceutical schedule, meaning automatic funded access for those who meet the eligibility criteria. The proposed listings are for ongoing treatment with Myozyme for infantile Pompe disease, short-term treatment with Aldurazyme prior to transplant for Hurler disease, and short term treatment with Elaprase prior to transplant for Hunter disease.

Also announced in background information to this proposal is advice that Pharmac's specialist advisory committee, PTAC, will this month be reviewing the evidence for Myozyme for adults with Pompe disease, in response to another submission from Sanofi to have this treatment listed on the schedule. About time too. And hardly a coincidence that our petition to get funded access to these therapies kicked off in earnest just 6 weeks ago.

Here's the submission we made in response to their proposal.

October 27th - Mother with Pompe a rare genetic disease petitions parliament

Samantha Lenik hopes to get access to a life changing treatment for Pompe disease, which isn't funded in New Zealand. Read more here.

6 October 2016 - Our mesage to all Members of Parliament, requesting support for our medicine access campaign

As we kicked off our petition about medicine funding and a fair deal for rare disease patients, we sent this message to all MPs, asking them to take particular note of the issues and show their support. We are pleased to note a we have had a small number of responses in the following days. Read more…

10th Oct 2016 - Press Release - Another Medicine Funding Petition launched

New Zealand Rare Disease Patients need funding too.

Samantha, Freda and Yakuta, a mother, a grandmother, and a young woman with her life ahead of her, have launched a petition to Parliament aimed at ensuring Pharmac have enough funding to implement its new policy on medicines for rare diseases, which was announced back in April 2014, and to give urgency to doing so. Read more here.

March 2016 Submission to the UN Secretary-General's panel on Access to Medicines

LDNZ contributed its years of experience with problems relating to medicines access in New Zealand, in a submission made by LDNZ Chair, John Forman, to a special panel set up by the UN Secretary-General. This panel is taking a global view of problems relating to the tension between the intellectual property rights of drug developers to set the price of their products, and the rights of patients to be treated for their health conditions under the "Right to Health" provisions of the international human rights framework.

The submission highlights the significant conflicts of interest held by most governments who are the bodies charged with meeting the rights of patients within their countries, but in most cases are also the payers and decision makers about health system costs. It calls on the UN to actively intervene to ensure governments give more attention to the right to health. Read more at this link.

March 19th - Funded drug shows change for Pharmac

Pharmac's decision to fund a new drug is an important breakthrough for rare disease treatment, an advocacy group says. Drug funding agency Pharmac has approved funding for Naglazyme, a treatment for the very rare Maroteaux-Lamy syndrome which is one of a series of Lysosomal diseases in New Zealand. Radio NZ news itemRead more

March 2016 - Pharmac approves funding of Naglazyme for Maroteaux-Lamy (MPS 6)

It was 11.22 am on 17 March 2016 when LDNZ got the briefing about this - the first listing in New Zealand of a novel treatment for a lysosomal disease since 1999 when Cerezyme was listed for Gaucher disease. This decision is an important breakthrough for LDNZ and represents a significant shift in approach by Pharmac. In the past 15 years Pharmac has been extremely reluctant to provide access to any of the new treatments for Lysosomal diseases. Their resistance required continued strong advocacy from LDNZ, and our efforts have finally been successful. Read more.

February 2016: LDNZ Submission to Pharmac on Naglazyme Consultation

Pharmac's third consultation under the orphan drugs fund is for Naglazyme, for the treatment of Maroteaux–Lamy syndrome (MPS6), which is one of our Lysosomal diseases. LDNZ submitted this response in February 2016 and we wait now for Pharmac's decision. This might be the breakthrough we have waited for, for so long – the possible funding for the first time from this special fund for rare diseases, of a treatment not previously accessible at all by patients in New Zealand. Read our submission.

August 2015 - Pharmac announce first orphan drug to be funded under the contestiable fund

There is a little bit of good news in Pharmac's announcement of approval of the first drug under the $5 million Orphan Drugs Fund. At least one has made it across the line. Although this is a good start, this drug has already been funded under the exceptional circumstances scheme, so this really just extends access rather than adding something new that was not previously available. And don't be misled by their claim of $25 million. It is just a $5 million fund, though guaranteed over time. Meanwhile there is no news about any new funding for orphan drugs not yet available to NZ patients, and even some worrying hints that progress for them is slowing and possibly grinding to a halt.

LDNZ has been contacting Pharmac almost weekly to see how progress is going and to try and push things along. Watch this space. Read more.

27th Feb 2015 - Pharmac in Negotiations over Rare Disorder Medicine

Pharmrac released their latest announcement today stating that they are still in negotiations with several drug companies in relation to orphan drug access. This announcement comes on the eve of International Rare Disease Day. Although this is not the message we had hoped for it’s good news that they are still talking with the companies.

Lets hope Pharmac does the right thing and funds our medicines. We would hate to see winners and losers. However there are grave concerns over the medicine budget for rare disorders. Read the press release here.

14th October 2014 - Pharmac reject entry and exit criteria for Myozyme

After 15 long months waiting for Pharmac to make a decision on a trial for Freda and Yakuta using Myozyme, Pharmac again rejects their appeal.

New evidence was submitted to Pharmac along with entry and exit criteria, all of which was reviewed by PTAC. The committee said that although there is a suggestion that alglucosidase alfa may confer an overall survival benefit they still considered the new evidence weak and insufficient to warrant a change in their previous recommendation to decline funding.

In regards to pricing they said: Given the substantially unchanged evidence of clinical benefit and in the absence of any new pricing information, the conclusion of our revised economic assessment is unchanged. 

So once again we come back to clinical evidence, cost and leaving our Pompe patients with no pathway forward. In our eyes there has to be some concern over what Pharmac will do when the drug companies put in their bids for the 5 million contestable fund for orphan drugs. Will our Lysosomal Enzymes get over the threshold or will they again be put into the too hard basket leaving our patients untreated and leaving us with no pathway to apply for funding?

25th July 2014 – High cost medicines for Rare Disorders – LDNZ submission

LDNZ submitted its submission to Pharmac advising them we are far from happy with their proposal for funding High cost medicines. Read more here.

July 2014 - Serious flaws in Pharmac's proposal for access to medicines.

Patient advocates are rallying support for submissions to Pharmac about the U-turn Pharmac has made on medicine funding for ultra-rare disorders. After repeated refusal to fund novel and expensive treatments for disorders that affect mostly just a handful of cases in New Zealand, Pharmac have announced a special $5 million fund so they do get a chance of being funded here. Read more here.

April 2014 - High cost Medicines for Rare Disorders

Who would believe that right before the next election Pharmac comes out with a discussion document for High cost Medicines for Rare Disorders, with the first line in their document stating - 'We've been doing some thinking about access to high cost medicines for rare diseases and we want your input to help us develop an alternative commercial approach'. Really come on Pharmac!!!!

We have to ask though is Pharmac starting to change the way they are thinking about orphan drugs? -  this would be fantastic to see - but do we trust them? Is this a pre-election bid to shut us down and keep us quite?

We can identify some fish hooks - the proposed funding level for this is $5 million a long way from the $20–25 million we have estimated that would fund all rare diseases where there is a therapy.

The policy document puts emphasis on maintaining the current Pharmac framework and states the usual emphasis on best health outcomes and value for money. This suggests the orphan drugs would still be compared to those for common diseases, which seems to contradict the purpose of a special fund. In that case, the orphan drugs are still likely to NOT stack up against other in terms of cost effectiveness.

February 2014 - Pharmac rejects call for change on rare diseases

Things are not looking good for people with rare diseases. Pharmac gives some explanation about what their proposal for change is about but its clear they have no intention of wanting to help with access to Orphan drugs. Read more.

February 2014 - Pharmac's Decision Criteria- Proposal for change

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

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

4th October 2013 - LDNZ, NZORD, MDA request Pharmac to reconsider declined applications for Freda and Yakuta

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.

September 2013 - LDNZ joins NZORD in seeking legal advice to Pharmac on Mandatory considerations

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 and NZORD’s letter to Pharmac at this link.

September 2013 Submission on Pharmac's decision criteria

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.

August 2013 - Submission on Pharmac's propsal to decline funding for eculizumab

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.

July 2013 - Ombudsman criticises Pharmac's approach to rare diseases

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

12th July 2013 - Freda Evans medical nightmare

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

July 5th 2013 - Pharmac plays fast and loose with facts and figures for vital life-saving treatments

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.

July 2013 -Big implications for funding of treatments for rare diseases

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

15th May 2013

TV3 Campbell Live featured Freda Evans and Allyson Lock, both patients with Pompe disease, one untreated the other treated. Their story showed how unfair our health system is as they will not fund the therapies for this very rare "orphan" diseases.  Pharmac was caught out fudging the facts and refusing to accept the evidence that over 45 countries rely on to provide access to this live-saving treatment. To see the interview click here.

28th Feb 2013

A Rare Disease Day Forum was held in Wellington on 28th Feb which bought together a number of patient groups and affected families to discuss the issues of access to medicines. Click here to see the presentations.

October 2012 - Complaint sent to Ombudsmen

  • We have become so frustrated trying to get anyone to seriously address the issues we have raised, we have taken  a fresh approach.  Working with Freda, NZORD, MDANZ and our Lawyers Elwood Law, we have pieced together a blow-by-blow account of the difficulties experienced and have assisted Freda with a complaint to the Ombudsmen's office. This was submitted on 23rd October 2012.

October 2012 - To the Health Select Committee, to the Minister, to the Ministry, back to the Minister, to Pharmac, to nowhere!!

  • Can you believe it? We asked the select committee to investigate the policy contradictions and we supplied them with extensive information about this. They then suggested we go directly to the Minister of Health, whose office agreed to seek a report from the Ministry of Health policy team.  But before we could brief the Ministry Officials we got a letter from the Minister saying he could not interfere in individual medicine funding decisions and referred us to Pharmac, even though we had asked him to hold Pharmac to account about how they implemented policy, and had not asked him to intervene in the decision.  Pharmac's response was predictably vague and obfuscatory (yes there is such a word - a polite alternative to bulls**t). We were sent into the circle of nowhere, the vortex of bureaucratic double-speak, the last refuge of scoundrels.  Sir Humphrey was a rank amateur.

September 2012 - The Named Patient Pharmaceutical Assessment Scheme does not work!

  • Freda's recent application for Enzyme Replacement Therapy has again been declined. It is clear from Pharmac's response to her application, and to another filed at the same time which was also declined, that the NPPA cannot actually be used as a pathway to access treatment for this condition, because Pharmac has already assessed the therapy for listing on the schedule. And that is in spite of no schedule decision being made.
  • In other words, the exceptional circumstances scheme does not provide for exceptions. It makes exceptions of them and excludes them from consideration under the exceptional circumstances scheme.  We think this has proved beyond any doubt our argument that the new NPPA scheme for exceptional circumstances is actually more restrictive when rare orphan diseases are concerned.

August 2012 - Application submitted for Freda Evans (Pompe)

  • The new Named Patient Pharmaceutical Assessment( NPPA) has been tested with an application submitted for Freda Evans.  The application was submitted in June 2012 and came back in early August with a postponement, giving the treating Dr the opportunity to respond to the Pharmac with further information. This has been submitted and we hope that Pharmac will respond in the correct way and fund treatment for Freda but if they don't we will again need to ramp up our campaign.

June 2012

  • On 20th June 2012 the Marquardt family take their son James (Hunter syndrome) story to Campbell live.  They had been trying to get Enzyme Replacement Therapy for James but were declined on their first application.  When this story went to air they had a second application in front of Pharmac.  This was another major breakthrough which saw Pharmac fund James short term prior to them commencing Bone Marrow Transplant. 

May 2012 - Medicine policy anomalies need to be investigated

  • The NZORD submission below led to us being invited to approach the Health select committee to ask them to investigate the anomalies in medicine funding that leave specialised medicines for rare diseases seriously disadvantaged. This request has resulted in the committee's agreement to ask questions of Pharmac. The link here is our request to them and sets out the main arguments.

April 2012 - NZORD makes a submission to Health Select Committee

  • NZORD  made a submission to the Health select committee about some of the detail of the Medicines Amendment Bill. They stated that while this bill is primarily about issues related to licensing and prescribing medicines, and adapting the rules to be more responsive to innovative practices, they focused in their submission on the inherent discrimination that occurs in many aspects of medicine access for rare diseases.  Click here to read their submission

November 2011 - Pompe patients march on Parliament

  • On Wednesday 16 November 2011, NZ Pompe patients will gather at Civic Square in Wellington at 12 noon to march (or roll in their wheelchairs) to Parliament to meet political party representatives. We will hear from them what their policies are about treating those with life-threatening diseases, and what they intend to do about setting up an orphan drugs policy for all rare diseases. You are welcome to join us. Remember, any family at any time can be affected by a disease like this.
  • Here is our latest press release about this march
  • Here are the political party commitments on Myozyme and orphan drugs (pdf file).

21 October 2011

18 October 2011

30 September 2011

  • Pharmac declines three more applications for Myozyme under Exceptional Circumstances.

August 2011

  • NZORD publishes an analysis of Pharmac's new Exceptional Circumstances policy.
  • This clarifies how Pharmac has failed to resolve the issue of access to specialised medicines. Follow this link to the article to the NZORD website.

9 September 2011

27 June 2011

  • Pharmac issued their new exceptional circumstances scheme and admit in a briefing to groups they "have not solved the problem" relating to specialised medicines for rare disorders.

22 June 2011

16 June 2011

  • John Forman and Chris Higgins MDANZ breifed health Minister Tony Ryall on the issues.
  • We emphasied that Pharmac's Exceptional Circumstances review will not solve the problem that he asked them to address about access to Highly Specialised Medicines.

7 June 2011

  • We called our families into action again asking them to call their local radio station, writing to their local National MP or their local papers, and on 7th June 2011 our Public and Media campaign began. Here is a link to our call to action. This campaign is not over yet, keep checking back for further information as we develop the next stage of our campaign.
  • Here are the media links to the interviews that took place.
    • Radio Interview - 7th June 2011, People with rare genetically inherited disorders are demanding a better deal from the drug-buying agency Pharmac. 
    • Dominion Post - 8th June 2011, Drugs for rare diseases may not be funded
    • Close up TV One - 8th June 2011, interview with Kirsty Peacock and Allyson Lock
    • Pharmac drug ruling "a death sentence" - Taranaki Daily News interview with Laurie Hill
    • TV One Breakfast interview with John Forman
    • Manukau Courier 19th September 2011 interview with Freda Evans - Her battle to get drug funded
    • Wairarapa Times - 15th September 2011 Double blow as rare disease strikes twins.  Allyson's twin brother diagnosed with Pompe disease.