Chairperson’s report to LDNZ Annual Meeting – 30 January 2008

Because this meeting is held later than usual in the annual cycle, I am reporting mainly on events during the 2007 calendar year, as well as referring to the financial report for the year ended 30 June 2007.


Financially we were very successful with grant income for 2006/07 exceeding $60,000 and a spectacularly successful charity dinner and auction bringing in a further $60,000 in September 2007. This puts us in a strong position to carry out many of the tasks ahead of us in the next few years, though future fundraising targets are also much higher because of the planned activities.

Family meetings

The second half of 2006 included a series of three family meetings in Tauranga, Palmerston North and Christchurch, resulting in a significant number of families having the opportunity to meet each other and learn about Lysosomal diseases and from each other, and in October 2006 we supported a delegation of more than 20 family members to attend the Australian MPS conference. These events provided plenty of opportunity for families to be involved and to get the information and support they needed. We continued this support for families to get to meetings in 2007 with two families attending the Australian Batten conference, three to the ISMRD conference in the US, and three going to the NZORD conference.

Support for families

Specific help was given to those experiencing particular difficulties with disability support assessments and with access to health services. Some of the individual cases can take up a lot of time and some involve travel costs for LDNZ. We are very fortunate that fundraising has enabled us to employ Jenny and meet costs to ensure this support continues to be provided.

Family contacts

Referrals of new families to LDNZ are very few in number. This continues to raise concern that families may not know about the information and support we can offer. In the past some have been in near crisis situations when contact was made. We need to get better communication with clinical services so appropriate referrals are made.

Access to quality services

There have been many significant problems emerging over the past few years with many of our families struggling to get adequate responses from the Needs Assessment system for disability support services, or suitable follow-through on clinical issues co-ordination between Paediatric and Adult health services, or between local DHB and regional and tertiary specialist services. Jenny and I put a huge amount of time into preparing information on these and submitting two complaints to the Health and Disability Commissioner early in 2007. The Commissioner was very concerned about the issues raised, took one case on for specific investigation by an appointed mediator, and referred the rest to the Director General of Health to look at the systemic issues involved.

The complaints process, in conjunction with Jenny’s direct advocacy work for families, seems to have resolved many of the disability support issues, but the health service delivery issues have not been well addressed by the Ministry, and the commissioner’s jurisdiction on some of them may be limited, meaning that considerable follow-up work will be needed this year as we directly approach the Ministry and politicians about these issues.

Promotion of LDNZ at Conferences

In November 2006 Jenny and I attended the Paediatric society of NZ and the NZ MedSci congress with our display posters and brochures, and followed up this publicity and promotion activity at the NZBio conference and the Human Genetics meetings in the first half of 2007. We also had one of our family members take the display to the Neurological Open Day in Auckland. LDNZ certainly gained a lot of profile from these events.

Publicity material

Our posters, brochures and banner were produced over this time to give us quality material for publicity opportunities as well as material to provide for genetic services and clinics to hand out to patients and families.

Medicines strategy

We had significant input during 2007 into the policy discussions on a medicines strategy for New Zealand. We contributed financially and put in a huge number of hours into the research and preparation of the Access to Medicines submission to the Ministry. Our efforts seem to have paid off and the strategy and action plan seem to have severely clipped Pharmac’s wings, set the scene for a more open and transparent budget setting and decision-making process for medicines. Significantly, the discussion paper on high cost therapies put up by Pharmac as part of the policy development has NOT been incorporated into the strategy. This is a major victory as their themes were very unhelpful to our aims of getting subsidy for therapies for our diseases. There is still work to do on how these decisions on high cost therapies will evolve, but blocking their narrow cost/utility approach is a triumph worth celebrating.

The medicine strategy and action plan is a platform for significant policy change but it also implies a lot of work on our part, in partnership with NZORD and the Access to Medicines Coalition, to ensure it is applied well. Access to Enzyme Replacement Therapies and other Lysosomal treatments will be absolutely dependent on good application of the strategy. The battle is certainly not won yet but we may have just staved off a major defeat.

Carers Strategy

During 2007 we participated in a lot of work associated with the consultation process on this strategy, through the NZ Carers Alliance, and progress has been very positive. We expect the government to announce a Carers Strategy in the next few months.

Maintaining Jenny’s role

Our success with grant writing has enabled us to continue funding Jenny’s role since April 2006 and we had news late in 2007 that the Todd Foundation will give core funding for this role for another two years. We still need some additional top-up funds to ensure the honorarium is a better reflection of her value to us, and that is absolutely undoubted. Our ability to secure Jenny’s continued role has certainly contributed hugely to the total activity of LDNZ and the immense value to affected families.

Trust accountability

The trustees met in person in September 2006 for the Annual Meeting and to plan activities for the year ahead. Since then, activities based on that budget and strategic plan have been carried out mainly by Jenny with regular reporting to the chair of the Trust, with additional issues being referred to the trustees by email for decisions. Jenny and John have also organised work planning meetings by phone or in person approximately every three months.

Conference hosting for 2008 and 2010

The crowning achievements for the past year were winning the hosting of two major conferences in this part of the world. The 25th anniversary conference of the Australian MPS Society in Christchurch will be held in parallel with the second LDNZ conference in November 2008, and we will invite other groups in the Asia-Pacific region to join us too. We aim to build significant links to improve research, clinical care, access to therapies, and policy improvements for rare diseases in the region.

The 2010 international MPS conference will be held in Adelaide thanks to a successful joint bid by LDNZ, LDA and the Australian MPS Society, to jointly host the meeting.

Both of these conferences will involve considerable work, fundraising and input from LDNZ and will keep us, and Jenny in particular, very busy for the next few years.

Other items of note

  • We are pleased to see that Callum Wilson’s paper on ERT treatment for Gaucher disease has been published. This work was supported by LDNZ.
  • Jenny’s work and experience with bisphosphonate treatment has resulted in more New Zealand families having access to this medicine and she is helping to set up an international consensus meeting on dosage levels for those with Lysosomal diseases.
  • Our newsletter production was down to two issues for 2007, as a result of extensive work needed for the charity dinner and the two conference bids.
  • More Enzyme Replacement Therapies were given marketing licences and funding approvals in various parts of the world last year, making a total of six treatments now available and more in development. As yet, only one of these is subsidised through Pharmac.
  • Jenny has filed an application for registration with the Charities Commission and is preparing an application for GST registration.

Challenges for the future

2008 will be a very busy year with the following topics at the top of our agenda:

  1. Promoting newborn metabolic screening of lysosomal diseases.
  2. Follow-up of service access and clinical care issues.
  3. Another charity dinner planned for this year.
  4. Organising the November 2008 conference and preparing the groundwork for the 2010 event.
  5. Continuing work on the medicine strategy implementation, as the key tool to gain access to ERTs in New Zealand.
  6. Plus all the usual work of family support, website maintenance, newsletter preparation and lobbying of officials and other decision makers.

Thank you

Thank you to LDNZ trustees for your continuing support and special thanks to Jenny Noble who makes so many things happen for our organisation and for our families.

John Forman