NZORD Newsletter 2014 #1 - 17 February 2014

Hello everyone,

In this 2014 Rare Disease Day special issue:
1 – Support the Rare Gems in our community.
2 – Genetics Otago hosts Rare Disease Day lectures in Christchurch and Dunedin.
3 – Many families organising events around the country.
4 – It’s a global effort.
5 – And amid the celebrations, a reality check.


1 – Support the Rare Gems in our community.
Yes, the annual rare disease day is on again, locally and internationally, and it’s a chance for individuals and small groups to raise awareness and raise funds for their chosen condition. See the NZ Rare Disease Day website for details on organising events, downloading posters, and getting funds to your group – either directly to them or, if they don’t have a bank account, we can hold the donations in trust for them. If you don’t have a particular rare disease group to support, donations can be made directly to our research efforts at

2 – Genetics Otago hosts Rare disease Day lectures in Christchurch and Dunedin.
Our grateful thanks to Genetics Otago and the University of Otago who are organising evening lectures on 28 February. The Dunedin event will showcase the work of some of New Zealand’s leading researchers on rare diseases, and the Christchurch event will have a keynote address about new genetic technologies and their impact on rare diseases.

3 – Many families organising events around the country.
The range of events includes:
·        Mufti days and coin trials at schools.
·        A fundraising barbeque outside a local Mitre 10. 
·        Rare disease day displays in many public libraries.
·        A fundraising movie night.
·        A quilt raffle.
·        Pampering sessions.
·        A scouts fundraiser.
·        A trip to the Bruce Springsteen concert for one lucky patient.
What activity can you organise to boost the profile of rare diseases and raise some funds for your favourite group?

4 – It’s a global effort.
Rare disease day is an international day, with over 60 countries and hundreds of patient groups participating. The international theme for this year is “Let’s join together for better care” emphasising the need for collaboration to get the best outcomes. So remember that while you celebrate the day here, many others around the world will also be raising their hands for rare diseases. Check out the official Rare Disease Day video with its emphasis on the caring role.

5 – And amid the celebrations, a reality check.
There are still many New Zealanders with rare diseases who need access to new treatments that have been developed for their condition, but which are not funded by our health system. While we raise awareness about rare diseases in a celebratory way, and raise funds for support groups, let us not forget the serious inequities that occur when patients are denied access to these therapies. The major block to these therapies is Pharmac and the extremely narrow view it takes of its responsibilities. It seems the greatest threat to the health and well-being of rare disease patients in New Zealand, is the health economists who don’t have a moral compass. Over the past year we have won support from most political parties that this situation is untenable and must change. Read more about this promise. The big challenge ahead for us to is cement in the promises from those parties of an Orphan Drugs access programme, and this year will see considerable work on that issue.


John Forman
Executive Director