NZORD Newsletter 2014 #6 - 21 November 2014

Hello everyone, 

In this issue:
1 – Preparations for Rare Disease Day 2015.
2 – Carer payments policy review initiated by Ministry of Health. Perhaps our voices are being heard at last?
3 – Political party commitments bode well for improvements to early and accurate diagnosis.
4 – Parenteral nutrition symposium in Auckland next week.
5 – LAM for GPs video series launched to assist diagnosis and management of Lymphangioleiomyomatosis. 
6 – Researcher is seeking young carers to participate in research study.
7 – Australian drug funder approves subsidy for two more orphan drugs. Will NZ ever catch up?
8 – This month’s good news item – Professor Margaret Brimble.

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1 – Preparations for Rare Disease Day 2015.
28 February 2015 marks the eighth Rare Disease Day and with just 100 days to go, preparations are already well under way.  At NZORD the NZ Rare Disease Day website has been updated and posters are being designed.  We are planning on holding several movie nights around the country during the week leading up to Rare Disease Day, more details will follow soon. Please share your event to help boost the total activity around this day.

Thank you to all who completed the Rare Disease Day Survey, we had a good response and the feedback is a big help as planning progresses for Rare Disease Day 2015.  One lucky survey respondent has been sent a Cadbury confectionary pack. 

2 – Carer payments policy review initiated by Ministry of Health. Perhaps our voices are being heard at last?
There is some good news on this subject, with the Ministry of Health doing an early review of the Carer payments policy. This policy, Funded Family Care, was bought in with a $23M provision in the 2013 budget but was widely criticised for its restrictive rules, lack of flexibility and complicated criteria for those affected. Analysis done after 6 months implementation showed that barely 100 of 1,600 intended care situations had been approved for funding. It is reassuring that the many concerns raised have been listened to with the review being started promptly after the election. It is hoped that the concerns we have raised will lead to revisions to the policy, to truly respond to carer interests and deliver the funding to those carers who need it the most.

3 – Political party commitments bode well for improvements to early and accurate diagnosis.
It was reassuring to learn that all of the political parties who were contacted prior to the general election with NZORD’s election manifesto were very supportive of NZORD’s early and accurate diagnosis policy.  It is positive that the key issue is understood by all of these political parties – the trick now will be holding the parties to their promises as the three year government term kicks off. We will report further on progress once we receive a response to our specific request for improvements to investments in genetic services, genetic testing budgets, and related service coordination and information resources.

4 – Parenteral nutrition symposium in Auckland next week.
The support group Parenteral Nutrition Down Under (PNDU) is hosting one and a half days of workshops, seminars and social gatherings particularly aimed at HPN consumers and carers in Auckland, on 24 and 25 November.

The workshops are free for all HPN consumers and carers, with modest charges for other interested attendees. For more information and to register click here

5 – LAM for GPs video series launched to assist diagnosis and management of Lymphangioleiomyomatosis. 
A web video series about a rare lung disease affecting women of child bearing age has been launched.  Currently there is no cure for Lymphangioleiomyomatosis (LAM) but it can be treated, particularly if diagnosed early. The LAM for GPs videos, introduced and narrated by Carol Hirschfeld, offers advice from people who really know about LAM. The short videos provide an opportunity for doctors to quickly gain more awareness of presenting symptoms and knowledge of a drug therapy.
You can find out more about LAM here.

6 – Researcher is seeking young carers to participate in research study.
Lauren Donnan is a student at Auckland University, researching the experiences of New Zealand young carers for her PHD.
Lauren is currently looking for participants for her research.  Participants will take part in a conversation style interview that will take around one hour.  This can take place face to face or via Skype, whatever is most convenient. 
It doesn’t matter what age you are now as long as you cared for someone when you were 25 years of age or younger who had a disability, illness, drug or alcohol misuse or was elderly. Those interested can contact Lauren on (09) 623 8899 ext 46387 or email her at l.hitchin@auckland.ac.nz

7 – Australian drug funder approves subsidy for two more orphan drugs. Will NZ ever catch up?
The Australian government has approved the listings of Kalydeco for Cystic Fibrosis, and Soliris for aHUS, on the Australian Pharmaceutical Benefits Scheme from 1 December 2014. Kalydeco treats patients with a specific gene mutation and is the first medicine to treat the underlying cause of Cystic Fibrosis in these patients. Soliris is already funded in Australia for the rare disease PNH and the new listing extends its use to aHUS. It is estimated that the listing decisions will provide access to approximately 250 Australians who would otherwise face prohibitive costs and effectively be denied access to these important treatments.
Meanwhile, New Zealand patients affected by rare orphan diseases wait anxiously while Pharmac considers proposals submitted recently for its new $5 million rare disease fund, and wonder if any NZ patients will ever be treated with the seven orphan drugs that will now be routinely administered to Australian patients, but bluntly denied for so long to NZ patients.

8 – This month’s good news item.
Distinguished Professor Margaret Brimble of Auckland University scored an award in the innovation and science category at the  Women of Influence Awards last month, adding one more to the impressive list of awards she has received throughout her very significant career. This month there was also an announcement by Neuren Pharmaceuticals of success with the phase 2 trial of an investigational compound NN-2566 for the treatment of Rett syndrome, a severe neurological disease primarily affecting young girls. Professor Brimble was the leader of the group that discovered the compound. Her many accolades are richly deserved.

Edited by Lyndal Bremer and John Forman