NZORD Newsletter 2011 #5 – 10 October 2011

Hello everyone,

In this issue:
1 – Current reviews of Non-Government organisations. What implications for support groups?
2 – Pharmac has dropped the ball on highly specialised medicines. Who will pick it up?
3 – Decisions made on long term storage of Guthrie card blood spots from newborn screening.
4 – Government response to select committee report on clinical trials. Positive, but limited by resource constraints.
5 – Display your rare disease information at Paediatric Society conference.
****************************************************************************************************************

1 – Current reviews of Non-Government Organisations. What implications for support groups?
Two reviews are under way at present. The Department of Internal Affairs has surveyed groups in the health and disability sector on behalf of the Lottery Grants Board, and the Law Commission is consulting on the law relating to Incorporated Societies, and by extension, to Charitable Trusts. Both review have significant implications for support groups. The first has implications for future funding and we have asked DIA for discussions before they reach any conclusions. We are concerned that limited understanding of what support groups do and what their needs are, might mean tough financial times getting even tougher.

The Law Commission review is about legal structures, governance and accountability for all societies, and thus is much wider than health and disability groups. We agree that the law in this area needs updating but once again we are concerned that the understanding of many different types of groups may not be as clear as it should. The Law Commission has an opportunity to take a strategic approach that helps efficiency and good governance for all Non-Government Organisations, as well as enabling them to be effective partners with government in benefiting all of New Zealand society. Read our submission to the Law Commission.

2 – Pharmac has dropped the ball on highly specialised medicines. Who will pick it up?
We’ve had over 6 years of promises from politicians and from Pharmac about improving access to highly specialised medicines for rare “orphan” diseases, yet the issue drags on like a festering sore. Pharmac either can’t or won’t address the issue. Read our analysis of their failed attempt in their recent review of the exceptional circumstances scheme. Nor have political leaders shown the commitment to action that they have promised.

Meanwhile New Zealand patients with life-threatening diseases like Pompe disease, which affects just a handful of patients, are denied life-saving treatment because Pharmac is agonising over the relative cost per quality-adjusted life year. They have again denied funding, while patients is 45 other countries are receiving treatment with the drug. Read more on the website of Lysosomal Diseases New Zealand.

In other countries the critical issue regarding access to these specialised medicines is that decisions are made at the highest levels of government that patients with orphan diseases should not be abandoned by the health system. The decisions cannot be left to officials alone. With nearly a year passing since Pharmac first considered an application to fund Myozyme for Pompe disease, it is time for the process to be sorted out. A decision must be made politically about an orphan drugs access programme, to ensure there is equitable access to medicines for all patients and to ensure patients like those with Pompe disease actually get treated when therapy becomes available. The Prime Minister is the one who needs to sort this problem out. Every other avenue has failed. He has the authority to direct that a solution is put in place, and we believe he now has the responsibility.

3 – Decisions made on long term storage of Guthrie card blood spots from newborn screening.
After nearly a decade of uncertainty, reports and consultation, decisions have finally been made about the retention, storage and use of Guthrie cards at the end of the newborn screening process. The issues were complex. Careful consultation and deliberation was needed. NZORD considers the outcomes to be a good policy framework for the future with a good balance of interests between personal autonomy and privacy, protection of the screening programme, and future opportunities to gain knowledge for the benefit of society.

Within a broad-ranging policy framework for the entire operation of the programme, the essential decisions on the retention, storage and future use issues are 1) that parents may opt to have the card returned to them, 2) other cards will be kept indefinitely, 3) research on previously stored cards will require specific consent from individuals, 4) new consent procedures will allow future research on stored cards after scrutiny by an ethics committee and approval by the Ministry of Health.

NZORD is very pleased to see these decisions made and published. Full details are available on the website of the National Screening Unit, linked from the quality standards page. We are also pleased to note a strong correlation between the final policy decisions and the 2008 submission made by NZORD.

4 – Government response to select committee report on clinical trials. Positive, but limited by resource constraints.
Boosting opportunities for clinical trials in New Zealand was the focus of a detailed report from the Health select committee in June this year. The government’s response to this report was released in August with substantial support for most of the recommendations, especially the focus on improving the efficiency of ethical review. However the current economic constraints did not result in any indication of extra investment in infrastructure that would enable such trials to be undertaken more routinely within our universities and medical institutions. Time will tell if the changes that are indicated to ethical review will be enough to improve clinical trials opportunities in New Zealand.

5 – Display your rare disease information at Paediatric Society conference.
NZORD has arranged a double booth space at the Paediatric Society conference in Auckland from 22 to 25 November 2011. We are offering an opportunity for all rare disease support groups to send us 100 copies of their 3-fold brochure or A4 flyer and we will display these free of charge in our booth. All you need to do is post them to the NZORD PO Box number (below) or courier them to our Thorndon office, before mid-November. We will gladly do the rest. Groups wishing to have one of their staff or volunteers join us in the booth to assist with questions, may also bring a stand-up banner to display. Contact us for further details of modest exhibitor registration costs involved in attending.

Regards, John

John Forman
Executive Director