15th May 2013

15 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 To see the interview click here (link no longer active).

28th Feb 2013

28 February 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.

Complaint sent to Ombudsmen

18 October 2012

  • 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.

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

5 October 2012

  • 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.

The Named Patient Pharmaceutical Assessment Scheme does not work!

18 September 2012

  • 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.

Application submitted for Freda Evans (Pompe)

18 August 2012

  • 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

18 June 2012

On 20th June 2012 the Marquardt family (link no longer active) 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.

Medicine policy anomalies need to be investigated

18 May 2012

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 (link no longer active) is our request to them and sets out the main arguments.

NZORD makes a submission to Health Select Committee

20 April 2012

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 (link no longer active).

Pompe patients march on Parliament

30 November 2011

  • 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).