Samantha’s submission to the Health Select Committee

10 March 2017

Following a request from the select committee that Samantha provide a written submission about her petition to the committee, Samantha was assisted by LDNZ to provide them with a submission that was submitted in March 2017. We were inspired by Samantha’s clear messages and personal courage in doing this, and very pleased to have supported her with the submission. Read the submission here.

Pharma Company gives up on Pharmac

28 February 2017

Sanofi Genzyme developed Myozyme, a complex biological treatment for Pompe disease, a lysosomal disease, over ten years ago. Since that time funded treatment of affected patients has spread to 76 countries. Over that decade, NZ’s drug purchasing agency Pharmac has consistently refused to fund it, leaving the 10 patients here abandoned and facing relentless decline in their health and quality of life.

After substantial renewed efforts to negotiate with Pharmac, and with a recent recommendation from Pharmac’s PTAC committee, released early February, that funding should be declined, the company has decided to provide the treatment free to four NZ patients. Read more here.

Petition delivered to Government

13 December 2016

Samantha Lenik and patients with Pompe and Fabry disease and their supporters delivered the petition to the government yesterday requesting that the government look under urgency to ensure Pharmac has enough funds to properly deliver on the rare disease drug policy.

Here the the media interviews:

The reading of the petition in the House

Radio NZPatients Call for Funding of Rare Disease Medicines

NZ Herald: Children of women with rare disease ask Santa for mummy’s medicine

Rodney Times: Petition for rare disease funding delivered to Parliament

CheckOrphan (link no longer active): The presentation of the petition made it to CheckOrphan

Samantha and the affected patients along with LDNZ thank everyone who participated in the signing of the petition and left some truly amazing comments that we will be forwarding onto Pharmac. Thank you everyone.

Enzyme Replacement therapies – 3 new decisions and one more review

30 November 2016

There is some very positive news to report this month, and some disappointment as well. LDNZ is delighted that Pharmac has approved the listing of Myozyme for infantile Pompe disease on the schedule. This is the first approval for this treatment and putting it on the schedule means that any baby diagnosed with this disease, and who fits the treatment guidelines, will be automatically given treatment, avoiding delays and anxious waits for special consideration under the exceptional circumstances scheme. Read more.

Rare Disease Mum Abandoned by her local MP

8 November 2016

For Samantha Lenik’s potential life-changing Petition to make it to Parliament, Samantha needs an MP to personally present the Petition to the House of Representatives. Unfortunately, Samantha’s local MP Mark Mitchell (National MP for Rodney) declined to receive the Petition, and with one week to go she is scrambling to find an MP who is willing to help her and other rare disease patients. Read more here.

LDNZ submission on proposed funding of 3 enzyme replacement therapies

1 November 2016

We are making progress and it is very satisfying to see some results. Just 2 weeks ago Pharmac issued for consultation a proposal to list 3 additional ERTs on the Pharmaceutical schedule, meaning automatic funded access for those who meet the eligibility criteria. The proposed listings are for ongoing treatment with Myozyme for infantile Pompe disease, short-term treatment with Aldurazyme prior to transplant for Hurler disease, and short term treatment with Elaprase prior to transplant for Hunter disease.

Also announced in background information to this proposal is advice that Pharmac’s specialist advisory committee, PTAC, will this month be reviewing the evidence for Myozyme for adults with Pompe disease, in response to another submission from Sanofi to have this treatment listed on the schedule. About time too. And hardly a coincidence that our petition to get funded access to these therapies kicked off in earnest just 6 weeks ago.

Here’s the submission we made in response to their proposal.

Mother with Pompe a rare genetic disease petitions parliament

27 October 2016

Samantha Lenik hopes to get access to a life changing treatment for Pompe disease, which isn’t funded in New Zealand. Read more here.

Press Release – Another Medicine Funding Petition launched

10 October 2016

New Zealand Rare Disease Patients need funding too.

Samantha, Freda and Yakuta, a mother, a grandmother, and a young woman with her life ahead of her, have launched a petition to Parliament aimed at ensuring Pharmac have enough funding to implement its new policy on medicines for rare diseases, which was announced back in April 2014, and to give urgency to doing so. Read more here.

Our message to all Members of Parliament, requesting support for our medicine access campaign

6 October 2016

As we kicked off our petition about medicine funding and a fair deal for rare disease patients, we sent this message to all MPs, asking them to take particular note of the issues and show their support. We are pleased to note a we have had a small number of responses in the following days. Read more…

Submission to the UN Secretary-General’s panel on Access to Medicines

20 March 2016

LDNZ contributed its years of experience with problems relating to medicines access in New Zealand, in a submission made by LDNZ Chair, John Forman, to a special panel set up by the UN Secretary-General. This panel is taking a global view of problems relating to the tension between the intellectual property rights of drug developers to set the price of their products, and the rights of patients to be treated for their health conditions under the “Right to Health” provisions of the international human rights framework.

The submission highlights the significant conflicts of interest held by most governments who are the bodies charged with meeting the rights of patients within their countries, but in most cases are also the payers and decision makers about health system costs. It calls on the UN to actively intervene to ensure governments give more attention to the right to health. Read more at this link.