Funded drug shows change for Pharmac

19 March 2016

Pharmac’s decision to fund a new drug is an important breakthrough for rare disease treatment, an advocacy group says. Drug funding agency Pharmac has approved funding for Naglazyme, a treatment for the very rare Maroteaux-Lamy syndrome which is one of a series of Lysosomal diseases in New Zealand. Radio NZ news itemRead more

Pharmac approves funding of Naglazyme for Maroteaux-Lamy (MPS 6)

18 March 2016

It was 11.22 am on 17 March 2016 when LDNZ got the briefing about this – the first listing in New Zealand of a novel treatment for a lysosomal disease since 1999 when Cerezyme was listed for Gaucher disease. This decision is an important breakthrough for LDNZ and represents a significant shift in approach by Pharmac. In the past 15 years Pharmac has been extremely reluctant to provide access to any of the new treatments for Lysosomal diseases. Their resistance required continued strong advocacy from LDNZ, and our efforts have finally been successful. Read more.

February 2016: Submission to Pharmac on Naglazyme Consultation

18 February 2016

Pharmac’s third consultation under the orphan drugs fund is for Naglazyme, for the treatment of Maroteaux–Lamy syndrome (MPS6), which is one of our Lysosomal diseases. LDNZ submitted this response in February 2016 and we wait now for Pharmac’s decision. This might be the breakthrough we have waited for, for so long – the possible funding for the first time from this special fund for rare diseases, of a treatment not previously accessible at all by patients in New Zealand. Read our submission.

Pharmac announce first orphan drug to be funded under the contestiable fund

18 August 2015

There is a little bit of good news in Pharmac’s announcement of approval of the first drug under the $5 million Orphan Drugs Fund. At least one has made it across the line. Although this is a good start, this drug has already been funded under the exceptional circumstances scheme, so this really just extends access rather than adding something new that was not previously available. And don’t be misled by their claim of $25 million. It is just a $5 million fund, though guaranteed over time. Meanwhile there is no news about any new funding for orphan drugs not yet available to NZ patients, and even some worrying hints that progress for them is slowing and possibly grinding to a halt.

LDNZ has been contacting Pharmac almost weekly to see how progress is going and to try and push things along. Watch this space. Read more.

Pharmac in Negotiations over Rare Disorder Medicine

27 February 2015

Pharmrac released their latest announcement today stating that they are still in negotiations with several drug companies in relation to orphan drug access. This announcement comes on the eve of International Rare Disease Day. Although this is not the message we had hoped for it’s good news that they are still talking with the companies.

Lets hope Pharmac does the right thing and funds our medicines. We would hate to see winners and losers. However there are grave concerns over the medicine budget for rare disorders. Read the press release here.

Pharmac reject entry and exit criteria for Myozyme

14 October 2014

After 15 long months waiting for Pharmac to make a decision on a trial for Freda and Yakuta using Myozyme, Pharmac again rejects their appeal.

New evidence was submitted to Pharmac along with entry and exit criteria, all of which was reviewed by PTAC. The committee said that although there is a suggestion that alglucosidase alfa may confer an overall survival benefit they still considered the new evidence weak and insufficient to warrant a change in their previous recommendation to decline funding.

In regards to pricing they said: Given the substantially unchanged evidence of clinical benefit and in the absence of any new pricing information, the conclusion of our revised economic assessment is unchanged. 

So once again we come back to clinical evidence, cost and leaving our Pompe patients with no pathway forward. In our eyes there has to be some concern over what Pharmac will do when the drug companies put in their bids for the 5 million contestable fund for orphan drugs. Will our Lysosomal Enzymes get over the threshold or will they again be put into the too hard basket leaving our patients untreated and leaving us with no pathway to apply for funding?

High cost medicines for Rare Disorders – LDNZ submission

25 July 2014

LDNZ submitted its submission to Pharmac advising them we are far from happy with their proposal for funding High cost medicines. Read more here.

Serious flaws in Pharmac’s proposal for access to medicines.

18 July 2014

Patient advocates are rallying support for submissions to Pharmac about the U-turn Pharmac has made on medicine funding for ultra-rare disorders. After repeated refusal to fund novel and expensive treatments for disorders that affect mostly just a handful of cases in New Zealand, Pharmac have announced a special $5 million fund so they do get a chance of being funded here. Read more here (link no longer active).

High cost Medicines for Rare Disorders

18 April 2014

Who would believe that right before the next election Pharmac comes out with a discussion document for High cost Medicines for Rare Disorders, with the first line in their document stating – ‘We’ve been doing some thinking about access to high cost medicines for rare diseases and we want your input to help us develop an alternative commercial approach’. Really come on Pharmac!!!!

We have to ask though is Pharmac starting to change the way they are thinking about orphan drugs? –  this would be fantastic to see – but do we trust them? Is this a pre-election bid to shut us down and keep us quite?

We can identify some fish hooks – the proposed funding level for this is $5 million a long way from the $20–25 million we have estimated that would fund all rare diseases where there is a therapy.

The policy document puts emphasis on maintaining the current Pharmac framework and states the usual emphasis on best health outcomes and value for money. This suggests the orphan drugs would still be compared to those for common diseases, which seems to contradict the purpose of a special fund. In that case, the orphan drugs are still likely to NOT stack up against other in terms of cost effectiveness.

Pharmac rejects call for change on rare diseases

18 February 2014

Things are not looking good for people with rare diseases. Pharmac gives some explanation about what their proposal for change is about but its clear they have no intention of wanting to help with access to Orphan drugs. Read more.