Medicines for rare disorders
PHARMAC is working on policy and funding for medicines for rare disorders.
Policy
Confirming adjusted policy settings
PHARMAC has published adjusted policy settings on medicines for rare disorders, to help guide its work in this area and give clarity to stakeholders.
The policy settings include three principles, which are based on the differences of medicines for the treatment of rare disorders. These differences address the current market challenges for these medicines, and enable (where appropriate) a different entry into the pharmaceutical funding process.
The principles are:
- The medicine has been approved by Medsafe, or an approved international regulatory authority, for the identified indication.
- The disorder is a clinically defined disorder affecting an identifiable and measurable patient population with a prevalence of less than 1:50,000 in New Zealand.
- The medicine is only registered for the treatment of the rare disorder, or if it is registered for other disorders (or is part of phase three clinical trials for other disorders), the cumulative prevalence across all indications still meets principle 2.
PHARMAC will determine whether funding applications meet the principles. Those that do will then be considered through the process determined by PHARMAC. Other applications that do not meet the principles can still be considered through our usual Schedule listing process.
Establishing a clinical subcommittee
PHARMAC has established a Rare Disorders Subcommittee of the Pharmacology and Therapeutics Advisory Committee (PTAC). We sought applications through colleges and clinical networks (both New Zealand & Australian based), and specifically sought applicants who had a special interest in rare disorders.
The committee will provide clinical input on funding applications PHARMAC receives for medicines for rare disorders.
The members include some of the country’s leading experts in treating rare disorders, and include specialties such as paediatric nephrology, metabolic disorders, blood disorders and neurology. They also include an Australian specialist in rare disorders.
The Subcommittee includes two PTAC members, to maintain links between this new Subcommittee and our primary clinical advisory committee.
Funding
Engaging with suppliers and progressing proposals to fund medicines for rare disorders
We're calling for more funding applications for medicines for rare disorders. This call for applications closes at 5 pm on Monday 5 August 2019.
Call for funding applications for medicines for rare disorders
In June 2018, PHARMAC called for funding applications for medicines for rare disorders.
By the 3 September 2018 deadline, we had received 13 funding applications for ten rare disorders (there was more than one application for some medicines).
These funding applications were assessed by our Rare Disorders Subcommittee at their first meeting on 5 and 6 November 2018.
They were then assessed by our main body of clinical advisors, the Pharmacology and Therapeutics Advisory Committee (PTAC) in February 2019.
You can see what the Subcommittee and PTAC recommended, as well as their full discussions on our website and Application Tracker.
Applications received
| Application | Indication | Supplier | Rare Disorders Subcommittee Recommendation | PTAC Recommendation |
|---|---|---|---|---|
| Agalsidase alfa (Replagal) | Fabry disease | Shire | Recommended for funding with a medium priority |
Recommended for decline Our advisors recommended the priority change from 'medium' to ‘recommended for decline’ due to low quality evidence consistent with only modest long-term health benefits |
| Alglucosidase alfa (Myozyme) | Late-onset Pompe disease | Sanofi-Genzyme | Recommended for decline | Recommended for decline |
| Carglumic acid | Hyperammonaemia due to urea cycle disorders | Max Health | Recommended for funding with a high priority | Recommended for funding with a high priority |
| Organic hyperammonaemias | Te Arai BioFarma | Recommended for funding with a medium priority | Recommended for funding with a medium priority | |
| Elosulfase (Vimizim) | Mucopolysaccharidosis (MPS) type IVA | BioMarin | Recommended for decline | Recommended for decline |
| Ivacaftor (Kalydeco) | Cystic fibrosis - with G551D mutation | Vertex | Recommended for funding with a medium priority |
Recommended for funding with a low priority Our advisors recommended the priority change from 'medium' to 'low' due to moderate quality evidence of a health benefit, the limited availability of long-term data, concerns regarding markers of surrogacy, and the high cost |
| Migalastat (Galafold) | Fabry disease | Amicus | Recommended for decline | Recommended for decline |
| Miglustat | Gaucher disease | Te Arai BioFarma | Recommended for decline | Recommended for decline |
| Miglustat | Niemann Pick Type C | Te Arai BioFarma | Recommended for decline | Recommended for decline |
| Nitisinone | Tyrosinaemia type 1 |
Max Health Te Arai BioFarma |
Recommended for funding with a high priority | Recommended for funding with a high priority |
| Nusinersen (Spinraza) | Spinal muscular atrophy | Biogen |
Recommended that the funding application be deferred, pending longer-term follow-up analyses of two trials We expect to receive more evidence by the end of 2019. |
Recommended that the funding application be deferred, pending longer-term follow-up analyses of two trials We expect to receive more evidence by the end of 2019. |
| Teduglutide (Revestive) | Short bowel syndrome intestinal failure | Shire | Recommended for decline | Recommended for decline |
A recommendation is not guarantee that PHARMAC will fund the medicine – PHARMAC decides what will be funded in the context of the options for funding, to ensure we are getting the best possible health outcomes for New Zealand.
PHARMAC has considered these funding applications using our Factors for Consideration framework, and compared and ranked them against other funding applications we have received.
We do not generally assess funding applications that have been recommended for decline any further. We will, however, consider any new evidence and information that we may receive.
We are unable to give a timeframe for if, or when, we may make a funding decision about these funding applications.
Call for funding applications - medicines for rare disorders (2018)
Background - rare disorders contestable funding RFP 2014-16
Rare Disorders Subcommittee minutes - 5 and 6 November 2018 [PDF, 713 KB]
Call for funding applications for medicines for rare disorders (2019)
Last updated: 10 May 2019
