Medicines for rare disorders
PHARMAC has a current focus on policy and funding work for medicines for rare disorders.
Work is underway in three areas:
- Policy: Confirming adjusted policy settings and establishing a clinical subcommittee.
- Commercial: Engagement with suppliers and progressing proposals to fund medicines for rare disorders.
- Stakeholder engagement: Keeping stakeholders informed about our work.
Policy
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.
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.
Commercial
In June 2018, PHARMAC called for supplier funding applications for medicines for rare disorders.
By the 3 September 2018 deadline, we had received 13 supplier applications. In some cases, more than one application was received for the same medicine and indication from different suppliers (such applications will be considered together). See below for a full list of medicines, indications and supplier applications.
These applications were considered at the Rare Disorders Subcommittee's inaugural meeting in November 2018.
Minutes of the Subcommittee’s discussion will be published on our website and Application Tracker. PHARMAC may decide it also needs advice from PTAC on the applications.
Following clinical advice and completion of our assessment processes, including prioiritisation, PHARMAC will then determine the next appropriate steps for the application, such as commercial processes.
Applications received
| Application | Indication | Supplier |
|---|---|---|
| Agalsidase alfa (Replagal) | Fabry disease | Shire |
| Alglucosidase alfa (Myozyme) | Late-onset Pompe disease | Sanofi-Genzyme |
| Carglumic Acid | Hyperammonaemia due to urea cycle disorders | Max Health |
| Te Arai BioFarma | ||
| Elosulfase (Vimizim) | Mucopolysaccharidosis (MPS) type IVA | BioMarin |
| Ivacaftor (Kalydeco) | Cystic fibrosis - with G551D mutation | Vertex |
| Migalastat (Galafold) | Fabry disease | Amicus |
| Miglustat | Gaucher disease | Te Arai BioFarma |
| Miglustat | Niemann Pick Type C | Te Arai BioFarma |
| Nitisinone | Tyrosinaemia type 1 | Max Health |
| Te Arai BioFarma | ||
| Nusinersen (Spinraza) | Spinal muscular atrophy | Biogen |
| Teduglutide (Revestive) | Short bowel syndrome intestinal failure | Shire |
Timelines for medicines for rare disorders applications
- November 2018 Consideration by Rare Disorders Subcommittee
- Early 2019 Next steps determined for applications received
Last updated: 5 December 2018
