Medicines for rare disorders
PHARMAC understands that people living with rare disorders face many challenges. These include access to suitable health care and effective medicines.
We encourage funding applications from suppliers or clinicians for medicines for rare disorders. We also scan the horizon for new medicines and might start an application ourselves.
On this page
- Funding medicines for rare disorders
- Policy principles for rare disorder medicine funding applications
- About the Rare Disorders Subcommittee
- Factors for Consideration for rare disorders
- Horizon scanning for new medicines
- History of funding rare disorder medicines
- Who to contact
Anyone can make an application for a medicine to be funded for a rare disorder.
Funding applications for rare disorder medicines need to meet these three policy principles.
- Medsafe, or an approved international regulatory authority, has approved the medicine for the specific indication or condition.
- The disorder is a clinically defined disorder that affects an identifiable and measurable patient population of less than 1:50,000 in New Zealand.
- The medicine is only registered to treat the rare disorder. If it is registered for other disorders (or is part of phase three clinical trials for other disorders), those other disorders must meet principle 2.
If the intent of all three principles is met, PHARMAC will accept an application for a rare disorders medicine and progress it. From there, we seek clinical advice from our expert advisory committees.
Suppliers of rare disorders medicines don't need Medsafe approval before applying to PHARMAC (Principle 1 notwithstanding). This differs from the funding application process for most other medicines. However, the medicine must be Medsafe-approved before a final decision can be made to fund it.
This flexibility enables us to consider funding applications before a Medsafe approves a medicine.
The Rare Disorders Subcommittee considers the funding application. It reviews the evidence for health benefit and need, then makes a recommendation to the Pharmacology and Therapeutics Advisory Committee (PTAC) and PHARMAC.
As with all medicines, if the Rare Disorders Subcommittee or PTAC recommend the application be declined, we may not progress the application any further. We would only reconsider the funding application if we receive additional information or new evidence.
If the Rare Disorders Subcommittee or PTAC make a positive recommendation, PHARMAC then ranks the funding application against all other funding applications to determine the next best investment.
The members include some of New Zealand’s leading experts in treating rare disorders. The members’ specialties include:
- paediatric nephrology
- metabolic disorders
- blood disorders
One member is an Australian genetics and metabolic disorders specialist. They have been involved in Australia’s Life Saving Drugs Programme (LSDP).
The Rare Disorders Subcommittee assesses applications in the context of rare disorders treatments and bases its recommendations on:
- PHARMAC’s Factors for Consideration and
- their own clinical expertise.
The subcommittee met in September 2019 to discuss:
- Coenzyme Q10 for mitochondrial diseases(external link)
- Levocarnitine for ketogenic diet support and metabolic indications(external link)
- Cysteamine (mercaptamine) eye drops for cystinosis(external link)
- Nusinersen (Spinraza) resubmission for spinal muscular atrophy(external link)
- Migalastat resubmission for Fabry disease(external link)
PHARMAC applies its Factors for Consideration to all funding decisions we make. The factors most relevant to our consideration of medicines for rare disorders are:
- Health need of the person – rare disorders can often be debilitating and severe. People with a rare disorder are often considered to have a high health need.
- The availability and suitability of existing medicines, medical devices and treatments. People with rare disorders often have limited alternative treatment options available.
- Health needs of others. Caring for a person with a rare disorder can impact on the health of those caring for them.
PHARMAC is always looking for new rare disorders medicines. As we learn of new information, we may approach suppliers or clinicians and encourage them to submit a funding application. On occasion we may start a funding application ourselves.
Our clinical advisers also stay up to date with the latest medicines on the market or in clinical trials through their professional practice.
We haven't always taken this approach. After wide consultation in 2014, we ran a contestable funding pilot with an RFP and separate pool of funds.
The pilot was reviewed by PHARMAC and an independent consultant. As a result, new medicines were funded and listed on the Pharmaceutical Schedule. While we didn't continue with the RFP approach, the pilot informed our current policy principles (above), which have been in place since 2018.
If you have any questions about PHARMAC’s work on rare disorders, email firstname.lastname@example.org
Last updated: 13 August 2020