Medicines for rare disorders

Useful Information

How we fund rare disorder medicines

We fund over 100 medicines for treating rare disorders through the Combined Pharmaceutical Budget (CPB). The decisions to fund these medicines were made through three different mechanisms:

Pharmaceutical Schedule listing

We use our normal processes for deciding which medicines to fund from within our fixed budget, that will achieve the best health outcomes for New Zealanders.

The Exceptional Circumstances Framework

This framework outlines the ways PHARMAC considers funding decisions for exceptional circumstances that fall outside of the Pharmaceutical Schedule funding process.

The rare disorders medicines contestable funding pilot

This pilot process was a request for proposals (RFP) initiated in 2014 to stimulate competition amongst suppliers of rare disorders medicines. We committed to spending up to a maximum of $5 million per year. While we continue to fund all the medicines listed in the Pharmaceutical Schedule as a result of the pilot, we are not currently planning similar RFPs.

Funding breakdown

This table shows the different ways PHARMAC has funded medicines for rare disorders. This table is updated annually.

This table includes medicines only used for the treatment of rare disorders. There are many other general medicines that are prescribed to people to treat their rare disorders that are not accounted for in this table.

Rare Disorders Subcommittee

PHARMAC has established a Rare Disorders Subcommittee of the Pharmacology and Therapeutics Advisory Committee (PTAC).

Subcommittee members review applications received and make recommendations to PTAC and PHARMAC about whether the treatments should be funded or not. Their recommendations are based on PHARMAC’s Factors for Consideration and their clinical review of the information provided about the medicine.

The appointed members include some of New Zealand’s leading experts in treating rare disorders. The members specialties range from paediatric nephrology and metabolic disorders, to blood disorders and neurology. One member is an Australian genetics and metabolic disorders specialist who has been involved in Australia’s Life Saving Drugs Programme (LSDP).

Click here to see the latest rare disorders subcommittee minutes and to see who sits on the committee.

The Rare Disorders Subcommittee will meet on 24 September 2019 and consider the following funding applications:

• 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)

Different viewpoints of PTAC and Subcommittees

The Rare Disorders Subcommittee assesses applications in the context of rare disorders treatments.

PTAC has a moderating function. PTAC looks at medicines within the context of every medicine PHARMAC is considering funding applications for.

Because of these different points of view, it is not uncommon for PTAC to make a different recommendation to a subcommittee. There are 21 subcommittees of PTAC in total including rare disorders.

PTAC and Rare Disorders Subcommittee recommendations

Clinical input is an important part of the process and these committees can make recommendations to fund a medicine or not. If they recommend funding, they are asked to rank the funding as high, medium, low. A recommendation from either the subcommittee or PTAC is not a decision, and doesn’t guarantee that PHARMAC will fund the medicine.

This is because the PHARMAC Board (or its delegate) is the final decision maker. To make this decision the PHARMAC Board compares funding applications against all other medicines that have received a positive recommendation and consider our available budget.

PHARMAC’s list of medicines which is in ranked order of preference is commercially sensitive. This is because PHARMAC wants to fund as many medicines as we possibly can from our available budget. Not disclosing exactly where a medicine sits on our list improves our negotiating power.

Rare Disorders policy information

PHARMAC revised its application criteria for rare disorders medicines in 2018, to encourage suppliers to make more applications and potentially lead to improved access to medicines for rare disorders.

Unlike other medicines, suppliers of rare disorders medicines are not required to have gained Medsafe approval for the medicine before it can be considered by PHARMAC for funding. However, Medsafe approval is required before PHARMAC will fund the medicine.

This change is designed to make it easier for pharmaceutical suppliers to submit an application to PHARMAC.

There are three principles that guide funding applications for rare disorders:

1. The medicine has been approved by Medsafe, or an approved international regulatory authority, for the identified indication or condition.
2. The disorder is a clinically defined disorder affecting an identifiable and measurable patient population of less than 1:50,000 in New Zealand.
3. 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), it still meets principle 2.

If all three of the above principles have been met, PHARMAC will accept an application for a medicine regardless of whether it is approved by Medsafe and will assess it as per our standard processes. 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.

In the context of rare disorders, we know that some aspects of our Factors for Consideration are particularly relevant including:

• Health need of the person – rare disorders can often be debilitating and severe, and so individuals 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 need of others – caring for a person with a rare disorder can have impacts on the health of those with this responsibility.

Horizon scanning for new medicines

PHARMAC is continuously looking for new rare disorders medicines. As we become aware of new information, we may approach suppliers or even self-initiate funding applications.

Our clinical advisors also stay up to date with clinical advancements, and regularly discuss the development of rare disorders medicines with suppliers and international colleagues or peers.

Through our relationships with suppliers and our clinical advice network we are aware of the current rare disorder’s medicines on the market and new treatments in clinical trials in New Zealand and internationally.