Call for funding applications for medicines for rare disorders

Medicines Consultation Closed

What we’re requesting

PHARMAC invites pharmaceutical suppliers to submit funding applications for medicines for rare disorders.

This call for applications closes at 5pm on Monday 3 September 2018 and applications can be emailed to

Who we think will be interested

  • Pharmaceutical suppliers of medicines for rare disorders.
  • Clinicians, patients, support groups with an interest in medicines for rare disorders.

How will it work?

PHARMAC has developed policy settings that will underpin our commitment to ‘regularly call’ for applications for rare disorders medicines. A standing Rare Disorders Subcommittee of the Pharmacology and Therapeutics Advisory Committee (PTAC) is being established to provide advice on applications.

We invite funding applications for medicines for rare disorders that meet the following Principles:

  1. The medicine has been approved by Medsafe, or an approved international regulatory authority, for the identified indication.(external link) Medsafe approval must still be gained before a pharmaceutical is listed on the Pharmaceutical Schedule.
  2. 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.
  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), the cumulative prevalence across all indications still meets Principle 2.

Applications should also meet the requirements of a full funding application as best they can, addressing the Factors for Consideration. Refer to Guidelines for Funding Applications for pharmaceutical suppliers. Applications should include a commercial proposal.

Next steps following submission

Applications that meet the Principles will then be considered through an assessment process in line with our usual processes, including seeking clinical advice.  A Rare Disorders Subcommittee of PTAC is in the process of being established to provide advice on applications. The policy settings are available on the PHARMAC website.

For suppliers, the key difference to the usual process is the ability to submit an application prior to Medsafe approval. Medsafe approval would still be required before a pharmaceutical was listed on the Schedule.

Applications will likely be considered by the Rare Disorders Subcommittee of PTAC as the first step in the application assessment process. Following clinical advice, PHARMAC would then determine the next appropriate steps for the application, such as commercial processes.

Funding applications for medicines for rare disorders can be submitted at any time through our usual processes.

Timelines for this call for applications for medicines for rare disorders


Call for supplier applications

3 September 2018

Applications for medicines for rare disorders close

November 2018

Consideration by Rare Disorders Subcommittee

Early 2019

Next steps determined for applications received

About medicines for rare disorders and PHARMAC

In 2014, PHARMAC tested a commercial process aimed at improving people’s access to medicines for rare disorders. A lack of competition was seen as a barrier to better access to medicines for rare disorders and so up to $5 million per year for the next five years was made available to fund a contestable pilot.  10 medicines were approved for listing on the Pharmaceutical Schedule.

In June 2017, PHARMAC published the external evaluation of the contestable funding pilot [PDF, 514 KB]. The conclusions drawn from the report were favourable - as a result of the pilot better commercial proposals were received and funded access to treatments for rare disorders improved.

In late 2017 PHARMAC announced that it was introducing a set of dedicated features for considering rare disorders medicines, so we can get the best out of our existing processes.  These include:

  • A standing Pharmacology and Therapeutics Advisory Committee (PTAC) expert subcommittee for rare disorders will be established. 
  • PHARMAC will regularly call for rare disorder funding applications.
  • PHARMAC will undertake dedicated pre-engagement with new, as well as existing, suppliers prior to each call for funding applications.
  • PHARMAC will formally adopt adjusted policy settings for rare disorders treatments.
  • The portfolio of rare disorders treatments would be regularly reviewed and good or reasonable opportunities for investment would progress through our routine process. Additionally, if a portfolio of rare disorders investments offers an opportunity to better obtain health gain, then we would run another round of contestable funding or develop an alternate commercial approach, dependent on circumstances.

These steps are now all underway.

More information regarding our work in rare disorders is available on the PHARMAC website.

To submit applications

Send us an email: 5pm on Monday, 3 September 2018.

PHARMAC staff will be meeting with suppliers of medicines for rare disorders over the coming months.

If you would like to discuss the application process please contact the Therapeutic Group Manager leading this process, Caroline De Luca, Senior Therapeutic Group Manager/Team Leader.