Call for funding applications for medicines for rare disorders

What we’re proposing

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

This call for applications closes at 5pm on Monday 5 August 2019 and applications can be emailed to raredisorders@pharmac.govt.nz.

Who we think will be interested

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

How will it work?

PHARMAC is committed to regularly calling for applications for medicines for rare disorders. PHARMAC’s Rare Disorders Subcommittee of the Pharmacology and Therapeutics Advisory Committee (PTAC) was established last year to provide advice on funding applications for medicines for rare disorders and had its first meeting in November 2018.

The next Rare Disorders Subcommittee meeting will be on 24 September 2019.

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 also include a commercial proposal.

Next steps following submission

Applications that meet the principles will be considered through an assessment process in line with our usual processes, including seeking clinical advice.  

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

Applications will generally be considered by the Rare Disorders Subcommittee as the first step in the application assessment process. Following clinical advice, the applications would be ranked against other medicines funding options and 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. Applications received by 5 August 2019 will be considered for inclusion on the agenda of the 24 September meeting of the Rare Disorders Subcommittee, as shown in the table below.

Timelines for this call for applications for medicines for rare disorders

About medicines for rare disorders and PHARMAC

In 2014, PHARMAC tested a commercial process aimed at improving access to medicines for rare disorders. A lack of competition was seen as a barrier to getting 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.  Ten medicines were approved for listing on the Pharmaceutical Schedule through this process.

In June 2017, PHARMAC published an external evaluation of the contestable funding pilot [PDF, 514 KB]. 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 medicines for rare disorders, so we can get the best out of our existing processes. These included:

• A standing Pharmacology and Therapeutics Advisory Committee (PTAC) expert subcommittee for rare disorders will be established. 
• Regularly calling for rare disorder funding applications.
• Undertaking dedicated pre-engagement with new, and existing, suppliers prior to each call for funding applications.
• Formally adopting adjusted policy settings for rare disorders treatments.
• Regularly reviewing the portfolio of medicines for rare disorders and progressing good or reasonable opportunities for investment through our routine process. Additionally, if a portfolio of rare disorders investments offered 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 features were implemented in 2018 and are now part of our usual process. We are now planning for our second Rare Disorders Subcommittee in September 2019. This will include consideration of new funding applications from suppliers or clinicians and reviewing existing funded medicines for rare disorders, including medicines considered via our Named Patient Pharmaceutical Assessment (NPPA) process for individual patients.

More information regarding our work in medicines for rare disorders is available on PHARMAC’s website.

To submit applications

Send us an email at raredisorders@pharmac.govt.nz by 5pm on Monday, 5 August 2019.

If you would like to discuss the application process, please contact the Therapeutic Group Managers leading this process:

• Sarita Von Afehlt - Therapeutic Group Manager
• Caroline De Luca - Senior Therapeutic Group Manager/Team Leader