Call for funding applications for medicines for rare disorders
What we’re doing
Te Pātaka Whaioranga - Pharmac invites pharmaceutical suppliers to submit funding applications for medicines for rare disorders.
We understand that people living with rare disorders face many challenges. These include access to suitable health care and effective medicines. We are taking steps to encourage funding applications from pharmaceutical suppliers and clinicians for medicines for rare disorders. Our next Rare Disorders Advisory Committee is scheduled for 29 May 2024.
This call for applications closes at 5pm on Friday 8 March 2024 and applications can be submitted through our application portal – PharmConnect.
More information about how to do this is on our website: Medicine funding application.
Input from people with rare disorders
We also want to hear from the rare disorders community about the medicines Te Pātaka Whaioranga - Pharmac is considering for funding for the treatment of rare disorders. We will share the agenda of the Rare Disorders Advisory Committee meeting in early April 2024. We welcome input from people with rare disorders to support our assessment of applications at any time throughout the funding assessment process. Queries and feedback about applications can be emailed to raredisorders@pharmac.govt.nz
Anyone can make an application for a medicine to be funded for a rare disorder at any time. You can find out about the progress of applications Pharmac is already considering here: Pharmac's application tracker(external link). If you are thinking about submitting an application the first step is to get in touch with us so we can help avoid any duplication of effort.
More information to support applicants to submit a funding application is available here: using PHARMConnect for Prescribers and patients [PDF, 136 KB]
Who we think will be interested
- Pharmaceutical suppliers of medicines for rare disorders
- People with rare disorders and their whānau, family and caregivers
- Healthcare professionals and support groups with an interest in medicines for rare disorders.
How will it work?
Te Pātaka Whaioranga - Pharmac is committed to regularly calling for applications for medicines for rare disorders ahead of meetings of our Rare Disorders Advisory Committee(external link). This Committee provides Pharmac with clinical advice about medicines for rare disorders for which funding is sought.
The next Rare Disorders Advisory Committee meeting is scheduled for 29 May 2024.
We invite funding applications for medicines for rare disorders that meet the principles of our Rare Disorders policy:
- The medicine has been approved by Medsafe, or an approved international regulatory authority[1], for the identified indication[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.
- The medicine is only regulatory approved for the treatment of the rare disorder, or if it is approved 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 [PDF, 630 KB] for pharmaceutical suppliers. Supplier applications should also include a commercial proposal.
For suppliers, a key difference to the usual Pharmac process is the ability to submit a funding application for a rare disorder medicine prior to Medsafe submission and/or approval. Medsafe approval would still be required before a pharmaceutical could be listed on the Pharmaceutical Schedule.
If the principles are not met, then the application may not go to the Rare Disorders Advisory Committee. Depending on which principle is not met, we may not accept the application at this time, or we may refer it to another relevant Pharmac Specialist Advisory Committee for advice.
More information regarding our work in medicines for rare disorders is available on the Pharmac website.
Next steps following submission
Applications accepted in line with our policy and received by Friday 8 March will be considered for inclusion on the May agenda for the Rare Disorders Advisory Committee meeting and assessed as per our usual processes.
Should applications receive positive funding recommendations following clinical advice, they would be assessed and ranked against other medicines on the Pharmac options for investment list. Pharmac would then determine the appropriate next steps for the application, such as commercial processes.
Applications received after 8 March will be considered for future Advisory Committee meetings.
Timelines for applications for medicines for rare disorders for consideration at the May 2024 Rare Disorders Advisory Committee meeting
|
Now |
Call for applications issued |
|---|---|
|
8 March 2024 |
Closing date for applications to be considered at the May meeting |
|
Early April 2024 |
Confirmed agenda for the May meeting published |
|
29 May 2024 |
Applications by Rare Disorders Advisory Committee considered |
|
August/September 2024 |
Records of meeting published and next steps determined for applications received |
About medicines for rare disorders and Pharmac
The Rare Disorders Advisory Committee last met in March 2023 at a joint meeting with members from our Gastrointestinal and Neurological Advisory Committees to discuss treatments for spinal muscular atrophy, Fabry disease, mucopolysaccharidosis and short bowel syndrome. The record of this meeting [PDF, 525 KB] and the Committee’s previous meetings are available on the Pharmac website.
The agenda for the Rare Disorders Advisory Committee May 2024 meeting will include consideration of new funding applications and review existing funded medicines for rare disorders, including medicines considered via our Named Patient Pharmaceutical Assessment (NPPA) process for individual patients.
As outlined in Pharmac’s full response to the Pharmac Review, available on our website, we agree the system needs to do more to improve the lives of people with rare disorders. We stated we will prioritise engagement with our Rare Disorders Advisory Committee and we look forward to supporting Manatū Hauora in its important work to develop a rare disorders strategy.
More information regarding our work in medicines for rare disorders(external link) is available on the Pharmac website.
To submit applications
Applications can be submitted through our application portal – PharmConnect by 5pm on Friday 8 March 2024.
Pharmac is available to meet with interested suppliers or other applicants prior to submission of any application.
More information about how to do this is on our website: Medicine funding application.
If you would like to discuss the application process, please contact the Funding Application Advisors at raredisorders@pharmac.govt.nz
[1] Refer to Medsafe website for guidance on recognised regulatory authorities(external link)
[2] Medsafe approval must still be gained before a pharmaceutical is listed on the Pharmaceutical Schedule.