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 March 2023.
This call for applications closes at 5pm on Monday 23 January 2023 and applications can be submitted through our application portal – PharmConnect.
More information about how to do this is on our website: Make a 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 late January 2023. 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
- Health 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. This Committee provides Pharmac with clinical advice about medicines for rare disorders for which funding is sought.
The next Rare Disorders Advisory Committee meeting will be on 7 March 2023.
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, 570 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 Monday 23 January will be considered for inclusion on the March agenda for the Rare Disorders Advisory Committee meeting and assessed as per our usual processes.
Following the receipt of clinical advice, the applications would usually be ranked against other medicines funding options and Pharmac would then determine the next appropriate steps for the application, such as commercial processes.
Applications received after 23 January will be considered for future Advisory Committee meetings.
Timelines for applications for medicines for rare disorders for consideration at the March 2023 Rare Disorders Advisory Committee meeting
| Now | Call for applications issued |
|---|---|
| 23 January 2023 | Closing date for applications to be considered at the March meeting |
| 27 January 2023 | Confirmed agenda for the March Meeting published |
| 7 March 2023 | Applications by Rare Disorders Advisory Committee considered |
| May/June 2023 | 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 July 2021 at a joint meeting with our Neurological Advisory Committee to discuss treatments for spinal muscular atrophy. Records of the Committee’s previous meetings are available on the Pharmac website.
The agenda for the Rare Disorders Advisory Committee March 2023 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.
In September 2022, Pharmac released its interim response [PDF, 835 KB] to Te Arotake i te Pātaka Whaioranga, the Pharmac Review. As outlined in this response, we agree the system needs to do more to improve the lives of people with rare disorders. We are looking forward to supporting Manatū Hauora in its important work to develop a rare disorders strategy. We stated we will also prioritise engagement with our Rare Disorders Advisory Committee and review our Exceptional Circumstances process to further test the policy settings we have in place. Pharmac’s full response to the Pharmac Review has been provided to the Minister of Health and will be available to public at a later date.
More information regarding our work in medicines for rare disorders is available on the Pharmac website.
To submit applications
Applications can be submitted through our application portal – PharmConnect by 5pm on Monday, 23 January 2023.
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: Make a medicine funding application
If you would like to discuss the application process, please contact the Therapeutic Group Managers at raredisorders@pharmac.govt.nz
- Joshua Cronin-Lampe - Therapeutic Group Manager
- Caroline De Luca - Senior Therapeutic Group Manager/Team Leader
Footnotes
[1] Refer to Medsafe website for guidance on recognised regulatory authorities [PDF](external link)
[2] Medsafe approval must still be gained before a pharmaceutical is listed on the Pharmaceutical Schedule.