Decision to decline inactive funding applications

Deflazacort - Duchenne muscular dystrophy(external link)

Feedback was overwhelmingly opposed to declining the deflazacort funding application and highlighted significant quality-of-life and tolerability differences between deflazacort and prednisone. Details of families’ experiences of both prednisone and deflazacort treatment were shared.

Many respondents considered that lived experience differed significantly to published clinical evidence used to inform PTAC’s recommendation to decline the application.

Respondents requested that, should deflazacort progress to being declined, that another treatment for Duchenne Muscular Dystrophy be made available and funded.

We acknowledge that there are high unmet health needs for people with Duchenne Muscular Dystrophy (DMD), and that there is high level of care provided by their whānau. 

We also acknowledge the feedback received from consumers and advocacy groups and the lived experiences shared regarding differences in treatment tolerability and quality of life between prednisone and deflazacort.

While PTAC has considered this application on multiple occasions and most recently (May 2024) recommended decline after reviewing the available clinical evidence, consultation feedback highlighted lived experiences suggesting deflazacort provides meaningful benefits for some people with Duchenne muscular dystrophy.

May 2024 PTAC meeting record [PDF, 995 KB]

Given this additional information and the significant differences between consumer experience and the clinical advice received to date, Pharmac has decided not to formally decline the application at this time.

The application will remain on the “Recommended for Decline” list, consistent with our most recent clinical advice. We also note that there is currently no Medsafe approved product. If an approved product were to become available, we could reconsider the application in light of any new evidence that may be available at the time, along with the information and feedback provided in response to consultation on this proposal.

We have also engaged with Rare Disorders New Zealand and Muscular Dystrophy New Zealand to identify priority treatments and seek funding applications from suppliers.

Meningococcal group B vaccine - Invasive meningococcal disease(external link)

No consultation feedback was received. However, information on meningococcal vaccines was recently reconsidered by the Immunisation Advisory Committee in May 2026.

Agenda for May 2026 Immunisation Advisory Committee (IAC) meeting

Pharmac has decided not to decline the application at this time. In light of the recent consideration of meningococcal vaccines by our advisory committee, Pharmac has decided not to decline the application while we consider the implications of the advice on the application.

A separate funding application for meningococcal group B vaccine for invasive meningococcal disease, widened access to current close living situations for people 13 – 25 years old(external link) remains active.

Micronutrient formulas - ADHD and or mood dysregulation(external link)

Feedback was opposed to declining the application. Submitters highlighted personal experiences of benefit from micronutrient formulas for ADHD and mood dysregulation. Key themes included improved wellbeing, fewer side effects than conventional treatments, greater treatment choice, potential health system savings, and concerns about affordability for families who currently self-fund treatment. Submitters also raised concerns that regulatory barriers, rather than lack of clinical value, were preventing access to funded treatment.

We acknowledge that micronutrient formulas may have a health benefit for treatment of ADHD and/or mood dysregulation as noted in the recommendation by PTAC in August 2022. We understand that more treatment options that work differently to currently funded options are needed for ADHD and mood dysregulation.

August 2022 PTAC meeting record [PDF, 680 KB]

However, for the requested product, Daily Essential Nutrients, as no application for approval as a medicine has been submitted to Medsafe and no Medsafe approved product is available, it does not currently meet Pharmac's usual requirements for funding consideration. Regulatory approval is important when Pharmac considers a treatment for funding as stated in our Guidelines for funding applications to Pharmac.

From application to funded medicine: Pharmac's process

Medsafe is the authority responsible for the regulation of therapeutic products in New Zealand. The role of Medsafe is to make sure treatments meet quality and safety standards and that the benefits of use outweigh the risks of harm in the intended treatment population.

About Medsafe | Medsafe(external link)

We note the product contains lithium orotate. Lithium is classified as a medicine in New Zealand. A recent application to reclassify low-dose lithium for general sale was declined by Medsafe’s Medicines Classification Committee.

Minutes for the 74th meeting of the Medicines Classification Committee | Medsafe(external link)

In light of the above information, we have decided to decline the application at this time. If a supplier applies for and obtains Medsafe approval, Pharmac could reconsider a funding application.

We have shared the summary of the consult feedback with Medsafe so that it is aware of responses.

We appreciate the feedback from people, and we understand that declining an application is not the outcome patients or their whānau may want.

Esketamine - treatment resistant depression, post augmentation(external link)

One respondent opposed declining esketamine and highlighted its potential benefits including:

• a rapid onset of action when compared to conventional antidepressants
• evidence of benefit for people who have not responded to multiple prior treatments
• endorsed by specialist psychiatric organisations for carefully selected patients
• the potential to generate health system efficiencies alongside improved patient outcomes.

We appreciate the feedback provided and acknowledge the unmet health needs experienced by people with treatment-resistant depression.

We note that the information submitted in this response was considered by PTAC when it recommended this for decline. Should new evidence become available that PTAC has not considered, the application could be reopened.

February 2024 PTAC meeting record [PDF, 847 KB]

Atezolizumab - Urothelial carcinoma, locally advanced or metastatic, 2nd line(external link)

Feedback supported the proposal to decline atezolizumab for this indication. Respondents noted that atezolizumab’s FDA approval for this use was withdrawn in 2022 due to efficacy data.

They also noted that in light of pembrolizumab funding, the need is met for this indication.

We appreciate the feedback.

Dabrafenib - Melanoma, stage III/IV with BRAF V600 mutation(external link)

We received feedback indicating there are no concerns with the proposal and thanking Pharmac for the opportunity to provide feedback and for providing rationale to the proposal.

We appreciate the feedback.

Vemurafenib - Melanoma, Stage IIIC/IV with BRAF V600 mutation(external link)

We received feedback indicating there are no concerns with the proposal and thanking Pharmac for the opportunity to provide feedback and for providing rationale to the proposal.

We appreciate the feedback.

SMA treatments - People with SMA type IV (aged 19 years and over at symptom onset)(external link)

We received feedback noting understanding behind why this recommendation had been made and thanking Pharmac for transparency in the process. No concerns with the proposal were noted.

We appreciate the feedback and are glad that the process has been transparent.