For the past 8 years MDDA has been proactively seeking subsidised access to new therapies for all PKU patients including adults. We advocated strongly for this during the last PBAC submission for BH4 by insisting adults be included in the submission and then ensuring we had adult representatives attend consumer meetings with the PBAC. Unfortunately, the application for adults was unsuccessful based on legislated cost effectiveness formulas used in evaluating new therapies. Originally the PBAC intended to recommend listing only for use in childhood with access ceasing at 18. The MDDA successfully argued that this would lead to an increase in adults not adhering with treatment if this therapy was taken away at aged 18. To help us make a stronger case in the future for this, and for other treatments, we will need to gather more evidence.

What MDDA are doing:

MDDA has continued to actively pursue all possible avenues for having pharmacological treatments for PKU subsidised for all Australians. We have developed some videos that form part of our awareness campaign which you can view here.

We have also taken the following steps:

  1. Meeting with PBAC consumer representatives to understand all ways an application can be made – this has included the MDDA investigating making its own public interest submission for subsidising BH4 for adults. After investigation, the current rules around public submissions means that this is not a viable option. We continue to provide evidence to the PBAC and government decision makers about the unmet need for people with PKU who only have access to the dietary therapy.
  2. Continuing discussions with the sponsor about the need for ALL Australians to have access to all safe and approved treatments that are available overseas.
  3. Researching alternative options for having BH4 subsidised.
  4. Promoting Australia as a good destination for promising clinical trials. Advocating for compassionate access to treatment for those who participate in clinical trials that are effective with pharmaceutical companies who are in the process of trials or are considering trials.
  5. Working with ASIEM and international PKU organisations to prepare surveys and research that will help us provide more evidence to the PBAC about the experience of PKU patients in Australia and the unmet need.
  6. Providing input into the National Rare Disease Action Plan, in particular the need for the PBAC to have a fit for purpose evaluation tool for rare disease therapies for conditions like PKU. While this is a long term strategy, recognition from the government that rare disease therapies are a specialised area and that they are being unfairly measured against therapies for common conditions is intended to create an easier reimbursement pathway for all future rare disease therapies. As there are many new treatments being developed for all IEMs this is an important part of our advocacy strategy.


What YOU can do:

WE NEED YOUR HELP!! We need more data to help decision makers understand the experience for PKU patients. We will shortly be looking for people to participate in research and surveys and we encourage all PKU patients to participate. In addition, it is always a good idea to develop a good relationship with your local federal member and discuss with them your concerns about access to therapies for your condition. You can find who your local member is here. While they are not able to do much without the PBAC process being completed it helps us when we do need support for specific submissions – which we hope to see in the coming months. You can also help by sharing the below video and be on the lookout for posts on Facebook asking for survey/research participants. If you have any questions or want to find out more ways in which you can help, please email [email protected].