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Better support for patients with rare diseases

The Turnbull Government is improving Australia’s Life Saving Drugs Program to ensure patients with rare and life threatening diseases continue to get access to vital medicines.

The Hon Greg Hunt MP
Former Minister for Health and Aged Care

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The Turnbull Government is improving Australia’s Life Saving Drugs Program to ensure patients with rare and life threatening diseases continue to get access to vital medicines.

Patients will be better supported by a new process for listing drugs on the Program, which includes expanded criteria to consider medicines which both extend a patient’s life and improve a patient’s quality of life.

A new independent panel will support the Chief Medical Officer in considering applications. This will mean pharmaceutical companies will have a clearer path for assessment, with more certainty.

This is ultimately about providing more support to some of our sickest Australians.

It is a big win for patients right across Australia and I hope we can support many more people who are facing incredibly tough health challenges.

Today’s improvements to the Life Saving Drugs Program have followed recommendations from an independent expert review (the Wilson Review).

The Government will draw on the Review’s findings and will adopt the following improvements to the Life Saving Drugs Program:

  • Specify that lifesaving medicines are those that extend lifespan, including through the measurement of substantial reduction to the level and duration of disability, which will lead to a significant increase in life extension.
  • Adopting a rare disease definition of 1:50,000 people or less in the Australian population (around 500 people).
  • Introducing a more structured system for consideration of the clinical benefits of very high cost medicines referred to the Life Saving Drugs Program by the Pharmaceutical Benefits Advisory Committee, including establishing an expert panel which will provide advice and assistance to the Chief Medical Officer.

We will also streamline administration with sponsor companies while introducing a mechanism to review the effectiveness of the medicines after 24 months.

The new Life Saving Drugs Program will include an appropriate assessment for medicines that treat rare diseases, recognising the challenges in listing these drugs.

This will mean better access to medicine for patients with rare and life threatening diseases.

These improvements also aim to put the program on a more robust, efficient and sustainable footing to ensure ongoing access to rare disease medicines into the future.

I welcome the findings of the Wilson Review and acknowledge the importance and need to continue to enable Australians with rare life threatening diseases to access lifesaving medicines that are safe and clinically effective.

The Government commissioned review explored a range of matters associated with rare diseases, and examined important issues such as access and equity, value for money and the future administration of the program.

The review group was chaired by Professor Andrew Wilson, Director of the Menzies Centre at the University of Sydney. Expert advice was provided to the Committee by specialist clinicians, medical ethicists, health economists, and consumers.

As a result of the Review’s extensive stakeholder engagement, interested parties made 90 submissions. We will continue to consult with key stakeholders in implementing the improvements announced today.

The battle against rare diseases is a fight we will continue to have in support of patients in need.

Only last week we announced $69 million for clinical trials targeting rare cancers, rare diseases and unmet need under the Medical Research Future Fund.

We are committed to continuing to invest in research to find the answers to these challenges, while also funding vital medicines to treat those in need and provide them with a better life.


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