Life Saving Drugs Program Expert Panel

The Life Saving Drugs Program Expert Panel (LSDPEP) considers all applications to list new medicines on the LSDP. The panel assesses medicines, listens to stakeholders, and advises the Chief Medical Officer about listing medicines. It also reviews medicines already on the program.

Role

The LSDPEP assesses applications to include new medicines on the Life Saving Drugs Program. It provides assistance and advice to the Australian Government Chief Medical Officer (CMO), including reviews of existing medicines. The CMO advises the Minister for Health and Aged Care on medicines suitable for listing.

The panel considers:

  • the sponsor’s application
  • the LSDP secretariat’s assessment (overview) of the submission
  • relevant materials from the Pharmaceutical Benefits Advisory Committee (PBAC) consideration, including:
    • ratified minutes/advice from PBAC and its sub-committees
    • responses from sponsors
    • stakeholder comments
  • additional stakeholder input
  • presentations made to the LSDPEP panel at the meeting.

Terms of reference

The LSDPEP advises the CMO on a range of matters relating to new medicines seeking funding through the LSDP, including:

  • assessing how the medicine addresses the LSDP criteria
  • guidelines for use, including testing requirements
  • pricing arrangements
  • data collection requirements for future review.

In reviewing LSDP medicines, the LSDPEP will:

  • determine the scope of the review
  • ensure relevant stakeholder input is included
  • review historical patient-level data in addition to any data available from international registries and in published literature
  • provide advice to the CMO regarding any changes to medicine funding arrangements.

The LSDPEP will advise on any other matters that may relate to the LSDP, as directed by the Minister or CMO.

Members

Member Background and experience
Professor Andrew Roberts (Chair)

Professor Roberts is an eminent researcher and clinical haematologist. He currently practices at the Royal Melbourne Hospital and Peter MacCallum Cancer Centre. Professor Roberts also served as a member of the Pharmaceutical Benefits Advisory Committee (PBAC) for 11 years.

Associate Professor Liliana Bulfone (industry nominee)

Ms Bulfone is the Managing Director of Shoten Pty Ltd and is an Associate Professor at the ANU College of Health and Medicine, Australian National University, and has a background in health technology assessment.

Professor John Christodoulou AM (clinical expert) Professor Christodoulou is the Co-Lead for brain and mitochondrial research at the Murdoch Children’s Research Institute.
Professor Jonathan Craig (clinical expert)

Professor Craig is Vice President and Executive Dean at the College of Medicine and Public Health, Flinders University and is a current member of Pharmaceutical Benefits Advisory Committee.

Nicole Millis (consumer nominee)

Ms Millis is the Chief Executive Officer at Rare Voices Australia, with over 10 years’ experience in rare disease consumer advocacy in the context of health technology assessment.

Professor Rosalie Viney (health economist)

Professor Viney is a Professor of Health Economics and Director of the Centre for Health Economics Research and Evaluation, University of Technology Sydney and is a member of the National Health and Medical Research Council Research Committee.

Dr Nick Simpson (ex officio) Dr Simpson is a Medical Officer at the Department of Health and Aged Care.

Meetings

The LSDPEP meets 3 times per year, usually in February, June and October.

Agendas

The LSDPEP secretariat publishes meeting agendas 4 weeks in advance of each meeting. The agenda lists all items the LSDPEP will consider at the upcoming meeting. This includes new medicines applications and considering the outcomes from LSDP medicine reviews.

If you require earlier agendas, please contact us.

2023 meetings

2022 meetings

2021 meetings

2020 meetings

Submissions

The LSDPEP welcomes input from the public, including patients, carers, family members, treating physicians and advocates on upcoming items. Once agenda is published, any interested parties can email their input to the LSDPEP secretariat. The LSDPEP secretariat must receive the input by 2 weeks before the meeting.

The LSDPEP secretariat keeps stakeholder input confidential on request. See full details of the consumer submission process in our procedure guidance.

All stakeholder comments received throughout the PBAC process are sent to the panel. Stakeholders do not need to duplicate responses.

Stakeholder forums

The LSDPEP conducts a stakeholder forum when considering new medicines or reviewing existing medicines. Stakeholders such as sponsors, consumers and clinicians can present their views.

Outcomes of new medicine applications

Avalglucosidase alfa

The LSDPEP considered this application on 18 February and 24 June 2022.
On 1 September 2022 avalglucosidase alfa was added to the LSDP. Read the avalglucosidase alfa outcome statement.

Asfotase alfa

The LSDPEP considered a reapplication on 10 October 2021. On 1 May 2022 asfotase alfa was added to the LSDP. Read the asfotase alfa outcome statement.

Asfotase alfa will be reviewed in mid 2024 as part of the 24 month review process.

The Chief Medical Officer previously considered this medicine before July 2018, when the Government introduced the LSDPEP and other changes. Read the previous asfotase alfa outcome statement.

Eliglustat

The LSDPEP considered this application on 15 November 2019. They deferred their decision until the Government-agreed outcomes from the Gaucher disease review are known. Read the eliglustat outcome statement.

Cerliponase alfa

The LSDPEP considered this application on 17 October 2018. On 1 May 2019, cerliponase alfa was added to the LSDP. Read the cerliponase alfa outcome statement.

Cerliponase alfa was reviewed in late 2021 as part of the 24 month review process.

Medicine reviews

In 2018 the LSDP commissioned 8 disease-based reviews of LSDP medicines.

The LSDPEP considered the review reports and made a total of 51 recommendations. The then Minister for Health, the Hon Greg Hunt MP, endorsed the LSDPEP's recommendations in March 2022. The Department has started work on the implementation of the recommendations.

The Department and the LSDPEP acknowledge the significant contributions of clinicians, patients, sponsors and other stakeholders to the reviews.

Condition

LSDP medicine(s)

Review documents

Stakeholder forum

Mucopolysaccharidosis type I

Laronisdase (Aldurazyme®)

MPS I review documents

Brisbane, 25 October 2019

Mucopolysaccharidosis type VI

Galsulfase (Nagalazyme®)

MPS VI review documents

Brisbane, 25 October 2019

Hereditary tyrosinaemia type 1

Nitisinone (Orfadin®)

HT-1 review documents

Melbourne, 24 October 2019

Pompe disease

Alglucosidase alfa (Myozyme®)

Pompe disease review documents

Sydney, 14 June 2019

Mucopolysaccharidosis type II

Idursulfase (Elaprase®)

MPS II review documents

Melbourne, 6 June 2019

Paroxysmal nocturnal haemoglobinuria

Eculizumab (Soliris®)

PNH review documents

Melbourne, 6 June 2019

Fabry disease

Agalsidase alfa (Replagal®)

Agalsidase beta (Fabrazyme®)

Fabry disease review documents

Melbourne, 21 February 2019

Gaucher disease (type 1)

Imiglucerase (Cerezyme®)

Velaglucerase (VPRIV®)

Taliglucerase (Elelyso®)

Gaucher disease review documents

Sydney, 22 February 2019

Similar reviews will be undertaken on all newly listed LSDP medicines two years after listing (called 24 month reviews). These reviews will be conducted in accordance with the agreed LSDP Procedure Guidance.

24 month reviews 

At its meeting of 17 June 2021 the LSDPEP commissioned 24 month reviews for migalastat (Galafold®) for Fabry disease and cerliponase alfa (Brineura®) for neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as Batten disease. It commissioned a 24 month review of elosulfase alfa (Vimizim®) for mucopolysaccharidosis type IVA at its 18 February 2022 meeting.

The purpose of 24-month reviews of newly listed medicines on the LSDP is to better understand the real-world use of a medicine by comparing the actual performance and use of the medicine to the recommendations and expectations at the time of listing. 

The LSDP procedure guidance details the 24 month reviews process. Stakeholders should note that more complex reviews or those requiring expert input may take longer.

Following the review process the expert panel will consider the report and make recommendations that align with the Terms of Reference (ToR) and the protocol questions outlined below. 

The ToRs below outline the main aims of each of the reviews. Some key protocol questions for consideration are listed below each ToR. 

Each review is not limited to the questions listed and evaluation may provide further advice to the Panel to inform the eventual recommendation(s) for each medicine. 

Condition LSDP medicine(s) Review documents

Fabry Disease

Galafold® (migalastat)

Migalastat Terms of Reference and Protocol Questions 

CLN2 Disease

Brineura® (cerliponase alfa)

Cerliponase alfa Terms of Reference and Protocol Questions 

Cerliponase alfa 24 month review

Mucopolysaccharidosis type IVA

Vimizim®

(elosulfase alfa)
Elosulfase alfa Terms of Reference and Protocol Questions

Life Saving Drugs Program Expert Panel secretariat

Contact the secretariat with submissions to the Life Saving Drugs Program Expert Panel.
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