Role

The Life Saving Drugs Program Expert Panel (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 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 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 panel 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 panel 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.

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.

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.

Professor Elizabeth Elliott AM (clinical expert)

Professor Elliott is a Professor of Paediatrics and Child Health, Sydney Medical School, University of Sydney, and a Consultant Paediatrician, Sydney Children’s Hospitals Network (Westmead).

Liliana Bulfone (industry nominee)

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

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.

Meetings

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

Agendas

We publish meeting agendas 4 weeks in advance of each meeting. The agenda lists all items the panel 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.

2022 meetings

2021 meetings

2020 meetings

Submissions

The LSDPEP welcomes input from the public, including patients, carers, family members, treating physicians and advocates. Once we publish the agenda 4 weeks before the upcoming meeting, any interested parties can email their input to the LSDP secretariat. We must receive the input by 2 weeks before the meeting.

We keep 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

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 will be reviewed in late 2021 as part of the 24 month review process.

Asfotase alfa

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

Medicine reviews

We have commissioned 9 disease-based reviews of LSDP medicines.

We have not yet commissioned a review of elosulfase alfa (Vimizim®, for mucopolysaccharidosis type IVA).

Similar reviews will be undertaken on all existing LSDP medicines over the first two years from the commencement of the new program (called 24 month reviews). These reviews will be conducted in accordance with the agreed LSDP Procedure Guidance.

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

Gaucher disease (type 1)

Due to its limited use, miglustat will be reviewed separately

Miglustat (Zavesca®)

Miglustat review plan

 

24 month reviews 

In their meeting of 17 June 2021 the LSDEP commissioned the 24 month reviews process to begin for migalastat (Galafold®) for Fabry disease and cerliponase alfa (Brineura®) for neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as Batten disease. 

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. It is anticipated that the Panel will provide recommendations to these reviews in late 2021. However, 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 

Contact

Life Saving Drugs Program Expert Panel secretariat

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

LSDPEP [at] health.gov.au

View contact

Last updated: 
21 January 2022