Life Saving Drugs Program Expert Panel

Page last updated: 28 May 2019

An Expert Panel for the Life Saving Drugs Program (LSDP) has been established to provide assistance and advice to the Commonwealth Chief Medical Officer (CMO). The Expert Panel will consider and advise on both new medicine applications and reviews of existing medicines. This advice will support the CMO in the development of recommendations to the Commonwealth Minister for Health on the suitability of medicines for funding (either new or ongoing) under the LSDP. The Expert Panel includes suitably qualified experts including a consumer representative, an industry nominee, a health economist and clinicians.

Terms of Reference

1. The Expert Panel provides advice the CMO on a range of matters relating to new medicines seeking funding through the LSDP, including:
  • assessment of how the medicine addresses the LSDP criteria;
  • guidelines for use, including testing requirements;
  • pricing arrangements; and
  • data collection requirements for future review.
2. In reviewing LSDP medicines, the Expert 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; and
  • Provide advice to the CMO regarding any appropriate changes to medicine funding arrangements.
3. The Expert Panel will advise on any other matter that may relate to the LSDP as directed by the Minister or CMO.

Current Membership

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 eleven 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.

Dr James Butler – HEALTH ECONOMIST
Dr Butler works as a health economics consultant and currently sits on the Commonwealth’s Prostheses List Advisory Committee and its Health Economics Sub 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 a Senior Research Fellow at the Health Economics Unit, Deakin University and has a background in health technology assessment.

Nicole Millis- CONSUMER NOMINEE
Ms Millis is the Executive Officer at Rare Voices Australia.

Expert Panel meeting agenda

The Expert Panel meets three times per year, usually February, June and October.

The agenda of the LSDP Expert Panel will be published four weeks in advance of the meeting and will list all items that will be considered at the upcoming meeting. This includes new medicines for funding under the LSDP and consideration of outcomes from LSDP medicine reviews.

Current Meeting Agenda

28 June 2019 Meeting Agenda (PDF 555 KB)
28 June 2019 Meeting Agenda (Word 18 KB)

Previous Meeting Agendas

17 October 2018 meeting Agenda (PDF 72 KB)
17 October 2018 meeting Agenda (Word 23 KB)

8 March 2019 Meeting Agenda (PDF 274 KB)
8 March 2019 Meeting Agenda (Word 16 KB)

Expert Panel Stakeholder Forum

A Stakeholder Forum will be conducted during the consideration of a new medicine or the review of existing medicines, where stakeholders such as sponsors, consumer and clinicians are invited to provide their views to the Expert Panel.

Expert Panel consumer submissions

Once the agenda for the upcoming Expert Panel meeting is published, any interested parties are welcome to provide their input directly to the LSDP Expert Panel Secretariat via email. Contact the LSDP Expert Panel Secretariat if you would like to be notified of any changes to the LSDP webpages, including when meeting agendas are published.

Stakeholder input is kept confidential on request. Full details around the consumer submission process can be found in the Procedure Guidance document on the main LSDP webpage.

For new medicines

Public submissions to the Panel are welcomed in the Expert Panel’s consideration of the needs of, and benefits to, patients living with the rare disease being discussed. These can be from any member of the public: patient, family member, carer, physician, advocate, etc.

As all medicines to be considered by the LSDP Expert Panel will have already been considered by the PBAC for listing on the PBS, all consumer comments received throughout the corresponding PBAC process will be made available to the Panel. Stakeholders do not need to duplicate responses.

The status of new medicine applications can be found on the New Medicine Applications for the LSDP webpage.

For reviews of existing LSDP medicines

There are multiple opportunities for consumer input throughout the review of existing medicines:
  1. When the Panel considers the draft Terms of Reference
  2. After the draft Terms of Reference have been published
  3. During the stakeholder forum. This is usually held on the day of the scheduled Expert Panel meeting, after the terms of reference have been endorsed by the CMO. Forums are based on discussion questions that have been developed in consultation with the Expert Panel.
The flowchart below provides an overview of the review process.

This flow chart outlines that at the first panel meeting for a review, the expert panel will consider the main issues to inform the draft terms of reference. The draft terms of reference will be published at this time for stakeholder comment.

Submission Timings


Once the Expert Panel meeting agenda has been published, any interested parties are welcome to provide their input directly to the LSDP Secretariat via email.

This input must be provided two weeks prior to the Expert Panel meeting to be considered.

Status of LSDP medicine reviews

Tranche 1


1. Fabry Disease – SUBMISSIONS CLOSED FOR TERMS OF REFERENCE AND REVIEW PROTOCOL
Agalsidase alfa (Replagal®) and Agalsidase beta (Fabrazyme®)

Terms of Reference
The Terms of Reference for the Fabry Disease medicine review were endorsed by the Commonwealth Chief Medical Officer on 10 December 2018 and can be found below.

Terms of Reference for Review of Fabry Disease Medicines (PDF 285 KB)
Terms of Reference for Review of Fabry Disease Medicines (Word 22 KB)

Review Protocol
A stakeholder forum to discuss the Fabry disease medicine review protocol was held in Melbourne on 21 February 2019. Stakeholders made a number of comments which were considered by the Expert Panel. The Summary of Changes to the Draft Review Protocol and the finalised Review Protocol are available below.

Review Protocol for Fabry disease (PDF 1240 KB)
Review Protocol for Fabry disease (Word 394 KB)

Summary of Changes to Draft Review Protocol for Fabry disease (PDF 652 KB)
Summary of Changes to Draft Review Protocol for Fabry disease (Word 35 KB)

2. Gaucher Disease (Type 1) – SUBMISSIONS CLOSED FOR TERMS OF REFERENCE AND REVIEW PROTOCOL
Imiglucerase (Cerezyme®), Velaglucerase (VPRIV®), Taliglucerase (Elelyso®) and Miglustat (Zavesca®)

Terms of Reference
The Terms of Reference for the Fabry Disease medicine review were endorsed by the Commonwealth Chief Medical Officer on 10 December 2018 and can be found below.

Terms of Reference for Review of Gaucher Disease Medicines (PDF 287 KB)
Terms of Reference for Review of Gaucher Disease Medicines (Word 23 KB)

Review Protocol
A Stakeholder forum to discuss the Gaucher disease medicine review protocol was held in Sydney on 22 February 2019. Stakeholders made a number of comments which were considered by the Expert Panel. The Summary of Changes to the Draft Review Protocol and the finalised Review Protocol are available below.

Review Protocol for Gaucher disease (PDF 1285 KB)
Review Protocol for Gaucher disease (Word 497 KB)

Summary of Changes to Draft Review Protocol for Gaucher disease (PDF 679 KB)
Summary of Changes to Draft Review Protocol for Gaucher disease (Word 41 KB)

Due to its limited utilisation, miglustat (Zavesca®) will not be assessed with the other Gaucher disease medicines, and will instead undergo a separate review by the Department. The miglustat Review Plan has been finalised by the LSDP Expert Panel and is available below.

Miglustat Review Plan (PDF 542 KB)
Miglustat Review Plan (Word 38 KB)

Tranche 2


3. Mucopolysaccharidosis Type II - SUBMISSIONS OPEN FOR REVIEW PROTOCOL
Idursulfase (Elaprase®)

Terms of Reference
The Terms of Reference for the Mucopolysaccharidosis Type II medicine review were endorsed by the Commonwealth Chief Medical Officer on 26 March 2019 and can be found below.

Terms of Reference for Review of MPSII medicines (PDF 480 KB)
Terms of Reference for Review of MPSII medicines (Word 18 KB)

Review Protocol
A Stakeholder Forum to discuss the draft Mucopolysaccharidosis Type II disease medicine Review Protocol will be held in Melbourne on 6 June 2019. Stakeholder comments are due to the LSDP Secretariat via email by 13 June 2019.

Draft Review Protocol for MPS II (PDF 1384 KB)
Draft Review Protocol for MPS II (Word 437 KB)

4. Mucopolysaccharidosis Type IVa – SUBMISSIONS CLOSED
Elosulfase Alfa (Vimizim®)

As Elosulfase Alfa was included on the LSDP in 2017, it will not have a full review at this time.

5. Pompe Disease – SUBMISSIONS OPEN FOR REVIEW PROTOCOL
Alglucosidase Alfa (Myozyme®)

Terms of Reference
The Terms of Reference for the Pompe disease review were endorsed by the Commonwealth Chief Medical Officer on 26 March 2019 and can be found below.

Terms of Reference for Review of Pompe disease medicines (PDF 482 KB)
Terms of Reference for Review of Pompe disease medicines (Word 16 KB)

Review Protocol
A Stakeholder Forum to discuss the draft Pompe disease Review Protocol will be held in Sydney on 14 June 2019. Stakeholder comments are due to the LSDP Secretariat via email by 13 June 2019.

Draft Review Protocol for Pompe (PDF 1300 KB)
Draft Review Protocol for Pompe (Word 577 KB)

6. Paroxysmal Nocturnal Haemoglobinuria – SUBMISSIONS OPEN FOR REVIEW PROTOCOL
Eculizumab (Soliris®)

Terms of Reference
The Terms of Reference for the Paroxysmal Nocturnal Haemoglobinuria review were endorsed by the Commonwealth Chief Medical Officer on 26 March 2019 and can be found below.

Terms of Reference for Review of PNH disease medicines (PDF 484 KB)
Terms of Reference for Review of PNH disease medicines (Word 18 KB)

Review Protocol
A Stakeholder Forum to discuss the draft Paroxysmal Nocturnal Haemoglobinuria Review Protocol will be held in Melbourne on 6 June 2019. Stakeholder comments are due to the LSDP Secretariat via email by 13 June 2019.

Draft Review Protocol for PNH (PDF 1327 KB)
Draft Review Protocol for PNH (Word 413 KB)

Tranche 3


7. Mucopolysaccharidosis Type I – SUBMISSIONS OPEN FOR TERMS OF REFERENCE
Laronidase (Aldurazyme®)

Terms of Reference
Comments on the draft Terms of Reference are due to the LSDP Secretariat via email by 17 June 2019.

Draft Terms of Reference for MPS I (PDF 480 KB)
Draft Terms of Reference for MPS I (Word 18 KB)

8. Mucopolysaccharidosis Type VI – SUBMISSIONS OPEN FOR TERMS OF REFERENCE
Galsulfase (Nagalazyme®)

Terms of Reference
Comments on the draft Terms of Reference are due to the LSDP Secretariat via email by 17 June 2019.

Draft Terms of Reference for MPS VI (PDF 483 KB)
Draft Terms of Reference for MPS VI (Word 19 KB)

9. Hereditary Tyrosinaemia Type I – SUBMISSIONS OPEN FOR TERMS OF REFERENCE
Nitisinone (Orfadin®)

Terms of Reference
Comments on the draft Terms of Reference are due to the LSDP Secretariat via email by 17 June 2019.

Draft Terms of Reference for HTI (PDF 481 KB)
Draft Terms of Reference for HTI (Word 18 KB)

Further Information

Life Saving Drugs Program Expert Panel Secretariat
Email: LSDP EP Secretariat