Life Saving Drugs Program medicine reviews

LSDP medicine reviews are designed to assess whether the use and performance of medicines is in line with the expectations at the time of listing.

2018-2020 Disease-based medicine reviews

In 2018 in response to the 2014 Post-market Review of the Life Saving Drugs Program the  Life Saving Drugs Program (LSDP) Expert Panel (the panel) commissioned 8 disease-based reviews of existing LSDP medicines. The purpose of these reviews was to explore:

  • the suitability of each medicine
  • the appropriateness of the eligibility or exclusion criteria and testing, and
  • avenues to improve the LSDP’s overall value for money.

The reviews were conducted by health experts, consumer representatives and an ethics advocate. The reports were drafted by contracted external evaluation groups. The panel considered the review reports and made 51 recommendations. The then Minister for Health, the Hon Greg Hunt MP, endorsed the panel’s recommendations in March 2022. We have started work on the implementation of the recommendations.

The panel acknowledges the significant contributions of clinicians, patients, sponsors and other stakeholders to the reviews.

Condition

LSDP medicine(s)

Review documents

Mucopolysaccharidosis type I

Laronisdase (Aldurazyme®)

MPS I review documents

Mucopolysaccharidosis type VI

Galsulfase (Nagalazyme®)

MPS VI review documents

Hereditary tyrosinaemia type 1

Nitisinone (Orfadin®)

HT-1 review documents

Pompe disease

Alglucosidase alfa (Myozyme®)

Pompe disease review documents

Mucopolysaccharidosis type II

Idursulfase (Elaprase®)

MPS II review documents

Paroxysmal nocturnal haemoglobinuria

Eculizumab (Soliris®)

PNH review documents

Fabry disease

Agalsidase alfa (Replagal®)

Agalsidase beta (Fabrazyme®)

Fabry disease review documents

Gaucher disease (type 1)

Imiglucerase (Cerezyme®)

Velaglucerase (VPRIV®)

Taliglucerase (Elelyso®)

Gaucher disease review documents

24-month reviews

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 to complete.

The panel considers the review report and makes recommendations that align with the Terms of Reference and the protocol questions for the review. 

The panel has conducted the following 24-month reviews:

Disease

LSDP medicine(s)

Review documents

Year of review

Fabry Disease

 Migalastat (Galafold®)

Migalastat Terms of Reference and Protocol Questions 

2021

CLN2 Disease

 Cerliponase alfa (Brineura®)

Cerliponase alfa Terms of Reference and Protocol Questions 

Cerliponase alfa 24 month review summaries

2021

Mucopolysaccharidosis type IVA

Elosulfase alfa (Vimizim®)

Elosulfase alfa Terms of Reference and Protocol Questions

Elosulfase alfa 24 month review summaries

2022

At its October 2023 meeting, the panel discussed the 24-month review of asfotase alfa (Strensiq®) for the treatment of perinatal- or infantile-onset hypophosphatasia (HPP). The panel agreed that the review should be deferred until the second half of 2025 to allow collection of additional data to inform the review.

Contact

Life Saving Drugs Program Expert Panel secretariat

Contact the secretariat with enquiries relating to the Panel, including new listing applications.
Date last updated:

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