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®) |
|
|
Mucopolysaccharidosis type VI |
Galsulfase (Nagalazyme®) |
|
|
Hereditary tyrosinaemia type 1 |
Nitisinone (Orfadin®) |
|
|
Pompe disease |
Alglucosidase alfa (Myozyme®) |
|
|
Mucopolysaccharidosis type II |
Idursulfase (Elaprase®) |
|
|
Paroxysmal nocturnal haemoglobinuria |
Eculizumab (Soliris®) |
|
|
Fabry disease |
Agalsidase alfa (Replagal®) Agalsidase beta (Fabrazyme®) |
|
|
Gaucher disease (type 1) |
Imiglucerase (Cerezyme®) Velaglucerase (VPRIV®) Taliglucerase (Elelyso®) |
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®) |
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®) |
2022 |
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