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.
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 |
Implementation
Following Government endorsement, the Department of Health, Disability and Ageing (the department) has been working to implement the recommendations. This has included work to streamline the testing requirements for Pompe disease, Gaucher disease (type 1), and mucopolysaccharidosis types I, II and VI, to help reduce the testing burden on patients, their families and carers and treating physicians.
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 |
|---|---|---|---|
| Pompe Disease | Avalglucosidase alfa (Nexviazyme®) | Avalglucosidase alfa Terms of Reference and Protocol Questions | 2024 |
| Mucopolysaccharidosis type IVA | Elosulfase alfa (Vimizim®) | 2022 | |
| Fabry Disease | Migalastat (Galafold®) | Migalastat Terms of Reference and Protocol Questions | 2021 |
| CLN2 Disease | Cerliponase alfa (Brineura®) | 2021 |
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.
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