Changes to products funded and supplied under the national blood arrangements
Interested parties, including patient groups or suppliers, can propose changes to the products or services that are publicly funded under the national blood arrangements.
Anyone wishing to make changes will be asked to provide evidence to support their proposal, including evidence-based evaluation of the product/service they nominate, as set out under Schedule 4 of the National Blood Agreement.
In order to make the application process consistent, a comprehensive Multi-Criteria Analysis Framework has been developed to assess proposals. Where appropriate, the Commonwealth’s Medical Services Advisory Committee (MSAC) health technology assessment process is also used to inform the cost effectiveness of proposals. In September 2011, the Jurisdictional Blood Committee (JBC) established a working group to review and update the Schedule 4 and health technology assessment processes.
For detailed information on the Schedule 4 / Multi-Criteria Analysis Framework, please contact the NBA at ProductReview@blood.gov.au.
Schedule 4 assessment - Alpha1-proteinase Inhibitor (A1-PI)
In July and September 2017, the National Blood Authority received National Blood Supply Change Proposals for purified human alpha1-proteinase inhibitor (A1-PI) for the treatment of A1-PI deficiency, from Grifols (for the product Prolastin-C) and from CSL Behring (for the product Zemaira).
On 1 December 2017, JBC referred the two proposals to MSAC for assessment. The assessment process included the opportunity for input through expert and public consultations.
MSAC considered the proposals on 22-23 November 2018. After considering the strength of available evidence put forward in relation to comparative safety, clinical effectiveness and cost-effectiveness, MSAC did not support A1-PI for the treatment of A1-PI deficiency.
The MSAC Public Summary Document setting out the basis for this MSAC advice can be found at http://www.msac.gov.au/internet/msac/publishing.nsf/Content/1530-public
On 14 March 2019, JBC agreed that, on the basis of the advice received from MSAC in relation to the submitted proposals, A1-PI products should not be funded under the National Blood Agreement at this time.
The advice from MSAC and the JBC decision based on that advice do not preclude resubmission of proposals with further evidence, in accordance with ordinary MSAC processes described at http://www.msac.gov.au/internet/msac/publishing.nsf/Content/msac-process-framework.
Schedule 4 assessment - emicizumab (Hemlibra) for haemophilia A
In 2017 the National Blood Authority received two National Blood Supply Change Proposals from Roche Products Pty Limited for the product emicizumab (Hemlibra), one for haemophilia patients with inhibitors to factor VIII, and one for haemophilia patients without inhibitors. The JBC referred each of these proposals to MSAC for assessment.
The proposal for haemophilia A patients with inhibitors was initially considered by MSAC on 22-23 November 2018 as MSAC application 1510. At this time MSAC advised that, while there was strong evidence to support the benefits of emicizumab, the product could not be shown to be cost-effective at the proposed price, and there were potential difficulties in ensuring that the use of the product would be limited to the target population. The Public Summary Document recording this advice can be found at http://www.msac.gov.au/internet/msac/publishing.nsf/Content/1510-public.
The proposal for haemophilia patients without inhibitors was considered by MSAC on 1-2 August 2019, as MSAC application 1579. The Public Summary Document recording MSAC’s advice on this application can be found at http://www.msac.gov.au/internet/msac/publishing.nsf/Content/1579-public. This advice supports the public funding of emicizumab for patients with moderate or severe haemophilia A without inhibitors. However, the MSAC advice indicates that the application did not justify the expense of emicizumab for all haemophilia A patients at the proposed price, and that the MSAC advice is subject to final negotiations of pricing and a financial risk sharing agreement.
At the same meeting, a revised proposal for haemophilia A patients with inhibitors was considered by MSAC, as MSAC application 1510.1. The Public Summary Document recording MSAC’s advice on this application can be found at http://www.msac.gov.au/internet/msac/publishing.nsf/Content/1510.1-public. This advice supports the public funding of emicizumab for patients with haemophilia A with inhibitors. However, again, the MSAC advice indicates that the application did not justify the expense of emicizumab for haemophilia A patients with inhibitors at the proposed price, and that the MSAC advice is subject to final negotiations of pricing and a financial risk sharing agreement.
Following these advices from MSAC, negotiations of pricing and a financial risk sharing agreement were conducted between the Department of Health on behalf of MSAC and Roche Products Pty Limited. Subsequently, supply of emicizumab under the national blood arrangements commenced on 1 November 2020. Further information on the supply arrangements is available at: https://www.blood.gov.au/national-supply-arrangements-hemlibra-emicizumab.