National supply arrangements for Hemlibra (emicizumab)

On 2 November 2020, Hemlibra became available under national supply arrangements.

Hemlibra is available to prevent or reduce the frequency of bleeding in severe or moderate haemophilia A patients without inhibitors, and in haemophilia A patients with inhibitors.

Hemlibra is available through specialist clinicians at Haemophilia Treatment Centres.

Distribution arrangements will support local delivery for patients who can appropriately self-administer Hemlibra at home, with ongoing clinical oversight from a Haemophilia Treatment Centre. This includes distribution through a community pharmacy, or home delivery on an exception basis within a defined set of eligibility criteria.

The timing of transition arrangements for Hemlibra took into account the need for appropriate clinical oversight of transitioning patients, and the efficient use of stock of current products held by Haemophilia Treatment Centres and patients, and stock required to be held in Australia under NBA contracts.

Learn more about our Hemlibra implementation, distribution and transition arrangements [PDF].

Read the Australian Haemophilia Centres Directors' Organisation clinical guidance on the appropriate use of Hemlibra(Opens in a new tab/window).

Background

All Australian governments have agreed to fund Roche’s Hemlibra (also known as emicizumab) under the national blood arrangements. This followed consideration of a proposal from Roche by the NBA in accordance with Schedule 4 of the National Blood Agreement, including an assessment by the Medical Services Advisory Committee (MSAC) to ensure comparative safety, clinical effectiveness, and cost effectiveness.

Hemlibra is a new type of blood-related product to be used for prophylaxis therapy for adult and paediatric patients with severe and moderate haemophilia A who would otherwise suffer life-threatening, severely disabling internal bleeding, or recurrent joint damage.

Currently, people with severe or moderate haemophilia A are usually treated with the clotting factor VIII (FVIII) multiple times a week to reduce rates of bleeding (prophylaxis), or when they have bleeding (treatment on demand). Factor VIII therapy requires frequent intravenous infusions, and can also lead to the development of antibody responses (known as inhibitors) which inhibit its effectiveness and subsequently require treatment with alternative high-cost bypassing agent products.

MSAC recommended public funding of Hemlibra under the national blood arrangements to prevent or reduce the frequency of bleeding in severe or moderate haemophilia A patients without inhibitors, and in haemophilia A patients with inhibitors.

MSAC concluded that Hemlibra prophylaxis significantly reduced the number of bleeds in patients without inhibitors previously treated on-demand with Short Half Life products (SHLs); and patients with inhibitors previously receiving bypassing-agents. Emicizumab prophylaxis also appeared to be more efficacious than prophylaxis with an SHL in non-inhibitor patients. Hemlibra is easier to administer and provides the significant advantage of less intrusive subcutaneous administration either once per week, every 2 weeks, or every 4 weeks.

The supply of Hemlibra through the national blood arrangements administered by the NBA will ensure patients and clinicians have holistic and consistent national access to therapies for haemophilia A. Hemlibra will be carefully prescribed by a specialist clinician in a recognised Haemophilia Treatment Centre, which will continue to provide coordinated access consistent with the current framework for access to therapies for haemophilia.

Implementation, distribution and transition

Supply of Hemlibra under NBA arrangements commenced on 2 November 2020. The supply of current FVIII and bypassing agent products under the national blood arrangements has continued. Services to provide local delivery for patients administering clotting factor products at home under the clinical management of a Haemophilia Treatment Centre have extended to Hemlibra, primarily through local pharmacy arrangements.

The prescription of Hemlibra for patients with haemophilia is a clinical matter which should be discussed and agreed between the patient (or their carer) and a specialist clinician in a recognised Haemophilia Treatment Centre. Clinical guidance for the use of Hemlibra, taking into account MSAC recommendations, has been provided by the Australian Haemophilia Centre Director’s Organisation (AHCDO). The Clinical Advisory Group of AHCDO provide peer advice to Haemophilia Treatment Centre clinicians considering the transition of relevant patients to Hemlibra.

Specific additional clinical governance and management arrangements for the provision of Hemlibra may also apply in each state and territory, or at a local health district or hospital level.

The NBA consulted with AHCDO, the Haemophilia Foundation Australia (HFA) and the Australian Haemophilia Nurses Group (AHNG) on transition arrangements for the introduction of Hemlibra. Transition arrangements should take account of the need for appropriate clinical oversight of transitioning patients, and the efficient use of stock already held by Haemophilia Treatment Centres and patients.

The Australian Bleeding Disorders Registry (ABDR) and the MyABDR App should be used to record the use of Hemlibra by patients in Australia, and have been modified for this purpose. The NBA will utilise data recorded in the ABDR for supply planning purposes, and to monitor utilisation and cost in order to confirm the benefits of the new therapy over time.

Schedule 4 assessment - emicizumab (Hemlibra) for haemophilia A

In 2017, the NBA received 2 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 Jurisdictional Blood Committee (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. Read MSAC's advice in Public Summary Document 1510(Opens in a new tab/window).

The proposal for haemophilia patients without inhibitors was considered by MSAC on 1-2 August 2019, as MSAC application 1579. Read MSAC's advice in Public Summary Document 1579(Opens in a new tab/window). 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. Read MSAC's advice in Public Summary Document 1510-1(Opens in a new tab/window). 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.