1592 – Review of immunoglobulin use for primary immunodeficiency diseases with antibody deficiency

Health Technology Assessment.

Immunoglobulin is a plasma-derived product manufactured to treat a range of medical conditions. Access to Government funded immunoglobulin is through the national blood arrangements and is determined by the National Blood Authority’s Criteria for Clinical Use of Immunoglobulin in Australia (the Criteria). Immunoglobulin is used as immunoreplacement therapy in PID.

Type: Therapeutic

Review of Immunoglobulin use in Australia

Demand for immunoglobulin in Australia has increased around 11% per year since 2009-10. Internationally, Australia is in the top three users of immunoglobulin, along with the US and Canada.

All Australian Governments, through the Jurisdictional Blood Committee (JBC), have agreed to conduct a robust Health Technology Assessment review to ensure Government funded immunoglobulin use within Australia is based on strong evidence of clinical and cost effectiveness. This is consistent with how other pharmaceutical and medical technologies are assessed for Government funding.

The review is to ensure treatment with immunoglobulin, funded by Governments, is both clinically and cost-effective compared to other treatment options.

The review complements the National Blood Authority Immunoglobulin Governance Program, which aims to strengthen clinical governance and authorisation of Government funded immunoglobulin in Australia.

The review is being conducted through the Medical services Advisory Committee (MSAC) assessment process supported by a bespoke reference group. The MSAC Immunoglobulin Review Reference Group will oversee all immunoglobulin HTA review applications.

Initially, the review will assess three immunoglobulin indications considered to have strong evidence and usage data (see MSAC Applications 1564, 1565, 1566). Phase 2 of the review will assess an additional three immunoglobulin indications, including primary immunodeficiency with antibody deficiency (PID).

Primary immunodeficiency is a broad term for immunodeficiencies where secondary causes have been excluded. There are over 280 identified PIDs and the condition usually presents with signs of infection that can be repetitive, severe and difficult to treat. Infection is the most common complication of PID and the most common reason leading to medical assessment. Autoimmune disease and malignancies are complications of many PIDs.

The journey of patient investigations, diagnosis and management of people with PID with antibody deficiency is very complicated. This patient group includes a wide range of rare conditions affecting both paediatric and adult patients. Patients often present with atypical features, have an autoimmune disease as their primary presentation and therefore are on multiple therapies.

The indications for use of Ig under the Criteria are:

• replacement therapy in common variable immune deficiency (CVID)
• replacement therapy in possible CVID (below normal serum IgG but normal IgA level)
• replacement therapy in transient hypogammaglobulinaemia of infancy (children aged less than 4 years)
• replacement therapy in transient hypogammaglobulinaemia for which immunoglobulin replacement is universally indicated (e.g. Severe Combined Immunodeficiency (SCID), Wiskott-Aldrich syndrome, etc.).

• Immunoglobulin Review Reference Group:
  • 24 July 2019
  • 9 October 2019
  • 25 March 2020
• ESC meeting: 8 to 9 October 2020
• MSAC meeting: 26 to 27 November 2020