Applications

1748 – Review of Tisagenlecleucel for treatment of confirmed relapsed/refractory CD19-positive acute lymphoblastic leukaemia in children and young adults up to 25 years old

Find out about the service or technology in this application and the medical condition it addresses. You can also view the application documents, the deadlines for providing consultation input and the outcome of the application when the MSAC process is complete.

  • Status Complete
  • Type Review
  • Pre-PASC consultation Not applicable
  • Pre-MSAC consultation Closed
  • Outcome Other (see outcome details)

Application details

Reason for application

Following consideration by MSAC at its November 2018 and March 2019 meetings, and an out-of-session meeting in April 2019, the committee supported funding of tisagenlecleucel (Kymriah®) for treatment of acute lymphoblastic leukaemia in paediatric and young adult patients (pALL) as a highly specialised therapy through the National Health Reform Agreement (NHRA).

A condition of MSAC’s support was that a full review of the clinical effectiveness, cost-effectiveness and budget impact would be provided to MSAC by the applicant after a specified amount of time had passed from the therapy being made available. In addition to the submission from the applicant, the Department has also invited States and Territories, as suppliers and joint funders of the treatment to provide submissions to the review.

Service or technology in this application

Tisagenlecleucel (Kymriah®) is an autologous, murine anti-CD19 Chimeric Antigen Receptor T cell (CAR-T) therapeutic process, involving harvesting, modifying, expanding and re-infusing a patient's own immune T-cells, to target and destroy certain cancerous cells. This therapy is used when patients do not respond to other treatment, such as chemotherapy, or relapse.

Medical condition this application addresses

Acute lymphoblastic leukaemia is the malignant proliferation of lymphoid progenitor cells in the bone marrow.

Previous applications

1519 – Tisagenlecleucel (CTL019) for treatment of refractory CD19-positive leukaemia and lymphoma

Application documents

Public summary document

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Consultation survey and deadlines

  • PASC consultation: Bypassing PASC
  • MSAC consultation: Closed Friday, 9 June 2023

Meetings to consider this application

  • PASC meeting: Bypassing PASC
  • ESC meeting: 8–9 June 2023
  • MSAC meeting: 27–28 July 2023

Outcome details

MSAC considered the new data was not enough to improve understanding of how well treatment with TIS works long term. Many patients in the clinical studies and Australian patients had other treatments including stem cell (bone marrow) transplants. MSAC considered that TIS was not curing most patients. Cost data from state and territory governments showed the true cost of providing TIS was substantially higher than expected. MSAC considered another review was needed in 3 years.

See the public summary document for the full details of our decision.