Tisagenlecleucel (Kymriah®) for the treatment of DLBCL

Zorginstituut Nederland has completed its assessment of tisagenlecleucel (Kymriah®) for the treatment of DLBCL. Due to its expected high costs the Minister of Health, Welfare and Sport (VWS) has placed tisagenlecleucel in the so-called ‘waiting room’ or ‘sluice’ for expensive drugs.
These products can only be accepted into the insured package after the Zorginstituut has advised on their inclusion in the insured package and, where applicable, subject to Ministry negotiations to arrive at a financial arrangement with the supplier.

2 indications

The Minister placed tisagenlecleucel in the waiting room for two indications:

  1. Paediatric and young adult patients up to the age of 25 years with B cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or later relapse (hereafter referred to as r/r B cell ALL).
  2. Adult patients with a relapsed or refractory diffuse large-cell B-cell lymphoma (r/r DLBCL) after two or more lines of systemic therapy.

Zorginstituut Nederland has in the meantime completed its assessment of tisagenlecleucel for the first indication mentioned, r/r B cell ALL, and informed the Minister about it in our letter dated 18 December 2018.

Tisagenlecleucel is an autologous immunocellular cancer therapy. In order to produce tisagenlecleucel, patients’ own T-cells have to be obtained via leukapheresis. These cells are subsequently genetically modified, ex vivo, using a lentiviral vector that codes for an anti-CD19 chimeric antigen receptor (CAR). After the patient has been pretreated with lympho-depletion-chemotherapy, the patient's own (autologous) CAR-T-cells (tisagenlecleucel) are returned to the patient via a single intravenous infusion.

Zorginstituut's advice

Tisagenlecleucel does not fulfil the statutory criterion, ‘established medical science and medical practice’, for the above-named indication. For this reason we advise the Minister not to include tisagenlecleucel in the insured package.
New published data, if the results point towards a clinically relevant benefit  of effectiveness, can be a starting point for reviewing whether tisagenlecleucel does comply with established medical science and medical practice.

This report is a summary of recommendations by Zorginstituut Nederland. The original text of this excerpt is in Dutch.