Axicabtagene ciloleucel (Yescarta®) for the treatment of adult patients with recurring or refractory diffuse large-cell B-cell lymphoma (DLBCL) and primary mediastinal large-cell B-cell lymphoma (PMBCL)
Zorginstituut Nederland carried out an assessment of the medicinal product axicabtagene ciloleucel (axi-cel, Yescarta®) for the treatment of DLBCL and PMBCL. Due to its expected high costs the Minister placed tisagenlecleucel in the 'waiting room' or 'sluice'.
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.
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Axi-cel is a personalised immunotherapy whose purpose is to stimulate the immune system of patients with haematological tumours to recognise and eliminate cancer cells. It is a gene-therapeutic treatment (changing the DNA of the body's own T-cells), in principle a one-off treatment. The therapy is intended for a sub-group of cancer patients with a high burden of disease (0.83 on a scale of 0-1), partly because no further effective treatment is available for this sub-group of patients and as a result only 17% will still be alive after one year.
As there is currently a lack of sufficient data on survival in the longer term, cost-effectiveness cannot be estimated reliably. However, it is highly likely that the actual ICER of axi-cel will exceed the relevant reference value that applies to this severe disease, i.e. €80,000/QALY. For this reason we advise the Minister not to include axi-cel in the insured package unless a price reduction can be agreed. We cannot say, however, by what percentage the price should fall in order to facilitate cost-effective deployment. Due to uncertainty about the long-term effect, and because it involves a one-off treatment, a pay-for-performance agreement would seem a good option. Particularly because the medical specialists have indicated that they will set up a national tumour board and follow patients via existing registers of the IKNL and the EBMT in the event of acceptance into the insured package. We suggest that the Minister considers coupling the level of the reimbursement to certainty about the effect in the long term. We also advise the Minister, when making agreements about the duration of a price arrangement, to take into account the possible arrival of competing drugs, so that health care insurers can take this into account in their purchasing policy.
Having listened to the Package Advisory Committee (ACP), the Zorginstituut emphasises that axi-cel should already become available during the negotiation period. After all, it is an effective therapy, that may even be curative for some patients.
This report is a summary of recommendations by Zorginstituut Nederland. The original text of this excerpt is in Dutch.