Positive appeal decision gives second chance for inotuzumab ozogamicin

In August 2017, NICE rejected the use of inotuzumab ozogamicin for the treatment of relapsed, or refractory, b-cell acute lymphoblastic leukaemia (ALL). This was based upon the treatment not being a cost-effective use of NHS resources, because as a life-extending palliative care treatment inotuzumab ozogamicin did not significantly improve survival compared to the current, cheaper, standard of treatment.

The model for cost-effectiveness was based upon a maximum of six cycles of treatment as per the marketing authorisation of inotuzumab ozogamicin. In the case of six-cycles, the treatment is used for palliative care.

UK practice, however, would largely consist of using inotuzumab ozogamicin as a step to enable patients to have a stem cell transplant (SCT). A clinical trial named INO-VATE showed that far more patients were able to have a SCT following treatment with inotuzumab ozogamicin, compared to those treated with current therapies.

When used as a step towards STC normally only two cycles of inotuzumab ozogamicin are necessary, plus an additional cycle in some patients and others are able to have a SCT after just one cycle. This means that in the UK the maximum number of cycles used to treat patients would be three instead of six, significantly reducing the cost of treatment that was used in the appraisal model.

For this reason, Leukaemia Care sought to appeal the NICE recommendation based upon the grounds that the decision to not recommend the treatment is unreasonable in light of the evidence submitted to NICE. A joint appeal from the Royal College of Pathologists, Royal College of Physicians and the Association of Cancer Physicians was submitted on the same grounds. Pfizer, the manufacturer of inotuzumab ozogamicin, also appealed the decision.

Director of Campaigns and Advocacy, Zack Pemberton-Whiteley, represented Leukaemia Care at the appeal hearing in November, alongside two patients and a carer who had experienced treatment with inotuzumab ozogamicin. On December 28^th the appeal committee published their decision to uphold the appeals.

The HTA for inotuzumab ozogamicin has now gone back to the appraisals committee, who are required to address the issues which were successfully appealed against. One of which will require the appraisal committee to reconsider of the cost-effective modelling to ensure that it aligns with UK practice. You can read the full appeal decision at the following link: https://www.nice.org.uk/guidance/gid-ta10091/documents/appeal-decision