Pfizer has said it is "very disappointed" by NICE’s decision to not recommend inotuzumab ozogamicin as a treatment for acute lymphoblastic leukaemia (ALL) – and that it believes NICE “inappropriately assessed the value of the medicine in making their determination of cost-effectiveness, using assumptions that are inconsistent with previous appraisals of other medicines in this disease area”.

“Recent decisions made by NICE led us to believe that there were clear parameters in place to evaluate new targeted therapies in ALL, however it appears that major errors in the evaluation of this drug will now significantly impact patients living with this difficult-to-treat disease,” said Professor David Marks, Department of Haematology and BMT, University Hospitals Bristol NHS Foundation Trust.

“Of specific concern in the final appraisal determination for inotuzumab ozogamicin is a gross misrepresentation of clinical practice in the UK. The ICER has been calculated incorrectly, based on clinicians using six courses of the drug. This is totally wrong. No patient in the UK will require more than two to three courses of the drug before a potentially curative transplant. Despite NICE being informed about this, it appears not to have been taken into account.

David Montgomery, Pfizer UK’s oncology medical director, added: “Today’s frustrating decision for inotuzumab ozogamicin is another example of how NICE is not appropriately assessing the value of modern cancer medicines, leaving patients without access to new treatments that could transform their lives.

“For this difficult-to-treat type of blood cancer, patients need to achieve complete remission in order to be eligible for a potentially curative bone marrow transplant. At the moment, treatment options that can achieve complete remission are few, and without new medicines like inotuzumab ozogamicin, patients have a very poor prognosis. We will continue to work with NICE in the hope that this decision can be overturned.”

NICE has stood by the decision, though. Professor Carole Longson, director of the centre for health technology evaluation, said: “The committee found that inotozumab ozogamicin showed no survival benefit over treatments currently available to patients with this type of leukaemia. This means the committee could not recommend the drug as clinically and cost effective for routine use in the NHS.”