The National Institute of Healthcare and Excellence (NICE) is set to reject Pfizer’s orphan drug for leukaemia Besponsa (inotuzumab ozogamicin).Pfizer issued a robust defence of its product, saying the UK cost-effectiveness watchdog’s final appraisal determination had ‘inappropriately’ assessed the drug’s value.The firm added that assumptions made during the process were ‘inconsistent’ with previous appraisals of other medicines in the disease area.David Montogomery, oncology medical director at Pfizer UK, said: “[This] 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.“We will continue to work with NICE in the hope that this decision can be overturned.”The cost-effectiveness watchdog was assessing ’s use to treat adults with Philadelphia chromosome positive (Ph+) as well as Philadelphia chromosome negative (Ph-) relapsed or refractory B-cell CD22-positive B-cell precursor acute lymphoblastic leukaemia (ALL).NICE compared Besponsa to the current usual treatment of fludarabine, cytarabine and granulocyte (FLAG) chemotherapy with idarubicin, but found clinical trial evidence “did not show an overall survival benefit”.For its part Pfizer pointed to the INO-VATE trial, which showed Besponsa more than doubled complete remission rates (81% versus 29%).When compared to chemotherapy the drug also showed that nearly four times as many patients were able to receive stem cell transplant (43% versus 11%).NICE accepted more Besponsa patients went on to have a stem cell transplant, but said its cost-effectiveness estimates were too high, despite a patient access scheme being in place.Besponsa has orphan status in Europe, where regulators approved it in June, and the treatment has just been in the US, where it has breakthrough status.NICE’s negative final appraisal determination is now out for consultation and Pfizer plans to appeal the ruling, which is currently due to be finalised by 27 September.. The National Institute of Healthcare and Excellence (NICE) is set to reject Pfizer’s orphan drug for leukaemia Besponsa (inotuzumab ozogamicin). Pfizer issued a robust defence of its product, saying the UK cost-effectiveness watchdog’s final appraisal determination had ‘inappropriately’ assessed the drug’s value.. Release. The U.S. Food and Drug Administration today approved Besponsa (inotuzumab ozogamicin) for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). “For adult patients with B-cell ALL whose cancer has not responded to initial treatment or has returned after treatment,.