NICE to reassess Pfizer blood cancer drug

NICE will re-assess Pfizer’s blood cancer drug Besponsa after the company successfully appealed a decision rejecting regular NHS funding last summer.

Pfizer said a NICE committee will re-assess evidence for Besponsa (inotuzumab ozogamicin), approved last year in Europe for adults with relapse or refractory CD22-positive B-cell precursor acute lymphoblastic leukaemia (ALL).

An antibody-drug conjugate, Besponsa could provide a treatment option for ALL, a rare and aggressive type of leukaemia that can be fatal within a matter of months if left untreated.

The phase 3 INO-VATE 1022 trial showed that more than 80% of ALL patients treated with Besponsa achieved complete remission, compared with only 29% of patients treated with standard intensive chemotherapy.

Complete remission paves the way for a potentially curative stem cell transplant – and four times as many patients (41%) were able to receive this compared with standard chemo (11%).

But in final draft guidance published in August, NICE rejected the drug, despite the strong clinical evidence.

In a statement sent by Pfizer at the time, experts said NICE’s cost estimates were too high as they were based on calculations from doctors using six courses of the drug.

Professor David Marks, a haematologist from University Hospitals Bristol NHS Foundation Trust, said the assessment was a “gross misrepresentation” of clinical practice in the UK, and that most patients will require around two or three courses of Besponsa before the transplant.

An appeal hearing held at the beginning of November heard similar arguments from a medical expert and representatives of Pfizer.

While the marketing authorisation is for around six cycles, UK doctors used the drug for up to three cycles as a bridge to stem cell transplant.

A document on NICE’s website shows that a NICE panel upheld Pfizer’s appeal on grounds that it provided no explanation for rejecting a revised analysis from Pfizer and that an assumption used for an assessment of Amgen’s rival Blincyto (blinatumomab) did not explain choices made for Besponsa.

The appeal panel also said the decision to reject Besponsa was unreasonable in the light of evidence submitted jointly by the charity Leukaemia CARE.

NICE said its appraisal committee must now explain clearly the reasons for its rejection and must reconsider Besponsa’s cost-effectiveness based on the UK practice of up to three cycles.

Craig Eagle, head of oncology, Pfizer UK, said: “NICE’s decision to uphold the appeal and re-assess the evidence for inotuzumab ozogamicin is good news for some of the most vulnerable leukaemia patients in the UK. Pfizer is committed to ongoing collaboration with NICE to ensure that eligible patients are granted access to inotuzumab ozogamicin.”