NICE rejects Pfizer’s ALL drug Besponsa

The National Institute for Health and Care Excellence has today published draft guidance not recommending Pfizer’s Besponsa to treat some patients with leukaemia on the NHS in England and Wales.

The drug is being appraised by the Institute for treating adults with relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukaemia (ALL).

Besponsa (inotuzumab) is an antibody drug conjugate which is comprised of a monoclonal antibody targeting CD22, a cell surface antigen expressed on around 90 percent of B-cell malignancies, linked to a cytotoxic agent.

When the drug binds to CD22 on malignant B-cells it is taken into the cell, where the cytotoxic agent calicheamicin is released to destroy it.

Data from the INO-VATE trial, which compared the drug to standard of care chemotherapy in 326 adult patients with relapsed or refractory CD22-positive ALL, and showed that the therapy more than doubled complete remission rates.

However, in draft guidelines, NICE says evidence from clinical trials showed no survival benefit from Besponsa compared to current treatment, but more people who were treated with the drug were able to subsequently have a stem cell transplant and go into remission than those on standard care.

Because of the "highly uncertain" survival benefits, the incremental cost effectiveness ratio (ICER) was over £100,000 per QALY for Besponsa, which is substantially higher than the range considered cost effective for routine use in the NHS.

ALL is an aggressive type of leukaemia with high unmet need and a poor prognosis. The current standard treatment is intensive, long-term chemotherapy, but currently only 10 percent of adults with ALL who relapse after first-line therapy survive five years or more.

It is estimated that 117 patients would be eligible for treatment with the drug in its first year if it were recommended for routine use.

In a statement, David Montgomery, Oncology Medical Director of Pfizer UK, said: "Whilst NICE, in its initial assessment of inotuzumab ozogamicin, has acknowledged the benefit of this medicine for people with this aggressive  leukaemia, the draft decision is disappointing and yet again we are concerned for patients who so urgently need new treatment options. We remain focused on finding a solution so that eligible patients can access this medicine and we will work closely with NICE as the technology appraisal for inotuzumab ozogamicin progresses."