Pfizer facing NICE showdown over leukaemia drug

Pfizer’s Besponsa leukaemia drug has been approved in Europe, but the company is heading for a showdown with England’s NICE, which has said in a draft decision that the medicine it is too expensive for regular NHS funding.

Besponsa (inotuzumab ozogamicin) has been approved in Europe for relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukaemia (ALL).

ALL is a rare but aggressive form of leukaemia that can be fatal within a matter of months if left untreated, and around 760 people in the UK are affected every year.

Of these, around 117 patients will be eligible for treatment with Besponsa, an antibody-drug-conjugate, comprised of a monoclonal antibody targeting CD22, an antigen expressed in almost all B-ALL patients, linked with a cytotoxic agent.

NICE is now issuing draft appraisals of cancer drugs much earlier -in some cases even before they gain European approval – and last month published first draft guidance rejecting the drug on cost grounds.

According to the first draft document, Pfizer’s estimates that the cost per Quality Adjusted Life Year (QALY) gained is between £48,000 and £68,000 – but NICE estimated that the likely cost per QALY is around  £114,000.

Pfizer will meet NICE next week and submit further evidence, and representatives of patient groups will also attend to make representations in an attempt to get NICE to rethink its decision.

Neither NICE nor Pfizer have given information on the drug’s price, but it looks likely that the manufacturer will have to offer a discount in order to get NICE’s backing.

A final draft decision is expected in late summer, or early autumn following the negotiations, with final guidance expected at the end of September.

Pfizer said the outcome of the first draft was “disappointing” but said its priority is to find a solution so that eligible patients can get access to the medicine.

Besponsa originates from a collaboration between Pfizer and Celltech, now UCB. Under the terms of this agreement, Pfizer has sole responsibility for all manufacturing and clinical development activities.

The goal of treatment in R/R ALL is to achieve complete remission without excessive toxicity so patients may proceed to additional therapeutic intervention, particularly stem cell transplant, which is the most recognised option to prolong patient survival, maintenance therapy or other therapy.

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