5th December 2023
Pfizer is extremely pleased that the National Institute for Health and Care Excellence (NICE) Appeal Panel has upheld the appeal for Oxbryta® (voxelotor) and is committed to working with NICE to find a path forward to ensure that this treatment can reach eligible patients.
Sickle cell disease (SCD) is a lifelong, inherited blood disorder, affecting approximately 17,500 people in the UK.1 People with SCD live with painful episodes, called sickle cell crises, which are one of the most common and distressing symptoms of SCD.2 The impact of the condition means that life expectancy of people with SCD is significantly lower than the general public, primarily owing to early mortality during adulthood.3
There is a significant unmet need for people living with SCD in the UK, who face stigma and misunderstanding linked to their condition, and unwarranted variations in the standard of care they receive.4 It's positive that the appeal panel recognised the substantial disadvantage for people with SCD and that reasonable adjustments should be made.5
The Appeal Panel upheld the appeal on the following grounds:
Pfizer 1a.1 It was procedurally unfair for the committee not to give, at any point during the appraisal process, any indication of what it considered to be the plausible cost-effectiveness of voxelotor.
Pfizer 1a.2 It was procedurally unfair for the committee not to inform Pfizer, in sufficient time in advance of the second appraisal committee meeting, of the estimates generated by the exploratory scenario analyses of the external assessment group (‘EAG’)
Pfizer 1b.1 The adjustment made by the committee to reflect the health inequalities associated with SCD, while welcome, was inadequate and should not have been so limited in scope
Pfizer 2.1 The committee's conclusion that there was too much uncertainty, such that it could not assess the cost-effectiveness of voxelotor, was irrational
Pfizer 2.2 The committee misunderstood the relationship between the proposed positioning/NHS population and the trial population. It drew incorrect conclusions
Sickle Cell Society 1b.1 The committee breached its duties under the Equality Act 2010 by failing to recognise barriers to access and/or take into account health inequalities for patients with SCD and therefore exceeded its powers.5