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HomeNewsMediaMedia statement regarding the NICE appeal hearing decision on Oxbryta® (voxelotor)Media statement regarding the NICE appeal hearing decision on Oxbryta® (voxelotor)

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:

References

  1. Sickle Cell Society. About Sickle Cell. Accessed December 2023.
  2. Centers for Disease Control and Prevention. What is Sickle Cell Disease? Accessed December 2023.
  3. Lubeck D, Agodoa I, Bhakta N, et al. Estimated Life Expectancy and Income of Patients With Sickle Cell Disease Compared With Those Without Sickle Cell Disease. JAMA Network Open. 2019;2(11):e1915374
  4. SCTAPPG The Sickle Cell Society and All-Party Parliamentary Group on Sickle Cell and Thalassaemia. No One’s Listening report. Accessed December 2023.
  5. NICE. Voxelotor for treating sickle cell disease [ID1403]. Accessed December 2023.
PP-UNP-GBR-7732 / December 2023
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