Disappointing news for the sickle cell disease community; the benefits of Oxbryta (voxelotor) were determined to not outweigh the risks of the treatment and Pfizer have now pulled Oxbryta from markets globally. This is especially discouraging following the revoking of marketing authorisation of Adakveo (crizanlizumab) for sickle cell disease last year.
After some work in the sickle cell space, and after closely following the HTA appraisal of Oxbryta by NICE, it is evident that there is huge unmet need for people living with sickle cell disease. A lack of funding for research in the area, combined with the challenges of evidence generation in a rare disease, means that available treatment options for people living with SCD are limited, and are often invasive and associated with substantial side effects.
September is hashtag #SickleCellAwarenessmonth and after this news it is even more important to raise awareness and support people living with SCD. The Sickle Cell Society provide some great resources on raising awareness for SCD, such as learning more about the condition and how to support people living with SCD, as well as how to donate blood which is vital for current SCD treatment. https://lnkd.in/eSvXxh2k
Evidence generation to meet HTA requirements is challenging and even more so in rare diseases. Small clinical trial populations, heterogeneity in outcome reporting, and difficulties in measuring changes in health-related quality of life mean innovative approaches to meet evidence requirements may be required for successful reimbursement.
Rare diseases rely on a bespoke approach to evidence generation, specific to each situation and Tolley offer support in determining evidence needs for successful UK HTA submissions, as well as generating the evidence to meet these needs. We have experience across a range of rare diseases and through this have learnt a great deal about the challenges facing reimbursement of new treatments for rare disease.