List prices of novel sickle cell drugs too high, report suggests

The Institute for Clinical and Economic Review (ICER; MA, USA) has published an evidence report concerning the comparative and cost—effectiveness of three novel interventions for sickle cell disease.

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In a recent evidence report, the Institute for Clinical and Economic Review (ICER; MA, USA) evaluated the comparative and cost—effectiveness of three novel interventions for sickle cell disease – crizanlizumab (Adakveo®), voxelotor (Oxbryta™) and L-glutamine (Endari®). The report suggests that the list prices of these therapies may have been set too high for the clinical benefits they confer and significant discounts from these are required.

Historically, research into sickle cell disease has been notably underfunded, despite the stigma and substantial lifelong burden associated with the disease.

In this evidence report, ICER investigators linked pain crises and individuals’ hemoglobin levels with acute health conditions, chronic conditions and mortality, to generate a comprehensive model for use in determining the health and economic impacts of crizanlizumab, voxelotor and L-glutamine. Model input data were largely real-world data obtained from published Center for Medicare and Medicaid Services (CMS; MD, USA) national level data and the Aetion Evidence Platform™.

Utilizing the model, investigators determined that crizanlizumab reduces the frequency of pain crises for individuals with sickle cell disease, compared with standard of care treatment. However, there is ultimately inconclusive evidence to determine the comparative clinical effectiveness of voxelotor and L-glutamine.

Further, ICER investigators generated health—benefit price benchmarks for each of the three therapies under evaluation; these price ranges, net of any manufacturer discounts and/or rebates, reflect the added health and quality of life benefits conferred by the treatments for individuals over their lifetimes.

Results of the analyses suggest that, at the treatments’ current list prices, only 21% and 16% of eligible patients could be treated with crizanlizumab and voxelotor respectively each year, before ICER’s potential budget-impact threshold of US$819 million per year were crossed.

Aggregate benchmark ranges for crizanlizumab, voxelotor and L-glutamine would require list price discounts of 61—89%, 71—93% and 18—76% respectively, to be deemed cost—effective.

Pamela Bradt, Chief Scientific Officer at ICER, stated: “What isn’t in question is the importance of the context here: it is difficult to imagine a disease that ravages patients’ and families’ lives more than sickle cell disease. It is also true that this community has borne the burden of systemic racism and stigma, with reduced investment in research and the broad effects of an inadequate system of care, particularly as pediatric patients grow into adulthood.”

“Our report takes an objective look at the evidence while highlighting that insurers and other policymakers must be fully aware of the broader clinical and social perspectives when developing prior authorization coverage criteria and negotiating prices with drug makers,” Bradt concluded.”


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Ilana Landau

Editor, Future Science Group

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