ICER Releases Draft Evidence Report for Sickle Cell, Scoping Document for Hemophilia


The Institute for Clinical and Economic Review (ICER) published a draft evidence report on crizanlizumab (Adakveo), voxelotor (Oxbryta), and L-glutamine (Endari) for sickle cell disease, as well as a draft scoping document on valoctocogene roxaparvovec, an investigational gene therapy, and emicizumab (Helimbra) for hemophilia.

The Institute for Clinical and Economic Review (ICER) published a draft evidence report on therapies crizanlizumab (Adakveo, Novartis), voxelotor (Oxbryta, Global Blood Therapeutics) and L-glutamine (Endari, Emmaus Life Sciences) for sickle cell disease (SCD), as well as a draft scoping document on therapies valoctocogene roxaparvovec (BioMarin Pharmaceutical, Inc.) and emicizumab (Hemlibra, Genentech) for hemophilia.

Sickle Cell Disease

In the draft evidence report,1 the study authors note that care for patients with SCD has been historically poor and represents a failure in the US healthcare system. “Deep dysfunction in care is driven by poor coordination within provider systems and by barriers to access that arise from a broad range of factors including systemic racism, uninformed clinicians, poverty, and insurance systems poorly designed to coordinate coverage for patients with multisystem chronic conditions,” they said.

In the past 3 years, innovations within the treatment of patients with SCD have occurred, with FDA approvals for crizanlizumab, voxelotor, and L-glutamine providing new treatment options for patients. ICER sought to assess the comparative clinical effectiveness and value of these treatments for SCD, with an emphasis on treatment costs and acute pain crises. The scope of the assessment followed the population, intervention, comparators, outcomes, timing, and settings (PICOTS) framework. Results shown are at the midpoint of ICER’s 8-month process of assessments on these treatments, indicating that final results may alter conclusions.1

Study results indicated that treatment costs were the main driver of the cost-effectiveness analyses, with average annual costs of the treatments being $88,000 for crizanlizumab; $84,000 for voxelotor; and $24,000 for L-glutamine. In the ICER analyses, all therapies were estimated to be over $1 million per quality-adjusted life year (QALY), combined with relatively small improvements in QALYs gained (0.85 for crizanlizumab, 0.96 for voxelotor, and 0.10 for L-glutamine). The researchers stated that none of the scenario analyses undertaken lowered the estimated cost per QALY to less than $150,000.1

In examining the clinical value of these treatments for patients, the researchers highlight, “Although a reduction in acute pain crises and increase in hemoglobin will provide relief to patients, they will continue to suffer from other acute and chronic conditions that will have a significant impact on their quality of life.”

As these therapies have yet to be proved effective in clinical trials against these other acute and chronic conditions, there is currently a large difference in the cost per QALY and the cost per life-year and equal value life years gained (evLYG), with cost per evLYG ranging from approximately $520,000 for crizanlizumab to $651,000 for voxelator to $847,000 for L-glutamine.1

Based on scenario analyses, the researchers suggested that treatment is most cost-effective for patients with higher rates of acute pain crises, in which patients who experience 10 acute pain crises per year may have a cost per QALY as low as $615,000 with crizanlizumab.1

The report is open to public comment until February 13, 2020.


The draft scoping document2 outlined the proposed scope of the comparative clinical effectiveness and value assessment of valoctocogene roxaparvovec and emicizumab for the treatment of hemophilia A.

In the planned review, using the PICOTS framework, researchers will incorporate both quantitative and qualitative comparisons, public health effects, reduction in disparities, innovation, and patient experience in the analysis to determine the clinical and economic value of these therapies. Additionally, the study authors plan to develop a comparative simulation model to assess the cost-effectiveness of using valoctocogene roxaparvovec versus prophylaxis using emicizumab and prophylaxis using factor VIII preparations.2

Results from the model will include the estimated mean life expectancy; quality-adjusted life expectancy; health outcomes, such as number of additional bleeds prevented; and health care costs, which will be used to estimate the incremental cost per bleed prevented and the incremental cost per life-year gained, per evLYG, and per QALY gained.2

The scope is open to public comment until February 13, 2020.


1. Bradt P, Spackman E, Synnott P, et al; ICER. Crizanlizumab, voxelotor, and L-glutamine for sickle cell disease: effectiveness and value. ICER website. Published January 23, 2020. Accessed January 24, 2020.

2. ICER; New England Comparative Effectiveness Public Advisory Counsil. Valoctocogene roxaparvovec and emicizumab for hemophilia A: effectiveness and value. ICER website. Published January 24, 2020. Accessed January 24, 2020.

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