FDA-Approved Therapies for Sickle Cell Disease May Not be Cost-Effective, ICER Draft Report Says

FDA-Approved Therapies for Sickle Cell Disease May Not be Cost-Effective, ICER Draft Report Says
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FDA-approved therapies for sickle cell disease (SCD) may not be cost-effective at their current prices, according to a draft report issued by the Institute for Clinical and Economic Review (ICER).

The report focused on the clinical benefits and cost-effectiveness of NovartisAdakveo (crizanlizumab), Global Blood TherapeuticsOxbryta (voxelotor) — both approved in the U.S. last year — and Emmaus Life SciencesEndari (L-glutamine).

At their current estimated costs, all three medications exceed $1 million per quality-adjusted life year (QALY), which is well above the threshold of $150,000 per QALY that’s considered cost-effective by the group. Of note, QALY measures how many years of good health a person may gain by taking a specific medication, and the added cost associated with that health gain.

In order to become cost-effective, ICER said, the average annual cost of Adakveo must drop from an estimated $88,000 to $25,410. The same applies to the other medications, with Oxbryta needing to come down from $84,000 to $9,218, and Endari from $24,000 to $3,859.

The group also attributed ratings to reflect the clinical effectiveness of all three medications compared to optimal usual care (placebo group in each of their respective clinical trials).

Adakveo was found to have a small to moderate incremental effect, meaning the group was highly certain that the use of the medication was associated with a small — and possibly a substantial — net health benefit compared to optimal usual care.

In contrast, Oxbryta and Endari were both found to be promising but inconclusive, meaning ICER was only moderately certain these medications could lead to a comparable, small, or substantial net health benefit compared to optimal usual care.

ICER noted that these findings are not definitive, as the draft report marks the midpoint of an eight-month treatment assessment process that is still underway.

The document will be open to public comment until 5pm ET on Feb. 20. General guidelines for submitting comments can be found on ICER’s website. More details about what information could be more relevant to the report can be found on ICER’s Manufacturer Engagement Guide and Patient Participation Guide.

Based on feedback received from stakeholders during this period, ICER may decide to make changes to the preliminary document. All comments and the group’s responses will be made public, along with the revised document — the Evidence Report — which will be released on March 12.

The Evidence Report will be analyzed at a public meeting of the New England Comparative Effectiveness Public Advisory Council in Boston, on March 26. Registration is now open for the public meeting and live webcast.

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
Total Posts: 94
Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
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Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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