On May 15, the U.S. Food and Drug Administration approved Adakveo (crizanlizumab-tmca), a treatment to reduce the frequency of vaso-occlusive crisis – a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells – for patients age 16 years and older.
| Unmet Medical Need Parameters | Current Unmet Medical Need in vaso-occlusive crisis in sickle cell disease | Unmet Medical Need after Adakveo (crizanlizumab-tmca) |
|---|---|---|
| Low to moderate unmet medical need | Low to moderate unmet medical need | |
| Disease Severity | Vaso-occlusive crisis and chronic pain are associated with considerable economic loss and disability | Vaso-occlusive crisis and chronic pain are associated with considerable economic loss and disability |
| Treatment Adequacy | Current treatment (Endari) affects episodes frequency | New treatment also affects episodes frequency |
| Concerns of Payers & Providers | • Median number of sickle cell crises • Median number of hospitalizations for sickle cell pain • Median cumulative days in hospital • Median time (days) to first sickle cell crisis • Patients with occurrences of acute chest syndrome | • Annual rate of VOC (vaso-occlusive crises) • Annual rate of days hospitalized |
| Net Unmet Medical Need Score | 2.46/5 | 2.34/5 |
Adakveo has disease modifying effect, affects disease severity and provides symptomatic relief as well as addresses patients, payers and providers concerns.
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We help researchers in unmet medical need analysis of diseases which helps them in disease prioritization for any new drug/device/target/MOA development. We do unmet medical need analysis of any disease based on various parameters which is not limited to treatment adequacy, patient perspective, payer & providers perspective.
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