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.
To get detailed analysis of unmet medical need and to understand our approach which we are using for unmet medical analysis of any disease please contact at gupta.pk@pharmintel.co.in
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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