Sickle Cell Disease | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2018


Sickle cell disease (SCD) is a rare genetic blood disorder characterized by a polymerization of hemoglobin in red blood cells (RBCs) that causes the cells to become distorted into a sickle shape. The distorted RBCs lead to several complications such as vaso-occlusion (VOC) associated with pain attacks, acute chest syndrome, and anemia. No curative therapy exists for SCD other than an allogenic hematopoietic stem cell transplantation with a matched (most often sibling) donor. Prophylactic penicillin to prevent commonly occurring infections, analgesics for pain episodes, hydroxyurea to increase the expression of an alternative form of hemoglobin, and blood transfusions are the cornerstones of SCD management, but a high unmet need remains for improved therapies. Drug developers have recognized the commercial opportunity in SCD, developing agents with novel mechanisms of action that target the underlying genetic defect and reduce VOC. A robust and diverse late-stage pipeline will be a key driver for substantial expansion of the SCD market by 2027 while patients await potentially curative options in early-stage development.


  • How large is the diagnosed prevalent SCD population in the United States and EU5? How will the population change through 2027?
  • What is the current treatment landscape for SCD patients and how will it change in the next ten years? What clinical needs remain unfulfilled?
  • What pipeline products are most promising, and what sales/uptake could they secure in SCD? How will new therapies impact medical practice?


Niche & Rare Disease Landscape & Forecast: Comprehensive market intelligence providing world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.


October 2018


United States and EU5


  • Six country-specific interviews with thought-leading hematologists
  • Supported by survey data collected for this study


Diagnosed prevalent and drug-treatable cases of sickle cell disease by country, segmented by clinical subtypes.


Drug-level sales and patient shares of key sickle cell disease therapies in 2027.


Phase III: 5 drugs. Phase II: 4 drugs. Coverage of select preclinical and Phase I and Phase I/II products.

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