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.

Table of contents

  • Disease Landscape & Forecast
    • Commercial Outlook
      • Key Findings
        • Key Findings
      • Drivers and Constraints
        • What Factors Are Driving the Market for Sickle Cell Disease?
        • What Factors Are Constraining the Market for Sickle Cell Disease?
      • Downstream Symptomatic Therapies
      • Selectin Inhibitors
      • Hemoglobin Modifiers
    • Forecast
    • Etiology and Pathophysiology
      • Disease Overview
        • Disease Overview
      • Etiology
      • Pathophysiology
      • Clinical Features of Sickle Cell Disease
        • Symptoms and Complications of Sickle Cell Disease
      • Key Pathways and Drug Targets
    • Epidemiology Overview
      • Introduction
        • Key Findings
      • Epidemiology Populations
        • Diagnosed Prevalent Cases
        • Drug-Treated Cases of Sickle Cell Disease
        • Diagnosed Prevalent Cases of SCD Subtypes
    • Current Treatment Overview
      • Key Findings
      • Diagnosis
        • Treatment Providers and Referral Patterns
      • Treatment Goals
      • Key Current Therapies
        • Overview
        • Prophylactic Penicillin
        • Hydroxyurea
        • L-Glutamine
        • Analgesics
        • Blood Transfusion
        • Hematopoietic Stem Cell Transplantation
      • Medical Practice
        • Overview
        • Treatment Guidelines
        • Region-Specific Treatment Practices
    • Unmet Need Overview
      • Current and Future Attainment of Unmet Needs in Sickle Cell Disease
    • Emerging Therapies Overview
      • Key Findings
        • Key Findings
      • Key Emerging Therapies
        • Notable Developments Among Key Emerging Therapies for Sickle Cell Disease
        • Downstream Symptomatic Therapies
        • Selectin Inhibitors
        • Hemoglobin Modifiers
      • Early-Phase Pipeline Analysis
        • Notable Developments in the Early-Phase Pipeline for Sickle Cell Disease
      • Key Discontinuations and Failures in Sickle Cell Disease
        • Key Discontinuations and Failures in Sickle Cell Disease
      • Patient Registries
        • Patient Organizations
      • Orphan Drug Designation
        • Orphan Drug Provisions: United States
        • Orphan Drug Provisions: Europe
    • Access and Reimbursement Overview
      • Looking for More?
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
    • Methodology
      • Bottom-Up Forecasting Overview
        • Patient Populations
        • Drug-Specific Assumptions
      • Bottom-Up Forecast Assumptions
        • General Sources of Data
        • Drug-Treatable Rate in Sickle Cell Disease
        • General Statements About Pricing
        • Dosing, Days of Therapy, and Compliance
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Sickle Cell Disease Bibliography

Author(s): Steven F. Trueman, PhD; Sunali D. Goonesekera, SM

Steve is a member of Decision Resources Group’s Infectious, Niche, and Rare Diseases (INRD) team. Currently, he provides analyses and content production on infections caused by the human immunodeficiency virus (HIV) and the hepatitis C virus (HCV). Steve conducted his postdoctoral research on models of neurodegenerative disease in the Department of Biochemistry at Brandeis University. He earned a doctorate in biochemistry from the University of Massachusetts Medical School, Graduate School of Biomedical Sciences, where he studied protein translocation into the endoplasmic reticulum.

Sunali Goonesekera is an Associate Epidemiologist at Decision Resources Group. Sunali holds a Master’s degree in Epidemiology from the Harvard School of Public Health and a in Biology (Honors) from Dartmouth College. Prior to joining Decision Resources Group, Sunali conducted epidemiological research and lead authored two manuscripts on racial/ethnic disparities in metabolic diseases at the New England Research Institutes. She has contributed to multiple publications in peer-reviewed journals in epidemiology and in the biological sciences.