Beta-Thalassemia | Landscape & Forecast | Disease Landscape & Forecast

Publish date: August 2019

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Beta thalassemia is a rare genetic blood disorder characterized by a substantial reduction in or lack of β-globin protein, which results in chronic anemia, failure to thrive, jaundice, pallor, poor musculature, and skeletal deformities. Allogenic hematopoietic stem cell transplantation with a matched (most often sibling) donor is the only available curative therapy; however, with the recent approval of bluebird bio’s Zynteglo in Europe and expected commercial launch starting in 2020, patients will soon have an additional potentially curative option. Most patients are currently managed with a combination of blood transfusions and iron chelators. The most severe thalassemia major patients require chronic blood transfusions to survive, which substantially impacts quality of life. A high unmet need exists for therapies to reduce or eliminate transfusion burden. Drug developers have recognized the commercial opportunity in the beta thalassemia market, and have focused on developing agents that target the underlying genetic defect and reduce transfusion burden.

Questions Answered:

  • How large is the diagnosed prevalent beta thalassemia population in the United States and EU5? How will the population change through 2028?
  • What is the current treatment landscape for beta thalassemia patients, and how will it change in the next ten years? How will the launches of Zynteglo and luspatercept impact beta thalassemia treatment?
  • What pipeline products are most promising, and what sales/uptake could they secure in beta thalassemia? How will new therapies affect medical practice?

Product Description

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.

Table of contents

  • Disease Landscape & Forecast
    • Executive Summary
      • Key Findings
    • Commercial Outlook
      • Key Findings
      • Drivers and Constraints
        • What Factors Are Driving the Market for Beta Thalassemia?
        • What Factors Are Constraining the Market for Beta Thalassemia?
      • Gene Therapy-Specific Trends
      • Ligand Trap Receptor-Specific Trends
      • Alternative Market Scenarios
    • Forecast
    • Etiology and Pathophysiology
      • Disease Overview
      • Etiology
        • The HBB Gene and β-Globin
        • Genotype/Phenotype Relationship
      • Pathophysiology
        • Ineffective Erythropoiesis
        • Iron Overload
        • Symptoms of Beta Thalassemia
      • Key Pathways and Drug Targets
    • Epidemiology
      • Key Findings
      • Epidemiology Populations
        • Diagnosed Prevalent Cases of Beta Thalassemia
        • Beta Thalassemia by Severity
        • Beta Thalassemia Major by Genotype
        • Diagnosed Prevalent Cases of Beta Thalassemia Major by HSC Transplantation
        • Diagnosed Prevalent Cases of Beta Thalassemia Intermedia by the Need for Transfusion
        • Drug-Treated Prevalent Cases of Beta Thalassemia
    • Current Treatment
      • Key Findings
      • Diagnosis
        • Treatment Providers and Referral Patterns
      • Treatment Goals
      • Key Current Therapies
        • Overview
        • Iron Chelators
        • Transfusion Therapy
        • Hematopoietic Stem Cell Transplantation
      • Medical Practice
        • Overview
        • Region-Specific Treatment Practices
    • Unmet Need Overview
      • Current and Future Attainment of Unmet Needs in Beta Thalassemia
    • Emerging Therapies
      • Key Findings
        • Expert Insight
      • Key Emerging Therapies
        • Cell-Based Gene Therapies
        • Ligand Trap Receptors
      • Early-Phase Pipeline Analysis
        • Notable Developments in the Early-Phase Pipeline for Beta Thalassemia
      • Patient Registries
        • Patient Organizations
      • Orphan-Drug Designation
        • Orphan-Drug Provisions: United States
        • Orphan-Drug Provisions: Europe
    • Access and Reimbursement Overview
      • Region-Specific Reimbursement Practices
        • General Reimbursement Environment: United States
        • General Reimbursement Environment: EU5
      • Looking for More?
    • Methodology
      • Bottom-Up Forecasting Overview
        • Patient Populations
        • Drug-Specific Assumptions
      • Bottom-Up Forecast Assumptions
        • Patients Eligible for Zynteglo and Luspatercept
        • Drug-Treatable Rate in Beta Thalassemia
        • General Statements About Pricing
        • Dosing, Days of Therapy, and Compliance
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Beta Thalassemia Bibliography

Author(s): Archita Kukreja; Narendra Parihar

Archita is a member of Decision Resources Group’s Infectious, Niche, and Rare Diseases (INRD) team. In this role she works on a range of antibacterial and antiviral indications as well as numerous niche and rare diseases. Archita holds a Masters in Science degree in biotechnology from Jamia Millia Islamia, New Delhi and has completed her MBA from Amity University, Noida.

Narendra is an associate epidemiologist within the epidemiology team at Decision Resources Group. Narendra specializes in developing epidemiological forecasts for multiple indications within the DRG syndicated portfolio. His qualifications include an MPH with specialization in Health Policy, Economics and Finance from the Tata Institute of Social Sciences, Mumbai, and a Bachelor’s degree in Dentistry from the Rajasthan University of Health Sciences.