DRG anticipates the next ten years to be an exciting time in the hemophilia A space as the number of treatment options grow for patients with, and without, inhibitors. The hemophilia A pipeline is diverse, both in terms of technology type and administration route, and we are witnessing a shift in R&D emphasis; there is growing interest in nonfactor treatment approaches to restore coagulation. Examples include the launch of Roche/Chugai’s bispecific antibody Hemlibra, and the development of gene therapies, such as BioMarin’s BMN 270, and the anti-TFPI drug class, with agents like Novo Nordisk’s concizumab. Despite novel nonfactor hemophilia agents forecast to launch, DRG expects the high demand for factor concentrates to persist. We anticipate the factor concentrate market will remain highly competitive, and pipeline agents are looking to further evolve this treatment approach.


  • How will physicians treating hemophilia A differentiate between the various emerging therapies, and which will gain the greatest market and patient share in an increasingly stringent regulatory environment?
  • What clinical role will Hemlibra, fitusiran, and gene therapy play in the evolving hemophilia A treatment landscape?
  • How will the management of patients with inhibitors change with the arrival of new treatment options? How will Hemlibra’s head start on its competitors play out over the forecast period?
  • What will be the market impact of gene therapies in hemophilia A?


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.


United States, EU5, Japan


20 country-specific interviews with thought-leading hematologists, specializing in hemophilia. Supported by survey data collected for this and other DRG research.


Alnylam, Bayer, BioMarin, Bioverativ, Chugai, CSL Behring, Novo Nordisk, Octapharma, OPKO, Pfizer, Roche, Sanofi, Shire, Spark


Advate, Adynovate/Adynovi, Afstyla, BMN 270, concizumab, Eloctate/Elocta, FEIBA, fitusiran, FVIIa marzeptacog, Helixate, Hemlibra, Kogenate, Kovaltry, N8-GP, Novoeight, NovoSeven, Nuwiq, OPK88005, ReFacto/Xyntha, SPK-8011

Table of contents

  • Disease Landscape & Forecast
    • Key Updates
      • September 2019
      • June 2019
    • Market Outlook
      • Key Findings
        • Key Findings - Hemophilia A +/- Inhibitors
        • Market Overview - Hemophilia A +/- Inhibitors
      • Market Drivers and Constraints
        • What Factors Are Driving the Market for Hemophilia A+/- Inhibitors?
        • What Factors Are Constraining the Market for Hemophilia A +/- Inhibitors?
      • Segment-Specific Trends
        • Hemophilia A Without Inhibitors
        • Hemophilia A with Inhibitors
    • Forecast
    • Etiology and Pathophysiology
      • Disease Overview
        • Disease Overview - Hemophilia A +/- Inhibitors
      • Etiology
        • Etiology
      • Pathophysiology
        • Pathophysiology
      • Key Pathways and Drug Targets
    • Epidemiology
      • Key Findings
        • Key Findings - Hemophilia A +/- Inhibitors
      • Epidemiology Populations
        • Diagnosed Prevalent Cases of Hemophilia A
        • Diagnosed Prevalent Cases of Severe Hemophilia with Inhibitors
        • Drug-Treated Prevalent Cases of Hemophilia
    • Current Treatment
      • Key Findings
        • Key Findings - Hemophilia A +/- Inhibitors
      • Treatment Goals
      • Key Current Therapies
        • Overview
        • Recombinant Factor VIII
        • Bypass Agents
        • Substitutive Therapy
      • Medical Practice
        • Overview
        • Treatment Guidelines
        • Region-Specific Treatment Practices
    • Unmet Need Overview
      • Current and Future Attainment of Unmet Needs in Hemophilia A +/- Inhibitors
    • Emerging Therapies
      • Key Findings
        • Key Findings - Hemophilia A +/- Inhibitors
      • Key Emerging Therapies
        • Notable Developments Among Key Emerging Therapies for Hemophilia A+/- Inhibitors
        • Factor-Based Agents
        • Substitutive Therapies
        • Hemostatic Rebalancing Therapies
        • Gene Therapies
      • Early-Phase Pipeline Analysis
        • Notable Developments in the Early-Phase Pipeline for Hemophilia A+/- Inhibitors
    • Access & Reimbursement Overview
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
        • Japan
      • Looking for More?
    • Methodology
      • Market Forecast Assumptions File - Hemophilia A +/- Inhibitors
      • Bottom-Up Forecasting Overview
        • Patient Populations
        • Drug-Specific Assumptions
      • Bottom-Up Forecast Assumptions
        • General Sources of Data
        • General Statements About Pricing
        • Dosing, Days of Therapy, and Compliance
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Hemophilia Bibliography

Author(s): Kerri Brown, M Pharm; Abey John, MPH

Kerri Brown, is an analyst with Decision Resources Group, focused on finding stories in data and developing products to inform strategic decisions in the hemophilia and diabetes spaces. Kerri holds a master of pharmacy degree from King’s College London with 1 st class honors. Prior to joining DRG, she was a national clinical educator at a UK diabetes start-up, and has extensive experience of diabetes medical devices. Kerri has worked at AstraZeneca, and as a pharmacist in a national psychiatric clinical trial center. In addition to her work at DRG, Kerri is a practicing pharmacist with extensive clinical experience of UK physician prescribing in diabetes.

Abey John is a medical graduate with a Master’s in Public Health and has been associated with DRG since September 2015. He works with a global team of epidemiologists in performing systematic reviews of assigned diseases and prepare forecast models for clients. He also is involved in producing analyses for pharmaceutical drug developers on the descriptive epidemiology of major drug indications in mature and developing markets and have an overall experience of three years working in different healthcare sectors across the country. Prior to joining DRG, Abey had been working with Jhpiego (an affiliate of Johns Hopkins Medical University) in implementing Family Planning Health Programs in India with collaborating with the Government of India. He also has worked with a grassroots level NGO as a health team manager which worked for the benefit of the rural population living in the foothills of the Himalayas.

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