The primary goal of hemophilia A treatment is to prevent bleeding. Hemophilia A patients are a heterogeneous cohort; the tendency to bleed is highly individual. Currently available treatments including FVIII replacement therapies (e.g., Takeda’s Advate, Sanofi’s Eloctate) and Roche’s Hemlibra have helped significantly to reduce bleeding rates; nevertheless, breakthrough bleeds remain almost inevitable for severe hemophiliacs. Although full correction of coagulation is not necessary to stop bleeding, having a greater correction than is achievable using the current prophylactic treatments remains an unmet need, alongside an improved QOL and reduced dosing burden.

QUESTIONS ANSWERED

  • What are the key treatment drivers and goals within severe hemophilia A? How do the current therapies perform on these goals?
  • Which unmet needs are viewed by physicians as most important? Is further reduction in ABR the ultimate goal?
  • Are the impact on QOL and dosing burden viewed as important attributes of emerging therapies?·
  • Based on a conjoint analysis and TPP simulation, what trade-offs across different clinical attributes and prices are acceptable to U.S. and European hematologists for a hypothetical hemophilia drug?

PRODUCT DESCRIPTION

Unmet Need supports clinical development decisions by identifying key attributes and assessing areas of unmet need for a specific disease or subpopulation. Based on surveys with U.S. and European physicians, this report provides insight into key treatment drivers and goals, the performance of current therapies, and the remaining commercial opportunities. One market scenario is profiled in detail by DRG experts, and additional customized market scenarios can be evaluated with the corresponding TPP simulator.

Markets covered: United States, United Kingdom, France, Germany

Primary research: Survey of 60 U.S. and 30 European hematologists fielded in March 2021

Key companies: Bayer, Novo Nordisk, Roche, Sanofi, Takeda

Key drugs: Advate, Eloctate, Esperoct, Hemlibra, plasma-derived FVIII

Table of contents

  • Hemophilia - Unmet Need - Detailed, Expanded Analysis : Hemophilia A – Severe Without Inhibitors: US/EU
    • Executive Summary
      • Unmet Need - Severe Hemophilia A Without Inhibitors - Executive Summary - May 2021
    • Introduction
      • Overview
      • Methodology
      • Rationale for Treatment Drivers and Goals Selection
        • Rationale for Drug Selection
          • Products for Severe Hemophilia A Without Inhibitors and Rationale for Drug Selection
      • Treatment Drivers and Goals
        • Key Findings: Attribute Importance
        • Relative Importance of Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Attributes to Surveyed Hematologists' Prescribing Decisions in Severe Hemophilia A Without Inhibitors
        • Importance of Efficacy Attributes to Prescribing Decisions in Severe Hemophilia A Without Inhibitors: United States
        • Importance of Efficacy Attributes to Prescribing Decisions in Severe Hemophilia A Without Inhibitors: Europe
        • Importance of Safety and Tolerability Attributes to Prescribing Decisions in Severe Hemophilia A Without Inhibitors: United States
        • Importance of Safety and Tolerability Attributes to Prescribing Decisions in Severe Hemophilia A Without Inhibitors: Europe
        • Importance of Convenience of Administration Attributes to Prescribing Decisions in Severe Hemophilia A Without Inhibitors: United States
        • Importance of Convenience of Administration Attributes to Prescribing Decisions in Severe Hemophilia A Without Inhibitors: Europe
        • Importance of Nonclinical Factors to Prescribing Decisions in Severe Hemophilia A Without Inhibitors: United States
        • Importance of Nonclinical Factors to Prescribing Decisions in Severe Hemophilia A Without Inhibitors: Europe
        • Key Findings: Stated vs. Derived Importance
        • Stated vs. Derived Importance of Key Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Attributes to Prescribing Decisions in Severe Hemophilia A Without Inhibitors: United States
        • Stated vs. Derived Importance of Key Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Attributes to Prescribing Decisions in Severe Hemophilia A Without Inhibitors: Europe
      • Product Performance Against Treatment Drivers and Goals
        • Key Findings
        • Overall Performance of Key Therapies for Severe Hemophilia A Without Inhibitors: United States
        • Overall Performance of Key Therapies for Severe Hemophilia A Without Inhibitors: Europe
        • Mean Overall Performance of Key Therapies for Severe Hemophilia A Without Inhibitors: United States and Europe
        • Relative Performance of Key Therapies for Severe Hemophilia A Without Inhibitors Across Select Efficacy Attributes: United States
        • Relative Performance of Key Therapies for Severe Hemophilia A Without Inhibitors Across Select Efficacy Attributes: Europe
        • Relative Performance of Key Therapies for Severe Hemophilia A Without Inhibitors Across Select Safety and Tolerability Attributes: United States
        • Relative Performance of Key Therapies for Severe Hemophilia A Without Inhibitors Across Select Safety and Tolerability Attributes: Europe
        • Relative Performance of Key Therapies for Severe Hemophilia A Without Inhibitors Across Select Convenience of Administration Attributes: United States
        • Relative Performance of Key Therapies for Severe Hemophilia A Without Inhibitors Across Select Convenience of Administration Attributes: Europe
        • Relative Performance of Key Therapies for Severe Hemophilia A Without Inhibitors Across Select Nonclinical Attributes: United States
        • Relative Performance of Key Therapies for Severe Hemophilia A Without Inhibitors Across Select Nonclinical Attributes: Europe
      • Assessment of Unmet Need
        • Key Findings: Unmet Need in Severe Hemophilia A Without Inhibitors
        • Surveyed Hematologists’ Satisfaction with the Performance of Key Therapies for Severe Hemophilia A Without Inhibitors on Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Factors: United States
        • Surveyed Hematologists’ Satisfaction with the Performance of Key Therapies for Severe Hemophilia A Without Inhibitors on Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Factors: Europe
        • Surveyed Hematologists' Ascribed Level of Unmet Need Across Key Efficacy Attributes in Severe Hemophilia A Without Inhibitors: United States
        • Surveyed Hematologists' Ascribed Level of Unmet Need Across Key Efficacy Attributes in Severe Hemophilia A Without Inhibitors: Europe
        • Surveyed Hematologists' Ascribed Level of Unmet Need Across Key Safety and Tolerability Attributes in Severe Hemophilia A Without Inhibitors: United States
        • Surveyed Hematologists' Ascribed Level of Unmet Need Across Key Safety and Tolerability Attributes in Severe Hemophilia A Without Inhibitors: Europe
        • Surveyed Hematologists' Ascribed Level of Unmet Need Across Key Convenience of Administration Attributes in Severe Hemophilia A Without Inhibitors: United States
        • Surveyed Hematologists' Ascribed Level of Unmet Need Across Key Convenience of Administration Attributes in Severe Hemophilia A Without Inhibitors: Europe
        • Surveyed Hematologists' Ascribed Level of Unmet Need Across Key Nonclinical Factors in Severe Hemophilia A Without Inhibitors: United States
        • Surveyed Hematologists' Ascribed Level of Unmet Need Across Key Nonclinical Factors in Severe Hemophilia A Without Inhibitors: Europe
        • Key Findings: Unmet Need in Severe Hemophilia A Without Inhibitors and Related Indications
        • Surveyed Hematologists' Ascribed Level of Unmet Need in Severe Hemophilia A Without Inhibitors and Related Indications: United States
        • Surveyed Hematologists' Ascribed Level of Unmet Need in Severe Hemophilia A Without Inhibitors and Related Indications: Europe
      • Opportunity Analysis
        • Areas of Opportunity in the Severe Hemophilia A Without Inhibitors Market and Emerging Therapy Insights
          • Opportunity: A New Agent that Reduces Bleeding
          • Opportunity: A Novel Therapy That Improves QOL Measures
      • Target Product Profiles
        • Assessing Drug Development Opportunities
        • Target Product Profile Methodology
          • Attributes and Attribute Levels
        • Attribute Importance and Part-Worth Utilities
          • Severe Hemophilia A Without Inhibitors Target Product Profile: Attribute Importance
          • Annualized Bleed Rate
          • Haem-A-QoL Total Score Change (Lower is Better)
          • Proportion of Patients Developing De-Novo FVIII Inhibitors
          • Suitability for Use in Different Age Groups
          • Route and Setting for Drug Administration
          • Dosing Frequency
          • Price per Year
        • Conjoint Analysis-Based Simulation of a Market Scenario
          • Severe Hemophilia A Without Inhibitors Market Simulation: Share of Preference of Target Product Profiles Included in the Market Scenario
          • Severe Hemophilia A Without Inhibitors Market Simulation: Likelihood to Prescribe Target Product Profiles Included in the Market Scenario
          • Severe Hemophilia A Without Inhibitors Market Simulation: Target Product Profiles Included in the Market Scenario
      • Appendix
        • Key Abbreviations

    Author(s): Dominika Rudnicka-Noulin, Ph.D., M.Sc.

    Dominika Rudnicka-Noulin, Ph.D., M.Sc., is a senior business insights analyst on the Cardiovascular, Metabolic, Renal, and Hematologic (CMRH) Disorders team at. She has authored reports on heart failure, acute coronary syndrome, and NASH. Previously, Dr. Rudnicka-Noulin was an associate editor at Nature Communications, working in a variety of therapy areas. She also worked for three years as a postdoctoral research associate on a joint project between Imperial College London and MedImmune aimed at developing more-potent antibody-based drugs. She earned her Ph.D. at the Institut Pasteur in Paris, France, where her work was funded by the European Commission's Marie Skłodowska-Curie Actions.


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