Acute Myeloid Leukemia – Unmet Need – Detailed, Expanded Analysis (US & EU)

MARKET OUTLOOK

Acute myeloid leukemia (AML) is the most common and aggressive form of leukemia in adults, with the lowest survival rates of all blood cancer types. Despite significant advances in understanding the disease, current treatment is dominated by chemotherapeutic agents offering limited efficacy benefits, after which recurrence is almost guaranteed. After years of dormancy and high levels of unmet need, four new drug approvals in 2017—Rydapt (Novartis), Idhifa (Celgene/Agios Pharmaceuticals), Mylotarg (Pfizer), and Vyxeos (Jazz Pharmaceuticals)—are expected to transform the way AML is treated. We assess how current therapies perform on key drug attributes and measure the impact of attributes on physicians’ prescribing behavior. We also consider which unmet needs novel therapies should address to distinguish themselves from their rivals and maximize uptake.

QUESTIONS ANSWERED

  • What are the treatment drivers and goals for AML? What impact does duration of response and event-free survival rates have on prescribing decisions in AML?
  • How do current therapies, such as Celgene’s Vidaza and Otsuka/Janssen’s Dacogen, and other chemotherapy regimens perform on key clinical drug attributes for this indication?
  • What are the prevailing areas of unmet need and opportunity in first-line, older AML patients? Are late-phase emerging therapies targeting these areas?
  • What trade-offs across different clinical attributes and price are acceptable to U.S. and European oncologists for a new treatment for first-line, older AML patients?

PRODUCT DESCRIPTION

Unmet Need provides quantitative insight into U.S. and European physician perceptions of key treatment drivers and goals and the current level of unmet need for a specific disease. Commercial opportunities are analyzed, and the extent to which emerging therapies may capitalize on these opportunities is evaluated.

GEOGRAPHIES

United States, France, Germany, United Kingdom

PRIMARY RESEARCH

Survey of 60 hematologist-oncologists in U.S. and 30 hematologists in Europe

KEY DRUGS COVERED

Dacogen, gilteritinib, guadecitabine, Mylotarg, Venclexta/Venclyxto, Vidaza, quizartinib

KEY METRICS INCLUDED

  • Stated versus derived analysis of U.S. and European physician prescribing behavior.
  • Conjoint analysis with U.S. and European physicians; includes market simulator.
  • Assessment of current drug performance against treatment drivers and goals.
  • Physician perceptions of unmet need in subpopulation covered.
  • Analysis of remaining drug development opportunities.

Table of contents

  • Acute Myeloid Leukemia - Unmet Need - Detailed, Expanded Analysis (US & EU)
    • Introduction
      • Overview
      • Methodology
      • Rationale for Treatment Drivers and Goals Selection
        • Efficacy
        • Safety and Tolerability
        • Nonclinical Factors
      • Rationale for Drug Selection
        • Therapies for AML
    • Treatment Drivers and Goals
      • Key Findings: Attribute Importance
      • Relative Importance of Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Attributes to Surveyed Hematologist-Oncologists' Prescribing Decisions in AML
      • Importance of Efficacy Attributes to Prescribing Decisions in AML: United States
      • Importance of Efficacy Attributes to Prescribing Decisions in AML: Europe
      • Importance of Safety and Tolerability Attributes to Prescribing Decisions in AML: United States
      • Importance of Safety and Tolerability Attributes to Prescribing Decisions in AML: Europe
      • Importance of Convenience of Administration Attributes to Prescribing Decisions in AML: United States
      • Importance of Convenience of Administration Attributes to Prescribing Decisions in AML: Europe
      • Key Findings: Stated vs. Derived Importance
      • Stated vs. Derived Importance of Key Efficacy, Safety and Tolerability, and Convenience of Administration Attributes to Prescribing Decisions in AML: United States
      • Stated vs. Derived Importance of Key Efficacy, Safety and Tolerability, and Convenience of Administration Attributes to Prescribing Decisions in AML: Europe
    • Product Performance Against Treatment Drivers and Goals
      • Key Findings
      • Overall Performance of Key Therapies for AML: United States
      • Overall Performance of Key Therapies for AML: Europe
      • Relative Performance of Key Therapies for AML Across Select Efficacy Attributes: United States
      • Relative Performance of Key Therapies for AML Across Select Efficacy Attributes: Europe
      • Relative Performance of Key Therapies for AML Across Select Safety and Tolerability Attributes: United States
      • Relative Performance of Key Therapies for AML Across Select Safety and Tolerability Attributes: Europe
      • Relative Performance of Key Therapies for AML Across Select Convenience of Administration Attributes: United States
      • Relative Performance of Key Therapies for AML Across Select Convenience of Administration Attributes: Europe
    • Assessment of Unmet Need
      • Key Findings: Unmet Need in AML
      • Surveyed Hematologist-Oncologists' Satisfaction with the Performance of Key Therapies for AML on Efficacy, Safety and Tolerability, and Convenience of Administration: United States
      • Surveyed Hematologist-Oncologists' Satisfaction with the Performance of Key Therapies for AML on Efficacy, Safety and Tolerability, and Convenience of Administration: Europe
      • Surveyed Hematologist-Oncologists' Ascribed Level of Unmet Need Across Key Efficacy Attributes in AML: United States
      • Surveyed Hematologist-Oncologists' Ascribed Level of Unmet Need Across Key Efficacy Attributes in AML: Europe
      • Surveyed Hematologist-Oncologists' Ascribed Level of Unmet Need Across Key Safety and Tolerability Attributes in AML: United States
      • Surveyed Hematologist-Oncologists' Ascribed Level of Unmet Need Across Key Safety and Tolerability Attributes in AML: Europe
      • Surveyed Hematologist-Oncologists' Ascribed Level of Unmet Need Across Key Convenience of Administration Attributes in AML: United States
      • Surveyed Hematologist-Oncologists' Ascribed Level of Unmet Need Across Key Convenience of Administration Attributes in AML: Europe
      • Key Findings: Unmet Need in AML and Related Indications
      • Surveyed Hematologist-Oncologists' Ascribed Level of Unmet Need in AML and Related Indications: United States
      • Surveyed Hematologist-Oncologists' Ascribed Level of Unmet Need in AML and Related Indications: Europe
    • Opportunity Analysis
      • Areas of Opportunity in the AML Market and Emerging Therapy Insights
        • Opportunity: A Novel Therapy with a Substantial Survival Benefit
        • Opportunity: A Therapy That Improves Complete Response Rate
        • Opportunity: A Therapy with a Better Safety and Tolerability Profile
    • Target Product Profiles
      • Assessing Drug Development Opportunities
      • Target Product Profile Methodology
        • Attributes and Attribute Levels
      • Attribute Importance and Part-Worth Utilities
        • AML Target Product Profile: Attribute Importance
        • Complete Remission
        • Event-Free Survival
        • Median Overall Survival
        • Duration of Response
        • Incidence of Grade 3/4 Febrile Neutropenia
        • Incidence of Grade 3/4 Thrombocytopenia
        • Price per 28-Day Cycle
      • Conjoint Analysis-Based Simulations of Market Scenarios
        • First-Line, Elderly AML Market Simulations: Share of Preference of Target Product Profiles Included in Scenario 1
        • First-Line, Elderly AML Market Simulations: Likelihood to Prescribe Target Product Profiles Included in Scenario 1
        • First-Line, Elderly AML Market Simulations: Target Product Profiles Included in Scenario 1
        • First-Line, Elderly AML Market Simulations: Share of Preference of Target Product Profiles Included in Scenario 2
        • First-Line, Elderly AML Market Simulations: Likelihood to Prescribe Target Product Profiles Included in Scenario 2
        • First-Line, Elderly AML Market Simulations: Target Product Profiles Included in Scenario 2
    • Appendix
      • Key Abbreviations
      • Bibliography

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