Myelodysplastic Syndromes – Unmet Need – Detailed, Expanded Analysis (US & EU)

No disease-modifying agents have been developed to treat myelodysplastic syndromes (MDS). All pharmacological approaches to treat the disease—growth factor stimulants (e.g., erythropoietin-stimulating agents), lenalidomide, luspatercept, and hypomethylating agents—are designed to ameliorate the symptoms and extend patients’ survival. The MDS pipeline is diverse in its approaches to address both lower- and higher-risk MDS; it includes injectable biologics, small-molecule inhibitors with novel MOAs, and reformulations / modifications of current therapies. In this report, we assess the clinical and commercial opportunities in the MDS space through a comprehensive analysis of surveyed hematologists’ treatment drivers and goals and their perceptions of the performance of key current therapies against these goals.

QUESTIONS ANSWERED

  • What are the key treatment drivers and goals in MDS treatment? How well do current therapies perform on these goals?
  • What drug attributes are key influences, which have limited impact, and which are hidden opportunities?
  • Which unmet needs do hematologists-oncologists consider most important in the treatment of MDS?
  • Based on conjoint analysis and TPP simulation, what trade-offs across different clinical attributes and prices are acceptable to U.S. and European hematologists-oncologists for a hypothetical MDS 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 Clarivate 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-oncologists fielded in March 2022

Key companies: Acceleron, Bristol-Myers Squibb (BMS), Novartis, Otsuka, Sanofi, Taiho Oncology

Key drugs: Antithymocyte globulin, azacitidine, decitabine / cedazuridine, deferasirox, growth factor stimulants, lenalidomide, luspatercept

Table of contents

  • Myelodysplastic Syndromes - Unmet Need - Detailed, Expanded Analysis (US & EU)
    • Executive summary
      • Unmet need - Myelodysplastic syndromes (MDS) - Executive summary - May 2022
    • Introduction
      • Overview
      • Methodology
      • Rationale for treatment drivers and goals selection
        • Rationale for drug selection
          • Products for MDS 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-oncologists' prescribing decisions in myelodysplastic syndromes
        • Importance of efficacy attributes to prescribing decisions in myelodysplastic syndromes: United States
        • Importance of efficacy attributes to prescribing decisions in myelodysplastic syndromes: Europe
        • Importance of safety and tolerability attributes to prescribing decisions in myelodysplastic syndromes: United States
        • Importance of safety and tolerability attributes to prescribing decisions in myelodysplastic syndromes: Europe
        • Importance of convenience of administration attributes to prescribing decisions in myelodysplastic syndromes: United States
        • Importance of convenience of administration attributes to prescribing decisions in myelodysplastic syndromes: Europe
        • Importance of nonclinical factors to prescribing decisions in myelodysplastic syndromes: United States
        • Importance of nonclinical factors to prescribing decisions in myelodysplastic syndromes: 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 myelodysplastic syndromes: United States
        • Stated vs. derived importance of key efficacy, safety and tolerability, convenience of administration, and nonclinical attributes to prescribing decisions in myelodysplastic syndromes: Europe
      • Product performance against treatment drivers and goals
        • Key findings
        • Overall performance of key therapies for myelodysplastic syndromes: United States
        • Overall performance of key therapies for myelodysplastic syndromes: Europe
        • Mean overall performance of key therapies for myelodysplastic syndromes: United States and Europe
        • Relative performance of key therapies for myelodysplastic syndromes across select efficacy attributes: United States
        • Relative performance of key therapies for myelodysplastic syndromes across select efficacy attributes: Europe
        • Relative performance of key therapies for myelodysplastic syndromes across select safety and tolerability attributes: United States
        • Relative performance of key therapies for myelodysplastic syndromes across select safety and tolerability attributes: Europe
        • Relative performance of key therapies for myelodysplastic syndromes across select convenience of administration attributes: United States
        • Relative performance of key therapies for myelodysplastic syndromes across select convenience of administration attributes: Europe
        • Relative performance of key therapies for myelodysplastic syndromes across select nonclinical attributes: United States
        • Relative performance of key therapies for myelodysplastic syndromes across select nonclinical attributes: Europe
      • Assessment of unmet need
        • Key findings: Unmet need in myelodysplastic syndromes
        • Surveyed hematologists-oncologistsu2019 satisfaction with the performance of key therapies for myelodysplastic syndromes on efficacy, safety and tolerability, convenience of administration, and nonclinical factors: United States
        • Surveyed hematologists-oncologistsu2019 satisfaction with the performance of key therapies for myelodysplastic syndromes on efficacy, safety and tolerability, convenience of administration, and nonclinical factors: Europe
        • Surveyed hematologists-oncologistsu2019 ascribed level of unmet need across key efficacy attributes in myelodysplastic syndromes: United States
        • Surveyed hematologists-oncologistsu2019 ascribed level of unmet need across key efficacy attributes in myelodysplastic syndromes: Europe
        • Surveyed hematologists-oncologists' ascribed level of unmet need across key safety and tolerability attributes in myelodysplastic syndromes: United States
        • Surveyed hematologists-oncologists' ascribed level of unmet need across key safety and tolerability attributes in myelodysplastic syndromes: Europe
        • Surveyed hematologists-oncologists' ascribed level of unmet need across key convenience of administration attributes in myelodysplastic syndromes: United States
        • Surveyed hematologists-oncologists' ascribed level of unmet need across key convenience of administration attributes in myelodysplastic syndromes: Europe
        • Surveyed hematologists-oncologists' ascribed level of unmet need across key nonclinical factors in myelodysplastic syndromes: United States
        • Surveyed hematologists-oncologists' ascribed level of unmet need across key nonclinical factors in myelodysplastic syndromes: Europe
        • Key findings: Unmet need in myelodysplastic syndromes and related indications
        • Surveyed hematologists-oncologists' ascribed level of unmet need in myelodysplastic syndromes and related indications: United States
        • Surveyed hematologists-oncologists' ascribed level of unmet need in myelodysplastic syndromes and related indications: Europe
      • Opportunity analysis
        • Areas of opportunity in the myelodysplastic syndromes market and emerging therapy insights
          • Opportunity: A therapy that extends overall survival compared with current therapies
          • Opportunity: A therapy that improves time to progression to AML
          • Opportunity: A therapy that improves quality of life (QOL)
          • Opportunity: Additional effective therapies that have good safety and tolerability profiles
      • Target product profiles
        • Assessing drug development opportunities
        • Target product profile methodology
          • Attributes and attribute levels
          • Attributes of key current therapies for myelodysplastic syndromes
        • Attribute importance and part-worth utilities
          • Myelodysplastic syndromes target product profile: attribute importance
          • Change from baseline in global EORTC-QLQ-C30 score
          • Median time of progression to acute myeloid leukemia (AML)
          • Overall survival (OS) vs. conventional care regimens (CCRs) or placebo
          • % of patients experiencing GI side effects (i.e., vomiting)
          • % of patients experiencing blood and lymphatic system side effects (i.e., anemia)
          • Delivery burden (route of administration and dosing frequency)
          • Price per cycle
        • Conjoint analysis-based simulation of a market scenario
          • Myelodysplastic syndromes market simulation: share of preference of target product profiles included in the market scenario
          • Myelodysplastic syndromes market simulation: likelihood to prescribe target product profiles included in the market scenario
          • Myelodysplastic syndromes market simulation: target product profiles included in the market scenario
      • Appendix
        • Key abbreviations
        • Bibliography

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