Myelodysplastic Syndromes – Unmet Need – Detailed, Expanded Analysis (US/EU)
Myelodysplastic syndromes (MDS) are a heterogeneous collection of hematopoietic stem-cell disorders that disrupt normal hematopoiesis in the bone marrow. All pharmacological approaches to treating the disease—growth factor stimulants (e.g., erythropoietin-stimulating agents), lenalidomide (Revlimid, generics), Bristol Myers Squibb / Acceleron’s Reblozyl (luspatercept), Astex Pharmaceuticals’ Inqovi (decitabine and cedazuridine), and hypomethylating agents (e.g., azacitidine)—are designed to ameliorate its symptoms and extend patients’ survival. The MDS pipeline is diverse in its approaches to addressing both lower- and higher-risk MDS; it includes injectable biologics, small-molecule inhibitors with novel mechanisms of action (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 / oncologists’ treatment drivers and goals and their perceptions of the performance of key current therapies against these goals.
QUESTIONS ANSWERED
- What are the treatment drivers and goals for MDS? 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 February 2023
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 - executive summary - April 2023
- 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
- 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 / oncologists' ascribed level of unmet need across key efficacy attributes in myelodysplastic syndromes: United States
- Surveyed hematologists / oncologists' 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
- Opportunity: additional effective therapies that have good safety and tolerability profiles
- Areas of opportunity in the myelodysplastic syndromes market and emerging therapy insights
- Target product profiles
- Assessing drug development opportunities
- Target product profile methodology
- Attributes and attribute levels
- Attributes of key current and late-phase emerging 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 of target product profiles included in the market scenario
- Myelodysplastic syndromes market simulation: target product profiles included in the market scenario
- Appendix
- Key abbreviations
- Bibliography
- Executive summary
Vidhi Khosla, M.B.A., M.Sc., Healthcare Research & Data Analyst, Infectious, Niche, and Rare Diseases. Ms. Khosla has several years of experience providing consulting, research, and analytic support in the pharmaceutical and healthcare domains and has worked in numerous therapy areas, including neurology, women’s health, metabolic disorders, and rare diseases. Prior to joining Clarivate, she worked at WNS Global Services and DelveInsight Business Research LLP. She earned her bachelor’s and master’s (gold medalist) degrees in biochemistry from Delhi University and Kurukshetra University, respectively, and an M.B.A. from Symbiosis International University.