Myelodysplastic Syndromes | Unmet Need | US/EU | 2022

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.


  • 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?


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

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