Myelofibrosis is a hematological malignancy characterized by disrupted production of blood cells and bone marrow scarring; symptoms include severe anemia, weakness, fatigue, and splenomegaly. Incyte/Novartis’s Jakafi/Jakavi is the first and only drug approved for myelofibrosis (MF) in the United States and EU5. Although it offers a therapeutic option, the drug is associated with high discontinuation rates and a loss of response. Furthermore, not all patients benefit from Jakafi/Jakavi, nor does the agent address all MF symptoms. Therefore, significant market opportunity remains for safe and tolerable treatments that can effectively manage the full spectrum of symptoms—thereby minimizing the need for polypharmacy over long periods of time—as well as for treatment options for patients with baseline thrombocytopenia and anemia.

QUESTIONS ANSWERED

  • What are the key diagnostic challenges in MF? What are common misdiagnoses and how frequently does misdiagnosis occur?
  • What are the sizes of the U.S. and EU5 diagnosed prevalent and incident primary and secondary MF populations?
  • What are the distributions of risk level among diagnosed incident and prevalent cases of primary MF?
  • How will novel therapies impact the treatment algorithm for MF, and which MF patients will be served by these treatment options in 2028?
  • What are the key unmet needs and areas of commercial opportunity for MF? How well will the MF pipeline address these needs?

Geographies: United States and EU5

Primary Research: Six country-specific interviews with thought-leading hematologists with expertise in treating myelofibrosis. Supported by survey data collected for this study.

Epidemiology: Diagnosed incidence and prevalence of primary and secondary MF by country, diagnosed incidence and prevalence of primary MF by risk (DIPSS and IPSS, respectively), comorbidities and biomarkers (JAK2 mutation, peripheral blasts > 1%, splenomegaly), with population-specific drug-treatment rates

Forecast: Drug-level sales and patient share of key MF therapies in 2028

Emerging Therapies: Phase III / PR: 3 drugs. Phase II: >10 drugs; coverage of select preclinical and Phase I products

PRODUCT DESCRIPTION

Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.

Table of contents

  • Disease Landscape & Forecast
    • COVID-19
    • Commercial Outlook
      • Key Findings
      • Drivers and Constraints
        • What Factors Are Driving Sales in Myelofibrosis?
        • What Factors Are Constraining Sales in Myelofibrosis?
      • JAK Inhibitor-Specific Trends
      • Alternative Market Scenarios
    • Forecast
    • Etiology and Pathophysiology
      • Disease Overview
      • Etiology
      • Pathophysiology
      • Clinical Presentation of Myelofibrosis
      • Key Pathways and Drug Targets
    • Epidemiology
      • Key Findings
        • Key Updates
      • Epidemiology Populations
        • Diagnosed Incident Cases of Myelofibrosis
        • Diagnosed Prevalent Cases of Myelofibrosis
        • Diagnosed Incident Cases of Primary Myelofibrosis
        • Diagnosed Prevalent Cases of Primary Myelofibrosis
        • Diagnosed Incident Cases of Secondary Myelofibrosis
        • Diagnosed Prevalent Cases of Secondary Myelofibrosis
        • Diagnosed Incident Cases of Myelofibrosis by IPSS Risk Group
        • Diagnosed Prevalent Cases of Myelofibrosis by DIPSS Risk Group
        • Diagnosed Incident Cases of Primary Myelofibrosis by Comorbidity
        • Diagnosed Prevalent Cases of Primary Myelofibrosis by Comorbidity
        • Diagnosed Incident Cases of Primary Myelofibrosis by Biomarker Status
        • Diagnosed Prevalent Cases of Primary Myelofibrosis by Biomarker Status
        • Diagnosed Incident Cases of Secondary Myelofibrosis by MYSEC-PM Risk Group
        • Drug-Treated Prevalent Cases of Myelofibrosis
    • Current Treatment
      • Key Findings
      • Diagnosis
        • Treatment Providers and Referral Patterns
      • Disease Staging
      • Treatment Goals
      • Key Current Therapies
        • Overview
        • JAK Inhibitors
        • Cytoreductive Therapies
        • Erythropoiesis-Stimulating Agents
        • Immunomodulatory Drugs
        • Interferons
        • Androgens
        • Red Blood Cell Transfusions
        • Hypomethylating Agents
        • Allogeneic Hematopoietic Stem Cell Transplantation
      • Medical Practice
        • Overview
        • Region-Specific Treatment Practices
    • Unmet Need Overview
      • Current and Future Attainment of Unmet Needs in Myelofibrosis
    • Emerging Therapies
      • Key Findings
      • Key Emerging Therapies
        • JAK Inhibitors
        • TGFβ Inhibitors
      • Early-Phase Pipeline Analysis
      • Patient Registries
        • Patient Organizations
    • Access & Reimbursement Overview
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
      • Looking for More?
    • Methodology
      • Bottom-Up Forecasting Overview
        • Patient Populations
        • Drug-Specific Assumptions
      • Bottom-Up Forecast Assumptions
        • General Sources of Data
        • General Statements About Pricing
        • Dosing, Days of Therapy, and Compliance
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Myelofibrosis Bibliography

Author(s): Nuno Tiago Giao Antunes, PhD; Narendra Parihar

Nuno T. Antunes, ,  is senior business insights analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, specializing in antibacterial agents. Previously, he was a Latin America Market Access senior analyst in the Global Market Access Insights Team, where he developed expertise in market access, pricing and reimbursement, health technology assessment, and health policy. Nuno holds a in animal health from the Universidad de las Palmas de Gran Canaria, Spain, and a degree from the Universidade de Trás-os-Montes e Alto Douro, Portugal. Prior to joining DRG, he conducted research in antimicrobial resistance and antimicrobial development, and worked in the medical devices industry as a scientist.

Narendra is an associate epidemiologist within the epidemiology team at Decision Resources Group. Narendra specializes in developing epidemiological forecasts for multiple indications within the DRG syndicated portfolio. His qualifications include an MPH with specialization in Health Policy, Economics and Finance from the Tata Institute of Social Sciences, Mumbai, and a Bachelor’s degree in Dentistry from the Rajasthan University of Health Sciences.


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