Systemic sclerosis (SSc) is a rare progressive autoimmune disorder characterized by skin fibrosis, systemic inflammation, and vasculopathy that manifests as Raynaud’s disease and, often, painful digital ulcers. Risks of pulmonary arterial hypertension(PAH), interstitial lung disease (ILD), and renal crisis are high in SSc patients. The only disease-modifying therapy (DMT) approved to treat SSc is Ofev, targeted for a subpopulation of SSc patients suffering from ILD.  Boehringer Ingelheim’s Ofev awaits approval in the European Union (EU) for SSc-ILD. Ofev has the potential to advance the treatment of SSc-ILD, which partially underlies the high mortality rate in SSc.ILD and skin fibrosis are treated with systemic immunosuppressants; symptoms of PAH and vasculopathy are treated with varying combinations of vasodilators; and renal crisis is treated acutely with angiotensin-converting exzyme (ACE) inhibitors. SSc is a debilitating condition, and treatments are only marginally effective, leaving tremendous need for effective DMTs and antifibrotics.

QUESTIONS ANSWERED

  • How large is the diagnosed SSc population in the United States and EU5, and how will it change over time?
  • What is the current treatment landscape in SSc, and how will it change in the next ten years?
  • What do key opinion leaders view as the greatest unmet medical needs in the management of SSc?
  • What is the commercial potential of key emerging therapies? How will they fit into the SSc treatment landscape?

CONTENT HIGHLIGHTS

  • Geographies: United States and EU5.
  • Primary research: Six country-specific interviews with thought-leading rheumatologists supported by survey data collected for this study.
  • Epidemiology: Diagnosed and drug-treated prevalent cases of SSc by country, diagnosed prevalent cases segmented by degree of cutaneous involvement.
  • Forecast: Drug-level sales and patient share of key SSc therapies in 2018 and 2028.
  • Emerging therapies: Phase III: 2 drugs; Phase II: 9 drugs; coverage of select Phase I products.

Table of contents

  • Scleroderma (Systemic Sclerosis) - Landscape & Forecast - Disease Landscape & Forecast
    • COVID-19
      • Commercial Outlook
        • Key Findings
          • Regional Sales of Key Therapies to Treat Scleroderma (Systemic Sclerosis): 2028
          • Scleroderma (Systemic Sclerosis) SWOT Analysis
        • Drivers and Constraints
          • What Factors Are Driving Sales in Scleroderma (Systemic Sclerosis)?
          • What Factors Are Constraining Sales in Scleroderma (Systemic Sclerosis)?
        • Drug-Class-Specific Trends
          • Tyrosine Kinase Inhibitors
          • Cannabinoid CB2 Receptor Agonists
          • Antioxidant Inflammation Modulators
        • Alternative Market Scenarios
          • Alternative Scenarios for the Scleroderma Market Through 2028
      • Forecast
        • Sales of Key Therapies in Scleroderma (Systemic Sclerosis)
      • Etiology and Pathophysiology
        • Disease Overview
          • Etiology
            • Key Risk Factors for the Development of Scleroderma (Systemic Sclerosis)
          • Pathophysiology
            • Clinical Presentation
              • Expert Insight
                • Key Pathways and Drug Targets
                • Epidemiology
                  • Key Findings
                    • Epidemiology Populations
                      • Disease Definition
                      • Methods
                      • Sources for Diagnosed Prevalent Cases of Scleroderma (Systemic Sclerosis)
                      • Number of Diagnosed Prevalent Cases of Scleroderma (Systemic Sclerosis) in the Major Pharmaceutical Markets: 2018-2028
                      • Number of Diagnosed Drug-Treated Prevalent Cases of Scleroderma (Systemic Sclerosis) in the Major Markets: 2018-2028
                      • Disease Definition
                      • Methods
                      • Sources Used for Subtypes by Severity
                      • Number of Diagnosed Prevalent Cases of Scleroderma (Systemic Sclerosis) in the Major Pharmaceutical Markets by Severity: 2018-2028
                  • Current Treatment
                    • Key Findings
                      • Diagnosis
                        • ACR-EULAR Criteria for the Classification of Systemic Sclerosis
                        • Treatment Providers and Referral Patterns
                      • Treatment Goals
                        • Key Endpoints Used in Clinical Trials for Scleroderma (Systemic Sclerosis)
                      • Key Current Therapies
                        • Overview
                        • Mechanism of Action of Key Current Drug Classes Used for Scleroderma (Systemic Sclerosis)
                        • Current Treatments Used for Scleroderma (Systemic Sclerosis)
                        • Use of Key Drug Classes for the Manifestations of Systemic Sclerosis
                        • Clinical Trial Outcomes for ACE Inhibitors
                        • Key Results of Select Clinical Trials Investigating ACE Inhibitors for the Treatment of Systemic Sclerosis
                        • Advantages and Disadvantages of ACE Inhibitors
                        • Expert Insight: ACE Inhibitors
                        • Clinical Trial Outcomes for Autologous Hematopoietic Stem-Cell Transplantation
                        • Key Results of Select Clinical Trials Investigating Autologous Stem-Cell Transplantation for the Treatment of Systemic Sclerosis
                        • Ongoing Clinical Development of Autologous Hematopoietic Stem-Cell Transplant for Scleroderma (Systemic Sclerosis)
                        • Key Ongoing Trials of Autologous Hematopoietic Stem-Cell Transplants for Scleroderma (Systemic Sclerosis)
                        • Advantages and Disadvantages of Autologous Stem-Cell Transplantation
                        • Expert Insight: Autologous Hematopoietic Stem-Cell Transplant
                        • Clinical Trial Outcomes for Rituximab
                        • Key Results of Select Clinical Trials Investigating Rituximab for the Treatment of Systemic Sclerosis
                        • Advantages and Disadvantages of Rituximab
                        • Ongoing Clinical Development of Rituximab
                        • Key Ongoing Trials of Rituximab in Scleroderma (Systemic Sclerosis)
                        • Expert Insight: Rituximab
                        • Clinical Trial Outcomes for Calcium-Channel Blockers
                        • Key Results of Select Clinical Trials Investigating Calcium-Channel Blockers for the Treatment of Systemic Sclerosis
                        • Advantages and Disadvantages of Calcium-Channel Blockers
                        • Expert Insight: Calcium-Channel Blockers
                        • Clinical Trial Outcomes for Endothelin Receptor Antagonists
                        • Key Results of Select Clinical Trials Investigating Endothelin Receptor Antagonists for the Treatment of Scleroderma (Systemic Sclerosis)
                        • Advantages and Disadvantages of Endothelin Receptor Antagonists
                        • Key Ongoing Trials of Endothelin Receptor Antagonists in the Treatment of Scleroderma (Systemic Sclerosis)
                        • Expert Insight: Endothelin Receptor Antagonists
                        • Clinical Trial Outcomes for Adempas
                        • Key Results of Select Clinical Trials Investigating Adempas for the Treatment of Scleroderma (Systemic Sclerosis)
                        • Advantages and Disadvantages of Guanylate Cyclase Stimulants
                        • Ongoing Clinical Development
                        • Key Ongoing Trials of Guanylate Cyclase Stimulants in the Treatment of Scleroderma (Systemic Sclerosis)
                        • Expert Insight: Adempas
                        • Clinical Trial Outcomes for Immunosuppressants
                        • Key Results of Select Clinical Trials Investigating Immunosuppressants for the Treatment of Scleroderma (Systemic Sclerosis)
                        • Advantages and Disadvantages of Immunomodulatory Agents
                        • Ongoing Clinical Development
                        • Key Ongoing Trials of Immunomodulatory Agents in the Treatment of Scleroderma (Systemic Sclerosis)
                        • Expert Insight: Immunosuppressants
                        • Clinical Trial Outcomes for PDE-5 Inhibitors
                        • Key Results of Select Clinical Trials Investigating PDE-5 Inhibitors for the Treatment of Scleroderma (Systemic Sclerosis)
                        • Advantages and Disadvantages of PDE-5 Inhibitors
                        • Clinical Trial Outcomes for Prostacyclin Analogues
                        • Key Results of Select Clinical Trials Investigating Prostacyclin Analogues for the Treatment of Scleroderma (Systemic Sclerosis)
                        • Advantages and Disadvantages of Prostacyclin Analogues
                        • Ongoing Clinical Development
                        • Key Ongoing Trials of Prostacyclin Analogues in the Treatment of Scleroderma (Systemic Sclerosis)
                        • Expert Insight: Prostacyclin Analogues
                        • Clinical Trial Outcomes for Ofev
                        • Key Results of Select Clinical Trials Investigating Ofev for the Treatment of Systemic Sclerosis
                        • Advantages and Disadvantages of Ofev
                        • Ongoing Clinical Development of Ofev
                        • Key Ongoing Clinical Trials of Ofev in the Treatment of Scleroderma (Systemic Sclerosis)
                        • Expert Insight: Ofev
                      • Medical Practice
                        • Overview
                        • Treatment Guidelines
                        • Regional Scleroderma (Systemic Sclerosis) Treatment Guidelines
                        • Factors Influencing Drug Selection in Scleroderma (Systemic Sclerosis)
                        • Generalized Treatment Decision Tree for Scleroderma (Systemic Sclerosis)
                    • Unmet Need Overview
                      • Current and Future Attainment of Unmet Needs in Scleroderma (Systemic Sclerosis)
                      • Top Unmet Needs in Scleroderma (Systemic Sclerosis): Current and Future Attainment
                      • Expert Insight: Unmet Need in Scleroderma (Systemic Sclerosis)
                    • Emerging Therapies
                      • Key Findings
                        • Pipeline Trends for Scleroderma (Systemic Sclerosis)
                      • Key Emerging Therapies
                        • Notable Developments Among Key Emerging Therapies for Scleroderma (Systemic Sclerosis)
                        • Key Therapies in Development for Scleroderma (Systemic Sclerosis)
                        • Estimated Launch Dates of Key Emerging Therapies for the Treatment of Scleroderma (Systemic Sclerosis)
                        • Lenabasum Profile
                        • Analysis of the Clinical Development Program for Lenabasum
                        • Key Ongoing Clinical Trials of Lenabasum in the Treatment of Scleroderma (Systemic Sclerosis)
                        • Expert Insight: Lenabasum
                        • Expectations for Market Authorization and Sales Opportunity for Lenabasum in Scleroderma (Systemic Sclerosis)
                        • Bardoxolone Methyl Profile
                        • Key Ongoing Clinical Trials of Bardoxolone Methyl in the Treatment of Scleroderma (Systemic Sclerosis)
                        • Analysis of the Clinical Development Program for Bardoxolone Methyl
                        • Expert Insight: Bardoxolone Methyl
                        • Expectations for Launch and Sales Opportunity for Bardoxolone Methyl in Scleroderma (Systemic Sclerosis)
                        • Esbriet Profile
                        • Key Ongoing Clinical Trials of Esbriet in the Treatment of Scleroderma (Systemic Sclerosis)
                        • Analysis of the Clinical Development Program for Esbriet
                        • Expert Insight: Esbriet
                        • Expectations for Market Authorization and Sales Opportunity for Esbriet in Scleroderma (Systemic Sclerosis)
                        • Orencia Profile
                        • Orencia Clinical Development
                        • Expert Insight: Orencia
                        • Expectations for Launch and Sales Opportunity for Orencia in Scleroderma (Systemic Sclerosis)
                        • Celution System Profile
                        • Analysis of the Clinical Development Program for the Celution System
                        • Expert Insight: Celution System
                        • Expectations for Market Authorization and Sales Opportunity for the Celution System in Scleroderma (Systemic Sclerosis)
                      • Early-Phase Pipeline Analysis
                        • Select Compounds in Early-Phase Development for Scleroderma (Systemic Sclerosis)
                      • Key Discontinuations and Failures in Systemic Sclerosis (Scleroderma)
                        • Expert Insight: Key Discontinuations and Failures
                      • Patient Registries
                        • Patient Registries for Scleroderma (Systemic Sclerosis)
                        • Prominent Scleroderma (Systemic Sclerosis) Patient Organizations
                      • Orphan Drug Designation
                      • Access & Reimbursement Overview
                        • Region-Specific Reimbursement Practices
                          • Key Market Access Considerations in Scleroderma (Systemic Sclerosis): United States
                          • General Reimbursement Environment: United States
                          • Key Market Access Considerations in Scleroderma (Systemic Sclerosis): EU5
                          • General Reimbursement Environment: EU5
                        • Looking for More?
                      • Methodology
                        • Bottom-Up Forecasting Overview
                          • Patient Populations
                          • Drug-Specific Assumptions
                        • Bottom-Up Forecast Assumptions
                          • General Sources of Data
                          • Drug-Treatment Rate Assumptions in Scleroderma (Systemic Sclerosis)
                          • Patient-Share Assumptions in Scleroderma (Systemic Sclerosis)
                          • General Statements About Pricing
                          • Sources for Drug Prices
                          • 2018 Exchange Rates
                          • Price per Treated Day: 2028
                          • Dosing, Days of Therapy, and Compliance
                          • Generic Erosion
                          • Generic Erosion Assumptions
                          • Out-Year Forecasting
                          • Emerging Therapy Prices
                        • Primary Market Research
                          • Experts Interviewed
                      • Appendix
                        • Key Abbreviations Related to Scleroderma (Systemic Sclerosis)
                        • Brands, Marketers, and Generic Availability of Key Therapies for Scleroderma (Systemic Sclerosis), by Market
                        • Scleroderma (Systemic Sclerosis) Bibliography

                    Author(s): Raina Priyadarshini, Ph.D; Sunali D. Goonesekera, SM

                    Raina is a senior analyst on the Infectious, Rare, and Niche Diseases team at Decision Resources Group.

                    Before joining DRG, Raina worked in drug development for novel disease targets. She gained experience assessing market research and competitive intelligence in rare disorders in multiple therapy areas.

                    She holds a Ph.D. in molecular biology from the National Institute of Immunology in India.

                    Sunali Goonesekera is an Associate Epidemiologist at Decision Resources Group.

                    Sunali holds a Master’s degree in Epidemiology from the Harvard School of Public Health and a B.A. in Biology (Honors) from Dartmouth College. Prior to joining Decision Resources Group, Sunali conducted epidemiological research and lead authored two manuscripts on racial/ethnic disparities in metabolic diseases at the New England Research Institutes. She has contributed to multiple publications in peer-reviewed journals in epidemiology and in the biological sciences.


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