Scleroderma (Systemic Sclerosis) – Landscape & Forecast – Disease Landscape & Forecast
Scleroderma, also known as systemic sclerosis (SSc), is a rare, progressive, debilitating autoimmune disorder characterized by skin fibrosis, systemic inflammation, and vasculopathy that manifests as Raynaud’s disease, skin thickening, and, often, painful digital ulcers. Mortality risks of pulmonary arterial hypertension (PAH), interstitial lung disease (ILD), and renal crisis are high in SSc patients. The only disease-modifying therapies (DMTs) approved for SSc are Boehringer Ingelheim’s Ofev (nintedanib) and Roche / Genentech’s Actemra / RoActemra (tocilizumab), both of which target the SSc-ILD subpopulation and have demonstrated the ability to slow the progressive loss of lung function. Other therapies, such as bosentan (Actelion’s Tracleer, generics) and Bayer’s Adempas (riociguat), are commonly prescribed for overlapping symptoms of PAH. The SSc pipeline is large and diverse, but most agents are in earlier phases of development. Although tremendous need exists for additional effective DMTs, drug development in SSc has been historically challenging owing to the heterogeneity of SSc patients and the commensurate challenges with trial design.
Questions answered
- How large is the diagnosed SSc population in the United States and EU5, and how will it change over the next 10 years?
- What is the current treatment landscape in SSc?
- What is the impact of recently approved therapies, and how will they change the treatment landscape over the next 10 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 SSc specialists supported by survey data collected for this study.
- Epidemiology: Diagnosed prevalence of SSc cases by country, disease severity, and drug-treatment rates.
- Forecast: Drug-level sales and patient share of key SSc therapies through 2031.
- Emerging therapies: Phase III / PR: 0 drugs; Phase I / II: 11 drugs; coverage of select early-phase 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.
Solution enhancement
Niche & Rare Disease Landscape & Forecast introduces a new Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.
Table of contents
- Scleroderma (Systemic Sclerosis) - Landscape & Forecast - Disease Landscape & Forecast
- Key findings
- Scleroderma (systemic sclerosis) key findings - November 2022
- Title
- Key takeaways
- Uptake of nintedanib and tocilizumab for SSc-ILD will drive U.S. and EU5 sales beyond $1.7 billion by 2031.
- Generics / biosimilars will capture substantial market share of the branded drugs.
- Current management of systemic sclerosis is tailored to each patientu2019s individual symptoms.
- The greatest unmet need in the treatment of SSc is for an overall DMT.
- About Clarivate
- Scleroderma (systemic sclerosis) key findings - November 2022
- Commercial outlook
- Key findings
- Regional sales of key therapies to treat scleroderma (systemic sclerosis): 2031
- 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
- Interleukin-6 inhibitors
- Key findings
- 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
- Key pathways and drug targets
- Disease overview
- 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: 2021-2031
- Number of diagnosed drug-treated prevalent cases of scleroderma (systemic sclerosis) in the major markets: 2021-2031
- 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: 2021-2031
- Key findings
- Current treatment
- Key findings
- Diagnosis
- ACR and 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)
- Market events impacting the use of key current therapies for 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 transplantation
- Key ongoing trials of autologous hematopoietic stem-cell transplantation in the treatment of systemic sclerosis
- Advantages and disadvantages of autologous hematopoietic stem-cell transplantation
- Expert insight: autologous hematopoietic stem-cell transplantation
- 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 the treatment of 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 systemic sclerosis
- Advantages and disadvantages of endothelin receptor antagonists
- Expert insight: endothelin receptor antagonists
- Key results of select clinical trials investigating Adempas for the treatment of systemic sclerosis
- Advantages and disadvantages of Adempas
- Ongoing clinical development of Adempas
- Key ongoing trials of Adempas in the treatment of systemic sclerosis
- Expert Insight: Adempas
- Actemra / RoActemra
- Clinical trial outcomes for Actemra / RoActemra
- Advantages and disadvantages of Actemra / RoActemra
- Key results of select clinical trials investigating Actemra / RoActemra for the treatment of systemic sclerosis
- Expert insight: Actemra / RoActemra
- Clinical trial outcomes for immunosuppressants
- Key results of select clinical trials investigating immunosuppressants for the treatment of systemic sclerosis
- Advantages and disadvantages of immunosuppressants
- Ongoing clinical development of immunosuppressants
- Key ongoing trials of immunosuppressants in the treatment of 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 systemic sclerosis
- Advantages and disadvantages of PDE-5 inhibitors
- Expert insight: PDE-5 inhibitors
- Clinical trial outcomes for prostacyclin analogues
- Key results of select clinical trials investigating prostacyclin analogues for the treatment of systemic sclerosis
- Advantages and disadvantages of prostacyclin analogues
- Ongoing clinical development of prostacyclin analogues
- Key ongoing trials of prostacyclin analogues in the treatment of 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 trials of Ofev in the treatment of 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)
- Key findings
- 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)
- Drug Pipeline
- Pipeline
- Regulatory Milestones
- Indication Comparison
- Emerging therapies
- Key findings
- Key emerging therapies
- Key therapies in development for scleroderma (systemic sclerosis)
- Dersimelagon profile
- Key ongoing clinical trials of dersimelagon in the treatment of scleroderma (systemic sclerosis)
- Expert insight: dersimelagon
- Expectations for launch and sales opportunity of dersimelagon in scleroderma (systemic sclerosis)
- Early-phase pipeline analysis
- Select compounds in early-phase development for scleroderma (systemic sclerosis)
- Key discontinuations and failures in scleroderma
- Patient registries
- Patient registries for scleroderma (systemic sclerosis)
- Prominent scleroderma (systemic sclerosis) patient organizations
- Orphan drug designation
- Key findings
- 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
- Region-specific reimbursement practices
- Appendix
- Abbreviation table
- Scleroderma (systemic sclerosis) bibliography
- Key findings
Rafael Widjajahakim, M.S., is an analyst on the Infectious, Niche, and Rare Diseases team at Clarivate. Prior to joining the company, he was a clinical research coordinator at University of Massachusetts Medical School. He received his master of science degree in clinical investigation from Boston University and his bachelor of science degree in biology from Suffolk University.
Deepika Chauhan, M.B.B.S., M.P.H., is an associate epidemiologist at Clarivate. Her focus is on oncology indications such as non-small-cell lung cancer, colorectal cancer, and gastric cancer. Prior to this role, she was an intern with DRG Analytics, where she worked with cross-functional teams to deliver analytical solutions to clients. Ms. Chauhan also worked at Drexel University as a research assistant on the EPA’s project to prevent Legionnaire’s disease in residential buildings. She graduated with an M.P.H. degree in epidemiology from Drexel University and holds a bachelor’s degree in medicine.