Scleroderma (Systemic Sclerosis) – Unmet Need – Detailed, Expanded Analysis (US & EU)
The systemic sclerosis (SSc) pipeline is replete with therapies targeting pathways underlying autoimmunity, inflammation, and fibrosis, particularly in the early phases of development. Currently, patients are managed on an individualized basis, depending on the severity of the patient’s disease and the organs or organ systems involved. Boehringer Ingelheim’s Ofev, which was approved in 2019, is a potent tyrosine kinase inhibitor that slows the rate of decline in pulmonary function in adults with interstitial lung disease (ILD) associated with SSc. Two novel therapies are in late-phase development: Corbus’s CB2 agonist lenabasum and Reata’s bardoxolone methyl (for pulmonary arterial hypertension [PAH]). We will assess the commercial opportunity in SSc through a comprehensive look at the key efficacy, safety, and delivery attributes driving prescribing choices; the gaps in treatment left by current therapies; and the interplay of clinical and nonclinical attributes in determining the appeal of potential SSc products in the patient population.
QUESTIONS ANSWERED
- What are the treatment drivers and goals for SSc?
- What drug attributes are key influences, which have limited impact, and which are hidden opportunities?
- How do current therapies perform on key treatment drivers and goals for SSc?
- What are the prevailing areas of unmet need and opportunity in SSc?
- What trade-offs across different clinical attributes and prices are acceptable to U.S. and European rheumatologists for a hypothetical new SSc drug?
Markets covered: United States, United Kingdom, France, Germany
Primary research: Survey of 60 U.S. and 30 European rheumatologists fielded in February 2020
Key companies: Boehringer Ingelheim, Genentech, Roche, Teva
Key drugs: Ofev (nintedanib), Rituxan/MabThera (rituximab), methotrexate, cyclophosphamide, mycophenolate mofetil
KEY METRICS INCLUDED
- Target Product Profile (TPP) simulator based on conjoint analysis methodology.
- Stated versus derived importance of product attributes on prescribing behavior.
- Assessment of current drug performance against treatment drivers and goals.
- Physician perceptions of unmet needs in the indication and related indications.
- Drug development opportunities.
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. Two market scenarios are profiled in detail by DRG experts, and additional customized market scenarios can be evaluated with the corresponding TPP simulator.
Table of contents
- Scleroderma (Systemic Sclerosis) - Unmet Need - Detailed, Expanded Analysis (US & EU)
- Executive Summary
- Unmet Need - Systemic Sclerosis/Scleroderma - Executive Summary - April 2020
- Introduction
- Overview
- Methodology
- Rationale for Treatment Drivers and Goals Selection
- Rationale for Drug Selection
- Products for Systemic Sclerosis/Scleroderma and Rationale for Drug Selection
- Treatment Drivers and Goals
- Key Findings: Attribute Importance
- Relative Importance of Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Attributes to Surveyed Rheumatologists' Prescribing Decisions in Systemic Sclerosis
- Importance of Efficacy Attributes to Prescribing Decisions in Systemic Sclerosis: United States
- Importance of Efficacy Attributes to Prescribing Decisions in Systemic Sclerosis: Europe
- Importance of Safety and Tolerability Attributes to Prescribing Decisions in Systemic Sclerosis: United States
- Importance of Safety and Tolerability Attributes to Prescribing Decisions in Systemic Sclerosis: Europe
- Importance of Convenience of Administration Attributes to Prescribing Decisions in Systemic Sclerosis: United States
- Importance of Convenience of Administration Attributes to Prescribing Decisions in Systemic Sclerosis: Europe
- Importance of Nonclinical Factors to Prescribing Decisions in Systemic Sclerosis: United States
- Importance of Nonclinical Factors to Prescribing Decisions in Systemic Sclerosis: 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 Systemic Sclerosis: United States
- Stated vs. Derived Importance of Key Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Attributes to Prescribing Decisions in Systemic Sclerosis: Europe
- Product Performance Against Treatment Drivers and Goals
- Key Findings
- Overall Performance of Key Therapies for Systemic Sclerosis: United States
- Overall Performance of Key Therapies for Systemic Sclerosis: Europe
- Mean Overall Performance of Key Therapies for SSc: United States and Europe
- Relative Performance of Key Therapies for Systemic Sclerosis Across Select Efficacy Attributes: United States
- Relative Performance of Key Therapies for Systemic Sclerosis Across Select Efficacy Attributes: Europe
- Relative Performance of Key Therapies for Systemic Sclerosis Across Select Safety and Tolerability Attributes: United States
- Relative Performance of Key Therapies for Systemic Sclerosis Across Select Safety and Tolerability Attributes: Europe
- Relative Performance of Key Therapies for Systemic Sclerosis Across Select Convenience of Administration Attributes: United States
- Relative Performance of Key Therapies for Systemic Sclerosis Across Select Convenience of Administration Attributes: Europe
- Relative Performance of Key Therapies for Systemic Sclerosis Across Select Nonclinical Attributes: United States
- Relative Performance of Key Therapies for Systemic Sclerosis Across Select Nonclinical Attributes: Europe
- Assessment of Unmet Need
- Key Findings: Unmet Need in Systemic Sclerosis
- Surveyed Rheumatologistsu2019 Satisfaction with the Performance of Key Therapies for Systemic Sclerosis on Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Factors: United States
- Surveyed Rheumatologistsu2019 Satisfaction with the Performance of Key Therapies for Systemic Sclerosis on Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Factors: Europe
- Surveyed Rheumatologists' Ascribed Level of Unmet Need Across Key Efficacy Attributes in Systemic Sclerosis: United States
- Surveyed Rheumatologists' Ascribed Level of Unmet Need Across Key Efficacy Attributes in SSc: Europe
- Surveyed Rheumatologists' Ascribed Level of Unmet Need Across Key Safety and Tolerability Attributes in Systemic Sclerosis: United States
- Surveyed Rheumatologists' Ascribed Level of Unmet Need Across Key Safety and Tolerability Attributes in Systemic Sclerosis: Europe
- Surveyed Rheumatologists' Ascribed Level of Unmet Need Across Key Convenience of Administration Attributes in Systemic Sclerosis: United States
- Surveyed Rheumatologists' Ascribed Level of Unmet Need Across Key Convenience of Administration Attributes in Systemic Sclerosis: Europe
- Surveyed Rheumatologists' Ascribed Level of Unmet Need Across Key Nonclinical Factors in Systemic Sclerosis: United States
- Surveyed Rheumatologists' Ascribed Level of Unmet Need Across Key Nonclinical Factors in Systemic Sclerosis: Europe
- Key Findings: Unmet Need in Systemic Sclerosis and Related Indications
- Surveyed Rheumatologists' Ascribed Level of Unmet Need in Systemic Sclerosis and Related Indications: United States
- Surveyed Rheumatologists' Ascribed Level of Unmet Need in Systemic Sclerosis and Related Indications: Europe
- Opportunity Analysis
- Areas of Opportunity in the Systemic Sclerosis Market and Emerging Therapy Insights
- Opportunity: Therapies That Are Effective In Treating ILD Associated With Systemic Sclerosis
- Opportunity: Therapies That Are Effective In Treating Skin Fibrosis
- Opportunity: Therapies That Improve Quality of Life of Systemic Sclerosis patients
- Areas of Opportunity in the Systemic Sclerosis 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 Systemic Sclerosis
- Attribute Importance and Part-Worth Utilities
- Systemic Sclerosis Target Product Profile: Attribute Importance
- Annual Rate of Decline in FVC over 52 Weeks
- Percent Relative Change from Baseline of MRSS at Week 52
- Absolute Change from Baseline of HAQ-DI at Week 52
- Percent of Responders Based on CRISS at Week 52
- Delivery Burden (Route of Administration and Dosing Frequency)
- Percentage of Patients with Adverse Events up to 100 Weeks
- Price per Day
- Conjoint Analysis-Based Simulation of a Market Scenario
- Systemic Sclerosis Market Simulation: Share of Preference of Target Product Profiles Included in the Market Scenario
- Systemic Sclerosis Market Simulation: Likelihood to Prescribe Target Product Profiles Included in the Market Scenario
- Systemic Sclerosis Market Simulation: Target Product Profiles Included in the Market Scenario
- Appendix
- Key Abbreviations
- Executive Summary
Sonal Divekar, M.S., is an analyst on the Infectious, Niche, and Rare Diseases team at Clarivate. Previously, she was a senior analyst at Merck’s Global Centre for Analytics and Forecasting, where she created long-range forecasts and demand forecasts for therapeutic areas such as diabetes and immunology for the European market. She holds a master’s degree in marketing management from India’s Symbiosis Institute of Business Management and a master’s degree in microbiology from Pune University.