Hemophilia – Landscape & Forecast – Disease Landscape & Forecast
Clarivate expects the next 10 years to be transitional for patients with hemophilia A (with and without inhibitors) owing to the entry of several novel therapeutic options into the market. With the emergence of Roche / Chugai’s bispecific antibody Hemlibra and a diverse pipeline both in terms of technology type and administration route, we forecast a significant change in the approach to disease management of many patients. The emphasis in R&D has shifted away from factor-VIII-replacement-based approaches to new mechanisms aimed at restoring coagulation, including gene therapies. These new therapies include Sanofi’s fitusiran and BIVV001, Novo Nordisk’s concizumab, Pfizer’s marstacimab, and BioMarin’s Roctavian. Despite the various novel nonfactor hemophilia agents forecast to launch, we expect the demand for factor-based concentrates to persist.
QUESTIONS ANSWERED
- How has Hemlibra changed the treatment landscape for hemophilia A for patients with or without inhibitors?
- How will hematologists differentiate between the various emerging therapies?
- What clinical roles will BIVV001, fitusiran, concizumab, marstacimab, mim8, and gene therapy play in the evolving hemophilia A treatment landscape?
- How will the management of patients with or without inhibitors change when new treatment options become available?
- How will the factor VIII concentrate market evolve? How will nonfactor treatment options affect the multitude of brands vying for patient share in this space?
CONTENT HIGHLIGHTS
- Markets covered: United States, France, Germany, Italy, Spain, United Kingdom, Japan.
- Primary research: 25 country-specific interviews with thought-leading hematologists. Supported by survey data collected for this and other Clarivate research.
- Epidemiology: Diagnosed prevalence of hemophilia A +/- inhibitors by country; population segmentation by disease severity and inhibitor titer.
- Emerging therapies: Coverage of select emerging therapies from preclinical to preregistration stages.
- Market forecast features: 10-year, annualized, drug-level sales and patient share of key hemophilia A (and inhibitor) therapies through 2031, segmented by brands and epidemiological subpopulations.
PRODUCT DESCRIPTION
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
Disease Landscape & Forecast will feature continual updates in 2022 to provide timely insights and analyses as meaningful indication-specific news and events unfold.
Additionally, 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
- Hemophilia - Landscape & Forecast - Disease Landscape & Forecast
- Key updates
- June 2023
- March 2023
- December 2022
- November 2022
- August 2022
- July 2022
- Key findings
- Hemophilia A +/- inhibitors - key findings - June 2023
- Market outlook
- Key findings
- Market share of drug classes for hemophilia A: 2031
- Hemophilia A SWOT analysis
- Market drivers and constraints
- What factors are driving the market for hemophilia A?
- What factors are constraining the market for hemophilia A?
- Segment-specific trends
- Patient shares for prophylactic treatments of severe hemophilia A in the major markets: 2021-2031
- Sales for prophylactic treatments of severe hemophilia A in the major markets: 2021-2031
- Patient share of prophylactic treatments of hemophilia A with high-titer inhibitors in the major markets: 2021-2031
- Sales of prophylactic treatments of hemophilia A with high-titer inhibitors in the major markets: 2021-2031
- Patient share of on-demand treatments of hemophilia A with high-titer inhibitors in the major markets: 2021-2031
- Sales of on-demand treatments of hemophilia A with high-titer inhibitors in the major markets: 2021-2031
- Patient share of immune tolerance induction for hemophilia A with high-titer inhibitors in the major markets: 2021-2031
- Sales of immune tolerance induction for hemophilia A with high-titer inhibitors in the major markets: 2021-2031
- Key findings
- Forecast
- Market Forecast Assumption - Hemophilia A [Severe_Moderate_Mild - Prophylaxis] (2021-2031) - June 2023
- Market Forecast Assumptions - Hemophilia A [Severe - On Demand] (2021-2031) - October 2022
- Market Forecast Assumptions - Hemophilia A [Moderate - On Demand] (2021-2031) - October 2022
- Market Forecast Assumptions - Hemophilia A [Mild - On Demand] (2021-2031) - October 2022
- Market Forecast Assumption - Hemophilia A [High-Titer Inhibitors - Prophylaxis] (2021-2031) - March 2023
- Market Forecast Assumptions - Hemophilia A [High-Titer Inhibitors - ITI] (2021-2031) - October 2022
- Market Forecast Assumptions - Hemophilia A [High-Titer Inhibitors - On Demand] (2021-2031) - October 2022
- Market Forecast Assumption - Hemophilia A [Low-Titer Inhibitors - Prophylaxis] (2021-2031) - March 2023
- Market Forecast Assumptions - Hemophilia A [Low-Titer Inhibitors - On Demand] (2021-2031) - October 2022
- Market Forecast Dashboard - Hemophilia A [Severe_Moderate_Mild - Prophylaxis] (2021-2031) - March 2023
- Market Forecast Dashboard - Hemophilia A - [Severe - On Demand] (2021-2031) - October 2022
- Market Forecast Dashboard - Hemophilia A - [Moderate - On Demand] (2021-2031) - October 2022
- Market Forecast Dashboard - Hemophilia A - [Mild - On Demand] (2021-2031) - October 2022
- Market Forecast Dashboard - Hemophilia A [High-Titer Inhibitors - Prophylaxis] (2021-2031) - March 2023
- Market Forecast Dashboard - Hemophilia A - [High-Titer Inhibitors - ITI] (2021-2031) - October 2022
- Market Forecast Dashboard - Hemophilia A - [High-Titer Inhibitors - On Demand] (2021-2031) - October 2022
- Market Forecast Dashboard - Hemophilia A [Low-Titer Inhibitors - Prophylaxis] (2021-2031) - March 2023
- Market Forecast Dashboard - Hemophilia A - [Low-Titer Inhibitors - On Demand] (2021-2031) - October 2022
- Etiology and pathophysiology
- Disease overview
- Etiology
- Genetics of hemophilia A
- Pathophysiology
- Key pathways and drug targets
- Select drug targets for hemophilia A
- Select novel treatment approaches for hemophilia A
- Disease overview
- Epidemiology
- Key findings
- Epidemiology populations
- Disease definition, methods, and sources used
- Diagnosed prevalent cases of hemophilia A +/- inhibitors: 2021-2031
- Disease definition, methods, and sources used
- Diagnosed prevalent cases of hemophilia A with inhibitors: 2021-2031
- Disease definition, methods, and sources used
- Diagnosed prevalent cases of hemophilia A by severity and inhibitor status: 2021-2031
- Disease definition, methods, and sources used
- Diagnosed prevalent cases of hemophilia A with inhibitor by titre: 2021-2031
- Drug-treated prevalent cases of severe hemophilia A (noninhibitor): 2021-2031
- Drug-treated prevalent cases of moderate hemophilia A (noninhibitor): 2021-2031
- Drug-treated prevalent cases of mild hemophilia A (noninhibitor): 2021-2031
- Drug-treated prevalent cases of hemophilia A with high-titer FVIII inhibitors: 2021-2031
- Drug-treated prevalent cases of hemophilia A with low-titer FVIII inhibitors: 2021-2031
- Key findings
- Current treatment
- Key findings
- Treatment goals
- Key endpoints used in clinical trials for hemophilia
- Key current therapies
- Overview
- Mechanism of action of key current drugs or drug classes used for hemophilia
- Current treatments used for hemophilia A
- Advantages and disadvantages of SHL factor VIII
- Ongoing clinical development of SHL factor VIII
- Expert insight: SHL factor VIII
- EHL factor VIII
- Advantages and disadvantages of EHL factor VIII
- Ongoing clinical development of EHL factor VIII
- Expert insight: recombinant FVIII products
- Advantages and disadvantages of NovoSeven
- Ongoing clinical development of NovoSeven
- Expert insight: NovoSeven
- Advantages and disadvantages of FEIBA
- Ongoing clinical development of FEIBA
- Expert insight: FEIBA
- Ongoing clinical development of Byclot
- Expert insight: Byclot
- Advantages and disadvantages of Sevenfact
- Ongoing clinical development of Sevenfact
- Expert insight: Sevenfact
- Advantages and disadvantages of Hemlibra
- Ongoing clinical development of Hemlibra
- Expert insight: Hemlibra
- Advantages and disadvantages of desmopressin
- Medical Practice
- Overview
- Factors influencing drug selection in hemophilia A+/- inhibitors
- Generalized treatment decision tree for hemophilia A
- Key findings
- Unmet need overview
- Current and future attainment of unmet needs in hemophilia A
- Top unmet needs in hemophilia A: current and future attainment
- Expert insight: unmet need in hemophilia A
- Drug pipeline
- Pipeline
- Regulatory Milestones
- Indication Comparison
- Emerging therapies
- Key findings
- Key emerging therapies
- Key therapies in development for hemophilia A +/- Inhibitors
- Key results of select clinical trials investigating BIVV001 for the treatment of hemophilia A
- Key ongoing clinical trials of BIVV001 in the treatment of hemophilia A
- Analysis of the clinical development program of efanesoctocog alfa (BIVV001)
- Expert insight: efanesoctocog alfa (BIVV001)
- Expectations for launch and sales opportunity of efanesoctocog alfa (BIVV001) in hemophilia A
- Key results of select clinical trials investigating concizumab for the treatment of hemophilia A
- Key ongoing clinical trials of concizumab in the treatment of hemophilia A
- Analysis of the clinical development program for concizumab
- Expert Insight: Concizumab
- Expectations for launch and sales opportunity of concizumab in hemophilia A +/- inhibitors
- Key results of select clinical trials investigating fitusiran for the treatment of hemophilia A
- Key ongoing clinical trials of fitusiran in the treatment of hemophilia A
- Analysis of the clinical development program for fitusiran
- Expert insight: fitusiran
- Expectations for launch and sales opportunity of fitusiran in hemophilia A +/- inhibitors
- Key results of select clinical trials investigating marstacimab (PF-06741086) for the treatment of hemophilia A
- Key ongoing clinical trials of marstacimab (PF-06741086) in the treatment of hemophilia A
- Analysis of the clinical development program for marstacimab (PF-06741086)
- Expert insight: marstacimab (PF-06741086)
- Expectations for launch and sales opportunity of marstacimab (PF-06741086) in hemophilia A
- Mim8 (NNC0365-3769)
- Key results of select clinical trials investigating mim8 for the treatment of hemophilia A
- Key ongoing clinical trials of Mim8 (NNC0365-3769) in the treatment of hemophilia A
- Analysis of the clinical development program for Mim8 (NNC0365-3769)
- Expert Insight: Mim8 (NNC0365-3769)
- Expectations for launch and sales opportunity of Mim8 (NNC0365-3769) in hemophilia A
- SerpinPC (AP-0101)
- Key results of select clinical trials investigating SerpinPC (AP-0101) for the treatment of hemophilia A
- Key ongoing clinical trials of SerpinPC (AP-0101) in the treatment of hemophilia A
- Analysis of the clinical development program for SerpinPC (AP-0101)
- Expert insight: SerpinPC (AP-0101)
- Expectations for launch and sales opportunity of SerpinPC (AP-0101) in hemophilia A
- Key results of select clinical trials investigating Roctavian for the treatment of hemophilia A
- Key ongoing clinical trials of Roctavian (BMN 270; valoctocogene roxaparvovec) in the treatment of hemophilia A
- Analysis of the clinical development program for Roctavian (BMN 270; valoctocogene roxaparvovec)
- Expert insight: Roctavian (BMN 270; valoctocogene roxaparvovec)
- Expectations for launch and sales opportunity of Roctavian (BMN 270; valoctocogene roxaparvovec) in hemophilia A
- Key results of select clinical trials investigating giroctocogene fitelparvovec for the treatment of hemophilia A
- Key ongoing clinical trials of giroctocogene fitelparvovec in the treatment of hemophilia A
- Analysis of the clinical development program for giroctocogene fitelparvovec
- Expectations for launch and sales opportunity of giroctocogene fitelparvovec in hemophilia A
- Key results of select clinical trials investigating SPK-8011 for the treatment of hemophilia A
- Key ongoing clinical trials of SPK-8011 in the treatment of hemophilia A
- Analysis of the clinical development program for SPK-8011
- Expectations for launch and sales opportunity of SPK-8011 in hemophilia A
- Key results of select clinical trials investigating BAY2599023 for the treatment of hemophilia A
- Key ongoing clinical trials of BAY-2599023 (DTX-201) in the treatment of hemophilia A
- Analysis of the clinical development program for BAY-2599023 (DTX-201)
- Expectations for lunch and sales opportunity of BAY-2599023 (DTX-201) in hemophilia A
- Early-phase pipeline analysis
- Select compounds in early-phase development for hemophilia A
- Early-phase pipeline analysis
- Select compounds in early-phase development for hemophilia A
- Key discontinuations and failures in hemophilia A
- Expert insight: key discontinuations and failures
- Key findings
- Access & reimbursement overview
- Region-specific reimbursement practices
- Key market access considerations in hemophilia A+/- inhibitors: United States
- General reimbursement environment: United States
- Key market access considerations in hemophilia A+/- inhibitors: EU5
- General reimbursement environment: EU5
- Key market access considerations in hemophilia A+/- inhibitors: Japan
- General reimbursement environment: Japan
- Region-specific reimbursement practices
- Appendix
- Abbreviations
- Hemophilia bibliography
- Key updates
Yogesh Shelke, M.B.A. (Pharm.), Manager, Cardiovascular, Metabolic, Renal, and Hematology. Before joining Clarivate, he was instrumental in providing actionable insights to clients from various leading pharmaceutical companies regarding competitive intelligence, market sizing and forecasting, pipeline analysis, situational analysis, unmet need analysis, and market entry strategies in a variety of therapeutic areas. He holds a bachelor’s degree in pharmacy from Pune University and a master’s degree in pharmaceutical marketing from the National Institute of Pharmaceutical Education and Research in Mohali, India.
Shilpa Thakur, M.P.H., is an epidemiologist at Clarivate. Previously, she monitored HIV sentinel surveillance in Himachal Pradesh. She has also studied the patterns of antimicrobial resistance in India. She received her M.P.H. from the Postgraduate Institute of Medical Education and Research with a specialization in epidemiology and biostatistics.