Cold Agglutinin Disease (CAD) (U.S.), July 2022
The MarketVue®: Cold Agglutinin Disease market landscape report combines primary (KOL interviews and survey data) and secondary market research to empower strategic decision-making and provide a complete view of the market.
Every MarketVue® includes a disease overview, epidemiology (US and EU5), current treatment, unmet needs, pipeline and access and reimbursement chapter.
Topics covered in this report:
- Disease overview: Review the disease pathophysiology and potential druggable targets
- Epidemiology: Understand prevalence, diagnosed and drug-treated prevalence of the population and key market segments
- Current treatment: Understand the treatment decision tree and strengths and weaknesses of current on-label and off-label treatment
- Unmet needs: Identify opportunities to address treatment or disease management gaps
- Pipeline analysis: Compare current and emerging therapy clinical development strategy; their performance on efficacy, safety, and delivery metrics; and their potential to address unmet needs
- Value and access: Gain insights into the drug pricing landscape and payer controls within the disease market or analogous markets
Methodology:
Research for the MarketVue®: Cold Agglutinin Disease report is supported by 9 qualitative interviews with key opinion leaders, a quantitative survey with 25 U.S. physicians and secondary research.
Geographies covered:
United States plus epidemiology for EU5 (France, Germany, Italy, Spain, United Kingdom)
Key companies mentioned:
- Sanofi/Genzyme
- Swedish Orphan Biovitrum AB (SOBI)
- Apellis Pharmaceuticals
- Novartis
Key drugs mentioned:
- Rituximab (Rituxan)
- Bendamustine
- Fludarabine (Fludara)
- Sutimlimab (Enjaymo)
- Corticosteroids
- Venetoclax (Venclexta)
- Bortezomib (Velcade)
- Ibrutinib (Imbruvica)
- Iptacopan (Fabhalta)
- Pegcetacoplan (Empaveli)
- SAR445088
1. DISEASE OVERVIEW
A rare, cold-activated, autoimmune bone marrow disorder characterized by hemolytic anemia
Figure 1.1. Classical complement pathway in CAD
CAD is characterized by anemia-related and cold-associated symptoms
Table 1.1. Clinical presentation of CAD
Figure 1.2. Hematologist-reported segmentation of their CAD patient population
2. EPIDEMIOLOGY & PATIENT POPULATIONS
Disease definition
Figure 2.1 – Diagnosed prevalent cases of CAD by region
Table 2.1 – Diagnosed prevalent and drug-treated patients in the U.S. and EU5
3. DIAGNOSIS AND CURRENT TREATMENT
Diagnosis overview
Figure 3.1. Diagnostic pathway for CAD patients
B-cell and complement-directed therapy are the current standards of care
Overview
Figure 3.2. Treatment goals for CAD
Figure 3.3. Current treatment patient share
Treatment flow for CAD
Figure 3.4. Treatment algorithm for CAD
Rituximab-based regimens are the most widely used treatments for CAD in US and EU
Figure 3.5. Hematologist rating (n=25) of current therapy effectiveness in CAD
Figure 3.6. Hematologist-reported percentage of CAD patients who are refractory to rituximab
There is room for improvement over existing treatments
Hematologists’ opinions on current treatments
Severity of anemia and dosing are critical drivers of treatment choice between rituximab and Enjaymo
Enjaymo is the first and only USFDA-approved drug for primary CAD
Enjaymo is viewed favorably in the US and EU, with a few reservations
Figure 3.7. Enjaymo’s advantages observed in clinical practice
Figure 3.8. Percentage of hematologists who rated Enjaymo as “extremely favorable” for its attributes
Figure 3.9. Percentage of hematologists familiar with Enjaymo
Hematologists’ perception of Enjaymo
Key treatment dynamics that shape disease management and drug use in CAD
Table 3.1 Must-know CAD treatment dynamics
Figure 3.10. Hematologist-reported percentage of patients with unsatisfactory outcomes with current CAD treatments
Complement pathway inhibition is the future of CAD therapy
Figure 3.11. Important dynamics of CAD market evolution
4. UNMET NEED
Overview
Figure 4.1 – Top unmet needs in CAD
Figure 4.2 – Physician-reported unmet needs in CAD
Important unmet needs in treating CAD remain
Figure 4.3 – Three unmet needs in CAD despite the addition of sutimlimab as a treatment option
Non-clinical barriers to care – physician perspective
Figure 4.4 – Percent of hematologists who see this as a barrier to receiving drug therapy
5. PIPELINE ANALYSIS
Overview
Figure 5.1. Number of emerging CAD therapies whose clinical trial primary end points target top unmet needs
Pipeline for CAD is limited
Table 5.1. Comparison of ongoing trials of complement-mediated therapies in CAD
Emerging therapies are complement inhibitors
Figure 5.2. Percentage of hematologists who rated the following emerging therapies as “promising”
Figure 5.3. Percentage of hematologists who rated emerging target or approach as “promising”
6. VALUE & ACCESS
Overview
Table 6.1 – Current therapy pricing
Table 6.2 – Typical commercial payer coverage of Rituxan and Enjaymo (e.g., United Healthcare, Aetna, BlueCross BlueShield, Cigna)
Key market access dynamics that will continue to shape treatment use
Figure 6.1 – CAD patients by insurance type
Thoughts from european physicians surrounding current and future access to CAD therapies
Drug Analogue: Soliris, Ultomiris, and Empaveli in PNH
Overview
Commercial payer requirement of drug analogues
Table 6.3. Summary of commercial insurance requirements for Empaveli, Ultomiris, and Soliris
Drug Analogue: Soliris in PNH
Overview
Figure 6.2. Annual cost (WAC) of Soliris (000s), 2007 – 2021
Figure 6.3. Key market access pillars for Soliris in PNH
Drug Analogue: Empaveli and Ultomiris in PNH
Overview
Figure 6.4. Strategies used by Ultomiris and Empaveli to compete with Soliris
Thoughts from European physicians surrounding current and future access to CAD therapies
7. METHODOLOGY
Primary market research approach
Epidemiology methodology
What is the difference between purchasing a published report and requesting a new report?
Published reports have been pre-produced on the specified date and can be delivered immediately. If you prefer to refresh an existing report or request an entirely new disease, REACH can create and deliver any new report within 15 business days.
How does REACH produce new primary market research reports in 15 business days?
By deploying our senior team to each of our reports, we can begin insight generation on day 1. The REACH team also recruits Key Opinion Leaders from our internal panel rather than outsourcing recruitment which enables us to quickly begin primary market research interviews.
Who writes REACH reports?
Each REACH report is produced by one of our senior US-based team members with 10+ years of experience in life sciences market research. We are deeply committed to ensuring REACH reports are high quality and strategic to enable informed decision-making for our clients.
Does REACH cover both rare and non-rare diseases?
REACH specializes in covering diseases and subpopulations that are not addressed by traditional market research reports. As such, we focus on rare diseases and niche subpopulations of more common conditions (e.g., Dupixent-refractory atopic dermatitis). Our flexible model enables in-depth assessments across all disease types, allowing us to support research for non-rare conditions as well.
Can I meet with the team to discuss the report?
Contact us to set up a call directly with the analyst who has written the report to answer any questions and discuss the key takeaways.
Published Report
New Report
Undecided about purchasing this report?
Reach out to learn more about report bundle discounts