Market Research

Sickle Cell Disease (SCD)

Best Price Guarantee Length Publisher Published Date SKU
from $12,250 36 Pages Reach Market Research January, 2022 RMR19136512
Best Price Guarantee
Price from $12,250
Length 36 Pages
Publisher Reach Market Research
Published Date January, 2022
SKU RMR19136512

MarketVue®: Sickle Cell Disease (SCD)

The MarketVue®: Sickle Cell Disease (SCD) 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: Review the evidence needed to assess and communicate value to key stakeholders (e.g., providers, payers, regulators) and learn what competitors have done or are doing

Methodology:
Research for the MarketVue®: Sickle Cell Disease report is supported by 4 qualitative interviews with key opinion leaders, a quantitative survey with 24 U.S. physicians and secondary research.

Geographies covered:
United States plus epidemiology for EU5 (France, Germany, Italy, Spain, United Kingdom)

Key companies mentioned:
• Emmaus Medical
• Global Blood Therapeutics
• Novartis
• Bluebird Bio
• Forma Therapeutics / Novo Nordisk
• Agios
• Imara
• Aruvant Sciences
• Sangamo Therapeutics
• Vertex Pharmaceuticals
• CRSPR Therapeutics
• Novartis
• Intellia Therapeutics
• Novo Nordisk
• Pfizer

Key drugs mentioned:
• L-glutamine (Endari)
• Crizanlizumab (Adakveo)
• Voxelotor (Oxbryta)
• BB305
• Canakinumab (Ilaris)
• Inclacumab
• Etavopiat / FT-4202
• Mitapivat / AG-348 (Pyrukynd)
• Tovinotrine / IMR-687
• ARU-1801
• BIVV-003
• CTX001
• OTQ923
• EPI01
• GBT021601
• PF-07209326


Please note: the online download version of this report is for a global site license.


1. DISEASE OVERVIEW
A chronic and progressive inherited genetic disorder
Figure 1.1 – Hemoglobin switching
Figure 1.2 – Key features of SCD
Sickle RBC morphology and function precipitates complications
Figure 1.3 – Impact of abnormal red blood cells on disease pathophysiology
2. EPIDEMIOLOGY & PATIENT POPULATIONS
Disease definition
Figure 2.1 – G6 diagnosed prevalent cases of SCD by region
Table 2.1 – Diagnosed prevalent and drug-treated patients in the G6
Key SCD population dynamics
Distinct phenotypes demarcate two market-relevant subpopulations
Figure 2.2 – SCD population segmented by annual VOC frequency
Figure 2.3 – SCD population segmented by predominant phenotype
Table 2.2 – Hb levels in healthy and SCD adults
Figure 2.4 – SCD population segmented by Hb levels
3. CURRENT TREATMENT
Disease management and treatment goals
New market entrants offer much needed expansion of treatment options
Table 3.1 – There are six treatment options for SCD
Figure 3.1 – Hematologist rating of current therapy effectiveness in SCD
Adakveo and Oxbryta use is on the rise
Figure 3.2 – SCD current treatment patient share
Standard of care: Hydroxyurea, a chemotherapy repurposed for SCD
Figure 3.3 – Reasons for hydroxyurea use
Clinical phenotype is a critical driver of Adakveo and Oxbryta prescribing
Novartis’ Adakveo has carved out its place for reducing VOCs
Adakveo addresses a top unmet need, but barriers to its use remain
Figure 3.4 – Physician-reported barriers to Adakveo use
Oxbryta is viewed favorable for its fast response, efficacy, and tolerability
Figure 3.5 – Oxbryta advantages observed in clinical practice
Figure 3.6 – Hematologist-reported barriers to Oxbryta use
GBT’s Oxbryta is experiencing slow but steady uptake
Treatment decision in SCD – Oxbryta and Adakveo add new options and new complexity
Figure 3.7 – Treatment algorithm for the chronic management of SCD
Key treatment dynamics that shape disease management and drug use
Table 3.2 – Must-know treatment dynamics for now and the future
The outlook for current treatment and disease management is bright
Figure 3.8 – Timeline of SCD market evolution
4. UNMET NEED
Overview
Figure 4.1 – Top unmet needs in SCD
Figure 4.2 – Physician-reported unmet needs to SCD
Important unmet needs in treating SCD remain
Figure 4.3 – Three Cs of unmet need in SCD in wake of Adakveo and Oxbryta availability
The patient’s perspective on unmet need
Table 4.1 – FDA Voice of the Patient: insights from different SCD patients
Figure 4.4 – In the words of SCD patients
Non-clinical barriers to care – Physician and patient perspectives
Figure 4.5 – Hematologists’ perspective – barriers to patient care
Figure 4.6 – The patient’s perspective – barriers to care
5. PIPELINE ANALYSIS
Drug development for SCD is very active
Figure 5.1 – Number of emerging therapies addressing unmet needs in SCD
Figure 5.2 – Percentage of hematologists who rated emerging target or approach as “promising”
Summary of clinical-stage emerging SCD therapies
Table 5.1 – Emerging SCD therapies, Phase 2 and Phase 3
Table 5.2 – Emerging SCD therapies, Phase 1 and Phase 1/2
Figure 5.3 – Hematologists’ estimate of percent of SCD patients who are candidates for gene therapy
Figure 5.4 – Hematologists’ most likely use of gene therapy
Figure 5.5 – Types of patients who are candidates for gene therapy
Figure 5.6 – How physicians’ impression of Bluebird Bio’s technology changed in the past year
Figure 5.7 – Bluebird Bio’s BB305 Gene Therapy – upside and downside
6. VALUE & ACCESS
Overview
Table 6.1 – Current therapy pricing
Table 6.2 – Typical commercial payer coverage of Adakveo an Oxbryta
Key market access dynamics that will continue to shape treatment use
Figure 6.1 – SCD patients by insurance type
Figure 6.2 – Actionable market access takeaways and lessons learned from Adakveo and Oxbryta
Figure 6.3 – Percentage of payer lives covered for Oxbryta reimbursement
7. METHODOLOGY
Primary market research methodology
Epidemiology methodology
Disease definition
Prevalence estimates

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