MarketVue® Report
Sickle Cell Disease (SCD) (U.S.), 2024

Syndicated market assessment report driven by primary market research

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Sickle Cell Disease (SCD) (U.S.), August 2024

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The MarketVue®: Sickle Cell Disease market landscape report combines primary (KOL interviews) 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®: Sickle Cell Disease report is supported by 15 qualitative interviews with key opinion leaders and secondary research.
 
Geographies covered
United States plus epidemiology for EU5 (France, Germany, Italy, Spain, United Kingdom)
 
Key companies mentioned
  • Vertex Pharmaceuticals
  • CRISPR Therapeutics
  • Bluebird Bio
  • Pfizer
  • Novartis
  • Novo Nordisk
  • Agios
  • Roche
  • GSK
  • Asklepion Pharmaceuticals
  • Beam Therapeutics
  • Bioray Laboratories
  • Bristol Myers Squibb
  • CorrectSequence Therapeutics
  • Editas Medicine
  • Fulcrum Therapeutics
  • Hillhurst Biopharmaceuticals
  • Invenux
  • Kamau Therapeutics
  • Secura Bio
 
Key drugs mentioned
  • Hydroxyurea
  • Oxbryta (voxelotor)
  • Adakveo (crizanlizumab)
  • Endari (L-glutamine)
  • Casgevy (exagamglogene autotemcel)
  • Lyfgenia (lovotibeglogene autotemcel)
  • Inclacumab
  • Mitapivat
  • Etavopivat
  • Osivelotor
  • Tebapivat (AG-946)
  • Crovalimab
  • Epeleuton
  • Decitabine-Tetrahydrouridine (Ndec)
  • ITU512
  • GSK4172239D
  • Nulabeglogene autogedtemcel (nula-cel)
  • Pociredir (FTX-6058)
  • CS-101
  • BEAM-101
  • HBI-002
  • Panobinostat
  • Renizgamglogene autogedtemcel (Reni-cel, EDIT-301)
  • SCD-101
  • BMS-986470
  • L-citrulline
  • BRL-101
1. DISEASE OVERVIEW
Figure 1.1. Key biologic and clinical features of sickle cell disease that impact patient outcomes
Figure 1.2. Switching from fetal to adult hemoglobin
Figure 1.3. The protective role of HbF in sickle cell disease
Figure 1.4. Impact of abnormal RBCs on disease pathophysiology
2. EPIDEMIOLOGY
Table 2.1. Diagnosed prevalent and drug-treated patients in the G6
Figure 2.1. Geographic distribution of sickle cell disease across the U.S. and EU5
Table 2.2. Hb levels in healthy adults and those with sickle cell disease
Table 2.3. Percentage of sickle cell disease patients by genotype
Figure 2.2. Sickle cell disease population segmented by annual VOC frequency
Figure 2.3. Sickle cell disease population segmented by predominant phenotype
3. DIAGNOSIS & CURRENT TREATMENT
Table 3.1. Status of newborn screening programs for sickle cell disease in the U.S. and EU5
Figure 3.1. Longitudinal care of sickle cell disease patients
Table 3.2. Factors taken into consideration for evaluating the severity of SCD complications
Table 3.3. Hematologist-reported treatment goals for sickle cell disease patients
Figure 3.2. Hematologist-reported advantages and shortcomings of hydroxyurea
Figure 3.3. Sickle cell disease patient adherence to hydroxyurea therapy by age group
Table 3.4. FDA-approved therapies for sickle cell disease
Figure 3.4. Treatment algorithm for HbSS sickle cell disease in treated by SCD specialists
Figure 3.5. Current therapy patient share in HbSS sickle cell disease patients (n=9)
Figure 3.6. SCD specialists’ rating of the effectiveness of current therapies at treating sickle cell disease patients (n=9)
Figure 3.7. Management of sickle cell disease patients at risk of or having had a stroke
Figure 3.8. Hematologist-reported advantages and shortcomings of Oxbryta
Figure 3.9. Hematologist-reported advantages and shortcomings of Adakveo
Figure 3.10. Hematologist-reported advantages and shortcomings of L-glutamine
Figure 3.11. Hematologist criteria to consider a patient for gene therapy
Figure 3.12. Hematologists’ estimate of percent of patients who are gene therapy candidates
Table 3.5. Must-know treatment dynamics for now and the future of sickle cell disease treatment 
4. UNMET NEED
Figure 4.1. Top treatment-related unmet needs in sickle cell disease
Figure 4.2. Three Cs of unmet need in SCD in wake of Adakveo and Oxbryta availability
5. PIPELINE ANALYSIS
Table 5.1. Key players in late-stage sickle cell disease drug development
Table 5.2. Hematologist perceptions of emerging therapy approaches for sickle cell disease
Table 5.3. Therapies in Development for SCD (Phase 2 and 3)
Table 5.4. Therapies in Development for SCD (Phase 1)
Table 5.5. Comparison of efficacy of HbS polymerization inhibitors and PKR activators
6. VALUE & ACCESS
Table 6.1. Sickle cell disease drug prices, 2024 U.S.
Figure 6.1. Sickle cell disease patient population coverage by by insurance type
Table 6.2. Typical commercial payer coverage of branded therapies
Figure 6.2. Innovative payer coverage of gene therapies
Figure 6.3. Key market access dynamics that will continue to shape treatment use
Figure 6.4. Considerations for the Medicare/Medicaid CGT Access Model
Figure 6.5. Actionable market access takeaways and lessons learned from Adakveo and Oxbryta
7. METHODOLOGY
Primary Market Research Approach
Epidemiology 
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