2024 Sickle Cell Disease Report - Insight Into Expanding Treatment Landscape And Unmet Needs

(MENAFN- GlobeNewsWire - Nasdaq) Dublin, Oct. 28, 2024 (GLOBE NEWSWIRE) -- The "Sickle Cell disease market Assessment: Epidemiology, treatment Landscape, Unmet Needs, Emerging Therapies, and Value & Access" report has been added to ResearchAndMarkets.com's offering.

The 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.

It 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 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 in this Sickle Cell Disease market report:

• 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 in this Sickle Cell Disease market report:

• 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

Key Topics Covered:

1. Disease Overview

• Key biologic and clinical features of sickle cell disease that impact patient outcomes
• Switching from fetal to adult hemoglobin
• The protective role of HbF in sickle cell disease
• Impact of abnormal RBCs on disease pathophysiology

2. Epidemiology

• Diagnosed prevalent and drug-treated patients in the G6
• Geographic distribution of sickle cell disease across the U.S. and EU5
• Hb levels in healthy adults and those with sickle cell disease
• Percentage of sickle cell disease patients by genotype
• Sickle cell disease population segmented by annual VOC frequency
• Sickle cell disease population segmented by predominant phenotype

3. Diagnosis & Current Treatment

• Status of newborn screening programs for sickle cell disease in the U.S. and EU5
• Longitudinal care of sickle cell disease patients
• Factors taken into consideration for evaluating the severity of SCD complications
• Hematologist-reported treatment goals for sickle cell disease patients
• Hematologist-reported advantages and shortcomings of hydroxyurea
• Sickle cell disease patient adherence to hydroxyurea therapy by age group
• FDA-approved therapies for sickle cell disease
• Treatment algorithm for HbSS sickle cell disease in treated by SCD specialists
• Current therapy patient share in HbSS sickle cell disease patients (n=9)
• SCD specialists' rating of the effectiveness of current therapies at treating sickle cell disease patients (n=9)
• Management of sickle cell disease patients at risk of or having had a stroke
• Hematologist-reported advantages and shortcomings of Oxbryta
• Hematologist-reported advantages and shortcomings of Adakveo
• Hematologist-reported advantages and shortcomings of L-glutamine
• Hematologist criteria to consider a patient for gene therapy
• Hematologists' estimate of percent of patients who are gene therapy candidates
• Must-know treatment dynamics for now and the future of sickle cell disease treatment

4. Unmet Need

• Top treatment-related unmet needs in sickle cell disease
• Three Cs of unmet need in SCD in wake of Adakveo and Oxbryta availability

5. Pipeline Analysis

• Key players in late-stage sickle cell disease drug development
• Hematologist perceptions of emerging therapy approaches for sickle cell disease
• Therapies in Development for SCD (Phase 2 and 3)
• Therapies in Development for SCD (Phase 1)
• Comparison of efficacy of HbS polymerization inhibitors and PKR activators

6. Value & Access

• Sickle cell disease drug prices, 2024 U.S.
• Sickle cell disease patient population coverage by by insurance type
• Typical commercial payer coverage of branded therapies
• Innovative payer coverage of gene therapies
• Key market access dynamics that will continue to shape treatment use
• Considerations for the Medicare/Medicaid CGT Access Model
• Actionable market access takeaways and lessons learned from Adakveo and Oxbryta

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