Sickle Cell Disease (SCD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Sickle Cell Disease (SCD) Market Outlook

Thelansis’s “Sickle Cell Disease (SCD) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Sickle cell disease treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Sickle Cell Disease (SCD) Overview

Sickle cell disease (SCD) results from homozygous and compound heterozygote inheritance of a mutation in the β-globin gene. This genetic anomaly leads to the formation of dense and rigid red blood cells (RBCs), particularly those containing HbS or HbS in combination with other abnormal β alleles. When exposed to a deoxygenated environment, these RBCs undergo polymerization, becoming rigid and prone to haemolysis. The increased density of these cells can impact blood flow and compromise endothelial vessel wall integrity, causing vaso-occlusion, tissue ischemia, infarction, and further haemolysis. The consequences of haemolysis set off a complex cascade of events, including nitric oxide consumption, intricately linked to nitric oxide dysregulation and endothelial dysfunction. These underlying factors contribute to complications such as leg ulceration, stroke, pulmonary hypertension, and priapism. In a significant development in 2023, the U.S. Food and Drug Administration (FDA) approved two groundbreaking treatments, Casgevy and Lyfgenia, marking the introduction of the first cell-based gene therapies for treating SCD in patients 12 years and older. Notably, Casgevy represents a milestone as the first FDA-approved treatment utilizing a novel genome editing technology. This advancement opens new avenues for managing SCD and offers hope for improved outcomes in affected individuals.

• The incidence of sickle cell disease (SCD) ranges between 27 and 35 cases per 100,000 people in the USA.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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