Transfusion-Dependent β-Thalassemia (TDT) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Transfusion-Dependent β-Thalassemia (TDT) Market Outlook

Thelansis’s “Transfusion-Dependent β-Thalassemia (TDT) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" 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 Transfusion-Dependent β-Thalassemia treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Transfusion-Dependent β-Thalassemia (TDT) Overview

β-thalassemia is typically a result of a monogenic disorder, primarily caused by a decrease or absence in the synthesis of the β-globin chain. This particular chain is vital to adult hemoglobin (HbA, α2β2). Consequently, an imbalance occurs between α and β chains, accumulating unattached α-globin chains. These excess α-globin chains form insoluble Hemi-chromes within erythrocyte precursor cells, causing damage to the erythrocyte membrane. This damage, in turn, triggers severe intramedullary erythrocyte apoptosis, referred to as ineffective erythropoiesis (IE). As a result, red blood cells (RBCs) experience a significantly shortened lifespan due to extra-medullary hemolysis, leading to severe anemia. The most severe manifestation of this condition, known as transfusion-dependent β-thalassemia (TDT), is characterized by profound anemia. To maintain adequate hemoglobin levels, individuals with TDT require lifelong and regular blood transfusions. However, these transfusions pose a considerable risk of causing progressive multi-organ damage due to the inevitable iron overload. If left untreated, β-thalassemia can inflict harm on various organs, potentially resulting in fatal consequences.

• The estimated prevalence of β-thalassemia cases in the United States varies between 1.5 to 2.6 cases per 100,000 individuals, depending on geographic factors.

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: 2021-2032

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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