Beta-Thalassemia – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Beta-Thalassemia Market Outlook

Thelansis’s “Beta-Thalassemia Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 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 Beta-Thalassemia treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Beta-Thalassemia Overview

Beta-thalassemia (BT) is a genetic disorder characterized by a deficiency (Beta+) or absence (Beta0) of the beta-globin chains of hemoglobin (Hb). There are three main types of BT: BT-minor, BT-major, and BT-intermedia. BT-minor is the heterozygous form and is generally asymptomatic. At the same time, BT-major is the homozygous form that causes splenomegaly, microcytic, and hypochromic anemia and requires systematic transfusions to maintain Hb levels above 90-100 g/L. Long-term transfusion results in iron overload, which can cause significant morbidity and hamper the vital prognosis. BT-intermedia is less severe than BT-major and diagnosed later in life, and patients may or may not require occasional transfusions. Diagnosis of BT relies on analyzing Hb by electrophoresis or HPLC. Treatment options for BT include a combination of regular transfusions and iron chelation therapy, which has led to increased survival during the last 40 years. The prognosis depends on the severity of the condition, mainly if appropriate treatment is provided.

• The US, the estimated population-based prevalence of alpha- and beta-thalassemia combined was 5.7/100,000.

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: 2022-2032

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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