Hemophilia – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Hemophilia Market Outlook

Thelansis’s “Hemophilia 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 Hemophilia treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Hemophilia Overview

Hemophilia is a severe hereditary hemorrhagic disorder, with significant types of hemophilia A (classic hemophilia or factor VIII deficiency) and hemophilia B (also known as Christmas disease or factor IX deficiency). The disorder is inherited and is characterized by a deficiency of clotting factors in the blood. Hemophilia C is a rare condition caused by a deficiency of clotting factor XI. Approximately 25% of hemophilia A cases are caused by gross gene rearrangements, with 5% due to gross gene deletions and 20% to inversions of a 500-600 kb segment that breaks intron 22 of the F8 gene. Hemophilia B cases due to gross rearrangements of F9 are less than 2%. F9 mutations occur more frequently in male germline than in female germline. Hemophilia can manifest in mild, moderate, and severe forms, depending on the blood level or amount of clotting factor and the patient's symptoms. Mild hemophilia involves having 6-49% of the normal factor level, with bleeding typically occurring only after a significant injury, trauma, or surgery. Moderate hemophilia is characterized by having 1-5% of the normal factor level, with bleeding episodes, typically following injuries, major trauma, or surgery. Patients with moderate hemophilia may also experience occasional spontaneous bleeding episodes without an obvious cause. Differential diagnosis is required to exclude other conditions presenting with similar symptoms, such as von Willebrand disease, scurvy, diseases of platelet dysfunction, and deficiencies of other coagulation factors.

• In the United States, the estimated prevalence of hemophilia is 12 cases per 100,000 U.S. males for hemophilia A and 3.7 cases per 100,000 U.S. males for hemophilia B.

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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