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

Hemophilia B Market Outlook

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

Hemophilia B Overview

Hemophilia B is an uncommon congenital blood disorder characterized by a clotting factor IX (FIX) deficiency, resulting in spontaneous bleeding episodes, particularly in joints, and delayed blood clotting during external bleeding incidents. Repeated joint bleeding can lead to long-term joint deterioration, physical limitations, the necessity for joint replacements, chronic pain, and a diminished quality of life (QoL). The level of circulating FIX determines the severity of Hemophilia B (HB). The treatment approach revolves around FIX replacement therapy, which can be administered either preventively to avert bleeding episodes or reactively after a bleeding episode, known as "on-demand" treatment. Prophylactic FIX therapy is regarded as the standard of care for patients with severe and moderately severe HB. FIX is administered through intravenous injections in either standard half-life (SHL) or extended half-life (EHL) treatments every 2–3 or 7–14 days. However, the frequent infusions required by FIX treatment can impose a significant treatment burden, potentially affecting adherence and clinical effectiveness. The most prevalent complication of hemophilia is attributed to bleeding episodes. Repeated hemarthrosis leads to inflammation and hypertrophy of the synovial membrane, ultimately causing destructive arthropathy. Other complications include intracranial hemorrhages following minor trauma, hypovolemic shock events due to bleeding in the iliopsoas muscle, and airway obstruction due to retropharyngeal bleeding.

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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