Epidemiology Study - Overview

Hemophilia Epidemiology Study Objective:

To determine the prevalence and incidence of Hemophilia among individuals in a defined population. This objective outlines the primary aim of the study, which is to establish the frequency of Hemophilia within a specific age group and geographic area, while also seeking to understand potential factors contributing to its development. Moreover, the report is having geographic coverage including North America, Europe and rest of the world however customisation can be made in the geographic coverage.

Hemophilia Study USP:

• This epidemiological study on Hemophilia stands out due to its comprehensive approach in establishing precise prevalence rates, identifying novel risk factors, and exploring geographical variations in a diverse population.
• By integrating molecular diagnostic techniques with robust statistical analyses, the study aims to provide actionable insights to identify treatment opportunities, target population, and an overview on public health initiatives aimed at mitigating the impact of Hemophilia related health problems.
• Through meticulous data collection and rigorous methodology, the study not only contributes to the scientific understanding of Hemophilia but also serves as a foundation for future research events and healthcare policymaking in addressing this increasing Hemophilia cases.

Hemophilia related Study Overview:

The study defines Hemophilia as an advanced form of disease. The significance of studying Hemophilia epidemiology is due to its increasing prevalence in different age populations.

Hemophilia Study Design:

Population: The target population and the sampling method (e.g., random sampling from healthcare registries or population databases)

Data Collection: Detail methods for identifying Hemophilia cases (e.g., clinical examination, etc.) and demographic data (age, sex, ethnicity).

Hemophilia Epidemiological Parameters:

Prevalence: Prevalence rates considered per 1,000 or 10,000 population.

Incidence: Determine annual incidence rates per 1,000 person-years.

Risk Factors: Analyse associations between Hemophilia and potential risk factors (e.g., age, genetics).

Geographical Variations: Compare prevalence or incidence rates across different regions or countries.                  

Hemophilia study summary:

Hemophilia study summarizes the prevalence, incidence, possible risk factors, and geographic variations of the Hemophilia worldwide.

Hemophilia overview:

Hemophilia is a rare inherited bleeding disorder in which the blood does not clot properly due to deficiency or absence of specific clotting factor, factor VIII in hemophilia A and Factor IX in hemophilia B., which leads to prolonged bleeding after injuries, spontaneous internal bleeding and joint damage over time. The condition is typically X linked meaning it primarily affects males, while females are usually carriers.

The severity of hemophilia it is segmented into, mild hemophilia with clotting activity between 5% and 49% of normal, Moderate hemophilia is characterized by clotting activity ranging from 1% to 5% of normal, Severe hemophilia occurs when clotting activity is less than 1% of normal.

Hemophilia affects to individuals with an estimated frequency of about 1 in 10,000 live births. Approximately 400,000 people globally live with hemophilia. Hemophilia A is more common, accounting for 80% to 85% of cases, occurring in 1 in 5,000 live male births. Hemophilia B is less common, occurring in 1 in 30,000 live male births. Due to its X-linked inheritance, regions with higher rates of consanguineous marriages, such as Egypt, have a higher prevalence. Hemophilia C is rarer, affecting 1 in 100,000 people, but Ashkenazi Jews have a higher incidence of factor XI deficiency, around 8%. Advances in early diagnosis and treatment mean that individuals with hemophilia can expect a normal life expectancy.

Treatments available in for Hemophilia include Prophylactic Treatment, On-Demand Treatment, Gene Therapy, Desmopressin, Factor Replacement Therapy. Key market players manufacturing therapeutics for the treatment of hemophilia are as follows: Genentech, Pfizer, CSL Behring, Octapharma, Kedrion, Grifols, Roche, Novo Nordisk, Sobi, Boehringer Ingelheim, uniQure, Baxter, Amgen, Sanofi.

Key players operating in the market are developing new products. For instance, in October 2024, Pfizer has received FDA approval for its anti-TFPI antibody, marstacimab, branded as Hympavzi, to treat hemophilia A and B in patients aged 12 and older without factor VIII or IX inhibitors. This once-weekly injectable is the first in its class approved in the US and simplifies administration with a flat dose. Hympavzi aims to reduce treatment burdens and will compete with Roche's Hemlibra and Sanofi's Altuviiio.

Hemophilia Demographic and Environmental Risk Factors:

Age and Sex: Hemophilia is typically diagnosed in children, but it can also manifest in adults, particularly those over 40. It is more prevalent among individuals with autoimmune or other health issues. Hemophilia A occurs in about 1 in 5,000 male births, whereas hemophilia B is rarer, affecting approximately 1 in 25,000 males. Many women unknowingly carry the hemophilia gene, with around 1 in 500 women being carriers, which means they can pass the gene to their children.

Ethnicity: According to one of the study published by American Society of Hematology, ethnic distribution of hemophilia was approximately 17.5% Black, 9.1% Hispanic, and 72.0% White. About 45.5% of participants had severe hemophilia, with higher proportions among Black (76.0%) and Hispanic (53.8%) participants compared to White participants (37.9%). Among the participants, over one-third of Black individuals had an inhibitor, while none of the Hispanic participants and less than 5% of White participants had it.

Risk Factors causing Hemophilia: The primary risk factor for hemophilia is having family members with the disorder. Blood thinners, which are used to treat heart disease or prevent blood clots, can interfere with clotting and increase bleeding risk in people with clotting disorders. Hemophilia is more common in males because the gene responsible for it is located on the X chromosome. While females can carry the gene, they typically do not exhibit symptoms unless they inherit it from both parents.

Hemophilia Market Scope:

Drivers: Increasing Prevalence of Hemophilia Cases Globally

The Global Hemophilia Treatment Market Industry is significantly driven by the rising cases of hemophilia around the world. As global awareness and screening initiatives improve, more cases are being diagnosed, stimulating demand for effective treatment options. The increasing prevalence necessitates innovative treatments, and organizations such as Novartis and Pfizer, which are investing in advanced Research and Development of hemophilia therapies, play a crucial role in catering to this growing patient population. With advances in treatment methodologies, the Global Hemophilia Treatment Market Industry is poised for continued expansion.

Restraints: High cost of the treatment, side effects of the available treatments, strict regulatory framework leading to delay in product launch are some of the hurdles which can hamper the growth of the market.