Epidemiology Study - Overview

Sickle Cell Disease Epidemiology Study Objective:

To determine the prevalence and incidence of Sickle Cell Disease among individuals in a defined population. This objective outlines the primary aim of the study, which is to establish the frequency of Sickle Cell Disease 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.

Sickle Cell Disease Study USP:

• This epidemiological study on Sickle Cell Disease 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 advanced imaging 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 Sickle Cell Disease related health problems.
• Through meticulous data collection and rigorous methodology, the study not only contributes to the scientific understanding of Sickle Cell Disease but also serves as a foundation for future research events and healthcare policymaking in addressing this increasing Sickle Cell Disease cases.

Sickle Cell Disease related Study Overview:

The study Defines Sickle Cell Disease as an advanced form of disease. The significance of studying Sickle Cell Disease epidemiology is due to its increasing prevalence in different age populations.

Sickle Cell Disease 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 Sickle Cell Disease cases (e.g., clinical examination, imaging studies) and demographic data (age, sex, ethnicity).

Sickle Cell Disease 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 Sickle Cell Disease and potential risk factors (e.g., age, genetics).

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

Sickle Cell Disease study summary:

Sickle Cell Disease study summarizes the prevalence, incidence, possible risk factors, and geographic variations of the Sickle Cell Disease worldwide.

Sickle Cell Disease overview:

Sickle Cell Disease is a genetic blood disorder characterised by the sickle shaped red blood cells which are involved in carrying oxygen, these abnormally shaped cells can block blood flow, leading to pain, organ damage and increased risk of infection.

Based on the severity of the sickle cell disease is segmented into three categories including mild, moderate and severe sickle cell anaemia.

According to U.S centre for disease control and Prevention (CDC), Life expectancy of the individuals with SCD in US is estimated to be more than 20 years shorter than average expected, and quality adjusted life expectancy is expected to be more than 30 years shorter. SCD is seen to affect millions of individuals globally, and it is common in regions like Sub Saharan Africa, Spanish speaking regions in western Hemisphere like South America, the Caribbean, and Central America Saudi Arabi, India, Mediterranean countries such as Turkey, Greece, and Italy.

Approximately 300,000 babies are born each year with sickle cell anemia, with the majority of these cases occurring in Nigeria, the Democratic Republic of Congo, and India. In some African countries, up to 40 percent of the population carries the sickle cell trait and Every day, around 1,000 children in are born with sickle cell disease (SCD), and more than half of them will not survive past the age of five.

 Globally, the number of people living with sickle cell disease increased by 41.4, from 5.46 million in 2000 to 7.74 million in 2021. In 2021, it was estimated that there were 34,400 cause-specific deaths across all ages due to sickle cell disease. About 20 percent of children with SCD in India die by 2 years old. By 2050, the number of people with SCD will increase by 30 percent.

Various medical treatments are available in the market for the treatment of Sickle cell anaemia including Transfusions, Medications, Stem Cell Transplants, Gene Therapy. Key market players manufacturing Editas Medicine, Inc., Takeda Pharmaceutical Company Limited, Pfizer Inc., Spark Therapeutics Inc., X4 Pharmaceuticals Inc., CRISPR Therapeutics AG, Global Blood Therapeutics, Intellia Therapeutics, Inc., bluebird bio, Sangamo Therapeutics Inc., Translate Bio, Inc., Moderna, Inc., Vertex Pharmaceuticals Incorporated, Novartis Pharmaceuticals Corporation, Roche Holding AG.

April 2023, The FDA has approved Casgevy, a groundbreaking treatment for sickle cell disease, marking the first use of the gene-editing tool CRISPR in a therapy. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy edits the DNA in a patient's stem cells to remove the gene causing the disease, eliminating the need for a donor. This approval is significant for the over 100,000 Americans affected by the disease, predominantly Black individuals. However, the treatment's high cost of $2.2 million per patient raises concerns about accessibility.

Sickle Cell Disease Demographic and Environmental Risk Factors:

Age and Sex:  According to the Institute for Health Metrics And Evaluation, the global incidences of births of babies with SCD in 2021 was Estimated to be is 515,000 babies, sickle cell disease mortality rates were highest in the under-5 age group. For patients under age 5, SCD is said to be 12 th leading causing of death, with child mortality rates found to be 81,100 deaths in children with less than age of five in 2021. From 2015 to 2017, overall birth prevalence of SCD in U.S. was 1 in 2,024 newborns.

Ethnicity: In US 100,000 individuals are affected with SCD, 90% and more are found to be Hispanic Black or African American, and 3 to 9% are estimated to be Hispanic or Latino. SCD is found to be affecting 1 in every Black or African American Births and 1 in every 16,300 Hispanic American Births. Approximately 1 in 13 Black or African American infants is born with sickle cell trait (SCT), which means they inherit a sickle cell gene from one parent.

Risk Factors causing Sickle Cell Anaemia:  It is genetic disorder, therefore genetic inheritance is the major risk factor causing the Sickle cell Anemia, SCD occurs when child inherits two sickle cell genes, one from each parent. If both the parents are carriers, meaning they have sickle cell trait then each child has 25% chance of having SCD, 50% chance of being the carrier, 25% chance of being completely unaffected.

Sickle Cell Disease Market Scope:

Drivers: Advances in Gene Therapy

There is a great opportunity for gene therapy in relation to transforming therapeutic procedures used for sickle cell disease. Gene therapy is specifically aimed at the root of the disease the genetic level seeking to provide a potential fix, or long-term remission for individuals that have the disease. There are several gene therapy solution under development today and are currently in clinical trials. Some of these include gene replacement solutions such as CRISPR–Cas9.

Gene therapies effectively seek to correct or replace the faulty hemoglobin gene so as to allow for the production of healthy red blood cells. This therapy, if successful, will significantly increase the quality of life of patients and may eventually eradicate the need for support therapy such as blood transfusion for the rest of the life of the patient.

Restraints: High treatment cost, side effects of the available treatment, strict regulatory framework can cause delay in the product launch in the market, and non-compliance will not let the approval of the product, limited awareness about the disease are some of the hurdles which can hamper the growth of the market manufacturing the therapeutics for the treatment of Sickle Cell Disease.