Epidemiology Study - Overview

Cystic Fibrosis Study Objective:

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

Cystic Fibrosis Study USP:

• This epidemiological study on Cystic Fibrosis 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 Cystic Fibrosis related health problems.

• Through meticulous data collection and rigorous methodology, the study not only contributes to the scientific understanding of Cystic Fibrosis but also serves as a foundation for future research events and healthcare policymaking in addressing this increasing Cystic Fibrosis cases.

Cystic Fibrosis related Study Overview:

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

Cystic Fibrosis 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 Cystic Fibrosis cases (e.g., clinical examination, imaging studies) and demographic data (age, sex, ethnicity).

Cystic Fibrosis 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 Cystic Fibrosis and potential risk factors (e.g., age, genetics).

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

Cystic Fibrosis study summary:

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

Cystic Fibrosis Disease overview:

It is a genetic disorder, affecting children and adults, primarily affecting lungs and even showing its impact on digestive and reproductive system, characterised by chronic lung infections, pancreatic insufficiency, sinusitis, and infertility, it is the most common cause of death in patients having last stage lung cancer, resulting in shorter lifespan of patient.

Mainly it is caused due to possible 2000 mutations of CFTR gene, which leads to disruption of chloride ion support, causing mucus accumulation, dysfunction of organs and imbalances in electrolyte. Disease symptoms are seen in infants, children, adults, and diagnosed using newborn screening and genetic testing.

In US Cystic Fibrosis incidence is estimated to be 1000 cases occurring annually, same rates are seen in UK and Canada. one in every 2000- 3000 infants in European Union is seen to be diagnosed with cystic fibrosis, many researchers from different countries report that incidence of cystic fibrosis is seen to be decreasing due to implementation of screening of newborn babies. Higher incidence of CF is seen in Mid-Eastern Countries than East Asia, one in 2560.2 incidence is reported in Jordan, in Japan incidence is one in every 350,000 births. Incidence rates in India are seen from 1 in 10,000 to 1 in 100,000 people. The average incidence in Latin and South America is 1 in every 8000 individuals, ranging from 1 in 6100 in Argentina to 1 in 15,000 in Costa Rica.

Various treatment options are available for cystic fibrosis including, Pancreatic Enzyme Supplements, Mucolytic Bronchodilators, Antibiotics, CFTR modulators. Key market players involved in manufacturing the therapeutics for the treatment of cystic fibrosis. Allergan PLC, Chiesi Farmaceutici SpA, AbbVie, Hoffmann-La Roche AG, Gilead Sciences, PTC Therapeutics, Novartis AG, Vertex Pharmaceuticals Incorporated, Pharmaxis Ltd.

Cystic Fibrosis Industry Developments

October 2020: For the treatment of patients with cystic fibrosis regardless of CFTR genotype and other respiratory disorders, Roche announced the partnership with AbbVie and the acquisition of the TMEM16A potentiators portfolio.

April 2019: The KALYDECO (ivacaftor) medication from Vertex Pharmaceuticals Incorporated has been approved by the US Food and Drug Administration (FDA) in an effort to increase the number of treatment choices for the millions of youngsters who have cystic fibrosis (CF). With the potential to alter the course of the disease, this enables doctors to start treating the underlying cause of CF in eligible infants as young as six months of age

Cystic Fibrosis Demographic and Environmental Risk Factors:

Age and Sex: It affects equally to both male and female, but females having CF exhibit short life expectancy compared to males, due to early respiratory infection onset, female patients diagnosed with CF have less survival chances after 40 years of age, causing disparities in age related prevalence of CF. 75% of diagnosis of cystic fibrosis in US is seen to occur in 2 years age child age group, this diagnosis is possible due to implementation of newborn screening. 90% patients with CF with more than 40 years were diagnosed with the disease before 10 years of their age.

Ethnicity: Incidence rate of 1 in 3500 of cystic fibrosis is observed in Northern Europe, and 1 in 15000 is seen in black patients, lower rates of prevalence is seen in Asia with 1: 30000 ratio. [delta]F508 in exon 11 is the most common mutation found in 70% white patients of cystic fibrosis in US of all 2/3^rd cases globally.

Risk Factors causing Cystic Fibrosis: Major risk factor of cystic fibrosis is mutation in gene on chromosome 7, coding for cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is functions as transmembrane cAMP-activated chloride channel. 2000 different mutations in CFTR gene are found out causing CF and this mutations are divided into 5 different classes which include as follows:

Class I: Defect in protein synthesis

Class II: Defect in protein processing

Class III: Disordered regulation

Class IV: Defect in conductance of chloride

Class V: channel turnover accelerated

Cystic Fibrosis Market Scope:

Drivers: Growing cases of cystic fibrosis to propel market growth-In all parts of the world, CF is swiftly overtaking other hereditary diseases in terms of prevalence. More than 30,000 people in the United States currently have cystic fibrosis, and it is anticipated that 70,000 people worldwide have this disorder, according to the Cystic Fibrosis Patient Registry. It is also mentioned that the illness is diagnosed in about 1,000 new cases annually. Therefore, it is projected that there will be an increase in demand for the treatment of the condition throughout the course of the projection period due to the rising prevalence of respiratory problems, gastrointestinal problems, and reproductive illnesses in CF patients. The adoption of novel treatment approaches for early detection is also increasing the patient base for improved treatment, which is anticipated to affect the market's growth throughout the anticipated period. Thus, this factor is driving the market CAGR.

Furthermore, cystic fibrosis sufferers are becoming more prevalent worldwide, which has sparked the industry. Pharmacotherapy for cystic fibrosis has led to many advancements in managing this condition, helping the market grow. The industry's rise is also fueled by market trends that are now being used in treating the ailment. This is due to a growing number of candidates for therapeutic care are being produced by important corporations and are currently in the pipeline. As a result, pharmaceutical firms are being pushed to create novel therapies that deal with CFTR protein abnormalities. Additionally, the launch of Lumacaftor/Ivacaftor, a potentiator that improves the quality of life for patients with the disease, also assists in the market's growth.

However, the market is expanding due to the impending introduction of medications for treating CF. During the anticipated period, the market expansion will be aided by introducing medications that target the fault in diseased people's cells. The market expansion is being influenced by the likelihood that this medicine will change many people's lives. The market is also thought to be primarily driven by an increase in the disease's prevalence.

Restraints: limited access to latest advanced therapies for the treatment of cystic fibrosis, especially in in developing areas, furthermore, strict regulatory requirements for getting approval from the authorised regulatory bodies, possible side effects of the therapies available currently, are some of the factors hampering the growth of the cystic fibrosis market.