Cystic fibrosis (CF) is a genetic disorder caused by mutations in the CFTR gene, affecting chloride and bicarbonate transport in cells. This leads to thick mucus secretions in organs like the lungs and pancreas, causing blockages and health complications in the respiratory and digestive systems. Before the 1980s, many CF patients didn’t survive past their 20s. Recent advancements have gradually raised life expectancy, with a projected 75% increase in adult cystic fibrosis population by 2025. 

The worldwide prevalence of cystic fibrosis  

CF impacts an estimated 162,400 individuals worldwide, with a predominant occurrence among the White population. However, recent evidence indicates its presence in regions like the Middle East, Asia, and Latin America, although at lower rates. In the North American region, the US leads in cystic fibrosis cases, while Canada shows a higher prevalence rate per CF patient population. Europe sees variation, with the UK leading in prevalence, while Belgium and Switzerland show high rates despite lower CF patient populations. 

 In the Asia-Pacific, Australia and New Zealand report a significant number of cystic fibrosis cases, demanding strong healthcare initiatives. The rest of the world exhibits disparities, with Brazil leading in cystic fibrosis population and South Africa and Egypt following next, although with a moderate number of cases. These findings emphasise the importance of personalised approaches and strong healthcare initiatives to tackle cystic fibrosis on a global scale. 

The global clinical trials landscape 

Since 2018, the biotech and biopharma industry has initiated over 450 CF trials. Europe and North America have conducted the most trials, followed by Asia Pacific, while the rest of the world contributes moderately. The US leads in North America, with the UK and Germany prominent in Europe. Significant research efforts are also seen in Australia in the Asia-Pacific region, and Israel in the Middle East. When it comes to patient recruitment trends, Europe shows shorter recruitment durations and faster patient recruitment rates in comparison to the US and Asia-Pacific. 

Emerging treatments 

While cystic fibrosis lacks a cure, modern treatments prolong lives through comprehensive management, including nutritional support, airway clearance techniques, and targeted drugs such as CFTR modulators. CFTR modulators enhance lung function by targeting the defective CFTR protein, though their effectiveness depends on gene mutations. They have transformed cystic fibrosis treatment by tackling the root cause of the disease. 

The cystic fibrosis treatment market offers diverse products from companies like Advanz Pharma Corp. Ltd., Novartis AG, Pari GmbH, Pharmaxis Ltd., and Vertex Pharmaceuticals Inc., covering small molecules, enzymes, biologics, and stem cells. Vertex Pharmaceuticals Inc. is conducting Phase III trials for small molecule therapies targeting CFTR protein, reflecting ongoing efforts to improve cystic fibrosis treatment. This and several other research and development initiatives represent a comprehensive approach by the companies involved in their pursuit to address cystic fibrosis treatment complexities and enhance patient outcomes. 

In conclusion, cystic fibrosis remains a global health challenge, but recent treatment advances have improved patient outcomes compared to the previous years. Disparities in global prevalence rates and healthcare infrastructure highlight the need for tailored approaches and strong healthcare initiatives. The increase in cystic fibrosis clinical trials and diverse therapeutic options reflects ongoing efforts to address cystic fibrosis complexities and enhance patient well-being. Collaboration among researchers, healthcare providers, and pharmaceutical companies holds promise for further progress in its management and care.  

About Novotech 

Rare disease clinical studies require a full-service CRO with expertise in patient enrollment, site selection, access to patient registries, and comprehensive global regulatory knowledge for rare and orphan disease trials. 

Novotech, a global CRO, has extensive experience in rare disease studies, including cystic fibrosis, Alport syndrome, and Duchenne muscular dystrophy. With numerous trials in rare disease indications, Novotech is dedicated to advancing biotech research and treatments. Discover more about cystic fibrosis research by downloading the comprehensive disease report below.