Novotech Publishes Analyst Report on Cystic Fibrosis Clinical Trial Landscape to Inform Biotech Research Planning 

Boston, USA - Novotech, the global full-service clinical Contract Research Organization (CRO) that partners with biotech companies to accelerate the development of advanced and novel therapeutics at every phase, has released a comprehensive research report entitled Cystic Fibrosis - Global Clinical Trial Landscape. 

The report presents a data-backed review of the Cystic Fibrosis (CF) funding landscape, trial density and patient recruitment data across geographies, standard of care and emerging therapies, recent USFDA approvals, and regulatory trends. Importantly it also includes an in-depth SWOT analysis, to guide biotech firms on strategic decision-making, and research opportunities, and challenges. 

Globally, 162,430 individuals are estimated to be living with CF. 

CF is a hereditary autosomal recessive condition caused by CFTR (Cystic Fibrosis transmembrane regulator) gene mutations, disrupting chloride and bicarbonate transport in epithelial cells. This impairs the function of epithelial cells in the lungs, pancreas, and other organs which cause significant health issues. 

The Cystic Fibrosis - Global Clinical Trial Landscape report found that before the 1980s, half of CF patients didn't survive beyond their 20s, but recent advancements in treatment have significantly increased life expectancy, with a projected 75% rise in the adult CF population by 2025. 

CF demonstrates varying prevalence rates globally: 

• In North America, the US leads with the highest CF patient population at 31,199 individuals, or an estimated CF prevalence rate of 0.95 per 10,000.  
• Canada's smaller CF patient population of 4,344 has a higher prevalence rate of 1.17 per 10,000, indicating a greater burden per capita than the US.  
• In Europe, the UK has the highest estimated CF prevalence rate, at 1.61 per 10,000 people, and the second-largest CF patient population of 10,509 people, after the US. globally.  
• Other European countries with high CF patient populations include France, Germany, and Italy.  
• Belgium and Switzerland have high estimated prevalence rates of 1.13 and 1.18 per 10,000, respectively.  
• Within the Asia-Pacific region, Australia stands out with a significant CF patient population of 3,151 individuals. It had reported the second-highest prevalence rate (1.27 per 10,000 people), after the UK. 
• New Zealand follows closely with a prevalence rate of 1.04 per 10,000 individuals, despite its smaller CF patient population.  
• In the Rest of World (ROW), Brazil has the highest CF patient population at 6,240 individuals, with an estimated prevalence rate of 0.29 per 10,000. 

Currently there is no cure for CF, but this report highlights the wide-ranging and emerging treatments to support patients’ longevity and quality of life, such as nutritional support, airway clearance techniques, and additional drugs targeted at improving the function of the faulty CFTR protein and preventing possible consequences. In certain cases, respiratory support, or surgical interventions may be necessary. 

Since 2018, the biotech and the biopharma industry initiated over 450 CF clinical trials worldwide with the following breakdown: 

• Europe and North America leading at 37% and 35% respectively 
• Followed by Asia Pacific at 23% 
• While the Rest of World (ROW) contributed a moderate share with 5% 

The report noted that “In Europe, the United Kingdom and Germany led in CF trials, contributing 13% and 12% of trials respectively, highlighting the region's efforts and advancements in this area. In North America, the United States held the majority trial share with 68%, while Canada held 32%. Within Asia-Pacific, Australia held the highest trial share (64%), followed by New Zealand at 21%. Among the ROW countries, Israel led while Iran contributed a moderate share”. 

Further key takeaways from the report include: 
 

• The US experienced a significant influx of venture capital, with investments totalling $919.1 million. Countries like the Netherlands, Israel, and Italy also contributed to venture funding, albeit to a lesser extent. 
• Like many conditions precision medicine has seen growth in CF treatments including identifying precise gene mutations, drugs aiming to rectify the function of the defective protein due to the mutation, gene therapy substitutions.  
• There are more than 30 CF drugs in preclinical stages, 18 in Phase I trials, and 10 in combined Phase I/II studies.  
• Increased funding, emerging therapies and personalised medicine were noted as some of the key opportunities. 
• Regulatory hurdles, ethical concerns, and antibiotic-resistant bacteria were identified as threats. 

The Novotech research analyst team provides these expert reports on a monthly basis, completely free of charge. These reports offer current insights into global clinical trial activity, revealing which regions experience the highest trial volumes and the unique factors behind these trends. They tackle the potential and real hurdles faced by biotech firms in specific therapeutic areas in the hopes to positively impact and inform clinical trial decision making, eventually improving rates of success with new treatments.   

Download the report here