As a complex, inherited disease, cystic fibrosis impacts the respiratory, gastrointestinal and reproductive systems. Many clinical trials are examining therapies that target this rare disease from different therapeutic angles, such as restoring function to a faulty protein, reducing inflammation, clearing mucus buildup, addressing gastrointestinal manifestations or fighting cystic fibrosis-related infections.
We are currently supporting several complete development programs and ongoing cystic fibrosis studies from Phase I through IV. Christine Tiesler, senior director, inflammation, infectious disease and general medicine (iiGM), and Karina Putineanu, medical director, iiGM, recently shared their insights on how a united effort between sponsors, CROs and sites can help advance cystic fibrosis clinical trials on a global scale.
Working with well-informed patients
With 75,000 known cases around the world and 30,000 of those in the U.S., cystic fibrosis is considered a rare disease even though it is the most common genetic disease in Caucasians. Most people with cystic fibrosis were diagnosed by the age of two and screening programs often identified the disease shortly after birth.
Despite being a relatively small patient population, people with cystic fibrosis are very informed about their options for receiving standard treatments and joining clinical trials, thanks to widespread availability of cystic fibrosis-related information from a strong network of cystic fibrosis foundations in the U.S., Europe and Australia.
Accepting valuable input
While cystic fibrosis foundations provide guidance to patients on their available treatment choices, they can support pharmaceutical drug development and CROs by providing input on clinical trial protocols.
Cystic fibrosis foundations also advise on the rigorous standards of study conduct. Our clinical research associates (CRAs) and key project management functions are trained according to guidance provided by cystic fibrosis foundations. This training helps ensure that CRAs understand the challenges, can provide sufficient monitoring and ultimately fulfill many unique requirements required to run these specialized clinical studies.
Established drug development companies working in cystic fibrosis often have close cooperation and long-standing relationships with sites and key opinion leaders (KOLs). Sponsors that are new to the space will have to build this network or rely on their CRO partner to facilitate these connections.
The cystic fibrosis foundations also give crucial feedback about the CRO’s performance at the site through established communication channels. This ongoing sharing of knowledge minimizes critical issues, helps maintain close cooperation between the sites, CRO and sponsor and helps ensure that the CRO keeps pace in the rapidly changing landscape.
Easing the burden on sites
Given the complexity of how cystic fibrosis affects many systems in the body and patient care cannot be handled with a single provider, an interdisciplinary team is required at each clinical site, which often can include a dietician, respiratory specialist and psychologist.
CROs supporting cystic fibrosis clinical trials must strive to make the entire process very straightforward for a site to coordinate many aspects of the delivery. This level of service is especially important in a very competitive field where many companies are seeking experienced CF sites.
Beyond developing a strong working relationship with sites, we leverage our extensive site relationships within this specialized cystic fibrosis network, we and can open up potential opportunities to secure these locations for the studies we conduct on behalf of our clients.
Applying proven experience
Making gains in cystic fibrosis clinical trials requires comprehensive solutions, from processing lab results and spirometry readings to nurturing longstanding relationships between sponsors, CROs, labs and foundations. These alliances can make a difference in running efficient studies and addressing the urgent need to find viable treatments to improve the lives of people with cystic fibrosis.
To make the most of your cystic fibrosis drug development, take advantage of our solutions and expertise. From central laboratory testing with sweat chloride and spirometry to our full range of CRO service such as genotyping and comprehensive monitoring solutions through the Xcellerate® Informatics Suite, we are here to help advance your studies.
Reference: Cystic Fibrosis Foundation