According to an article at HealthEuropa, including patients and their families as an important component of cystic fibrosis research is a requirement when developing future treatments for the disease. That’s because patients and their families, the people living closest to the disorder, can provide valuable insight into clinical trial design.
About Cystic Fibrosis
Cystic fibrosis (CF) is a genetically-inherited condition that most commonly causes damage to the lungs and digestive system, though other organs can also be affected.
The damage is caused by mucus naturally produced by many of the body’s cells. In healthy individuals, these fluids are typically watery and slick, providing a light layer of protection and lubrication. In individuals with cystic fibrosis, the cells that product this mucus instead secrete a thick and sticky mucus that can clog ducts and other passages in major organs.
Management of the condition requires daily care. However, many people with CF can live into their 30s, 40s, or even 50s thanks to improvements in medical technology.
Patients in research
The number of advances in cystic fibrosis research have made life with the disease in recent years easier. One of these breakthroughs was the development of CFTR-modulators, small molecule drugs that are able to increase the activity of CFTR proteins. These proteins act as transport channels in the membranes of mucus-producing cells. Regularly behaving CFTR proteins are essential for the body to produce slick, lubricating mucus.
The development of CFTR-modulators represented a significant shift in the treatment of cystic fibrosis, in that it seeks to treat the underlying cause of the disease rather than the symptoms. A number of different new CFTR modulators are in development, and the field is a brave new world for cystic fibrosis research.
The discovery of new treatments like CFTR modulators is only possible with the input of patients and their families, according to advocacy group CF Europe – an alliance of 39 European national cystic fibrosis associations. That’s because patients can provide valuable insight to aid researchers in designing clinical trials. What seems practical and manageable to a scientist may be totally impractical and unmanageable to a cystic fibrosis patient. For example, patients responding to a questionnaire distributed by CF Europe pointed out that they could not participate in trials requiring frequent trips to the hospital (sometimes as often as every two days). Such practical considerations might be overlooked by research professionals.
Simply put, by being more considerate to the needs of the patients, researchers could improve clinical trial enrollment and retention rates – and thereby, likely, improve clinical trial success rates.
Do you think a greater emphasis should be placed on the patient experience in clinical trials? Do you think too much emphasis is placed on the researcher end? Why or why not? Share your thoughts with Patient Worthy!
