How innovative approaches are changing the way patients are recruited into rare disease trials
As every bio pharma company knows, rare disease trials may be among the most difficult projects to plan and deliver. The patient populations are small and scattered and some may be located in pockets in remote areas or regions where clinical research is not traditionally conducted and others are only identified after sophisticated molecular testing. Additionally, some rare conditions have a limited number of experts, often little to no peer reviewed research and a virtual absence of key opinion leaders to access for guidance and advice. This all creates a trial environment that is fraught with uncertainty and a risk of failure to meet objectives.
Thinking differently for rare disease research
Often the first thing parents and families do when a diagnosis is made is head to the internet. Harnessing this powerful tool is really at the center of good strategy from trial concept to recruitment. The best way to approach a clinical trial is to first understand who the thought leaders are, where patients go for information, and what their key concerns are about treatment and trial participation. All of this information can directly inform trial protocol development and efforts to recruit patients. Social listening addresses all of these issues, enabling researchers to gather valuable data in a matter of days.
Whitepaper written by Dr. Cynthia Jackson, Vice president and head of the Pediatric Center of Excellence and of the Rare Diseases Platform, Quintiles