This guest blog post was provided by Premier Research.
In our ongoing effort to gain more insight to the world of rare disease and orphan drugs, earlier this year, we commissioned an independently-conducted survey of clinical trial decision makers from 50 biotech and pharmaceutical firms in North America and Europe.
Some of the feedback was familiar and came as no surprise. For example, 69% of our respondents said that among the most difficult factors in recruiting patients into a rare disease clinical trial was not only finding and motivating patients to join and remain in trials, but identifying and setting up investigative sites for studies.
“Much of the success in patient recruitment is based on finding the appropriate sites for the small patient populations we have to work with,” Premier Research Executive Director, Clinical Trials Management, Angi Robinson said. “It’s worth noting that these studies become even more difficult to manage when they are conducted outside of western markets.”
An example of this was brought to life recently when we flew patients from the Middle East to an investigative site in the UK, which required oversight of countless details from arranging for transportation of patients and parents, to visa requirements, language issues, ethics committees and ongoing follow up with patients upon their return home.
Some of the feedback was surprising. We found that nine of 10 (88%) survey respondents said the number of patients they are required to enroll in a rare disease and orphan drug study is reasonable — a surprising finding given the inherently smaller universe of patients available to enter such a study and the respondents’ perception of the difficulty in finding qualified sites and recruiting patients.
Some of the feedback was downright gratifying. Perhaps the best finding from our study was that compared to traditional clinical trial populations, the vast majority (73%) of respondents indicated that working on rare disease/orphan drug trials was either “somewhat more rewarding” or “incredibly rewarding.”
“You can hardly overstate how gratifying it is to be a part of a team that develops a treatment for a rare medical condition, especially when it involves children, as many of these rare diseases do,” Robinson said. “Our fondest wish is to play a role in the effort that finds a cure for these illnesses.”
In the end, one could argue that working on rare diseases and orphan drugs are more complicated than most trials, but would further argue that complications are worth the reward. Would you agree?
Results of our latest survey covered several different issues, ranging from regulatory factors, adaptive design, to use of advocacy groups and even use of CROs.
To read more about what we learned, here’s our full Rare Disease and Orphan Drug study release.
We’re pleased to be a sponsor of the 4th Annual World Orphan Drug Congress, November 14-15, 2013. We’re currently developing our panel discussion, and would love to hear from you about what you’d like to see us cover with our Geneva panel. Let us know in the comments below, or send us an email at firstname.lastname@example.org.