CRO publishes survey on challenges of rare disease trials

Contract research organisation Premier Research has published the results of a survey of decision-makers in 50 pharma and biotech firms, which looks at the challenges of conducting clinical trials in rare and orphan diseases.

The survey highlighted that identifying and starting up appropriate trial sites is still a major challenge in this area of research. Advocacy groups and medical networks play a substantial role, with advocacy groups being used by 60% of the respondents to assist with their site or patient recruitment, 42% of them saying that the use of advocacy groups to meet patient recruitment goals was “successful” or “vital to the program.”

In addition, 43% of respondents noted that securing regulatory approval for the design of their clinical study was “difficult” or “extremely challenging.” However, while it might have been expected that adaptive methodologies might have been prevalent to maximise the information obtained from each participant, their use in rare disease research roughly mirrors levels in general clinical development.