Mitigating the impact of study-start delays in clinical trials for rare disorders: insights and lessons from a PKAN trial

Our study indicates that the delay in the start of the clinical trial had a significant effect on all the researchers and on some PKAN participants, especially those who urgently wanted the research to start, due to the pressure of the severe and progressive nature of the disorder. For these participants, there was a sense of discontent with how the researchers communicated, which made them feel that the researchers had different interests. This study also revealed that researchers had different perceptions of what information was needed than what participants wanted. To lessen the impact of such delays on participants and researchers, we recommend both honest and transparent communication and adjusting communication to meet participants' needs. Close collaboration between participants, patient organisations and researchers can help achieve this goal.