Abstract
BACKGROUND: Rare disease clinical trials are notorious for complexities that frequently result in study-start delays. However, there is limited knowledge about how participants and researchers perceive these delays and what factors shape their experiences. A clinical trial in the rare, progressive disorder Pantothenate Kinase-Associated Neurodegeneration-the PKAN trial-encountered an unexpected delay. As this delay caused noticeable challenges for both PKAN participants and researchers, we sought to unravel their experiences in order to mitigate the impact of a study-start delay in future trials. RESULTS: Fourteen semi-structured qualitative interviews were performed with PKAN participants (n = 9), represented by their caregivers or patient organisations, and researchers (n = 5) involved in the PKAN trial. During the delay, worries arose among some participants, which were directed towards the researchers. These participants expressed desperation to be included in the study in the early stages of the disease and held overly optimistic expectations for the PKAN trial. However, most participants did not experience a significant impact of the delay on their lives. On their side, the researchers mentioned the challenge of managing expectations while preserving hope. Most participants were satisfied with the communication; however, some participants highlighted concerns regarding the lack of transparency. Different interests between participants and researchers came to light during the delay. Some researchers advised providing background information on clinical trials, whereas all participants indicated that they did not need this information. CONCLUSIONS: 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.