Objective: To understand patient and caregiver preferences and perceptions of a proposed clinical trial design for an interventional drug targeting Multiple System Atrophy.
Background: Stakeholders in clinical trial design and drug regulatory bodies are increasingly noting the importance of incorporating patient and caregiver input into clinical trial design in order to increase accessibility of trials to a broader spectrum of patients. We conducted this project to understand both patient and caregiver preferences and perceptions to support the design of our proposed interventional drug clinical trial for patients with Multiple System Atrophy. Elements of patient and caregiver feedback from this project were incorporated into our study design in an effort to develop a more patient centric clinical trial, which may enhance the accessibility of MSA trials.
Method: This comparative project utilized data from qualitative interviews and a survey involving MSA patients and caregivers, carried out from October to December 2022. A comprehensive analysis was conducted to discern variances in preferences and perceptions between 49 participants of which 23 were patients and 26 were caregivers.
Results: This project highlights a wide range of patient and caregiver preferences in MSA clinical trial design. Universal preferences of burden reduction on patients and caregivers were noted as well as an understanding and tailoring of interventions, such as nurse to home visits to support the willingness of patients and caregivers to participate in and complete clinical trials. Differences in caregiver and patient preferences were also noted in some clinical trial aspects such as differential ranking of benefit and burden of participation in clinical trials and different disease management preferences.
Conclusion: Recognizing the distinct perspectives of patients and caregivers in MSA management is crucial. This understanding enables the development of more nuanced, effective care strategies in clinical trials, ensuring that both patients’ and caregivers’ voices are heard and implemented.
To cite this abstract in AMA style:
J. Marer, G. Krog, D. Houghton, J. Nettleship, M. Chen, L. Marinelli. Incorporating the Voice of the Patient and Caregiver into a Multiple System Atrophy (MSA) clinical trial [abstract]. Mov Disord. 2024; 39 (suppl 1). https://www.mdsabstracts.org/abstract/incorporating-the-voice-of-the-patient-and-caregiver-into-a-multiple-system-atrophy-msa-clinical-trial/. Accessed June 15, 2025.« Back to 2024 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/incorporating-the-voice-of-the-patient-and-caregiver-into-a-multiple-system-atrophy-msa-clinical-trial/