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The movement toward prevention trials in people at-risk for Parkinson's disease (PD) is rapidly becoming a reality. The authors of this article include a genetically at-risk advocate with the LRRK2 G2019 S variant and two patients with rapid eye movement sleep behavior disorder (RBD), one of whom has now been diagnosed with PD. These authors participated as speakers, panelists, and moderators in the "Planning for Prevention of Parkinson's: A Trial Design Forum" hosted by Massachusetts General Hospital in 2021 and 2022. Other authors include a young onset person with Parkinson's (PwP) and retired family physician, an expert in patient engagement in Parkinson's, and early career and veteran movement disorders clinician researchers. Several themes emerged from the at-risk participant voice concerning the importance of early intervention, the legitimacy of their input in decision-making, and the desire for transparent communication and feedback throughout the entire research study process. Challenges and opportunities in the current environment include lack of awareness among primary care physicians and general neurologists about PD risk, legal and psychological implications of risk disclosure, limited return of individual research study results, and undefined engagement and integration of individuals at-risk into the broader Parkinson's community. Incorporating the perspectives of individuals at-risk as well as those living with PD at this early stage of prevention trial development is crucial to success. [ABSTRACT FROM AUTHOR]

Clinical studies test whether a new treatment is safe and if the treatment works in people who have a particular condition. Most current questionnaires used in clinical studies investigating Parkinson's measure symptoms in people who have been diagnosed for many years. This means that these existing questionnaires may not be useful for people living with early-stage Parkinson's, where the symptoms experienced can be quite different to later stages, or may not show if a new treatment is helpful for them. The most common symptoms in Parkinson's are involuntary shaking of parts of the body ("tremor"), slow movement ("bradykinesia") and stiff, inflexible muscles ("rigidity"), which worsen with time. Symptoms specific to early-stage Parkinson's are not fully understood and research is ongoing in this area. New measures are therefore needed to assess the symptoms affecting people living with early-stage Parkinson's, especially the symptoms that they find most troublesome. This study investigated which symptoms are of most importance to people in the earlier stages of their condition and which would be appropriate to measure in future clinical studies. The research team that led the study was made up of people living with Parkinson's, as well as technical experts and representatives from Parkinson's patient organizations (Parkinson's UK and the Parkinson's Foundation). The participants in the study were people living with early-stage Parkinson's and their care partners. Slowness of movement (called "bradykinesia") was noted as a key symptom. "Functional slowness" was especially noted. This symptom caused people to feel slower during many daily tasks, such as brushing teeth, walking and cooking. The loss of ability to move easily and freely, termed "mobility", was also a key symptom. It was noticeable in walking abnormalities and difficulties performing "fine motor skills". These are tasks that require precision, dexterity and coordination. Other impactful symptoms were: tremor, rigidity/stiffness, feelings of exhaustion (fatigue), depression, sleeping problems and pain. The personal views gathered in this study show the wide-ranging effects of early-stage Parkinson's. The study also identifies functional slowness and loss of mobility as key symptoms that would be appropriate to measure in future early-stage Parkinson's clinical studies to test if treatments are working or not. [ABSTRACT FROM AUTHOR]

The wide application of patient engagement and its associated benefits has increased across government, academic and pharmaceutical research. However, neither an identified standard practice for the process of engagement, nor utilization of common metrics to assess associated outcomes, exists. Parkinson's Foundation developed a patient engagement framework and metrics to assess engagement within the academic research and drug development sectors. This approach was developed over the course of several years through assessing the literature, acquiring feedback from researchers and people with Parkinson's disease and adapting practices to be relevant and generalizable across patient engagement projects. This framework includes the: 1) creation of a scope of work, 2) establishment of guiding principles, 3) selection and training of participants, 4) co‐determination of project metrics, 5) execution of the project and 6) dissemination of project findings. Parkinson's Foundation has also worked with academic, government and pharmaceutical stakeholders to identify metrics that assess both the quality of patient engagement and outcomes associated with patient engagement on projects. By improving patient engagement project methodologies and metrics, global clinical trials can have access to evidence‐based patient engagement practices to more efficiently capture the needs of, and potentially benefit, the patient community. [ABSTRACT FROM AUTHOR]