Assessing Cumulative, Meaningful Benefits of Disease-Modifying Therapies Targeting Synucleinopathies: Conference Proceedings and Roadmap for Research.

This article summarizes the Workshop on Assessing Cumulative Benefits of Disease-Modifying Therapies (DMTs) Targeting Synucleinopathies, which was held in New York, N.Y., on November 18 and 19, 2024. This event was hosted by a coalition of nonprofit organizations and brought together representatives from academia, industry, and patient advocacy communities to discuss the cumulative, meaningful benefits of DMTs for synucleinopathies; to identify priorities for the field; and to explore opportunities for collaboration. Synucleinopathies, including Parkinson's disease, dementia with Lewy bodies, and multiple system atrophy, are neurodegenerative conditions that share underlying pathobiology and have many clinical similarities, including difficulties with movement, changes in cognition, sleep disturbances, and autonomic symptoms. Although there are some available symptomatic treatments for synucleinopathies, DMTs-medications that address the underlying biological processes of illness-are currently under development and might obtain regulatory approval in the near future. However, many other factors contribute to whether patients will have access to these novel therapies. Governments, private payers, clinicians, medical professional specialty societies, patients, and care partners think about meaningful benefits differently from regulators, who focus on safety and efficacy. The workshop attendees discussed these factors and considered next steps for understanding the benefits of DMTs for synucleinopathies.