Risk willingness in multiple system atrophy and Parkinson’s disease understanding patient preferences

Disease-modifying therapeutics in the α-synucleinopathies multiple system atrophy (MSA) and Parkinson's Disease (PD) are in early phases of clinical testing. Involving patients' preferences including therapy-associated risk willingness in initial stages of therapy development has been increasingly pursued in regulatory approval processes. In our study with 49 MSA and 38 PD patients, therapy-associated risk willingness was quantified using validated standard gamble scenarios for varying severities of potential drug or surgical side effects. Demonstrating a non-gaussian distribution, risk willingness varied markedly within, and between groups. MSA patients accepted a median 1% risk [interquartile range: 0.001-25%] of sudden death for a 99% [interquartile range: 99.999-75%] chance of cure, while PD patients reported a median 0.055% risk [interquartile range: 0.001-5%]. Contrary to our hypothesis, a considerable proportion of MSA patients, despite their substantially impaired quality of life, were not willing to accept increased therapy-associated risks. Satisfaction with life situation, emotional, and nonmotor disease burden were associated with MSA patients' risk willingness in contrast to PD patients, for whom age, and disease duration were associated factors. An individual approach towards MSA and PD patients is crucial as direct inference from disease (stage) to therapy-associated risk willingness is not feasible. Such studies may be considered by regulatory agencies in their approval processes assisting with the weighting of safety aspects in a patient-centric manner. A systematic quantitative assessment of patients' risk willingness and associated features may assist physicians in conducting individual consultations with patients who have MSA or PD by facilitating communication of risks and benefits of a treatment option.

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Publication Details

Journal
npj Parkinson s Disease
Published
2024-08-15
DOI
https://doi.org/10.1038/s41531-024-00764-5
Primary Topic
Health Systems, Economic Evaluations, Quality of Life
Type
article
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article

Risk willingness in multiple system atrophy and Parkinson’s disease understanding patient preferences

Stephan Breimann, Isabel Friedrich, Emre Kocakavuk, Alexander Bernhardt et al.
npj Parkinson s Disease
Health Systems, Economic Evaluations, Quality of Life
article

Risk willingness in multiple system atrophy and Parkinson’s disease understanding patient preferences

Stephan Breimann, Isabel Friedrich, Emre Kocakavuk, Alexander Bernhardt, Tino Prell, Armin Giese, Malte Roderigo, Annette Janzen, Kevin Peikert, Tobias Warnecke, Eliana Nachman, Johannes Levin, Carla Palleis, Markus Weiler, Florin Gandor, Björn Falkenburger, Marc Oeller, Sabrina Katzdobler, Christoph van Riesen, Wolfgang H. Oertel, Lea O. Wilhelm, Günter U. Höglinger, Heike Jacobi, Johanna Nieweler, A. M. Hermann, Moritz Brandt, Inga Claus, Jost‐Julian Rumpf, Joseph Claßen, Tabea Schröter, Andreas Roßmann
article en

Abstract

Disease-modifying therapeutics in the α-synucleinopathies multiple system atrophy (MSA) and Parkinson's Disease (PD) are in early phases of clinical testing. Involving patients' preferences including therapy-associated risk willingness in initial stages of therapy development has been increasingly pursued in regulatory approval processes. In our study with 49 MSA and 38 PD patients, therapy-associated risk willingness was quantified using validated standard gamble scenarios for varying severities of potential drug or surgical side effects. Demonstrating a non-gaussian distribution, risk willingness varied markedly within, and between groups. MSA patients accepted a median 1% risk [interquartile range: 0.001-25%] of sudden death for a 99% [interquartile range: 99.999-75%] chance of cure, while PD patients reported a median 0.055% risk [interquartile range: 0.001-5%]. Contrary to our hypothesis, a considerable proportion of MSA patients, despite their substantially impaired quality of life, were not willing to accept increased therapy-associated risks. Satisfaction with life situation, emotional, and nonmotor disease burden were associated with MSA patients' risk willingness in contrast to PD patients, for whom age, and disease duration were associated factors. An individual approach towards MSA and PD patients is crucial as direct inference from disease (stage) to therapy-associated risk willingness is not feasible. Such studies may be considered by regulatory agencies in their approval processes assisting with the weighting of safety aspects in a patient-centric manner. A systematic quantitative assessment of patients' risk willingness and associated features may assist physicians in conducting individual consultations with patients who have MSA or PD by facilitating communication of risks and benefits of a treatment option.

npj Parkinson s DiseaseVol. 10(1)
Allen Institute for Brain Science (US), Philipps University of Marburg (DE), Heidelberg University (DE), German Center for Neurodegenerative Diseases (DE), University Hospital Heidelberg (DE), Helmholtz Zentrum München (DE), MSB Medical School Berlin (DE), Munich Cluster for Systems Neurology (DE), Universitätsmedizin Göttingen (DE), Neurologisches Fachkrankenhaus für Bewegungsstörungen/Parkinson (DE), Jena University Hospital (DE), Klinikum Osnabrück (DE), Max Planck Institute of Biochemistry (DE), University Hospital in Halle (DE), VIB-KU Leuven Center for Brain & Disease Research (BE), University of Rostock (DE), Technical University of Munich (DE), Technische Universität Dresden (DE), Ludwig-Maximilians-Universität München (DE), Leipzig University (DE), Otto-von-Guericke-Universität Magdeburg (DE)
Openalex Percentile: Top 84%
Health Systems, Economic Evaluations, Quality of Life
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