Forecasts for the Attainment of Major Research Milestones in Parkinson's Disease.

Autor: Kane PB; Biomedical Ethics Unit, STREAM Research Group, McGill University, Montreal, QC, Canada., Benjamin DM; University of Southern California, Information Sciences Institute, Marina del Rey, CA, USA., Barker RA; Department of Clinical Neuroscience, John van Geest Centre for Brain Repair, WT/MRC Cambridge Stem Cell Institute, University of Cambridge, Cambridge, UK., Lang AE; Edmond J. Safra Program in Parkinson's Disease and the Morton and Gloria Shulman Movement Disorders Clinic, Toronto Western Hospital, Toronto, ON, Canada., Sherer T; The Michael J. Fox Foundation for Parkinson's Research, New York, NY, USA., Kimmelman J; Biomedical Ethics Unit, STREAM Research Group, McGill University, Montreal, QC, Canada.
Jazyk: angličtina
Zdroj: Journal of Parkinson's disease [J Parkinsons Dis] 2020; Vol. 10 (3), pp. 1047-1055.
DOI: 10.3233/JPD-201933
Abstrakt: Background: Projections about when research milestones will be attained are often of interest to patients and can help inform decisions about research funding and health system planning.
Objective: To collect aggregated expert forecasts on the attainment of 11 major research milestones in Parkinson's disease (PD).
Methods: Experts were asked to provide predictions about the attainment of 11 milestones in PD research in an online survey. PD experts were identified from: 1) The Michael J. Fox Foundation for Parkinson's Research data base, 2) doctors specializing in PD at top ranked neurology centers in the US and Canada, and 3) corresponding authors of articles on PD in top medical journals. Judgments were aggregated using coherence weighting. We tested the relationship between demographic variables and individual judgments using a linear regression.
Results: 249 PD experts completed the survey. In the aggregate, experts believed that new treatments like gene therapy for monogenic PD, immunotherapy and cell therapy had 56.1%, 59.7%, and 66.6% probability, respectively of progressing in the clinical approval process within the next 10 years. Milestones involving existing management approaches, like the approval of a deep brain stimulation device or a body worn sensor had 78.4% and 82.2% probability of occurring within the next 10 years. Demographic factors were unable to explain deviations from the aggregate forecast (R2 = 0.029).
Conclusions: Aggregated expert opinion suggests that milestones for the advancement of new treatment options for PD are still many years away. However, other improvements in PD diagnosis and management are believed to be near at hand.
Databáze: MEDLINE
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