Patients’ Preferences for Adjunctive Parkinson’s Disease Treatments: A Discrete-Choice Experiment
Bibliographic record
Abstract
Michael Serbin,1 Connie Marras,2 Carol Mansfield,3 Colton Leach,3 Charles Yonan,1 Margaret Sheehan,4 Anne Donnelly,5 Olga Klepitskaya1 1Neurocrine Biosciences, Inc., San Diego, CA, USA; 2The Edmond J. Safra Program in Parkinson’s Disease, University Health Network, University of Toronto, Toronto, ON, Canada; 3RTI Health Solutions, Research Triangle Park, NC, USA; 4Ashurst, Washington, DC, USA; 5Kellogg School of Management, Northwestern University, Evanston, IL, USACorrespondence: Carol Mansfield, RTI Health Solutions, 3040 East Cornwallis Road, PO Box 12194, Research Triangle Park, NC, 27709, USA, Tel +19195418053, Email carolm@rti.orgBackground: Several adjunctive medications are available to reduce OFF time between levodopa/carbidopa (LD/CD) doses for people with Parkinson’s disease (PD).Objective: To explore how individuals with PD balance benefits and burdens when considering adjunctive medications.Methods: US adults (30– 83 years) with self-reported PD, currently treated with LD/CD, who experienced OFF episodes were recruited through the Fox Insight study to complete a discrete-choice experiment survey. Respondents selected among experimentally designed profiles for hypothetical adjunctive PD treatments that varied in efficacy (additional ON time), potential adverse effects (troublesome dyskinesia, risk of diarrhea, risk of change in bodily fluid color), and dosing frequency or the option “No additional medicine”. Data were analyzed with random-parameters logit models.Results: Respondents (N=480) would require ≥ 60 additional minutes of daily ON time to accept either a 40% risk of change in bodily fluid color or 10 additional minutes with troublesome dyskinesia daily. Respondents would require 40 additional minutes of daily ON time to accept a 10% risk of diarrhea and 22 additional minutes of daily ON time to switch from 1 additional pill each day to 1 pill with each LD/CD dose. On average, respondents preferred adjunctive PD medication over no additional medication. Results predicted that 59.1% of respondents would select a hypothetical treatment profile similar to opicapone, followed by no additional medication (27.5%) and a hypothetical treatment profile similar to entacapone (13.4%).Limitations: The data collected were based on responses to hypothetical choice profiles in the survey questions. The attributes and levels selected for this study were intended to reflect the characteristics of opicapone and entacapone; attributes associated with other adjunctive therapies were not evaluated.Conclusion: Patients with PD expressed interest in adjunctive treatment to increase ON time and would accept reduced ON time to avoid adverse effects.Plain Language Summary: People with Parkinson’s disease may experience OFF time, when their symptoms return between doses of levodopa/carbidopa. Some medicines can reduce OFF time when taken in addition to levodopa/carbidopa. These additional medicines have their own benefits and side effects. In this study, researchers used a survey to understand what side effects people with Parkinson’s disease would accept to have more ON time. Respondents were presented with a series of choices between 2 hypothetical medicines that increased daily ON time or no additional medicine. The medicines were described by 5 features (benefits and side effects). Respondents’ answers to the survey questions allowed researchers to estimate which unwanted side effects people with Parkinson’s disease would accept in exchange for additional ON time. The most important features of a medicine to reduce OFF time, in order, were (1) Lower risk of a change in urine, sweat, or saliva color; (2) Fewer additional minutes with troublesome dyskinesia each day; and (3) Increase in daily ON time. Introducing a 40% risk of change in urine, sweat, or saliva color (from no risk) would require more than 60 minutes of additional daily ON time to offset. An additional 10 minutes of troublesome dyskinesia each day would require more than 60 minutes of additional daily ON time. Introducing a 10% risk of diarrhea (from no risk) would require 40 minutes of additional ON time. Individuals’ preferences for the key features of medications that reduce OFF time for people with Parkinson’s disease should be considered when making treatment decisions.Keywords: discrete choice, Parkinson’s, stated preferences
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.003 |
| Open science | 0.003 | 0.001 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.010 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".