Caregivers' and Physicians' Treatment Preference in Parkinson Patients Treated With Neupro® Requiring Caregiver Support: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Caregivers' and Physicians' Treatment Preference in Parkinson Patients Treated With Neupro® Requiring Caregiver Support (NCT01330290), a registered phase not stated study. The primary outcome is Mean Score of the Caregivers' Rating of Neupro® Compared to Oral Anti-Parkinson Medication.

2. Clinical Question

The study’s stated objective was to evaluate advantages and disadvantages of Neupro® versus oral anti-Parkinson medication from caregivers’ and physicians’ perspectives. The focus was on people with idiopathic Parkinson’s disease who needed caregiver support, rather than on a randomized treatment comparison.

The primary endpoint was the mean score of caregivers’ ratings of Neupro® compared with oral anti-Parkinson medication. The questionnaire covered caregiving aspects, and the registry describes the scores as reflecting perceived disadvantages or advantages. Physicians’ ratings and their rationale for choosing Neupro® were addressed through secondary outcomes, including ratings related to dysphagia, nausea and/or vomiting, compliance, multiple medication, surgery requiring general anaesthesia, and dose adaptation.

This question concerns reported perceptions in the study setting. It does not, by itself, estimate a causal treatment effect or establish that one route of administration is superior. The registry record does not report the primary mean score, its standard deviation, or the corresponding result for physicians’ ratings.

3. Trial Design

This was a multi-site, non-interventional, cross-sectional evaluation. The registry lists the study as completed, with a start date of 2011-03 and primary completion in 2012-08. Enrollment was 148. The condition listed was idiopathic Parkinson’s disease, and UCB Pharma was the sponsor.

Because the study was non-interventional, Neupro® use was routine treatment rather than a treatment assigned by the study. The registry describes treatment according to the approved European Union label or local marketing authorization. It lists a Neupro® Treatment group and does not describe a randomized allocation. The allocation method is not stated, and the record explicitly indicates that this is not a randomized comparison. The group therefore should not be read as one arm of a like-for-like controlled trial.

The cross-sectional assessment used a questionnaire completed at a single time-point during the run of the study, described as 16 months. For the primary endpoint, suitable patients had idiopathic Parkinson’s disease and had been treated with a combination of l-dopa or another oral idiopathic Parkinson’s disease drug and Neupro® for at least one month. Caregivers rated Neupro® in comparison with oral anti-Parkinson medication. Physicians also provided ratings and reasons for choosing Neupro®.

These features mean that the study records perspectives within an existing care context rather than comparing outcomes after prospectively assigned treatments. It can describe the ratings collected from caregivers and physicians, but the design does not separate treatment-related differences from other factors that may influence treatment choice or questionnaire responses. The registry record does not report a protocol for controlling such factors, a randomized comparator group, or a statistical analysis plan.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Before assignment: The physicians were instructed to select idiopathic Parkinson's Disease (iPD) patients with documented need of care with caregivers/nurses who attended patients at home as caregiving family members or as professional nurses in outpatient care or nursing homes.

Overall Study

Analysis population for the primary outcome, as defined in the registry: The Analysis Population refers to the Full Analysis Set (FAS). The Full Analysis Set (FAS) comprises all patients with at least 1 assessment in the physicians' or caregivers'/nurses' questionnaires. The average assessment of all patients assessed by a distinct caregiver was calculated and used for analysis.

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

Population: The Baseline Characteristics refer to the Enrolled Set (ES). All patients who were entered into the database are included in the Enrolled Set (ES).

6. Primary Endpoint

The registry lists the primary outcome as Mean Score of the Caregivers' Rating of Neupro® Compared to Oral Anti-Parkinson Medication, measured over Questionnaire completed at a single time-point during the run of the cross-sectional study (16 months). Suitable patients suffer from idiopathic iPD treated with a combination of l-dopa or another oral iPD drug and Neupro® for at least one month..

The primary endpoint was the mean score of caregivers’ ratings of Neupro® compared with oral anti-Parkinson medication. Caregivers completed a questionnaire covering caregiving aspects through 7 questions. Responses were rated from great disadvantages to great advantages on a 5-point scale scored from -2 to +2. The endpoint is therefore a continuous measure of caregiver-reported comparative assessments, not a clinical measure of disease severity or a direct measure of treatment efficacy.

The registry states that scores from each caregiver were averaged over all participants that caregiver assessed. The final mean was then calculated from the caregivers’ averaged assessments. This approach makes the caregiver the unit contributing an averaged assessment to the final mean, while allowing a caregiver to have assessed one or multiple participants. The registry does not report how many caregivers contributed or how many completed questionnaires were available.

A mean can describe the overall direction and average level of the recorded ratings. The standard deviation, which the registry names as the measure of dispersion, describes how spread out the scores were around that mean. General interpretation: a mean is most informative when considered alongside its scale, the dispersion of individual or averaged assessments, and the way the assessments were collected.

The registry record does not report the primary mean score or standard deviation. Consequently, the size, direction, and precision of the observed caregiver rating cannot be described from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The statistical quantities named for the primary endpoint are a mean and a standard deviation. The mean summarizes the average of the caregiver assessments after the averaging procedure described in the registry. The standard deviation describes the spread of those assessments around their mean. The registry record does not report the numerical values for either quantity, and it does not report an inferential analysis for the primary endpoint.

What does the mean represent?

Each caregiver responded to 7 questions about caregiving aspects. The response scale ran from -2 to +2, with verbal anchors ranging from great disadvantages to great advantages. The endpoint combined scores across the single responses. According to the registry, each caregiver’s scores were first averaged over the participants that caregiver assessed, and the final mean was calculated from those caregiver-level averages.

General statistical explanation: averaging can make a multi-question measure easier to summarize, but the resulting mean does not show the full pattern of responses. Different sets of individual ratings can have the same average. The standard deviation adds information about spread, but it does not reveal every feature of the response distribution. Interpretation also depends on how the questions were constructed and whether respondents understood and used the scale consistently. The registry record does not report additional information on these measurement properties.

Why does the design matter for analysis?

The study is described as non-interventional and cross-sectional. Neupro® was used as routine treatment; the registry does not describe randomized treatment assignment or an assigned oral-medication control group. The ratings compare perceptions of Neupro® with oral medication, but they are not measurements from two randomized treatment arms. A difference in ratings, if reported, would describe respondents’ assessments in this setting and would not by itself identify a causal effect of treatment.

The timing is also relevant. The questionnaire was completed at a single time-point during the study run, described as 16 months. That describes the study period, not repeated measurement of each participant across that period. The primary endpoint’s timeframe also states that suitable patients had received Neupro® together with l-dopa or another oral idiopathic Parkinson’s disease drug for at least one month. The registry record does not describe follow-up assessments or a longitudinal model.

What would uncertainty measures add?

General statistical explanation: a confidence interval gives a range of estimates compatible with the data and analysis assumptions, and communicates uncertainty around an estimated quantity. A p-value, when tied to a specified hypothesis test, addresses how unusual the data would be under that test’s null hypothesis. Neither is a substitute for the estimate itself, and neither alone establishes practical importance. The registry record does not report a confidence interval, p-value, hypothesis test, or model for the primary outcome.

The endpoint is based on assessments that may be clustered: a caregiver could assess one or multiple participants, and the described procedure averaged assessments within caregiver before calculating the final mean. General statistical explanation: when multiple observations come from the same respondent, they may be more similar to one another than observations from different respondents. Analyses that treat correlated observations as independent can misstate uncertainty. The registry describes the averaging procedure but does not report additional modeling or variance estimation details that would clarify how dependence was handled beyond that procedure.

How should the planning assumptions be read?

The registry’s sample-size assumptions describe a planning illustration based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated effect to detect is a fraction of the observed effect. These are planning assumptions, not evidence that the study randomized participants between two arms. The registry record does not report a sample-size justification for the study’s own design or numerical sample-size results. Such illustrations are not observed power; they describe hypothetical planning quantities under their assumptions.

General statistical explanation: planning calculations depend on the target effect, variability, significance level, allocation, and statistical method. Changing these assumptions can change the calculated number of participants. In a non-interventional study with no assigned comparison arm, a hypothetical equal-allocation illustration should not be confused with the actual enrollment or with an achieved treatment comparison. The registry record does not report the observed primary effect needed to assess such illustrations independently.

What analysis details are absent?

The registry lists 21 outcome measures and 0 analyses. It does not report the statistical test or model used for the primary endpoint, any adjustment for covariates, a confidence interval, or a plan for handling multiple outcomes. It also does not report the number of caregivers contributing to the primary endpoint or the number of endpoint observations. Without those details and the outcome values, the analysis cannot be reconstructed from the registry record.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Neupro® Treatment")
n   <- c(147)
m   <- c(1.32)
sp  <- c(0.67)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Neupro® Treatment  147  1.320  0.670  1.211  1.429

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 21 outcome measures, including the primary caregiver rating and secondary physician-related outcomes. It lists 0 analyses. The record does not report whether the outcomes were treated as a family of tests, whether any multiplicity adjustment was planned, or how the primary and secondary questions were prioritized in statistical testing. Without reported estimates or tests, the impact of multiple outcome assessment on interpretation cannot be evaluated.

The analysis population is described as the Full Analysis Set (FAS). It comprises all patients with at least 1 assessment in the physicians’ or caregivers’/nurses’ questionnaires. For the primary endpoint, caregiver assessments were averaged over the participants each caregiver assessed, followed by calculation of a final mean from the caregiver-level averages. The registry record does not report the number of patients, caregivers, or completed questionnaires contributing to that endpoint.

The enrollment of 148 does not establish how many participants had usable caregiver ratings. The record does not report the amount or pattern of missing endpoint data, reasons for missingness, or a method for handling missing responses. It also does not report whether the endpoint calculation required complete answers to all 7 questions or how partial questionnaires were treated. As a result, the potential effect of missing information on the reported endpoint cannot be assessed.

General statistical explanation: missingness can affect an estimate when the people or observations without data differ systematically from those with data. A complete-case result, an imputed result, and a result under other missing-data assumptions may differ. The registry does not describe which approach, if any, was used. It also does not report sensitivity analyses. These omissions limit assessment of robustness, independently of the absence of numerical outcome results.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

The registry reports safety figures for the Neupro® Treatment group. There were 148 participants at risk for serious adverse events, and 0 were reported as affected. For other adverse events, 148 were at risk and 1 was reported as affected. Deaths are not reported in the registry record.

These figures are counts of participants recorded as affected, not a complete description of the nature, timing, severity, or clinical course of events. The registry lists no top serious adverse-event terms. It does not provide further event details in the record, so the seriousness count cannot be used to infer which events were monitored, how they were defined, or how long each participant was observed for safety.

General statistical explanation: a count of zero observed serious adverse events does not establish that an event is impossible or that the treatment has no safety risks. Interpretation depends on the amount of observation, event definitions, reporting completeness, and study design. The safety figures are from a non-interventional study and do not provide a randomized safety comparison against oral medication. The registry does not report comparative safety estimates or uncertainty intervals.

13. What the Result Does, and Does Not, Mean

The study addresses how caregivers and physicians assessed Neupro® relative to oral anti-Parkinson medication among people with idiopathic Parkinson’s disease requiring caregiver support. Its cross-sectional questionnaires provide a framework for describing those perceptions. However, the registry record does not report the primary caregiver mean score or standard deviation, nor does it report numerical results for the physician-related secondary outcomes. The direction and size of the ratings therefore remain unknown from this record.

The design also determines what such ratings could mean. Neupro® was used in routine care, and the study was non-interventional rather than randomized. Caregiver and physician judgments may reflect treatment experience and care context, but the record does not provide a controlled comparison that separates the effect of treatment from other influences. The comparison with oral medication is part of the rating question; it is not evidence that participants were assigned to oral medication as a control group.

The primary measure is a questionnaire score on a 5-point scale from -2 to +2, constructed from 7 caregiving questions and averaged as described in the registry. It represents ratings, not a direct measure of symptom change, disease progression, or clinical benefit. Even if the numerical score were available, its interpretation would require attention to the scale, response variability, respondent selection, and missing data. The registry record does not report the score, its uncertainty, or the number of caregivers contributing.

The enrollment of 148 and the reported safety counts describe aspects of study conduct, not the missing preference result. The serious adverse-event count is 0 among 148 at risk, and 1 participant among 148 at risk is reported with another adverse event; deaths are not reported in the registry record. These counts do not create a comparison with oral medication.

Accordingly, the record can establish what the study sought to measure and how it was designed, but it cannot establish the observed caregiver preference or a causal treatment effect. No claim that Neupro® does or does not work follows from the information reported in the registry record.

14. Why This Trial Matters Statistically

This study is statistically notable because its main question is about reported preference, while its design is observational and cross-sectional. Caregivers and physicians compared Neupro® with oral anti-Parkinson medication in questionnaires, but participants were not randomly assigned by the study. Recognizing that distinction prevents a perception measure from being interpreted as a randomized estimate of treatment effect.

The primary endpoint also has a respondent-level structure. A caregiver could assess one or multiple participants, and the registry describes averaging assessments within caregiver before calculating the final mean. That procedure makes clear that the reported endpoint is not simply a raw average of all participant-level responses. It also raises an important general analytic issue: observations from the same respondent may be related, and the method used to account for that relationship can affect uncertainty. Further modeling details are not reported in the registry record.

Questionnaire interpretation depends on both the scale and the way the data are summarized. The caregiver measure combines 7 questions rated on a 5-point scale from -2 to +2. A mean can communicate the overall average direction, while the standard deviation can describe spread, but neither alone shows all response patterns. Because neither endpoint value is reported, the record does not permit evaluation of the distribution or precision of caregiver ratings.

The record’s 21 outcome measures and 0 analyses also illustrate why reporting of analysis plans and outcome results matters. Without reported results, missing-data handling, or multiplicity methods, readers cannot assess how the outcome set was analyzed or how stable any finding might be. The study can still be understood as a structured evaluation of caregiver and physician perspectives, but its statistical contribution to comparative treatment evidence is limited by the design and the absent results.

15. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The primary limitation for interpreting the stated research question is that the registry record does not report the primary endpoint result. Neither the caregiver mean nor its standard deviation is available, and numerical results for the physician outcomes are also not reported in the registry record. The preference direction, magnitude, and precision therefore cannot be assessed.

The design is non-interventional and cross-sectional, with no randomized allocation and no like-for-like oral-medication control group. The registry does not state an allocation method. As a result, comparisons in caregiver or physician ratings should be understood as judgments made in routine-care circumstances, not as causal estimates from assigned treatments. The registry does not report adjustment for differences between people receiving treatment or for other factors that could influence treatment choice and ratings.

The primary outcome relies on caregiver questionnaire responses. The record describes a 7-question instrument and an averaging process, but does not report how many caregivers responded, the response rate, the number of endpoint observations, or further details on questionnaire validation. It does not report how incomplete questionnaires or missing answers were handled. The enrollment of 148 cannot be assumed to equal the number contributing to the primary endpoint.

The registry lists 21 outcome measures and 0 analyses, but does not report inferential methods, confidence intervals, p-values, multiplicity procedures, or sensitivity analyses. The sample-size assumptions describe a planning illustration, not the study’s own design justification; a numerical planning result and the observed effect are not reported in the registry record.

Safety information is also limited: the record gives participant counts for serious and other adverse events but does not give event descriptions or deaths. These limitations constrain interpretation of both the preference question and the safety figures.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Caregivers' and Physicians' Treatment Preference in Parkinson Patients Treated With Neupro® Requiring Caregiver Support — NCT01330290.
• Registry record last updated 2013-12-10; status last verified 2013-11.
• Results first posted 2013-12-10.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

19. Record Summary

NCT01330290 is a completed, multi-site, non-interventional, cross-sectional evaluation sponsored by UCB Pharma. The registry lists idiopathic Parkinson’s disease as the condition, enrollment of 148, a study start of 2011-03, and primary completion in 2012-08.

The study assessed caregiver and physician perspectives on Neupro® compared with oral anti-Parkinson medication among people requiring caregiver support. The primary endpoint was the mean caregiver rating across 7 caregiving questions, scored from -2 to +2, with a standard deviation specified as the measure of dispersion. The registry lists 21 outcome measures and 0 analyses.

The primary endpoint value, its standard deviation, and numerical results for the physician outcomes are not reported in the registry record. Safety figures report 0 participants with serious adverse events among 148 at risk and 1 participant with another adverse event among 148 at risk. Deaths are not reported in the registry record.