Efficacy and Safety of Buprenorphine Transdermal Patch in Non-cancer Pain of Moderate Intensity: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Efficacy and Safety of Buprenorphine Transdermal Patch in Non-cancer Pain of Moderate Intensity (NCT02519387), a na phase 4 study. The primary outcome is Change in Box Scale-11 (BS-11) Pain Score.

2. Clinical Question

In practical terms, the study asked whether patients’ recorded pain intensity changed over the stated treatment period while they used the patch. The BS-11 is a self-selected rating from 0, representing no pain, to 10, representing the worst imaginable pain. Because there was no concurrent control group, a change over time would describe the observed course among participants, but would not by itself isolate the effect of the patch from other explanations. The registry record lists secondary questions about sleep quality, rescue pain medication, treatment satisfaction, and withdrawals due to adverse events. Numerical findings for those outcomes are not reported in the registry record.

3. Trial Design

This was a phase IV, open-label, single-arm study sponsored by Mundipharma Pharmaceuticals Sdn. Bhd. The registry record states that 78 participants were enrolled and that the study is completed. The recorded start is 2013-07, and the primary completion is 2015-01. The conditions named include osteoarthritis, rheumatoid arthritis, lower back pain, joint pain, and muscle pain.

All participants were assigned to the buprenorphine transdermal patch group in the registry’s description. The intervention is described as a 5mg or 10mg patch, applied every 7 days for 3 months. Allocation is listed as not applicable, masking as none, and the model as single group. These characteristics mean the study was not designed as a masked, randomized comparison between treatment arms. Instead, its primary endpoint compares pain scores at baseline and at the stated 3-month visit within the treated group.

The study summary describes the target population as patients with non-malignant pain who were not responding to non-opioid analgesics. The primary efficacy population is described as eligible patients included in the intent-to-treat population. The registry record does not report how many enrolled participants were included in that population, how many completed follow-up, or whether every participant had an observed score at both time points.

Five outcome measures are recorded, including the primary BS-11 pain endpoint and secondary outcomes covering sleep quality, rescue medication use, treatment satisfaction, and withdrawals due to adverse events. The record lists no analyses. Details of the statistical analysis plan, sample-size justification for the original study, and any interim analyses are not reported in the registry record. The study’s design supports a descriptive account of outcomes during treatment, but lacks a concurrent control group for separating changes associated with treatment from other influences over time.

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.

Overall Study

Analysis population for the primary outcome, as defined in the registry: These patients were eligible and included in the intent-to-treat efficacy population.

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.

6. Primary Endpoint

The registry lists the primary outcome as Change in Box Scale-11 (BS-11) Pain Score, measured over Baseline,3 months.

The primary endpoint is change in the Box Scale-11 (BS-11) pain score, a continuous measure of pain intensity. The scale ranges from 0, meaning no pain, to 10, meaning the worst imaginable pain. Participants selected the number corresponding to the pain intensity they were feeling. Scores were recorded at baseline and at Visit 6, described as 3 months from the baseline visit.

A continuous score preserves gradations in reported pain rather than reducing each participant’s experience to a yes-or-no outcome. Comparing baseline and follow-up scores can describe whether the group’s average reported pain intensity changed over the observation period. The registry identifies the endpoint summary as a mean with standard deviation, but does not report the numerical means, standard deviations, or mean change. It also does not report a confidence interval or statistical test for the endpoint.

Using the same scale at the two stated time points gives the endpoint a clear longitudinal interpretation: it concerns change in reported pain intensity within the study population. In general, a mean describes the group’s average score, while the standard deviation describes variation among individual scores around that average. Neither statistic alone shows how much individual participants changed, and a within-group change does not establish what would have happened over the same period without the intervention. The registry record does not report a threshold defining a clinically important change on the BS-11.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record describes a continuous primary outcome summarized by a mean and standard deviation. It also identifies two measurement occasions: baseline and 3 months. The numerical outcome summaries and any inferential analyses are not reported in the registry record. The following concepts explain how such results are generally interpreted; they do not assert that a particular analysis was conducted in this study.

What does a mean change describe?

For a continuous outcome, a mean is the arithmetic average of the recorded scores in the analyzed group. A mean change would summarize the average difference between participants’ follow-up and baseline scores, if calculated from paired observations. The direction depends on how change is defined. For a pain-intensity scale where larger values represent worse pain, a lower follow-up score would correspond to lower reported pain intensity for that observation, but the registry record does not provide the score values or state a change convention. No direction should therefore be inferred for this study.

A group mean can conceal variation. Participants may have different baseline scores and may change by different amounts. The standard deviation describes the spread of observed scores around a mean; it is not the uncertainty of the estimated mean. A confidence interval, when reported, expresses uncertainty around an estimate under the assumptions of the statistical method. A narrower interval indicates greater precision than a wider interval, but neither an interval nor a p-value is reported for this primary endpoint.

Why does the single-group structure matter?

A baseline-to-follow-up comparison within one group is not equivalent to a randomized comparison. In general, random allocation to concurrent groups helps balance, on average, factors that could affect outcomes, whether measured or not. This registry record lists no random allocation and no separate comparator. Thus, even if an average pain score changed, the design would not by itself determine how much of that change was attributable to the patch rather than time, expectations, changes in other care, or other influences. The open-label design also means masking was absent, so participants and study personnel were not described as blinded to treatment.

The absence of a control group does not make a within-group description meaningless. It can characterize recorded scores during the study period and can describe the degree of observed variation. But causal interpretation requires caution. The registry record supplies no adjusted comparison or other analysis intended to address these limitations.

What does the analysis population imply?

The primary outcome description says the efficacy population consisted of eligible patients included in the intent-to-treat population. The record does not report the number in that population or explain how participants with missing follow-up scores were handled. In general, an intent-to-treat approach aims to analyze participants according to their assigned study group, but this single-group record does not give enough detail to determine the exact operational definition, inclusion rules, or handling of incomplete measurements. Those details are not reported in the registry record.

Missing outcome data can affect both the number of observations and the interpretation of a mean change. If participants without follow-up differ systematically from those with follow-up, an analysis restricted to complete observations may not represent the full enrolled group. Statistical methods such as imputation can address some missing-data situations under specified assumptions, but no missing-data method is reported in the registry record. It is therefore not possible to assess whether missingness was present or how it was addressed.

How should significance and precision be read?

A p-value, if reported, would describe how compatible the data are with a specified null hypothesis under the test’s assumptions. It does not measure the size or clinical importance of a change. A confidence interval would give an estimated range of values compatible with the data and model assumptions, and would help convey precision. Neither a test result nor an interval is reported for the BS-11 endpoint. The absence of a reported result is not evidence that a change was absent, nor is it evidence that a change was present.

The registry record lists no analyses. The statistical analysis plan, including the exact model or test, covariate adjustment, and handling of repeated measurements, is not reported in the registry record. No method should be attributed to the investigators beyond the outcome description that is recorded.

What do planning illustrations mean?

Any sample-size illustration based on an observed effect is a planning exercise, not an observed trial result and not observed power. The stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the target effect is expressed as a fraction of the observed effect. For time-to-event outcomes, the illustration counts total events, while other illustrations are expressed as participants per arm. These assumptions describe hypothetical planning calculations, not the design of this single-group study. The registry record does not report the original study’s sample-size justification, and the absence of an observed endpoint estimate prevents an independent interpretation of the effect on which such an illustration might rely.

In general, over-dispersion matters when observed variability exceeds what a statistical model assumes. If ignored, standard errors may be too small and uncertainty may be understated. The registry record does not report an analysis requiring an over-dispersion assessment for this continuous endpoint. This is a general statistical consideration, not a claim about the study’s data or methods.

10. Multiplicity, Missing Data and Analysis Populations

The record lists the BS-11 pain score as the primary outcome and four categories of secondary outcomes: sleep quality, breakthrough pain medication use, treatment satisfaction, and withdrawals due to adverse events. No numerical results are reported for these secondary outcomes. The record lists no analyses, and the statistical analysis plan is not reported in the registry record.

Multiplicity arises when several outcomes, time points, or statistical tests are examined. In general, conducting many tests can increase the chance of finding at least one apparently notable result by chance if the tests are treated independently and no adjustment or hierarchy is used. A prespecified primary endpoint can help focus interpretation, but the registry record does not report a multiplicity strategy, testing hierarchy, or adjustment method. It is not possible to determine which secondary analyses, if any, were conducted or how they would have been interpreted together.

The registry record describes the primary efficacy population as eligible patients included in the intent-to-treat population. It does not report the size of that population, the number with complete baseline and follow-up pain scores, or the number with missing values. The missing-data strategy is not reported in the registry record. This leaves uncertainty about the amount of available information and whether the analyzed participants differed from those without observed outcomes.

In general, complete-case analysis uses only participants with the required measurements, while imputation or model-based approaches may use additional information but depend on assumptions about missingness. None of these approaches should be attributed to this study on the basis of the registry record. The number of enrolled participants, by itself, does not establish the number included in a particular endpoint analysis.

11. 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 serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: elbow fracture due to fall (1.3%), failed elbow implant (1.3%).

The safety record lists 78 participants at risk for serious adverse events and 1 participant affected by at least one serious adverse event. It identifies “Elbow fracture due to fall” and “Failed elbow implant” among the top serious event terms, each reported as 1.3%. The record does not provide enough detail to determine whether those terms refer to the same participant or to separate events, so they should not be added together or treated as a full event inventory.

For other adverse events, the record lists 78 at risk and 56 affected. These counts are distinct from the serious-event figures and should not be combined with them. The registry record does not provide a complete breakdown of the other adverse events, their timing, duration, severity, or relationship to treatment in the information. Deaths are not reported in the registry record.

In general, an adverse-event count describes reported events or affected participants according to the recorded definition; it does not by itself establish that treatment caused the event. The listed serious terms and counts are descriptive safety information, not a comparison with an untreated group. Because this was a single-arm study, the record offers no concurrent control rate for evaluating relative event frequency. The counts also do not by themselves describe the full balance of potential benefits and harms.

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

The registry record establishes that a completed phase IV study examined a buprenorphine transdermal patch in a single group of patients with moderate non-malignant pain. The intended primary assessment was change in BS-11 pain score between baseline and 3 months. It also records selected safety counts and identifies secondary outcomes related to sleep, rescue medication, satisfaction, and adverse-event withdrawals.

What the record does not establish is the size or direction of change in pain scores. Numerical baseline and follow-up means, standard deviations, a mean change, a confidence interval, and a statistical test are not reported in the registry record. The study’s primary outcome cannot therefore be characterized as showing an increase, a decrease, or no change on the basis of this information. Secondary outcome results are also not reported.

Even if a within-group change were available, the single-arm, open-label design would limit causal conclusions. In general, outcomes can change over time for reasons other than the intervention, and a concurrent comparison group can help separate treatment differences from those background changes. With no separate comparator or random allocation recorded, the study can describe outcomes among participants during treatment but cannot by itself establish what their outcomes would have been under another approach.

The safety counts indicate that serious and other adverse events were recorded among participants, but they do not establish treatment causation. The listed serious terms are limited entries and do not replace a complete safety analysis or a comparative assessment. The registry record does not report enough information to assess the primary estimate’s precision, missing-data effects, or the full outcome profile. These limits concern what can be concluded from the registry record; they do not imply that unreported results were favorable or unfavorable.

13. Why This Trial Matters Statistically

This study is statistically informative as an example of the distinction between a clearly defined endpoint and a reported result. The registry record specifies the BS-11 scale, its meaning, the baseline and 3-month measurement occasions, and the intended efficacy population. Those details clarify what the primary outcome was designed to measure. Yet the numerical endpoint results are not reported in the registry record, so the magnitude and precision of any change remain unknown.

The design also illustrates why a within-group comparison and a treatment comparison answer different questions. Measuring participants before and after treatment can describe the group’s trajectory. A randomized concurrent comparator would address a different question: how outcomes compare between groups assigned to different conditions. This study is listed as single-group with no random allocation and no masking, so its design does not provide that direct contrast.

Another statistical lesson is that enrollment and analysis populations are not interchangeable. The registry record reports 78 enrolled participants and describes an intent-to-treat efficacy population, but does not report the population size used for the primary endpoint or the number with complete measurements. Without those details, readers cannot assess the amount of outcome information or the possible role of missing data.

Finally, safety counts need context. The registry lists one participant affected by a serious adverse event and identifies serious event terms, alongside a separate count for other adverse events. Those descriptive figures do not provide a controlled comparison or establish causality. The study therefore highlights the need to interpret design, outcome reporting, uncertainty, and safety information together rather than treating any one count as a complete account of effectiveness or risk.

14. 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 main limitation of interpretation is that the registry record does not report numerical results for the primary endpoint. Although it defines the BS-11 scale and the baseline-to-3-month assessment, the mean scores, standard deviations, mean change, and inferential estimates are absent. The record therefore does not allow assessment of the direction, magnitude, or precision of the pain-score outcome.

The single-group, open-label structure is an additional limitation. With no concurrent comparator and no random allocation, a within-group change, if reported, could not by itself distinguish treatment-associated change from other changes over time. The lack of masking can also matter for self-reported pain ratings in general, although the registry record does not report whether or how expectations affected responses.

Further limitations arise from incomplete reporting. The size of the intent-to-treat efficacy population, follow-up completion, missing outcome counts, and missing-data methods are not reported in the registry record. The analysis plan, statistical model, any adjustment for baseline values, and any interim analyses are also not reported. The record lists no analyses, so there is no basis for assessing how the specified outcomes were statistically evaluated.

The registry record names several secondary outcomes but does not report their numerical results or a strategy for handling multiple outcomes. The serious-event information is limited to a count and selected terms, while the record does not report a complete description of event timing, treatment relationship, or deaths. These reporting gaps limit interpretation of both efficacy and safety; they do not establish that unreported outcomes were either positive or negative.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Efficacy and Safety of Buprenorphine Transdermal Patch in Non-cancer Pain of Moderate Intensity — NCT02519387.
• Registry record last updated 2016-07-20; status last verified 2016-07.
• Results first posted 2016-07-20.
• 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.

18. Record Summary

NCT02519387 is a completed phase IV, open-label, single-group study sponsored by Mundipharma Pharmaceuticals Sdn. Bhd. The record reports 78 participants enrolled, a start date of 2013-07, and primary completion in 2015-01. Participants had non-malignant pain associated with osteoarthritis, rheumatoid arthritis, lower back pain, joint pain, or muscle pain. The intervention was a buprenorphine transdermal patch described as 5mg or 10mg every 7 days for 3 months.

The primary outcome was change in BS-11 pain score from baseline to 3 months. The registry record does not report numerical results for that endpoint. It lists secondary outcomes concerning sleep, rescue medication, treatment satisfaction, and adverse-event withdrawals. Safety entries include 1 participant affected by a serious adverse event and 56 affected by other adverse events. The record does not report a comparator, statistical analysis results, or the missing-data approach.