Characteristics and Treatment Patterns of Patients With Chronic Obstructive Pulmonary Disease (COPD), Initiating Tio+Olo or Other Maintenance Therapies in the US and the UK: A Retrospective Claims Database Study: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Characteristics and Treatment Patterns of Patients With Chronic Obstructive Pulmonary Disease (COPD), Initiating Tio+Olo or Other Maintenance Therapies in the US and the UK: A Retrospective Claims Database Study (NCT04926233), a registered phase not stated study. The primary outcome is Age of the Chronic Obstructive Pulmonary Disease (COPD) Patients at the Time of COPD Diagnosis - US.

2. Clinical Question

3. Trial Design

This is a retrospective claims database study, not a randomized clinical trial. The registry identifies two data sources: the US IBM MarketScan database and the UK CPRD GOLD database. The record describes the US data as covering January 1, 2008 to March 31, 2018; a corresponding coverage period for the UK data is not reported in the registry record. The study is marked completed, with a start date of 2019-11-15 and a primary completion date of 2020-04-14.

The stated objective is to describe characteristics of people with COPD in the US and UK, including demographic, lifestyle, clinical, and medication-use characteristics. The title also identifies treatment patterns among patients initiating tiotropium plus olodaterol or other maintenance therapies. The record lists enrollment of 1371146, but it does not report how that enrollment is distributed across countries, treatment groups, or diagnosis-period strata.

The primary outcome is age at COPD diagnosis in the US database. It is described as a mean with standard deviation, stratified by diagnosis before versus after approval of tiotropium plus olodaterol in 2015. The listed arms are database populations, not randomized treatment groups. Allocation is not stated, and the registry cautions that the arms are not a like-for-like control comparison. No protocol details explaining cohort construction, treatment assignment, adjustment, or statistical testing are reported in the registry record.

The record lists 21 outcome measures and 0 analyses. It includes outcomes concerning exacerbations and timing of maintenance therapy, separately for the US and UK. Their outcome values are not reported in the registry record. The listed intervention includes tiotropium bromide plus olodaterol and the Sp(t)iolto® Respimat® device; the record does not describe a randomized intervention assignment.

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: Patients with chronic obstructive pulmonary disease (COPD) who met all inclusion criteria and none of the exclusion criteria initiating tiotropium + olodaterol or other maintenance therapies from either IBM MarketScan (United States) or CPRD GOLD (United Kingdom) databases were included.

Overall Study

Analysis population for the primary outcome, as defined in the registry: All chronic obstructive pulmonary disease (COPD) patients who meet all selection criteria from the United States (US) IBM MarketScan database (contained data collected from January 1, 2008 to March 31, 2018).

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: All chronic obstructive pulmonary disease (COPD) patients who meet all selection criteria from the United States (US) IBM MarketScan (contained data collected from January 1, 2008 to March 31, 2018) and the United Kingdom (UK) CPRD GOLD databases (contained data collected from November 21, 1987 to June 30, 2018).

6. Primary Endpoint

The registry lists the primary outcome as Age of the Chronic Obstructive Pulmonary Disease (COPD) Patients at the Time of COPD Diagnosis - US, measured over At index date of cohort entry (baseline; time of COPD diagnosis)..

The primary endpoint is the age of US patients with COPD at the time of diagnosis, expressed as a mean in years with standard deviation. Its stated timeframe is the index date of cohort entry, described as baseline and the time of COPD diagnosis. The outcome description specifies that the mean is to be reported separately for patients whose diagnosis occurred before versus after approval of tiotropium plus olodaterol in 2015.

Age is a continuous characteristic, so a mean gives a measure of the group’s central value, while the standard deviation describes how spread out individual ages are around that mean. These are descriptive statistics; by themselves, they do not establish that a difference between groups is caused by a treatment or by the approval period. In this study, the endpoint helps characterize the US patient population across the two diagnosis periods. It is not a direct measure of COPD severity, treatment effectiveness, or change in health.

The registry record does not report the mean age or standard deviation for either period, nor does it report a difference estimate, confidence interval, or test result. It also does not describe the corresponding age measure as a primary endpoint for the UK data. Consequently, the endpoint’s definition is clear, but its observed values and the precision of any comparison are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the primary statistic describe? The registry identifies the primary measure as a mean, with standard deviation, for a continuous variable: age at COPD diagnosis in the US database. The mean summarizes the central value of the recorded ages. The standard deviation describes variability among individual ages around that mean. Neither statistic alone shows how all individual observations are distributed, and neither establishes why any observed difference between groups exists.

The diagnosis-period groups are defined around approval in 2015. Such a division can support a descriptive comparison of populations recorded in different periods. It does not make the groups randomized or automatically comparable. Calendar-period changes in diagnosis, coding, treatment practice, or database composition can accompany a change in the mean. Whether those factors were measured, adjusted for, or assessed is not reported in the registry record.

What would uncertainty measures add? In general, a confidence interval expresses the uncertainty around an estimated quantity under the assumptions of the analysis. A narrow interval indicates greater precision under those assumptions, while a wide interval indicates less precision. A p-value, in general, relates the observed data to a specified null hypothesis and statistical model; it is not a measure of clinical importance or the probability that a hypothesis is true. The registry record does not report a confidence interval, p-value, or test for the primary outcome, so these concepts cannot be applied to an observed age comparison.

Why does the study design matter? Claims databases record information generated for administrative or healthcare purposes rather than through randomized assignment. In general, observational comparisons may be affected by confounding: characteristics associated with both treatment or calendar period and the measured outcome can contribute to apparent group differences. Claims-based variables may also depend on coding and the coverage of the database. These are general considerations for this design, not claims that a specific bias occurred. The registry does not report an adjustment strategy or enough detail to evaluate how such concerns were handled.

The study lists populations from US IBM MarketScan and UK CPRD GOLD. The primary outcome is specifically described for US MarketScan. The record does not give a UK primary age result or explain whether the same outcome definition and processing were used in both countries. Results from separate data sources can depend on the populations and information captured by each source, so a pooled interpretation should not be assumed.

What is reported about planning? 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 assumed effect is a stated fraction of the observed effect. For time-to-event planning, the stated quantity is the total number of events; for other outcomes, it is participants per arm. These are planning assumptions, not observed study results or observed power. The record does not provide the actual effect used, the stated fractions, or resulting planning values in the information available for interpretation, and it does not say that these assumptions were the study’s own design justification.

General sample-size calculations depend on the effect to be detected, variability, allocation, and chosen statistical assumptions. A calculation based on an observed effect is an illustration of the sample size that could be needed under those assumptions; it does not establish what the study was powered to detect before data collection. Because the study is observational and its database populations are not described as like-for-like controls, equal allocation in a planning calculation should not be mistaken for actual equal group sizes or randomized assignment.

The registry names a mean and standard deviation for the primary outcome but does not report how the age variable was checked, whether any transformations or robust methods were considered, or how missing ages were handled. In general, a standard deviation is most informative alongside the mean when the distribution is reasonably characterized; unusual skew or extreme values can make the mean less representative of a typical observation. The record does not report distributional assessments, so it is not possible to determine whether any particular modeling choice was appropriate.

The record lists 0 analyses. It does not report model specifications, covariates, standard-error methods, confidence intervals, hypothesis tests, or interim analyses. No recalculated comparison is reported in the registry record. Accordingly, the statistical interpretation should remain limited to the outcome definition and the study’s descriptive purpose, without inferring an unreported method or result.

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("US IBM Marketscan - COPD Diagnosis Before Tio+Olo ", "US IBM Marketscan - COPD Diagnosis After Tio+Olo A")
n   <- c(1021963, 258523)
m   <- c(65.6, 64.4)
sp  <- c(13.3, 12.8)
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  US IBM Marketscan - COPD Diagnosis Before Tio+Olo   1021963  65.600  13.300  65.574  65.626
CARM  US IBM Marketscan - COPD Diagnosis After Tio+Olo A  258523  64.400  12.800  64.351  64.449

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 21 outcome measures, including the primary age measure and secondary outcomes concerning exacerbations and timing of maintenance therapy in the US and UK. It records 0 analyses. The registry record does not explain whether outcomes were tested as a family, whether any hierarchy or multiplicity adjustment was planned, or how the primary outcome was distinguished analytically from the secondary measures. Without reported analyses or results, the effect of multiple comparisons on any inferential claims cannot be assessed.

Missing-data handling is not reported in the registry record. The outcome descriptions do not say how missing age, exacerbation, or timing information was treated, whether records with incomplete information were excluded, or whether any imputation was used. These decisions can affect both the population represented by a result and its precision. The registry’s enrollment figure does not establish the denominator for any individual outcome or analysis.

The population is described as patients meeting selection criteria in the US MarketScan or UK CPRD GOLD database, but the full selection criteria and analysis-population definitions are not reported in the registry record. There is no reported description of exclusions after cohort entry, subgroup denominators, or the handling of people appearing in more than one analysis. No conclusions about attrition or data completeness can therefore be drawn.

The record also does not report an interim-analysis plan, stopping rule, or the statistical analysis plan. These omissions do not establish that such procedures were absent; their status is not reported in the registry record. The listed zero entries in adverse-event fields are likewise not accompanied by a missing-data or event-ascertainment explanation.

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

The record defines a descriptive study of COPD patient characteristics and treatment patterns in US and UK claims data. Its primary outcome concerns mean age at diagnosis among eligible US patients, divided by diagnosis period around approval of tiotropium plus olodaterol in 2015. This information identifies what the researchers intended to describe; it does not supply the observed values for that outcome.

Because no primary means, standard deviations, or inferential results are reported in the registry record, the size and precision of any difference between diagnosis-period groups cannot be interpreted. The absence of results in the record is not evidence that the groups had equal ages, nor evidence that they differed. Similarly, the listed secondary outcomes identify measures of exacerbations and treatment timing, but do not show their observed values.

The design does not support treating the database populations as randomized treatment groups. The registry says allocation is not stated and cautions that the arms are not a like-for-like control comparison. A descriptive association across diagnosis periods or treatment categories would not, by itself, establish a causal treatment effect. Whether methods were used to address confounding is not reported in the registry record.

More generally, claims-based descriptions can help characterize the populations and recorded care represented by the databases, while remaining dependent on the information those databases capture. This study’s record does not provide enough detail to assess data completeness, selection into the cohorts, or the comparability of the US and UK sources. The adverse-event fields listing zero counts also do not establish absence of events because their ascertainment and interpretation are not described.

These limits concern what can be concluded from the registry record, not a clinical recommendation. The record supports describing the study’s aims, databases, and planned measures, but it does not report the outcome evidence needed to characterize an observed treatment or population difference.

13. Why This Trial Matters Statistically

Retrospective claims studies can describe characteristics and patterns of recorded care across large administrative databases. This registry record identifies US and UK COPD populations and a specific descriptive question about age at diagnosis in the US. It also names outcomes related to exacerbations and the timing of maintenance therapy. Those aims can be statistically useful because they clarify the populations and measures researchers intended to examine.

The record also illustrates why design labels and estimands matter. A database population is not equivalent to a randomized treatment group, and a comparison before and after an approval date is not automatically a causal estimate. The registry’s caution that the groups are not like-for-like controls is important when interpreting any eventual difference. Descriptive summaries, adjusted associations, and causal effects answer different questions and require different assumptions.

Precision is another essential part of interpretation. A mean without its variability or uncertainty would not fully characterize a group comparison; in this record, even the mean and standard deviation are not reported. Naming a primary measure is not the same as reporting its result. Likewise, a planning illustration based on an observed effect cannot be interpreted as observed power or as proof that a study was designed to detect a particular clinically meaningful effect.

For statistical readers, this record makes the boundary between a clearly specified descriptive outcome and an interpretable result especially visible. The registry identifies data sources, a primary measure, and several secondary measures, while leaving outcome estimates, analysis methods, missing-data procedures, and multiplicity handling unreported. That distinction prevents an objective or outcome label from being mistaken for evidence of a treatment effect.

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 registry record does not report the primary outcome values, including the mean age and standard deviation for either diagnosis-period group. It also does not report confidence intervals, p-values, or an estimated between-group difference. As a result, the primary outcome cannot be evaluated for magnitude or statistical precision from the record.

Although enrollment is listed as 1371146, separate denominators for the US and UK data sources, diagnosis periods, treatment groups, and individual outcomes are not reported in the registry record. The full selection criteria are not given, and neither are the methods used to define or validate COPD diagnosis, therapy initiation, or exacerbations. The US data coverage is specified as January 1, 2008 to March 31, 2018, but equivalent UK coverage dates are not reported.

The study is retrospective and based on claims databases. In general, such data may reflect coding and coverage practices and may not contain every clinical characteristic relevant to comparisons. The registry does not report how confounding was addressed, whether estimates were adjusted, or how missing information was handled. These are design-related uncertainties; the record does not establish that any particular bias occurred.

The registry cautions that allocation is not stated and the arms are not a like-for-like control comparison. It does not report an analysis plan, multiplicity approach, subgroup definitions, or interim analyses. The adverse-event entries list zero counts without describing ascertainment or interpretation. These limitations restrict conclusions about both comparative outcomes and safety from the registry record.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Characteristics and Treatment Patterns of Patients With Chronic Obstructive Pulmonary Disease (COPD), Initiating Tio+Olo or Other Maintenance Therapies in the US and the UK: A Retrospective Claims Database Study — NCT04926233.
• Registry record last updated 2021-11-15; status last verified 2021-11.
• Results first posted 2021-07-20.
• 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

NCT04926233 is a completed retrospective claims database study sponsored by Boehringer Ingelheim. The stated objective is to describe demographic, lifestyle, clinical, and medication-use characteristics of people with COPD in US and UK data. The record lists enrollment of 1371146 and identifies US IBM MarketScan and UK CPRD GOLD populations.

The primary outcome is mean age at COPD diagnosis, with standard deviation, among eligible US MarketScan patients, stratified by diagnosis before versus after approval of tiotropium plus olodaterol in 2015. The registry lists 21 outcome measures and 0 analyses. It does not report the primary outcome values, inferential statistics, analysis plan, or missing-data procedures. Allocation is not stated, and the record cautions that the populations are not a like-for-like control comparison.