Clinical Impact and Utility of Digital Health Solutions in Participants Receiving Systemic Treatment in Clinical Practice: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Clinical Impact and Utility of Digital Health Solutions in Participants Receiving Systemic Treatment in Clinical Practice (NCT05694013), a randomized phase 2, phase 3 study. The primary outcome is Mean Difference in Change of Week 12 Value From Baseline of Participant-reported Total Symptom Interference Score From the MD Anderson Symptom Inventory (MDASI) Core Items.

2. Clinical Question

3. Trial Design

This was a randomized, open-label, parallel platform study listed as phase 2 and phase 3. The study title and brief description frame its purpose as evaluating digital health solutions in participants receiving systemic anti-cancer treatment in clinical practice, with attention to health outcomes and health-care resource utilization. Hoffmann-La Roche is listed as the sponsor.

The registry record lists 49 participants enrolled. Cohort A compared an atezolizumab regimen with local standard-of-care support against an atezolizumab regimen accompanied by the Roche Digital Patient Management module. The cohort included participants with metastatic non-small cell lung carcinoma, extensive-stage small-cell lung carcinoma, and advanced or unresectable hepatocellular carcinoma who were prescribed an intravenous atezolizumab-containing regimen. Cohort B involved participants with resected Stage IIB-IIIB non-small cell lung carcinoma using the DPM Module with subcutaneous atezolizumab in hospital and flexcare, or home, settings. The registry record does not describe a separate Cohort B comparator.

The study began on 2023-02-27, had a listed primary completion date of 2024-06-30, and is marked terminated. The reason for termination is not reported in the registry record. Nor does the record explain how the platform cohorts relate to the primary comparison beyond the arm descriptions. The listed primary analysis population is the intention-to-treat population, defined as all randomized participants.

Although the study is randomized, masking is listed as none. Participants and study personnel therefore were not masked according to the registry record. The allocation procedure, randomization strata, and any additional protocol details are not reported in the registry record. The listed characteristics describe the design, but without numerical endpoint results they do not establish the direction or size of any treatment-group difference.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The ITT population included all randomized participants.

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 Mean Difference in Change of Week 12 Value From Baseline of Participant-reported Total Symptom Interference Score From the MD Anderson Symptom Inventory (MDASI) Core Items, measured over Baseline, Week 12.

The primary endpoint concerns the change from baseline to Week 12 in participant-reported total symptom interference, measured with the core items of the MD Anderson Symptom Inventory. The inventory asks about the extent to which symptoms interfere with aspects of daily life over the last 7 days. The six interference items address general activity, walking, work, mood, relations with other people, and enjoyment of life. Each item is rated from 0-10: 0 means symptoms are absent or do not interfere, while 10 means symptoms are as bad as imaginable or interfere completely.

This type of patient-reported measure focuses on how symptoms affect functioning, rather than recording symptom presence alone. As a general statistical point, a change score compares a participant’s follow-up value with their baseline value, so it describes change over the specified period. It can help characterize whether reported interference shifts during treatment, but interpretation depends on the observed values, their variability, and the analysis method.

The registry title describes a mean difference in change, while the outcome details list the summary parameter as median with full range. That mismatch matters when interpreting a result: a mean and a median describe different features of a distribution, and the registry record does not resolve which summary was used for a reported group comparison. No numerical primary-endpoint values or comparison estimates are reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The study is listed as randomized, open-label, and parallel. Randomization is intended to allocate participants among study groups using a chance-based procedure; as a general statistical principle, this can reduce systematic differences between groups at baseline. The registry record does not describe the allocation sequence, concealment procedures, or any stratification factors. Open-label means masking is listed as none, so expectations or knowledge of group assignment could influence participant reports or other aspects of outcome collection. The record does not report specific steps used to address that possibility.

What does the primary endpoint summarize?

The endpoint is based on participant ratings of symptom interference across six aspects of life. Each item uses a 0-10 scale, with higher values representing greater interference. The outcome is described as change from baseline to Week 12. In general, change scores express how a measurement differs from a participant’s earlier measurement; a group comparison of those changes asks whether the pattern of change differs between groups. The registry record does not post the underlying values, a group contrast, or a confidence interval.

There is a difference between the endpoint title and its listed summary parameter: the title refers to a mean difference in change, while the parameter is median and the dispersion is full range. In general, a mean is the arithmetic average, whereas a median is the central ordered observation. A range gives the endpoints of the observed values and does not convey how concentrated or dispersed values are within that interval. Because no numerical results or clarification are reported, readers cannot determine which summary governed a comparison or how to interpret its magnitude.

How should precision be understood?

In general, a confidence interval describes a range of values compatible with the data and the statistical model used, and it communicates uncertainty around an estimate. A narrow interval indicates more precision than a wide interval, all else equal. No primary-outcome estimate or confidence interval is reported in the registry record, so the precision of a treatment-group contrast cannot be assessed. The record also gives no p-value for the primary outcome. Without an estimate and its uncertainty, statistical significance and clinical importance cannot be evaluated from this record.

What does the analysis population mean?

The primary population is described as intention-to-treat and includes all randomized participants. As a general concept, intention-to-treat analysis aims to retain participants in their assigned groups, regardless of treatment received after assignment. This can preserve the balance created by randomization. The registry record does not report the number randomized in each group, the number contributing endpoint data, or whether the analysis actually followed this population definition. Other analysis populations are not reported in the registry record.

What can be inferred from the planning illustrations?

The planning illustrations are described as using the observed effect, a two-sided significance level of 0.05, equal allocation, and a normal approximation, with the target effect set as a stated fraction of the observed effect. These are planning assumptions, not a report of the trial’s original design justification. The registry record does not report that justification. Such illustrations are conditional on the chosen assumptions and do not represent observed power, nor do they establish that the study was planned around those values. The record provides no displayed numerical planning estimates in the registry record for this interpretation.

For a time-to-event outcome, event-based planning commonly focuses on the number of events rather than only the number of participants; for other outcomes, the stated illustrations are participants per arm. In general, the required information depends on the target effect, variability, significance threshold, allocation, and analysis assumptions. The registry record does not provide enough primary-outcome information to assess whether the completed study had adequate precision.

Why can over-dispersion matter?

As a general statistical concept, over-dispersion occurs when outcome variability is greater than assumed by a model. If a model underestimates variability, its uncertainty estimates may be too small and inference may appear more precise than warranted. The registry record does not report a model for the primary outcome, a dispersion assessment, or a correction for over-dispersion. It would therefore be inappropriate to claim that over-dispersion was assessed or addressed.

The registry record lists five outcome measures and zero analyses. The posted results include safety counts, but no numerical primary endpoint comparison. Thus, a description of possible statistical methods cannot substitute for a reported estimate, model specification, or uncertainty interval. Analysis details such as covariate adjustment, handling of repeated measurements, and sensitivity analyses are not reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The record lists a primary outcome and four additional outcome descriptions concerning quality of life, health status, symptom severity, and hospitalization due to serious adverse events. It also notes that the primary outcome has several categories or time points and was not reduced to one comparison. The registry record does not report a multiplicity strategy, such as a testing hierarchy or adjustment across outcomes or comparisons. Without that information, the chance of false-positive findings across multiple statistical tests cannot be evaluated. No numerical outcome analyses are reported in the registry record, so there are no posted estimates or p-values to interpret in light of multiplicity.

The primary population is described as all randomized participants. However, the registry record does not report group-specific numbers with Week 12 measurements, reasons for missing endpoint data, or how missing observations were handled. Missingness can matter because an analysis based only on participants with observed follow-up may differ from one that uses all randomized participants, particularly if availability of measurements relates to health status or treatment experience. The record does not identify an imputation method, a missing-data assumption, or sensitivity analyses.

Although the intention-to-treat population is defined, the record does not state whether any modified intention-to-treat, per-protocol, or safety populations were used for particular analyses. It also does not report how participants were assigned to analysis groups if treatment use differed from randomization. The registry record does not report a statistical analysis plan, interim analyses, or a sample-size justification. These omissions limit assessment of how the analysis was prespecified and how robust any potential findings would be.

11. Safety Results

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

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: pneumonia (12.5%), pulmonary embolism (8.0%), acute myocardial infarction (4.2%), atrial fibrillation (4.2%), cardiac failure acute (4.2%).

The posted safety figures distinguish the Atezolizumab Regimen group from the Atezolizumab Regimen + DPM group. The group sizes at risk for serious adverse events are 25 and 24, respectively; serious adverse events affected 10 and 13 participants. For other adverse events, the numbers at risk are 25 and 24, with 18 and 22 participants affected. Deaths are listed as 6 and 2. These are counts, not estimates of a causal effect of adding the DPM module.

The record also lists serious adverse event terms with percentages: Pneumonia, 12.5%; Pulmonary embolism, 8.0%; Acute myocardial infarction, 4.2%; Atrial fibrillation, 4.2%; Cardiac failure acute, 4.2%; and Hyperthyroidism, 4.2%. The record does not specify in this listing the denominator or whether participants could contribute to more than one term. These percentages should therefore be read as posted term values, not as a basis for additional calculations.

Safety counts alone do not show event timing, severity beyond the serious-event classification, exposure duration, or adjusted differences. The registry record does not report a safety-analysis method or explain the deaths. The study’s open-label design and its terminated status do not, by themselves, explain any particular safety count or establish a relationship with the digital module.

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

The registry record establishes that a randomized, open-label platform study was conducted to evaluate digital health solutions among people receiving systemic cancer treatment in clinical practice. It identifies the populations and interventions described for two cohorts, the MDASI symptom-interference endpoint, additional patient-reported and resource-use outcomes, and selected safety counts. It also records an enrollment of 49 and marks the study as terminated.

What the record does not establish is the size or direction of a primary-endpoint difference. No numerical primary results, confidence intervals, or p-values are reported. The record says the primary outcome includes several categories or time points and was not reduced to one comparison; it also lists a mismatch between the endpoint title’s reference to a mean difference and the parameter description of median with full range. Without a resolved analysis and numerical results, the effect and its precision cannot be characterized.

As a general principle, a patient-reported outcome can provide information about how participants experience symptoms and their effects on daily life. The MDASI interference items concern activity, walking, work, mood, relationships, and enjoyment of life. Such measurements are distinct from resource-use measures or clinician-recorded events. The registry lists these outcomes, but listing them is not equivalent to reporting their results.

The safety counts describe the number of affected participants in the named groups; they do not demonstrate that one group caused more or fewer events. Differences in raw counts can be difficult to interpret without details such as exposure, event timing, analysis methods, and uncertainty. Those details are not reported in the registry record.

The termination status should also be interpreted narrowly. The reason for termination is not reported, so the status cannot be used to infer the cause of stopping or the study’s scientific conclusions. Overall, the posted information supports a description of the design and selected counts, but not a conclusion about the primary endpoint’s result or the clinical impact of the digital solution.

13. Why This Trial Matters Statistically

This study raises a statistical question that is increasingly relevant to clinical practice: how to evaluate a digital support tool alongside systemic cancer treatment when patient experience and health-care use are among the outcomes of interest. Its randomized design provides a framework for comparing groups, while its open-label status is important because participants know their assigned support. For a self-reported outcome, expectations and awareness of care arrangements may be relevant to measurement and interpretation.

The primary endpoint focuses on symptom interference rather than symptom severity alone. That distinction matters because a person’s symptoms and the effect of those symptoms on daily activities are related but different constructs. The endpoint’s six life domains also make clear that a summary score can combine several kinds of impact. The record’s note that the outcome has several categories or time points, together with the mean-versus-median description, highlights the importance of clearly specifying the comparison and summary measure.

The platform structure includes different clinical populations and treatment settings. Cohort A contrasts local standard-of-care support with DPM support alongside an intravenous atezolizumab regimen; Cohort B describes DPM use with subcutaneous atezolizumab across hospital and home settings. These descriptions raise questions about which comparisons are intended and how outcomes should be interpreted across distinct cohorts. The registry record does not provide a single reduced primary comparison or enough analysis detail to answer those questions.

The lack of numerical endpoint results is itself important for statistical interpretation. A design description and safety counts cannot replace effect estimates and uncertainty measures. The record’s terminated status, without a reported reason, further limits what can be inferred about study completion. This study therefore illustrates why transparent reporting of analysis populations, missing data, multiplicity, endpoint definitions, and results is necessary for assessing evidence from digital health evaluations.

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 has important limitations for interpreting this study. It is marked terminated, but the reason for termination is not reported in the registry record. Numerical results for the primary endpoint are also not reported, and the record notes that the outcome has several categories or time points without reducing it to one comparison. Consequently, the primary treatment-group contrast and its precision cannot be assessed.

The endpoint description is internally unclear: the title refers to a mean difference in change, whereas the outcome details list median and full range. No result or explanatory note resolves this. The record does not report a statistical analysis plan, a sample-size justification, interim analyses, a multiplicity approach, or details of covariate adjustment. The sample-size illustrations are described as planning exercises based on an observed effect, not as the trial’s original design basis.

Missing-data methods and follow-up completeness are not reported. Although the primary population is defined as all randomized participants, the record does not report the number analyzed for the Week 12 endpoint or explain how missing measurements were handled. The randomization procedure and any stratification are also not reported.

The two cohort descriptions differ in disease setting and treatment context, and the Cohort B description does not identify a separate comparator. The arm description for one Cohort A group is incomplete in the registry record. Safety counts are available, but the record does not report exposure duration, event timing, or the analysis method; the serious-event term percentages also lack a stated denominator in the listing. These limitations restrict interpretation of both the clinical outcome and the safety information.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Clinical Impact and Utility of Digital Health Solutions in Participants Receiving Systemic Treatment in Clinical Practice — NCT05694013.
• Registry record last updated 2025-10-22; status last verified 2025-10.
• Results first posted 2025-10-22.
• 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

The registry record describes a randomized, open-label, parallel platform study of digital health solutions during systemic cancer treatment. It lists phase 2 and phase 3, an enrollment of 49, Hoffmann-La Roche as sponsor, and a status of terminated. The listed dates are a start on 2023-02-27 and primary completion on 2024-06-30.

Cohort A involved participants with specified advanced cancers receiving an intravenous atezolizumab-containing regimen, comparing local standard-of-care support with support that included the Roche DPM Module. Cohort B involved participants with resected Stage IIB-IIIB non-small cell lung carcinoma using the DPM Module with subcutaneous atezolizumab. The primary endpoint concerns change from baseline to Week 12 in MDASI symptom interference. Other outcomes include quality of life, health status, symptom severity, and hospitalization due to serious adverse events.

Numerical primary-endpoint results, analysis details, missing-data methods, and the reason for termination are not reported in the registry record. The record lists serious and other adverse event counts and deaths for two groups.