For clinical trials
Endpoint Development and Validation
Develop measures that can answer your trial's clinical question.
BioSensics designs and conducts clinical research to develop candidate measures and evaluate whether they are fit for a specified trial use. We combine digital assessments, clinical measures, participant reports, and selected biological biomarkers to study measurement performance, clinical relevance, and change over time.
Build the evidence behind your endpoint.
Purpose-designed studies and targeted analyses connect the measure, the patient population, and the clinical question.
Can this measure become an endpoint for your trial?
The answer requires evidence in the intended population, setting, and assessment schedule. We develop the measure and conduct studies to test its performance, establish its clinical interpretation, and identify the evidence needed before trial use. The result is a defined measure, documented validation findings, and a research path matched to the proposed use.
Clinical research to develop and validate the measure
Study designs follow the evidence question: reference comparisons, repeated assessments, cross-sectional clinical comparisons, and longitudinal follow-up.
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Develop and test the measure
Define what the measure captures, how it is collected and calculated, and the proposed endpoint variable. Pilot the assessment procedures and refine them before evaluating performance in the intended population.
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Establish accuracy and reliability
Compare measurements with appropriate reference methods and evaluate repeatability under defined conditions. Study sources of error and variability, including collection setting, device placement, and day-to-day variation.
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Evaluate clinical relevance
Examine relationships with clinical assessments, participant reports, and relevant biological biomarkers. Test whether the measure reflects the targeted aspect of function or disease and distinguishes clinically relevant groups.
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Measure longitudinal sensitivity
Use repeated assessments to quantify change, variability, and floor or ceiling effects over a relevant interval. Evaluate whether the collection schedule can capture disease-related change in the intended trial population.
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Interpret meaningful change
Study change against suitable clinical or patient-reported anchors, supported by distribution-based analyses. Estimate meaningful-change thresholds when the evidence supports them, with uncertainty and population-specific interpretation documented.
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Evaluate trial suitability
Assess participant burden, adherence, missing data, and operational consistency. Translate findings into collection and analysis requirements, and design further research where treatment responsiveness or another requirement remains unresolved.
Evidence your team can use
Deliverables reflect the agreed studies and proposed trial use.
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Measure and endpoint specification
A documented measure definition, assessment procedures, calculation rules, proposed endpoint variable, and intended population and setting.
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Study protocol and analysis plan
Research questions, study design, comparators, assessment schedule, sample-size rationale, and planned analyses aligned with the evidence needed.
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Validation findings
Results on measurement performance, clinical relationships, longitudinal change, and interpretation, with uncertainty, limitations, and applicability to the proposed use.
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Trial translation and next studies
Recommendations for trial implementation, remaining evidence gaps, and the next validation studies. Scientific and technical documentation can support sponsor regulatory discussions.
From research to trial use
Examples illustrate different stages of endpoint development and translation. Regulatory status is specific to each measure and proposed use.
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Natural history to Phase 2
Multiple system atrophy
PAMSys measured physical activity and posture in bioMUSE, Alterity Therapeutics' natural history study in multiple system atrophy. The measures were subsequently used as a secondary endpoint in the ATH434 Phase 2 trial.
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Cohort research to the FDA ISTAND program
Charcot-Marie-Tooth disease
Gait, balance, foot drop, and at-home activity measures were developed in cohort studies of adults with CMT in collaboration with the Hereditary Neuropathy Foundation, and defined as wearable foot drop metrics for adults with CMT1A. The FDA accepted the Letter of Intent into the FDA ISTAND program in August 2026.
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Longitudinal research to trial deployment
Progressive supranuclear palsy
Twelve-month natural history studies characterized wearable ambulation and digital speech measures in PSP. FDA has accepted PAMSys into the FDA ISTAND program as a drug development tool for measuring ambulation in PSP clinical trials. The Letter of Intent for BioDigit Speech measures in PSP has been accepted for review. The measures are being integrated into the PSP Trial Platform and have been deployed in a pharmaceutical sponsor's Phase 1 study.
A study program built around the evidence gap
Collaborate from the initial research question through study conduct, analysis, and interpretation.
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Define the intended use
Agree on the clinical question, population, trial setting, assessment interval, and proposed role of the endpoint.
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Design the research
Review available evidence and design focused studies. Broader disease-course questions can be addressed through Natural History Studies.
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Conduct and analyze
Collect data under the agreed protocol, monitor quality, and complete planned analyses with applicable ethics review, consent, and data controls.
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Translate the findings
Document what the evidence supports, specify trial implementation requirements, and define the next studies or regulatory-support activities.
Planning your research
How does this differ from Natural History Studies?
Natural History Studies characterize disease course, support discovery, and develop cohorts. Endpoint Development and Validation asks whether a particular measure is fit for a specified trial use. Natural history data can contribute evidence to that focused program.
Can the research include biomarkers and outcome measures?
Yes. The intended use determines whether a measure is evaluated as a biomarker or a clinical outcome assessment. Studies can combine digital measures, clinical assessments, participant reports, and biological biomarkers, including blood biomarkers.
Can existing data answer our validation questions?
Relevant BioDigit Database or sponsor data may address focused questions. We assess population, assessment, comparator, and follow-up coverage, then design prospective studies to fill evidence gaps.
Does sensitivity to disease progression establish treatment responsiveness?
No. Detecting longitudinal disease-related change does not establish sensitivity to treatment effects. Treatment responsiveness requires suitable interventional evidence; meaningful-change interpretation also requires appropriate clinical or patient-reported anchors.
What happens after the validation study?
Findings guide further validation, evaluation in an interventional study, or trial implementation. Regulatory Support can organize evidence for the proposed use, while Clinical Trial Operations supports deployment.
Connect your research with the right support
Coordinate complementary research, data, and scientific expertise around your program.
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Natural History Studies
Characterize disease course, investigate candidate biomarkers, and build longitudinal cohorts for future research.
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BioDigit Database
Assess existing digital, clinical, and selected biological data for relevance to your research question.
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Data Analysis and Scientific Reporting
Connect study data with documented analyses, clear findings, and scientific reports.
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Regulatory Support
Organize evidence for discussions about the proposed use of digital measures and study findings.
Develop the evidence for your next endpoint
Share your candidate measure, indication, target population, and intended trial use. We will shape the clinical research needed to develop and evaluate it.