Data Collection
Multinational HEOR study among patients
Respondents: Patients recruited through Patient Advocacy Groups or KOLs
Dissemination of results:
Typically, 4 manuscripts to publish all results
What for?
De novo data collection among patients to collect all HEOR endpoints for economic modelling, and document the burden of disease and unmet need
OBJECTIVES
Inform the economic model
Highlight the unmet need, through comparison with the general population
Examine associations with disease severity
Document the impact of the disease on different dimensions of HRQoL
Evaluate the burden of caregivers
Calculate country-specific utility values
Assess the economic and societal burden of an illness
PROCESS
Strategic advice on study design​
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Which outcomes to include
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Frequency and combination of measurements
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Clinical, PROM and HCRU outcomes
Ethical committee submission
Liaising with PAGs or doctors
Contracting, licensing
Professional online data collection tool
Analysis and publication of results
Concordance between patient and physician on scoring PROs
Respondents: Dyads of patients and their physician
What for?
Clinical data collection among patients and their physician, to validate the proxy versus self-measurement of a clinical outcome, and understand the concordance of items and total score
Caregiver Burden study
Respondents: Dyads of patients and their caregiver
What for?
Observational data collection among patients and caregivers to document the burden that caregivers experience, and whether this burden is associated with disease severity
Study among the General Population to measure HEOR endpoints
Respondents: Representative members of the general population
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​The Study Protocol for GENESIS: GENEral population normS—an International Survey to establish normative values for PROMs in CIDP, MMN, and TED. medRxiv. Submitted September, 2025
What for?
Obtain reference values of the PROMS and clinical endpoints measured in your RCT and observational studies, for better interpretation of the scores and documenting the unmet need
Time and Motion (T&M) Workflow Mapping
Interviews or Survey
Respondents: Practicing healthcare professionals.​
Workflow Mapping of Nebulized COPD Therapy in Inpatient and Long-Term Care (LTC) Settings in the U.S.: a Precursor to an Observational Time and Motion (T&M) Study. Respiratory Effectiveness Group Summit, March 18–20, 2021; virtual, United States.
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What for?
Gaining in-depth understanding of one or more healthcare delivery workflows and eliciting healthcare professional’s time on various tasks.
Insights can be disseminated and/or used to design a T&M observational study.
Time and Motion (T&M) Observational Study
(Single or Multi-Country)
Multi-country global study alongside a Phase 3 trial:
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Real-world multi-country study including activity diary approach:
What for?
For single or multiple countries, accurately quantify active healthcare professional time and patient time for one or more workflows, to generate compelling efficiency-based value messages.
Respondents: Healthcare professionals, patients (passive participants)
Synthetic Control Arm (SCA) Feasibility
In-depth feasibility allows to:
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Determine number of potentially
eligible patients during study
eligibility period.
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Determine which Phase III endpoints are part of routine care in population of interest (e.g., specific focus on survival outcomes)
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Understand how data are housed, type of Electronic Medical Records (EMR) system used, and possibility for automated data import
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Understand how data are housed, type of used, and possibility for automated data import
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Understand level of interest in participating in a retrospective chart SCA study
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Assess availability of local resources (data abstractors)
Respondents:
Practicing healthcare professionals​
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What for?
Facilitate a formal evaluation of the patient cohort, medical chart data availability and site infrastructure for the execution of a chart review study and build a synthetic control arm.
Retrospective Chart Review Study
Respondents:
Patients (pseudonymized secondary data)
What for?
Tailored extraction of existing health-related data via automated electronic medical record (EMR) abstraction or manual chart review
Evidence Generation
Strategic Planning
Specific objectives:
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Report insights from Sponsor
internal stakeholders regarding
product strategic imperatives and
identify key data/evidence gaps
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Report insights from external stakeholders (e.g., FDA/EMA, HTA, clinical KOLs) regarding their evidentiary needs related to Sponsor’s product and determine relative importance of data needs
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Determine relative suitability of study designs by data need
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Provide recommendations on potential research design(s), including rationale, objectives, and top-line methodology for each
What for?
To support the launch of an asset, determining what evidence is needed for which stakeholders at what time is critical but complex. This is achieved via strategic research resulting in a prioritized list of study designs.
Respondents:
Sponsor internal and external stakeholders​
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