Article by: Florian Eichmann, PhD, Principal Late Stage, Scientific Affairs and Real World Evidence, inVentiv Health Clinical, Munich, German
Regulators and Health Technology Assessment (HTA) agencies – and thus manufacturers and research stakeholders –
are showing increasing interest in real world information, optimally from the patient perspective, collected with high evidence validity, and readily available for analysis. This has led to competing health research trends.
Regulators and Health Technology Assessment (HTA) agencies – and thus manufacturers and research stakeholders –
are showing increasing interest in real world information, optimally from the patient perspective, collected with high evidence validity, and readily available for analysis. This has led to competing health research trends.
To meet stakeholders’ requirements, and to save time and money, we must identify the optimal study design for a given research question. Direct-to-subject studies collect clinical, subject-reported, or economic information directly from the research subject: Instead of relying on indirect data sources such as health care providers or administrative databases, direct-to-subject studies, virtual trials, or subject-based research provide direct access to individual health information from the subject’s perspective.
Regulatory and reimbursement stakeholders increasingly require subjects’ perspectives to be included into the evidence portfolio supporting their decisions. Nevertheless, direct-to-subject studies are still falling behind relative to database approaches or site based trials. This article outlines strengths of subject-based research and indicates how to enhance direct-to-subject designs to maximize real world evidence validity.
KEY POINTS:
- Direct-to-subject research collects information directly from the research subject and provides valuable disease and treatment pattern information for Public Health, communication, or reimbursement purposes.
- Active Surveillance and Pregnancy Registries are exemplary direct-to-subject study designs meeting regulatory post marketing requirements.
- Validating outcomes is a primary quality aspect for subject based research, as demonstrated in a Design/Quality Matrix (DQM).