Presentation (CTI)

OBJECTIVES: Real-World Evidence generation through direct-to-patient (DTP) studies research offers several strengths, i.e., customizable design that captures real-world patient experience in a timely and cost-efficient manner, providing robust insights into patients’ unmet needs, humanistic burden, and allows for validated Patient-Reported Outcome Measures (PROMs), which are increasingly valued by regulators, payers, and health technology assessment (HTA) bodies. DTP studies collect self-reported data through online self-administered, or in-person, surveys. Self-reported selection criteria may increase risk of misclassification bias which occurs when participants or variables are included and categorized (e.g., diseased vs. healthy), potentially impacting research results. To mitigate bias, measures could be implemented at the design stage to confirm diagnosis.
METHODS: In DTP studies, participants may be recruited for screening and inclusion from various sources, e.g., healthcare professionals’ referral, patient advocacy groups, or custom recruitment. An additional layer of robustness was added to screening, by requesting that consenting eligible participants, provide a confirmation of diagnosis (COD). A COD may be a physician note, prescription, or pathology report, and is objectively assessed by trained research personnel, to verify authenticity and confirm inclusion for study participation.
RESULTS: COD adequately balanced external with internal validity and mitigated misclassification bias. However, some important challenges were noted related to regulatory and compliance policies, and data privacy concerns. To address this, COD handling was meticulously protocolled following local regulatory bodies guidelines. CODs were managed by an independent third party who verified the information, redacted personal identifiers, then stored the anonymized COD on a restricted-access platform.
CONCLUSIONS: One of the strengths of decentralized DTP research design is the ability to access broader, more diverse populations. The use of a COD may add to the many strengths of DTP studies when employed in certain cases. Incorporating COD can raise the methodological standard for future real-world DTP studies, promoting best practices.