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P239: Choosing the Right Design: A Guiding Framework for Using Direct-to-Patient, Hybrid and Conventional Site-Based Study Designs

Poster Presenter

Mui Van Zandt

VP/Global Head, Data Strategy, Access and Enablement
IQVIA
United States

Objectives

Given the increasing importance and use of patient-centric and decentralized approaches, a framework was developed to help researchers choose and consider between direct-to-patient, hybrid and conventional site-based study designs for longitudinal real world research with primary data collection.

Method

Ten experts involved in recommending study design approaches for more than 100 real world studies annually for the life sciences industry were consulted on appropriate study designs for a range of real world study types and therapy areas. [Co-authors: A Pitcher, I Bonzani, S Kishnani, E Oakkar]

Results

A decision tree framework was created to guide clinical researchers on study design decisions considering study purpose, regulatory requirements and the patient and data journey for real world studies with the goal of minimizing study participation burden, maintaining data quality and optimizing study delivery efficiency. The study designs included: direct-to-patient (DtP), conventional site-based and hybrid design. DtP study designs are those that are conducted fully virtual, with no involvement of traditional research sites. Hybrid studies contain some element of site involvement, e.g., to recruit and consent participants. Conventional site-based studies are conducted fully via traditional research sites. The framework suggests the most appropriate study design model based on the answers to the following questions: Population •Can enough participants be identified and recruited without research site involvement? •Is there a concern about not being able to recruit a diverse/representative study population by focusing on traditional research sites? •Can enough sites be opened to identify and recruit the required number of patients? •Can study eligibility be determined without site involvement? Patient and data journey •Does the target population regularly visit traditional research sites in standard of care? •Are there any clinical assessments, tests or interventions required as part of the study design that require site visits? •Are routine patient care and needed data points spread across multiple care settings? •Can all relevant data points be reported or mediated via the patient, caregiver or via virtual site services? •Are site-based methods of engagement and retention sufficient for reliable data collection? Regulatory considerations •Is a face-to-face visit needed for informed consent based on the study population, study design and geographies? •If safety endpoints are being collected/monitored, can they be reported using a DtP approach?

Conclusion

While several internal and external factors may be taken into account when choosing direct-to-patient, hybrid or site-based study design models, clinical researchers should have a good understanding of the patient and data journey and seek to align data collection with a patient’s routine care to minimize the patient burden associated with participating in research. This may require the use of digital technologies and virtual services to engage patients and collect data away from traditional sites data collection or the provision of study services directly to a patient’s home or local care setting. Furthermore, country-specific regulatory considerations may impact which study elements may be possible with direct-to-patient approaches or how they should be operationalized. This decision framework will continue to be monitored and re-evaluated as the technology, healthcare management and regulatory landscape changes over time.