P217: Patient Disease Strategy: A Framework for Collecting and Applying Patient Experience Data into Clinical Development Programs

Poster Presenter

Catherine Coulouvrat

Head of Health Value Translation
Sanofi
France

Objectives

Authors: Catherine Coulouvrat, Keri Brady, Stephanie Bascle, Laurence Lucats, Sophie van Tomme, Benoit Arnould.

Method

Objective: Successful clinical development programs hinge on comprehensive, continuous collection of patient experience data (PED) to inform trial design, select meaningful endpoints for patients, support benefit/risk evaluations and identify unmet needs.

Results

Sponsors require additional guidance on the timing/process for eliciting and integrating PED into product development. The Patient Disease Strategy (PDS) provides one such framework. Methods: PDS is the gathering of PED for an indication that is meaningfully incorporated early in development to support asset value, differentiation and trial optimization. The PDS is initiated in the pre-clinical phase and applied/adapted throughout development. The three PDS phases include 1) developing the patient profile, 2) conducting PED gap analysis to identify patient priorities, differentiation criteria, labeling opportunities, diversity/inclusion (D&I) considerations, and recruitment/retention strategies, all within the context of development timelines, and 3) generating output to bridge gaps and inform decision making. Results: The PDS begins with characterization of the patient profile in terms of epidemiology (e.g., prevalence/incidence, average age of disease onset), demographics (e.g. racial/ethnic representation), patient journey, disease/treatment burden, unmet needs (e.g., symptoms untreated by available therapies) and patient expectations for new therapies. The PED gap analysis determines current levels of knowledge and identifies areas of further exploration (e.g., patient priorities, differentiation of development assets compared to available treatments, diversity of target populations). The final step focuses on bridging gaps with patient narratives, clinical outcome assessment (COA) evidence generation, D&I strategy, and other methodologies, e.g., patient preference studies, to inform development programs.

Conclusion

The PDS provides a framework for eliciting PED at the disease level throughout research and development to support an asset’s value, differentiation, and clinical operational challenges which impact program success and patient access. The PDS supports the design of patient-centered clinical development programs that can deliver treatments that meet the needs and expectations of patients.