Mitigating Evidence Gaps: A Survey Template to Inform Patient-Centered Value Assessment

Abstract

Background: Necessary and informative patient-experience data (PED) needed for value/health technology assessments (V/HTA) are rarely available as they are not routinely collected in clinical trials, the traditional data source for V/HTA. Patient organizations frequently collect PED; however, inconsistent approaches in the methods and type of data collected limit their usefulness for V/HTA. A universal framework, such as a standard, disease-agnostic survey template, could coalesce efforts for collecting patient-centric data for application to V/HTA for more efficiency, alignment, and better patient centricity.

Objective: Using a mixed-methods approach and by identifying PED concepts common across conditions (i.e., cross-cutting PED concepts), co-develop a disease-agnostic survey template that can be used to inform a standardized approach to fill patient-centered evidence gaps in V/HTA.

Methods: This mixed-methods study used a triangulation of data sources: (1) Voice-of-the-Patient reports, (2) peer-reviewed literature, (3) V/HTA reports, (4) publicly available surveys used by patient organizations to collect and submit data for V/HTA, (5) elicitation from patient experiences with conditions and associated treatments, and (6) patient-advisor and other expert input to identify cross-cutting PED concepts and draft a disease-agnostic survey template. Cognitive interviews with patients were conducted to test and refine survey items and response choices. Following, the draft survey was pilot tested to evaluate and finalize the disease-agnostic survey template by assessing usability from the patient perspective.

Results: The resulting survey template is comprised of thirty-one items asking patients about the impact of disease and treatment on health and daily life such as but not limited to symptoms, treatment and treatment-related experiences, ability to work, wellness, finances, healthcare utilization, condition stability, treatment preferences, healthcare and provider experiences, access to healthcare services and treatment, experiences living with the condition, impact on daily life, support from others, impact on others, and additional patient insights.

Conclusions: The resulting survey template supports standardized PED collection while offering flexibility to tailor to a condition or population. Continuous patient engagement throughout the development and testing process increases the survey template’s utility as it reflects what patients, who actually live with a condition, experience and think.