# Identification and Description of Regulatory and Health Technology Assessment Agencies’ Guidance Related to Patient Experience Data in North America, Europe, and Asia Pacific

## TL;DR

This paper examines how regulatory and health agencies in North America, Europe, and Asia Pacific use patient experience data, finding differences that could lead to complex trials and burdens on patients.

## Contribution

The study identifies and compares guidance from regulatory and HTA agencies on patient experience data across three regions, highlighting misalignment and suggesting harmonization.

## Key findings

- Regulatory agencies mostly expect data collected via clinical outcome assessments and validated instruments.
- HTA guidance varies between countries, with some focusing on utility estimates and others on disease impact.
- Misalignment in PED expectations can lead to complex trial protocols and significant patient burden.

## Abstract

To identify and describe patient experience data (PED)-related guidance issued by regulatory and health technology assessment (HTA) agencies in North America, Europe, and Asia Pacific.

National agency websites were manually searched (October 2021, updated October–December 2023) to identify standalone PED guidance, and general submission/clinical trial or disease-specific guidance in four therapy areas, including PED-related recommendations. Identified documents were reviewed for requirements/expectations on types of PED; concepts of interest and instruments/tools; validation, analysis, and interpretation; study design, and endpoint definitions.

A total of 34 regulatory and 21 HTA documents were reviewed across 7 and 11 agencies, respectively. Most regulatory agencies expected data collected via clinical outcome assessments; concepts of interest were similar; specific tools were not usually recommended, although the use of validated instruments was expected; and meaningful within-patient change was generally considered key to interpretation. HTA guidance on PED varied primarily based on country requirement for utility estimates to inform economic evaluation (little/no guidance beyond the concept of interest) versus interest in PED on signs, symptoms, or disease impact on functioning (generally more comprehensive details on PED expectations).

Through PED, patient voice is an increasingly important factor in regulatory, access, and reimbursement decision-making. Regulatory and HTA agencies in North America, Europe, and Asia Pacific have varying expectations regarding PED. This misalignment can necessitate the collection of multiple types of PED to meet all requirements (resulting in complex clinical trial protocols and significant patient burden), and lead to differences in data interpretation or gaps in the expected data. Therefore, PED measurement standards should be harmonized globally, and the collection of required PED should be ensured early during drug development to ensure decisions can be made based on valid and reliable data.