Partnering With Patients: Alignment on Terminologies and Definitions for Global Harmonization

OBJECTIVES: Measuring what is most meaningful to patients is increasingly recognized as an essential component of research design and conduct. To support this imperative, health authorities have recently published guidelines for industry best practices for patient centric research (PCR). This signifies a shift from past rhetoric and intended patient-centricity to a new model of active patient partnership for drug development. This abstract presents the first output of ongoing research aiming to landscape PCR related guidelines and recommendations and to identify terminologies and highlight discrepancies and commonalities among term definitions.

METHODS: A targeted review searching the web-based sites of participating regulatory authorities for the International Consortium for Harmonization (ICH) identified relevant publications for inclusion. A structured extraction template was developed. Thematic analysis methods were used to code elements in terminologies and in their respective definitions to categorize into subthemes and themes.

RESULTS: 25 full text publications were screened. 16 were included. Median publication year: 2022 (IQR: 2021, 2023). 28 PCR related definitions were identified, coded and analyzed for commonalities and discrepancies. They were related to 12 terms that were grouped into 4 thematic categories: 1. Patient-Focused Drug Development (PFDD) (1 term, 12 consistent definitions across 12 publications), 2. Patient Engagement (4 terms [patient engagement, patient involvement, patient voice, patient input], 6 definitions across 5 publications), 3. Patient-Experience Data (PED) (3 terms [patient experience data, patient evidence, patient experience evidence], 5 definitions across 3 publications) and 4. Patient-Reported Outcomes (4 terms [patient-reported outcomes, patient centered outcomes, patient preferences, patient preference studies] 5 definitions across 2 publications). The thematic category PFDD was the most recurrent overall with a single term and almost consistent definition.

CONCLUSIONS: Given the dynamic nature of patient priorities and evolving treatment landscapes, the global ICH multistakeholder collaboration to achieve alignment for PCR design and methods is timely and essential.