Page 137 - Let them speak Towards a patient reported outcome measure set for

Establishing the core PRO set
relevant PROs for patients, care, research, and policy can better align with
their concerns and needs. For example, if anxiety is identified as a relevant
PRO, care pathways can be developed to address it more effectively.
PROs can be measured with patient reported outcome measures (PROMs).
PROMs are standardized questionnaires that allow patients to report on
various aspects of their health status without interference of a clinician
or anyone else (FDA, 2006). Incorporating PROMs in care gives clinicians
greater insight into individuals’ daily functioning and overall well-being,
allowing them to offer personalized treatment and improve overall care
strategies (Basch et al. 2013). In addition, incorporating PROMs into
research provides comprehensive data on treatment efficacy and disease
progression beyond the traditional outcomes (FDA, 2006). This PRO data
can subsequently inform care and guidelines, shape research agendas and
health policy, and support drug approval and treatment reimbursement
decisions (Rivera et al. 2019).
Regulatory agencies such as the European Medicines Agency (EMA) and
the Food and Drug Administration (FDA), along with patient organizations,
increasingly advocate for integrating PRO data into drug development and
regulatory processes (Compendium, 2022; EMA, 2022; FDA, 2024). Recent
initiatives in the United States and Europe also reflect this priority. For
example, the European Rare Disease Research Coordination and Support
Action (ERICA) established a repository to harmonize and centralize clinical
outcome instruments, including PROMs, for rare diseases (ERICA, 2023).
Similarly, the Rare Disease Clinical Outcome Assessment Consortium (RD-
COAC) identified scientifically robust instruments, including PROMs, to
collect clinically meaningful data in rare disease trials (RD-COAC, 2023).
Clinical trials, including those involving individuals with GID, are also
incorporating PROMs to assess treatment efficacy from the patients’
perspective (Crossnohere et al. 2025; Djukic et al. 2016; Maeder et al. 2022;
Müller, Den Hollander, et al. 2024).
The growing focus on PROs amplifies patients’ voices and makes outcomes
potentially more relevant. However, currently a wide array of PROs is
being measured for GID (Müller, van Silfhout, et al. 2024; van Silfhout, van
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