Page 70 - Let them speak Towards a patient reported outcome measure set for

Chapter 3
steps outlined in this protocol should result in a classified and well-defined
generic core PRO set relevant to the whole GID population. Moreover, it will
also become clear which outcomes do not qualify as a PRO, and should
therefore be measured with alternative instruments. The next step involves
the selection of suitable PROMs with optimal psychometric properties
which sufficiently measure the generic core PRO set: the generic core PROM
set. As GID is a heterogeneous population, condition-specific PROs will be
necessary for specific GID subgroups. Additionally, there may also be a need
for specific PROs for other clinically significant subgroups (e.g., age, ID level)
that are not sufficiently covered by the generic core PRO set. Therefore, it is
crucial to explore these subgroups to identify potential condition-specific or
other subgroup-specific PROs. If necessary, specific questions or PROMs for
specific genetic conditions or other subgroups will be added. Subsequently,
the generic core PROM set will be validated within the GID population (e.g., by
measuring reliability, internal consistency, and responsiveness of the PROMs),
and implemented in care and research. The validation and implementation
of the core PROM set will be done in collaboration with experienced
methodologists and implementation scientists (53-55). Consideration will be
given to known PROM implementation barriers (55-57).
Necessity of a core PRO(M) set
The development of the generic core PRO(M) set forms the first step in
standardizing personalized measurement in care and research for children
and adults with GID (58). This novel and state-of-the-art generic core PRO(M)
set will enable consistent and standardized measurement of relevant PROs
across different GID groups in both care and research. This reduces the
great diversity of PROs measured, simplifying the aggregation and analysis
of PRO data. Moreover, this approach ensures an ongoing integration of
patient perspective in care and research, leading to a better understanding
of the overall impact and the variations in impact on daily functioning among
different GIDs, or other subgroups of interest such as those affected by
shared comorbidity such as autism spectrum disorders. The necessity to
integrate PRO data, especially in research areas such as drug development
and disease-modifying therapies (59), resounds not only from patients
themselves, but also from healthcare professionals and policymakers
including the European Medicines Agency (EMA) and the Food and Drug
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