Page 195 - Let them speak Towards a patient reported outcome measure set for

Evaluating the core PROM set
Study limitations
A limitation of this study is that we used adult self-report versions for
caregivers of adults, as proxy-report versions are not yet available for this
group. Although caregivers were instructed to complete the questionnaires
on behalf of the affected individual, the first person wording may have
caused confusion and some may have responded for themselves.
Nonetheless, most PROMs showed moderate to high correlations with
the legacy subscales, suggesting that caregivers largely responded for the
affected individual rather than themselves.
Another limitation is that we reported only whether scores of adults and
caregivers of children were within the normal range or deviated from the
general population. This approach was necessary as mean scores for
caregivers of children were not always present for PROMIS v1.0 and PROMIS
v2.0 instruments, and we aimed to present deviations in an unambiguous
way. Consequently, we cannot/did not establish whether these scores
differed significantly from those of the general population.
Strengths of the core PROM set in care and research
Eventually, we aim to implement the GenID core PROM set in both care
and research, to better capture the impact of GenID on daily life. Compared
with legacy questionnaires, the core PROM set offers several advantages. It
captures PROs that matter to individuals with GenID and, by reducing floor
and ceiling effects, it can allow more sensitive detection of health problems.
It often requires fewer items to reliably assess health and functioning,
reducing the burden for both affected individuals and caregivers. PROMIS
T-scores can be compared within and between GenID groups and with the
general population. In addition, the questionnaires are free or low-cost and
therefore more accessible to implement. This core PROM set is essential
for GenID care and research to reduce the wide variety of PROMs used and
to systematically include the patient perspective. In GenID care, the selected
core PROM set can be used to monitor relevant aspects of daily functioning,
detect health problems, and guide personalized treatment. In research, the
set can provide insight into disease progression and treatment effects, all
from the perspective of individuals with GenID or their caregivers. Both the
European Medicines Agency (EMA) and the Food and Drug Administration
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