Page 94 - Let them speak Towards a patient reported outcome measure set for

Chapter 4
Strengths and limitations
A strength of this study is the inclusion of not only clinical trials, but also
condition-specific COSs, which made it possible to provide an extensive
overview of PROs used for GID. A limitation of this study is that the clinical
trial data is extracted from a previous published scoping review, and it may
be possible that errors have occurred during the process of extraction and
subsequent summarization. Moreover, because there are so few GIDs for
which clinical trials exist, the yield of PROs used in clinical trials is limited
and biased toward a few conditions such as Down syndrome, Fragile
X syndrome, Prader-Willi syndrome, and Tuberous Sclerosis Complex.
Furthermore, despite the almost 2000 hits after the literature search, only
five condition-specific COSs were included. This could either mean overly
strict study eligibility criteria or poor quality of study reporting. Lastly, scoping
reviews have a descriptive nature and usually do not assess the quality of
the included studies (37). However, assessing the quality of the studies could
enhance our understanding and assessment of the PROs found.
Future steps
Eventually, by utilizing a generic core PRO set for GID it becomes feasible to
combine and compare PRO data within and between specific GID subgroups.
Additionally, it ensures the measurement of PROs that are relevant to the
GID population. Therefore, further work is needed to investigate whether
the PROs identified by these two scoping reviews are indeed most relevant
to individuals with GID, or whether there are still potentially relevant
PROs missing. Involving individuals with GID and their caregivers as both
participants and co-researchers is crucial. Future steps include focus
groups and interviews with individuals with GID, caregivers, and experts
(e.g., healthcare professionals). After identifying all potentially relevant
PROs, these PROs need to be classified within a conceptual framework,
defined in detail, and then prioritized by important stakeholders, such as
individuals with GID, their caregivers, and experts. Prioritizing PROs will be
done through a Delphi survey and consensus meetings. The prioritization
of PROs will lead to a well-defined generic core PRO set applicable to the
whole GID population. Potential condition-specific PROs should also be
identified and incorporated into the generic core PRO set for particular
GIDs. Subsequently, suitable instruments, PROMs, must be selected to
measure the generic core PRO set, which will form the generic core PROM
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