Page 49 - Let them speak Towards a patient reported outcome measure set for

Outcomes and instruments
Strengths and limitations
This scoping review is the first overview of outcomes and outcome
measurement instruments used in clinical trials in GNDs and ID, examining
the broad array of outcomes related to health manifestations common in
these patient populations, using state of the art classifications. However,
when conducting this review, we faced some challenges. We initially aimed
to cluster the outcomes and outcome measurement instruments according
to the Valderas and Alonso model.12 Domain assessment has rather been
an indication, as instruments should ideally be assessed per subscale
(unidimensional), which was not feasible due to the enormous amount of
different outcome measurement instruments. Furthermore, the terminology
used for the outcomes and outcome measurement instruments was
often unclear, lacking, or inconsistently reported. We clustered reported
outcomes based on frequency of used terminology, and thus do not refer
to a standardized terminology. Finally, we cannot recommend specific
outcome measurement instruments, because psychometric properties
were not investigated in this review.
2
Conclusion
This review provides insight into the large number of outcomes and
outcome measurement instruments reported in clinical trials for GNDs
and ID. The abundancy of available tools is problematic from an efficiency
and generalizability perspective, highlighting the need for a more universal
approach to the selection of outcomes and instruments. Moving forward,
further collaborative efforts are recommended to achieve consensus on
outcome selection. The output of this review may serve as a starting point
for discussion about relevant outcomes and instruments in GNDs and ID,
and to develop a core outcome set for these populations. Preferably, it will be
applicable for care as well as research purposes with possible implications
for market authorization and reimbursement of (orphan) drugs to improve
patient-centered care by measuring what matters to affected individuals.
Acknowledgements
None.
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