Page 48 - Let them speak Towards a patient reported outcome measure set for

Chapter 2
through short forms or CAT.41,63 Furthermore, it is recommended to attempt
to include (user-friendly) PROMs to acquire information from the patient
perspective, as also encouraged by regulatory authorities such as the FDA
and European Medicines Agency (EMA).72,73
Future outcome measure landscape
Because of the overgrowth of available outcome measurement instruments,
clinical researchers need guidance in choosing appropriate outcome
measures in clinical trials. Regulatory agencies, such as the EMA and
FDA, encourage maintaining consistency in assessment methods and are
placing focus on capturing the patient experience, but poorly defined PRO
objectives have hindered the utility of PROs in regulatory decisions.68,74 A
core outcome set or generic measure with disorder-specific or comorbidity-
specific extensions may provide a solution to ensure generalizability and
interpretation, and effectively target-specific phenotypes in individuals
with GNDs and ID. To move from this ‘mess of measures’ to a more unified
approach for future interventional research for GNDs and ID, the field could
take the following steps:
y Reach (international) consensus on outcomes (e.g. Delphi
procedure) and establish a core outcome set for individuals
with GNDs and ID: terminology and constructs should be
relevant, clear, harmonized and operationalized, in collaboration
with affected individuals, caregivers, and (methodological and
clinical) experts.
y Reach (international) consensus on the most suitable
instruments to be selected per outcome, taking into account
relevance, applicability, patient preferences, validity, reliability,
responsiveness to change, (strategies for controlling) learning
effects, and language and culture barriers. Some instruments
may need to be adapted to individuals with ID.
y Implement the core outcome set or (ID-friendly) generic
measure(s) with appropriate versions for different levels of
ID. This could be extended with disorder- or comorbidity-
specific measures (e.g. symptom checklists) to cover relevant
condition-specific aspects.
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