Additional resources 

Sophie Staniszewska , Kirstie L Haywood, Jo Brett, Liz Tutton

Patient and public involvement in patient-reported outcome measures: evolution not revolution *

*Please note that this article is behind a paywall. However, the abstract is freely available.

Abstract

This paper considers the potential for collaborative patient and public involvement in the development, application, evaluation, and interpretation of patient-reported outcome measures (PROMs). The development of PROMs has followed a well trodden methodological path, with patients contributing as research subjects to the content of many PROMs. This paper argues that the development of PROMs should embrace more collaborative forms of patient and public involvement with patients as research partners in the research process, not just as those individuals who are consulted or as subjects, from whom data are sourced, to ensure the acceptability, relevance, and quality of research. We consider the potential for patients to be involved in a much wider range of methodological activities in PROM development working in partnership with researchers, which we hope will promote paradigmal evolution rather than revolution.

 

Soren Eik Skovlund, Lise H. Troelsen, Lotte Klim, Poul Erik Jakobsen, Niels Ejskjaer

The participatory development of a national core set of person-centred diabetes outcome constructs for use in routine diabetes care across healthcare sectors

 

Abstract

Background

This study sought to utilise participatory research methods to identify the perspectives of people with diabetes regarding which diabetes outcomes were most important to them. These findings were then used to support an expert working group representing multiple health sectors and healthcare disciplines and people with diabetes to establish a core set of patient-important outcome constructs for use in routine diabetes care.

Methods

26 people with diabetes and family members were recruited through purposive sampling to participate in interviews, focus groups, voting and plenary activities in order to be part of identifying outcome constructs. Content and qualitative analysis methods were used with literature reviews to inform a national multi-stakeholder consensus process for a core set of person-centred diabetes outcome constructs to be used in routine diabetes care across health care settings.

Results

21 people with diabetes and 5 family members representing type 1 and 2 diabetes and a range of age groups, treatment regimens and disease burden identified the following patient-reported outcome constructs as an important supplement to clinical indicators for outcome assessment in routine diabetes care: self-rated health, psychological well-being, diabetes related emotional distress and quality of life, symptom distress, treatment burden, blood sugar regulation and hypoglycemia burden, confidence in self-management and confidence in access to person-centred care and support. Consensus was reached by a national multi-stakeholder expert group to adopt measures of these constructs as a national core diabetes outcome set for use in routine value-based diabetes care.

Conclusions

We found that patient-reported outcome (PRO) constructs and clinical indicators are needed in core diabetes outcome sets to evaluate outcomes of diabetes care which reflect key needs and priorities of people with diabetes. The incorporation of patient-reported outcome constructs should be considered complementary to clinical indicators in multi-stakeholder value-based health care strategies. We found participatory research methods were useful in facilitating the identification of a core prioritised set of diabetes outcome constructs for routine value-based diabetes care. The use of our method for involving patients may be useful for similar efforts in other disease areas aimed at defining suitable outcomes of person-centred value-based care. Future research should focus on developing acceptable and psychometrically valid measurement instruments to evaluate these outcome constructs as part of routine diabetes care.



 Lesson 2, Table 1
Major concepts measured in PROs’

Adapted from: https://eprovide.mapi-trust.org/about/about-eprovide


Lesson 2, Table 2
Important properties of PROMs



GUIDANCE DOCUMENT

Principles for Selecting, Developing, Modifying, and Adapting Patient-Reported Outcome Instruments for Use in Medical Device Evaluation Guidance for Industry and Food and Drug Administration Staff, And Other Stakeholders
Issued by:
Center for Devices and Radiological Health
Center for Biologics Evaluation and Research

FDA (The Food and Drug Administration) is issuing this guidance to describe principles that should be considered when using Patient-Reported Outcome (PRO) instruments in the evaluation of medical devices and provide recommendations about the importance of ensuring the measures are fit-for-purpose. This guidance is not meant to replace the Patient-Focused Drug Development (PFDD) guidance series.