Core Outcome Measures in Effectiveness Trials

Importance of hemodialysis-related outcomes: comparison of ratings by a self-help group, clinicians, and health technology assessment authors with those by a large reference group of patients

General Information

Background: The selection of important outcomes is a crucial decision for clinical research and health technology assessment (HTA), and there is ongoing debate about which stakeholders should be involved. Hemodialysis is a complex treatment for chronic kidney disease (CKD) and affects many outcomes. Apart from obvious outcomes, such as mortality, morbidity and health-related quality of life (HRQoL), others such as, concerning daily living or health care provision, may also be important. The aim of our study was to analyze to what extent the preferences for patient-relevant outcomes differed between various stakeholders. We compared preferences of stakeholders normally or occasionally involved in outcome prioritization (patients from a self-help group, clinicians and HTA authors) with those of a large reference group of patients.
Participants and methods: The reference group consisted of 4,518 CKD patients investigated previously. We additionally recruited CKD patients via a regional self-help group, nephrologists via an online search and HTA authors via an expert database or personal contacts. All groups assessed the relative importance of the 23 outcomes by means of a discrete visual analog scale. We used descriptive statistics to rank outcomes and compare the results between groups.
Results: We received completed questionnaires from 49 self-help group patients, 19 nephrologists and 18 HTA authors. Only the following 3 outcomes were ranked within the top 7 outcomes by all 4 groups: safety, HRQoL and emotional state. The ratings by the self-help group were generally more concordant with the reference group ratings than those by nephrologists, while HTA authors showed the least concordance.
Conclusion: Preferences of CKD patients from a self-help group, nephrologists and HTA authors differ to a varying extent from those of a large reference group of patients with CKD. The preferences of all stakeholders should form the basis of a transparent approach so as to generate a valid list of important outcomes.

To analyze to what extent the preferences for patient-relevant outcomes differed between various stakeholders.

Inger M Janssen, Fueloep Scheibler, Ansgar Gerhardus


Patient Preference and Adherence
Further Study Information

Not stated
Funding source(s):
We acknowledge support for the Article Processing Charge by the Deutsche Forschungsgemeinschaft and the Open Access Publication Fund of Bielefeld University.

Health Area

Disease Category
Kidney disease

Disease Name

Target Population

Age Range


Nature / type of Intervention



Stakeholders Involved

Clinical experts
Consumers (patients)
Patient/ support group representatives

Study Type

COS for clinical trials or clinical research
COS for practice

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