Patient-reported Outcomes as Endpoints in Clinical Trials of Kidney Transplantation Interventions

Allison Tong; Rainer Oberbauer; Maria Irene  Bellini; Klemens  Budde; Fergus J Caskey; Fabienne Dobbels; Liset  Pengel; Lionel  Rostaing; Stefan  Schneeberger; Maarten  Naesens

doi:10.3389/ti.2022.10134

Patient-reported Outcomes as Endpoints in Clinical Trials of Kidney Transplantation Interventions

Allison Tong, Rainer Oberbauer, Maria Irene Bellini, Klemens Budde, Fergus J Caskey, Fabienne Dobbels, Liset Pengel, Lionel Rostaing, Stefan Schneeberger, Maarten Naesens^*

^*Corresponding author for this work

Research output: Contribution to journal › Review article (Academic Journal) › peer-review

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Abstract

Patient-reported outcomes (PROs) that assess individuals’ perceptions of life participation, medication adherence, disease symptoms, and therapy side effects are extremely relevant in the context of kidney transplantation. All PROs are potentially suitable as primary or secondary endpoints in interventional trials that aim to improve outcomes for transplant recipients. Using PRO measures (PROMs) in clinical trials facilitates assessment of the patient’s perspective of their health, but few measures have been developed and evaluated in kidney transplant recipients; robust methodologies, which use validated instruments and established frameworks for reporting, are essential. Establishing a core PROM for life participation in kidney transplant recipients is a critically important need, which is being developed and validated by the Standardized Outcomes in Nephrology (SONG)-Tx Initiative. Measures involving electronic medication packaging and smart technologies are gaining traction for monitoring adherence, and could provide more robust information than questionnaires, interviews, and scales. This article summarizes information on PROs and PROMs that was included in a Broad Scientific Advice request on clinical trial design and endpoints in kidney transplantation. This request was submitted to the European Medicines Agency (EMA) by the European Society for Organ Transplantation in 2016. Following modifications, the EMA provided its recommendations in late 2020.

Original language	English
Article number	10134
Journal	Transplant International
Volume	35
DOIs	https://doi.org/10.3389/ti.2022.10134
Publication status	Published - 20 May 2022

Bibliographical note

Funding Information:
The authors thank the experts involved with the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) who participated in the Broad Scientific Advice request. The published information is based on EMA feedback received during the Broad Scientific Advice request. EMA/CHMP have not been involved in the drafting or review of the manuscript to be published. This publication does not constitute a formal EMA/CHMP endorsement of the manuscript. Medical writing support was provided by Linda Edmondson, independent medical writer, funded by ESOT.

Funding Information:
This initiative was supported by the European Society for Organ Transplantation.

Publisher Copyright:
Copyright © 2022 Tong, Oberbauer, Bellini, Budde, Caskey, Dobbels, Pengel, Rostaing, Schneeberger and Naesens.

Keywords

patient-reported outcome measure (PROM)
patient perspective
adherence
life participation
SONG-Tx
PROMIS®

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abstract = "Patient-reported outcomes (PROs) that assess individuals{\textquoteright} perceptions of life participation, medication adherence, disease symptoms, and therapy side effects are extremely relevant in the context of kidney transplantation. All PROs are potentially suitable as primary or secondary endpoints in interventional trials that aim to improve outcomes for transplant recipients. Using PRO measures (PROMs) in clinical trials facilitates assessment of the patient{\textquoteright}s perspective of their health, but few measures have been developed and evaluated in kidney transplant recipients; robust methodologies, which use validated instruments and established frameworks for reporting, are essential. Establishing a core PROM for life participation in kidney transplant recipients is a critically important need, which is being developed and validated by the Standardized Outcomes in Nephrology (SONG)-Tx Initiative. Measures involving electronic medication packaging and smart technologies are gaining traction for monitoring adherence, and could provide more robust information than questionnaires, interviews, and scales. This article summarizes information on PROs and PROMs that was included in a Broad Scientific Advice request on clinical trial design and endpoints in kidney transplantation. This request was submitted to the European Medicines Agency (EMA) by the European Society for Organ Transplantation in 2016. Following modifications, the EMA provided its recommendations in late 2020.",

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note = "Funding Information: The authors thank the experts involved with the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) who participated in the Broad Scientific Advice request. The published information is based on EMA feedback received during the Broad Scientific Advice request. EMA/CHMP have not been involved in the drafting or review of the manuscript to be published. This publication does not constitute a formal EMA/CHMP endorsement of the manuscript. Medical writing support was provided by Linda Edmondson, independent medical writer, funded by ESOT. Funding Information: This initiative was supported by the European Society for Organ Transplantation. Publisher Copyright: Copyright {\textcopyright} 2022 Tong, Oberbauer, Bellini, Budde, Caskey, Dobbels, Pengel, Rostaing, Schneeberger and Naesens.",

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AU - Bellini, Maria Irene

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AU - Caskey, Fergus J

AU - Dobbels, Fabienne

AU - Pengel, Liset

AU - Rostaing, Lionel

AU - Schneeberger, Stefan

AU - Naesens, Maarten

N1 - Funding Information: The authors thank the experts involved with the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) who participated in the Broad Scientific Advice request. The published information is based on EMA feedback received during the Broad Scientific Advice request. EMA/CHMP have not been involved in the drafting or review of the manuscript to be published. This publication does not constitute a formal EMA/CHMP endorsement of the manuscript. Medical writing support was provided by Linda Edmondson, independent medical writer, funded by ESOT. Funding Information: This initiative was supported by the European Society for Organ Transplantation. Publisher Copyright: Copyright © 2022 Tong, Oberbauer, Bellini, Budde, Caskey, Dobbels, Pengel, Rostaing, Schneeberger and Naesens.

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N2 - Patient-reported outcomes (PROs) that assess individuals’ perceptions of life participation, medication adherence, disease symptoms, and therapy side effects are extremely relevant in the context of kidney transplantation. All PROs are potentially suitable as primary or secondary endpoints in interventional trials that aim to improve outcomes for transplant recipients. Using PRO measures (PROMs) in clinical trials facilitates assessment of the patient’s perspective of their health, but few measures have been developed and evaluated in kidney transplant recipients; robust methodologies, which use validated instruments and established frameworks for reporting, are essential. Establishing a core PROM for life participation in kidney transplant recipients is a critically important need, which is being developed and validated by the Standardized Outcomes in Nephrology (SONG)-Tx Initiative. Measures involving electronic medication packaging and smart technologies are gaining traction for monitoring adherence, and could provide more robust information than questionnaires, interviews, and scales. This article summarizes information on PROs and PROMs that was included in a Broad Scientific Advice request on clinical trial design and endpoints in kidney transplantation. This request was submitted to the European Medicines Agency (EMA) by the European Society for Organ Transplantation in 2016. Following modifications, the EMA provided its recommendations in late 2020.

AB - Patient-reported outcomes (PROs) that assess individuals’ perceptions of life participation, medication adherence, disease symptoms, and therapy side effects are extremely relevant in the context of kidney transplantation. All PROs are potentially suitable as primary or secondary endpoints in interventional trials that aim to improve outcomes for transplant recipients. Using PRO measures (PROMs) in clinical trials facilitates assessment of the patient’s perspective of their health, but few measures have been developed and evaluated in kidney transplant recipients; robust methodologies, which use validated instruments and established frameworks for reporting, are essential. Establishing a core PROM for life participation in kidney transplant recipients is a critically important need, which is being developed and validated by the Standardized Outcomes in Nephrology (SONG)-Tx Initiative. Measures involving electronic medication packaging and smart technologies are gaining traction for monitoring adherence, and could provide more robust information than questionnaires, interviews, and scales. This article summarizes information on PROs and PROMs that was included in a Broad Scientific Advice request on clinical trial design and endpoints in kidney transplantation. This request was submitted to the European Medicines Agency (EMA) by the European Society for Organ Transplantation in 2016. Following modifications, the EMA provided its recommendations in late 2020.

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DO - 10.3389/ti.2022.10134

M3 - Review article (Academic Journal)

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SN - 0934-0874

VL - 35

JO - Transplant International

JF - Transplant International

M1 - 10134

ER -

Patient-reported Outcomes as Endpoints in Clinical Trials of Kidney Transplantation Interventions

Abstract

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Keywords

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