TY - JOUR
T1 - Challenges and key lessons from the design and implementation of an international haemophilia registry supported by a pharmaceutical company
AU - Hay, Charles R.M.
AU - Shima, Midori
AU - Makris, Michael
AU - Jiménez-Yuste, Victor
AU - Oldenburg, Johannes
AU - Fischer, Kathelijn
AU - Iorio, Alfonso
AU - Skinner, Mark W.
AU - Santagostino, Elena
AU - von Mackensen, Sylvia
AU - Kessler, Craig M.
N1 - Funding Information:
The authors thank the ECHO project managers for their support: Matthew Willis, Sabine Friedl and Yunyun Zhou. Prasad Mathew of Bayer assisted with the design, initiation of ECHO, registry close‐out and analysis planning. Stephan Rauchensteiner, formerly of Bayer, assisted with registry monitoring, close‐out and analysis planning. Medical writing and editorial assistance were provided by Karen L. Zimmermann from Complete Healthcare Communications, LLC (North Wales, PA, USA), and Niall Harrison from Sudler Medical Communications (London, UK), and were fully funded by Bayer.
Funding Information:
Charles RM Hay attended speaker bureau for Pfizer, Novo Nordisk, Shire, Sobi, and Biotest and advised for Alnylam, Bayer, Shire and Roche. Midori Shima received grants/personal fees from Shire, Bioverativ, Chugai, CSL Behring, Novo Nordisk, Bayer, Kaketsuken, Pfizer and Sysmex and personal fees from Roche, BioMarin, Octapharma and Sanofi. Michael Makris was the project lead of the EUHASS project, which receives partial funding from Bayer. Victor Jiménez‐Yuste was reimbursed for attending symposia/congresses and/or honoraria for speaking/consulting and/or funds for research from Shire, Bayer, CSL Behring, Grifols, Novo Nordisk, Sobi, Octapharma and Pfizer. Johannes Oldenburg was reimbursed for attending symposia/congresses and/or honoraria and/or funds for research from Bayer, Biogen Idec, Biotest, Chugai, CSL Behring, Grifols, Novo Nordisk, Octapharma, Pfizer, Roche, Shire and Swedish Orphan Biovitrum. Kathelijn Fischer received speaker fees from Bayer, Baxter, Biogen, Biotest, CSL Behring, Octapharma, Pfizer and Novo Nordisk; performed consultancy for Bayer, Baxter, Biogen, CSL Behring, Freeline, Novo Nordisk, Pfizer, Roche and Sobi; and has received research support from Bayer, Pfizer, Baxter and Novo Nordisk; she is the epidemiologist for EUHASS and PedNet registries. Alfonso Iorio's institution has received funds for research and service agreements from Bayer, Novo Nordisk, Octapharma, Pfizer and Roche. Mark W. Skinner received honoraria for educational presentations and advisory roles with Bayer, BioMarin, Roche, Pfizer, Novo Nordisk and Spark Therapeutics and received research support as the PROBE study principal investigator from Shire (part of Takeda), Bayer, Bioverativ (a Sanofi company), CSL Behring, Novo Nordisk, Roche and Sobi. Elena Santagostino was the member of the speaker bureau and/or advisory boards for Bayer, Shire/Takeda, Pfizer, Novo Nordisk, CSL Behring, Sobi, Bioverativ, Roche, Grifols, Kedrion, uniQure, Spark and Octapharma. Sylvia von Mackensen was the consultant for Bayer. Craig M. Kessler was the consultant and advisory board participant for Bioverativ, Bayer, Novo Nordisk, Octapharma, Pfizer, Roche and Shire and received research funding from Bioverativ, Bayer, Novo Nordisk, Octapharma and Roche.
Publisher Copyright:
© 2020 John Wiley & Sons Ltd
Copyright:
Copyright 2020 Elsevier B.V., All rights reserved.
PY - 2020/11
Y1 - 2020/11
N2 - Introduction: Real-world data are lacking regarding the relationship between prospectively collected patient-reported outcomes (PROs), clinical outcomes and treatment in people with haemophilia (PWH). The Expanding Communications on Hemophilia A Outcomes (ECHO) registry was designed to address this data gap, but a range of difficulties led to early study closure. Aim: To describe the challenges faced and lessons learned from implementing a multinational haemophilia registry. Methods: The Expanding Communications on Hemophilia A Outcomes was planned as a five-year observational cohort study to collect data from 2000 patients in nine countries. Based on direct observations, feedback from patients enrolled in ECHO, challenges of the study design and input from study-sponsor representatives, the ECHO Steering Committee systematically identified the challenges faced and developed recommendations for overcoming or avoiding them in future studies. Results: The study closed after two years because few countries were activated and patient recruitment was low. This was related to multiple challenges including delayed implementation, stringent pharmacovigilance requirements, objections of investigators and patients to the burden of multiple PROs, data collection issues, lack of resources at study sites, little engagement of patients and competing clinical trials, which further limited recruitment. At study closure, 269 patients had been enrolled in four of nine participating countries. Conclusions: Researchers planning studies similar to ECHO may want to consider the barriers identified in this global registry of PWH and suggestions to mitigate these limitations, such as greater patient involvement in design and analysis, clearer assessment and understanding of local infrastructure and potential changes to the administration of the study.
AB - Introduction: Real-world data are lacking regarding the relationship between prospectively collected patient-reported outcomes (PROs), clinical outcomes and treatment in people with haemophilia (PWH). The Expanding Communications on Hemophilia A Outcomes (ECHO) registry was designed to address this data gap, but a range of difficulties led to early study closure. Aim: To describe the challenges faced and lessons learned from implementing a multinational haemophilia registry. Methods: The Expanding Communications on Hemophilia A Outcomes was planned as a five-year observational cohort study to collect data from 2000 patients in nine countries. Based on direct observations, feedback from patients enrolled in ECHO, challenges of the study design and input from study-sponsor representatives, the ECHO Steering Committee systematically identified the challenges faced and developed recommendations for overcoming or avoiding them in future studies. Results: The study closed after two years because few countries were activated and patient recruitment was low. This was related to multiple challenges including delayed implementation, stringent pharmacovigilance requirements, objections of investigators and patients to the burden of multiple PROs, data collection issues, lack of resources at study sites, little engagement of patients and competing clinical trials, which further limited recruitment. At study closure, 269 patients had been enrolled in four of nine participating countries. Conclusions: Researchers planning studies similar to ECHO may want to consider the barriers identified in this global registry of PWH and suggestions to mitigate these limitations, such as greater patient involvement in design and analysis, clearer assessment and understanding of local infrastructure and potential changes to the administration of the study.
KW - clinical outcome
KW - haemophilia
KW - multinational
KW - patient-reported outcome
KW - registry
KW - study design
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U2 - 10.1111/hae.14144
DO - 10.1111/hae.14144
M3 - Article
C2 - 33094894
AN - SCOPUS:85093528655
VL - 26
SP - 966
EP - 974
JO - Haemophilia
JF - Haemophilia
SN - 1351-8216
IS - 6
ER -