Dataset Information

Challenges and key lessons from the design and implementation of an international haemophilia registry supported by a pharmaceutical company.

ABSTRACT:

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.

SUBMITTER: Hay CRM

PROVIDER: S-EPMC7894355 | biostudies-literature | 2020 Nov

REPOSITORIES: biostudies-literature

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Publications

Challenges and key lessons from the design and implementation of an international haemophilia registry supported by a pharmaceutical company.

Hay Charles R M CRM Shima Midori M Makris Michael M Jiménez-Yuste Victor V Oldenburg Johannes J Fischer Kathelijn K Iorio Alfonso A Skinner Mark W MW Santagostino Elena E von Mackensen Sylvia S Kessler Craig M CM

Haemophilia : the official journal of the World Federation of Hemophilia 20201023 6

<h4>Introduction</h4>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.<h4>Aim</h4>To describe the challenges faced and lessons learned from implementing a multinational haemophilia registry.<h4>Meth ...[more]

PMID: 33094894

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Project description:BackgroundExploring barriers to the uptake of research based recommendations into practice is an important part of the development of implementation programmes. Techniques to identify barriers can include use of theory-informed questionnaires and qualitative interviews. Conceptualising and measuring theory-informed factors, and engaging health professionals' to uncover all potential barriers, can be a difficult task. This paper presents a case study of the process of trying to identify, systematically, the key factors influencing health professionals' referrals for women diagnosed with mild to moderate postnatal depression for psychological treatment. The paper illustrates how the factors were conceptualised and measured and explores the real world challenges experienced, with implications for future implementation studies.MethodsTheory-informed factors were conceptualised and measured using a questionnaire and interviews. The questionnaire was piloted, before being administered to general practitioners, practice nurses and health visitors working in general practices in one area of the UK NHS. The interviews were conducted with a small sample of general practitioners who had not completed the questionnaire, further exploring factors influencing their referral decisions in the local context.ResultsThe response rate to the questionnaire was low (19%), despite selecting the recommendation to target through engagement with local stakeholders and surveying local health professionals, and despite using two reminders, an incentive prize, and phone calls to practice managers to bolster response rates.ConclusionsTwo significant challenges to achieving higher response rates and successfully exploring local context were identified: the difficulties of developing a robust- but feasible- questionnaire to explore theory-informed factors, and targeting recommendations that are important to policy makers, but which health professionals view as unimportant. This case study highlights the "trade-off" between scientifically rigorous collection of data against the pragmatism and flexibility requirements of "real world" implementation. Future implementation studies should explore different ways of identifying factors influencing the adoption of recommendations to bridge this gulf.

Dataset Information

Challenges and key lessons from the design and implementation of an international haemophilia registry supported by a pharmaceutical company.

Introduction

Aim

Methods

Results

Conclusions

Publications

Challenges and key lessons from the design and implementation of an international haemophilia registry supported by a pharmaceutical company.

Similar Datasets

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