World bleeding disorders registry: The pilot study

Coffin, Donna; Herr, Caroline; O’Hara, Jamie; Diop, S; Hollingsworth, Robert E.; Srivastava, Alok; Lillicrap, David; Berg, H. M. Van Den; Iorio, Alfonso; Pierce, GF

doi:10.1111/hae.13431

Cited by 13 publications

(14 citation statements)

References 12 publications

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“…Related to haemophilia specifically, the WBDR, which was launched in April 2018 after the ECHO study registry was already initiated, records real‐world patient‐level data on global clinical outcomes. The pilot study indicated good feasibility and a high level of patient interest, 7 and the first report was produced in 2019 28 . This registry has a privacy‐protected, web‐based data entry system that allows for the collection of individual patient data, providing a clinical profile for each PWH.…”

Section: Discussionmentioning

confidence: 94%

“…28 This registry has a privacy-protected, web-based data entry system that allows for the collection of individual patient data, providing a clinical profile for each PWH. Unlike ECHO, these registries did not focus exclusively on PRO data, 7,26 Although the multinational ECHO haemophilia registry was closed early, the global PROBE project is currently underway to collect PROs in PWH and control subjects without bleeding disorders. PROBE may overcome some common registry obstacles by administering a short questionnaire and employing a cross-sectional design.…”

Section: Industry Sponsor Perspectivementioning

confidence: 99%

“…Haemophilia registries and observational cohort studies can capture large amounts of data on a broad range of products in a real‐world setting, in contrast to clinical trials, which tend to be smaller and provide limited data 5 . The Patient Reported Outcomes, Burdens and Experiences (PROBE) study 6 and the World Bleeding Disorders Registry (WBDR) 7 are new global initiatives set‐up to gather prospective real‐world PRO data or clinical data, respectively, from people with haemophilia (PWH). PROBE is a patient‐led research project, and WBDR is a World Federation of Haemophilia database that had not been launched when the Expanding Communications on Hemophilia A Outcomes (ECHO) registry was initiated; both independent, investigator‐led projects are funded by research grants from multiple pharmaceutical companies.…”

Section: Introductionmentioning

confidence: 99%

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

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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.

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Section: Discussionmentioning

confidence: 94%

Section: Industry Sponsor Perspectivementioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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

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“…The discussion led to the second statement on the collection of data regarding known and unknown long‐term safety concerns associated with gene therapy, which was agreed upon by 80% of the 56 voters. This small group placed great importance on the existence of one global registry to capture safety and duration of efficacy data for all gene therapy products, and the WFH World Bleeding Disorders Registry (WBDR) framework was proposed to meet this need 21,22 . Collecting adverse event data prospectively over the long term is essential for identifying low incidence events associated with gene therapy.…”

Section: Discussionmentioning

confidence: 99%

Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table

et al. 2020

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Introduction:With approval of gene therapy for haemophilia likely in the near future, policy frameworks are needed to guide the path forward for this disruptive and novel therapeutic advance. Aim:The WFH has initiated a series of multi-stakeholder Gene Therapy Round Tables (GTRT) to better understand where guidance is needed and develop initial consensus statements to inform policy. Methods:The first day of the 2nd GTRT was devoted to didactic presentations on models of access to gene therapy, payment and health technology assessment considerations, regulatory issues and the generation of evidence on safety and durable efficacy of gene therapy products. On the second day, participants were tasked with developing and voting on consensus statements that reflected the information presented and multistakeholder views expressed during discussions in the 1st and 2nd WFH GTRTs. The statements covered global access to gene therapy for all people with haemophilia (PWH), collection of long-term safety and efficacy data, ensuring gene therapy is available for all subgroups of PWH including those who have been largely excluded from clinical trials and characterizing acceptable and ideal factor expression levels for gene therapy products. Results:The first 3 statements achieved consensus (at least 80% agreement) by this group of experts. The statement on identifying an ideal and an acceptable factor level expression elicited a lively discussion but failed to achieve consensus by this group.Conclusions: This issue of ideal and acceptable factor level expression and other unresolved issues will be brought to the 3rd WFH GTRT in 2020. K E Y W O R D Schallenges, consensus, gene therapy, global, haemophilia, round table 444 | PIERCE Et al.

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“…Among illustrations of these major changes figure the introduction of prophylaxis, including immune tolerance induction, the performance of increasingly complex invasive procedures, as well as the participation to international registries, and clinical trials in numerous developing countries . The active contribution made by these transformations is encouraging and motivating many physicians and other health professionals to collect local data from developing countries, analyse them and share their experiences and observations in scientific reports and publications.…”

Section: Introductionmentioning

confidence: 99%

Publishing in the Haemophilia Journal: Opportunities and challenges for developing countries

Hermans

2020

Haemophilia

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Better access to haemophilia treatment and care is currently changing the life of a great many patients with haemophilia or other inherited bleeding diseases in numerous developing countries. These changes are encouraging while stimulating local research initiatives and projects that deserve to be communicated through scientific publications. This paper addresses several opportunities and challenges, providing guidance to scientists, clinicians, researchers and health professionals from developing countries who are actively involved in multidisciplinary haemophilia care, while wishing to publish their reports in Haemophilia, the official, global, multidisciplinary journal of the World Federation of Haemophilia (WFH), the European Haemophilia and Allied Disorders (EAHAD) organization and the Hemostasis and Thrombosis Research Society (HTRS) of North America, focusing on inherited bleeding diseases. Several strategies and pathways designed to encourage, help and support successful publications from developing countries are herein discussed.

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World bleeding disorders registry: The pilot study

Cited by 13 publications

References 12 publications

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

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

Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table

Publishing in the Haemophilia Journal: Opportunities and challenges for developing countries

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