World Federation of Hemophilia Gene Therapy Registry

Konkle, Barbara A.; Coffin, Donna; Pierce, Glenn F.; Clark, Cary; George, Lindsey A.; Iorio, Alfonso; Mahlangu, Johnny; Naccache, Mayss; O’Mahony, Brian; Peyvandi, Flora; Pipe, Steve; Quartel, Adrian; Sawyer, Eileen K; Skinner, Mark W.; Tortella, Bartholomew J.; Watson, Crystal; Winburn, Ian

doi:10.1111/hae.14015

Cited by 39 publications

(48 citation statements)

References 15 publications

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“…Current AAV products are not part of FDA Risk Evaluation and Mitigation Startegies (REMS). Efforts to prospectively gather outcomes data on a global scale via the World Federation of Hemophilia Gene Therapy Registry are also underway 80 …”

Section: Current Management Of Hemophiliamentioning

confidence: 99%

2021 clinical trials update: Innovations in hemophilia therapy

Croteau

Wang²,

Wheeler

2020

American J Hematol

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Therapies engineered to prolong clotting factor protein circulation time, manipulate the balance of pro‐coagulant and anti‐coagulant proteins, or introduce new genetic material to enable endogenous factor protein production dominate the clinical trial landscape of hemophilia. The availability of clotting factor concentrates and the establishment of primary prophylaxis have dramatically improved health outcomes for hemophilia patients. But, the burden of hemostatic therapy remains significant, and many barriers to consistent longitudinal use of prophylaxis exist. Several types of emerging therapeutics including engineered factor concentrates, substitutive therapies, rebalancing therapies, and gene transfer/editing all aim to reduce the challenges of current hemophilia treatment. Emerging treatment options may reduce treatment frequency or need for intravenous administration. They may also introduce new challenges in laboratory assessment of hemostasis. These novel therapies must not introduce significant new health risks and continue to support similar or improved outcomes. The potential ramifications of rebalancing the coagulation cascade, particularly in a stress or inflammatory state, or introduction of new genetic material are not trivial. The focus of this review is to provide an overview of active and recently completed clinical trials as well as emerging preclinical data investigating new therapeutic possibilities for hemophilia patients and potentially other rare bleeding disorders.

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Section: Current Management Of Hemophiliamentioning

confidence: 99%

2021 clinical trials update: Innovations in hemophilia therapy

Croteau

Wang²,

Wheeler

2020

American J Hematol

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“…The need for consistency and uniformity when assessing efficacy and durability of different GT treatments is essential. Working from common outcome sets, such as CoreHEM and incorporating these into a global registry collecting post‐marketing data on individuals who undergo GT, is critical to provide uniform data on reliability, efficacy, durability, and most importantly, safety if these therapies are approved 23,30 …”

Section: Reimbursement Processmentioning

confidence: 99%

Reimbursing the value of gene therapy care in an era of uncertainty

Noone¹,

Coffin

Pierce

2020

Haemophilia

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Early‐stage gene therapy (GT) clinical trials are demonstrating exciting results for persons with haemophilia (PWH), with the first products possibly licenced over the next few years for haemophilia A and B. These new treatments offer the possibility of a one‐off approach to the treatment of haemophilia, with demonstrated increases in factor level expression and substantial reductions in both bleeds and factor utilization. However, clinical trial participants have demonstrated variable expression in factor levels, including decreases, over time, suggesting in some cases the effect may not last. The consequence of this uncertainty has led to challenging discussions on value and reimbursement. In most national healthcare systems, the relatively high cost of paying for GT on a one‐off basis may be prohibitive, resulting in a lack of access and less post‐marketing data generated, ultimately keeping these performance uncertainties high for payers. Economic models have demonstrated the cost‐effectiveness of GT in haemophilia based on current clinical trial inputs, but it is in the certainty of these inputs and concomitant budget impacts where the lack of available data will be a concern for payers. To overcome the ‘chicken and egg’ discussion in relation to reimbursement and data, GT will necessitate new pricing and reimbursement models that share the risk between the manufacturer and the payer. New models have been described for other conditions. The aim of this paper is to propose illustrative concepts of haemophilia reimbursement models that may be further considered in the assessment of a less predictable therapeutic such as GT.

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“…The numerous uncertainties, however, highlight the importance of a global longitudinal surveillance registry to capture outcomes for all PWH undergoing gene therapy. 17,18 Clotting factor levels considerably above the upper normal limits of 150 IU/dL were observed in some patients in some FVIII and FIX studies. 5,19,20 Variability may or may not be related to durability.…”

mentioning

confidence: 99%

“…Only in this environment, we can have confidence in this technology. A global registry is a vital and necessary foundation for tracking safety and efficacy, 17,18 but observational studies alone do not substitute for scientific investigations to further our biologic knowledge of this new and promising therapeutic modality.…”

mentioning

confidence: 99%