Converting factor and nonfactor usage into a single metric to facilitate benchmarking the resources consumed for haemophilia care across jurisdictions and over time

Background rVIII-SingleChain is a recombinant factor VIII (FVIII) with increased binding affinity to von Willebrand factor compared with other FVIII products. rVIII-SingleChain is indicated for the treatment and prevention of bleeding episodes in patients with hemophilia A. Objectives To collect real-world evidence data from patients treated with rVIII-SingleChain to confirm the efficacy and safety established in the clinical trial program and carry out a population pharmacokinetic (PK) analysis. Patients/Methods This interim analysis includes data, collected between January 2018 — September 2021, from patients treated with rVIII-SingleChain prophylaxis at French Hemophilia Treatment centers. Data on annualized bleeding rates, dosing frequency, and consumption before and after switching to rVIII-SingleChain were recorded. A population PK analysis was also conducted to estimate PK parameters. Results Overall, 43 patients switched to prophylaxis with rVIII-SingleChain either from a previous prophylaxis regimen or from on-demand treatment. Following the switch to rVIII-SingleChain, patients maintained excellent bleed control. After switching to rVIII-SingleChain, most patients maintained or reduced their regimen. Interestingly, a majority of patients treated >2 ×/weekly with a standard half-life FVIII reduced both injection frequency and FVIII consumption with rVIII-SingleChain. A PK analysis revealed a lower clearance of rVIII-SingleChain (1.9 vs. 2.1 dL/h) and a longer half-life both in adolescents/adults (n = 28) and pediatric (n = 6) patients (15.5 and 11.9 hours, respectively vs. 14.5 and 10.3 hours) than previously reported. Conclusions Patients who switched to rVIII-SingleChain prophylaxis demonstrated excellent bleed control and a reduction in infusion frequency. A population PK analysis revealed improved PK parameters compared with those reported in the clinical trial.

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“…According to several publications, 22 35 36 37 38 39 real-world evidence shows that rVIII-SingleChain is a full-fledged EHL FVIII.…”

Section: Discussionmentioning

confidence: 99%

A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis

Guillet

Hassoun²,

Wibaut³

et al. 2023

Thromb Haemost

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“…The quantum of CFC procured in any country and indeed in the world expressed in IU/capita has been used to determine the adequacy of product availability. 41 The definition of adequacy has been a moving target with about 3 IU/capita being considered sufficient in the 1990s to some higher income countries now moving to >8 IU/capita. 42 This calculation has become a little more complex with the introduction of non-CFC products which are not measured in IU like CFCs.…”

Section: Access To Therapeutic Products-measuring Adequacymentioning

confidence: 99%

“…Even though effective therapeutic products for haemophilia have been available for nearly five decades, its distribution and access has been highly uneven. The quantum of CFC procured in any country and indeed in the world expressed in IU/capita has been used to determine the adequacy of product availability 41 . The definition of adequacy has been a moving target with about 3 IU/capita being considered sufficient in the 1990s to some higher income countries now moving to >8 IU/capita 42 .…”

Section: Access To Therapeutic Products—measuring Adequacymentioning

confidence: 99%

“…The definition of adequacy has been a moving target with about 3 IU/capita being considered sufficient in the 1990s to some higher income countries now moving to >8 IU/capita 42 . This calculation has become a little more complex with the introduction of non‐CFC products which are not measured in IU like CFCs 41 . While the amount that should be considered sufficient for haemophilia can be difficult to define, it is important that this important parameter, which also serves as an indicator of the type of replacement therapy feasible, accurately reflect what is available for the PWH in that country.…”

Section: Access To Therapeutic Products—measuring Adequacymentioning

confidence: 99%

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Defining success in haemophilia care – Are we doing it right?

Srivastava

2024

Haemophilia

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IntroductionTransformational advances have occurred in the management of haemophilia in the last decade leading to much better outcomes. However, a detailed and critical examination of its assessment and reporting show gaps in many aspects. These are discussed in this review.MethodsThe relevant literature related to different aspects of management of haemophilia was reviewed to identify gaps which need to be addressed. These include detection and diagnosis of haemophilia, documentation and reporting of joint bleeding, its management and methods of reporting in clinical trials and practice, aspects of personalizing care as well as access to therapeutic products and the need for and organization of comprehensive care.ResultsCurrent diagnostic approaches have more than doubled the identified number of persons with haemophilia (PWH) over the last 25 years but still constitute only ∼30% of the expected number. Joint bleeding is the primary indicator of disease severity and treatment efficacy, but there is lack of consistency and standardization in the way it is recorded and reported. Its continued use as an efficacy measure of modern treatments which maintain steady state factor levels or equivalence of >5% will lack sensitivity. The treatment of acute haemarthrosis has focussed on haemostasis and pain control, ignoring the role of inflammation in joint damage. Phenotypic heterogeneity of severe haemophilia has recognized clinical and laboratory variations based on haemostasis but not differences in local response to blood in the joint. At the organizational level, IU/capita provides a relevant measure of access to therapeutic products when the detection rate is ∼100% but is fallaciously low when detection rates are very low. With highly effective modern therapies for haemophilia and nearly no bleeding, the concept of comprehensive care team will need modifications.ConclusionAs haemophilia care advances, a deeper dive is needed into the details of various aspects its management to ensure consistency and contemporary relevance.

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Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B

Olasupo,

Noronha,

Lowe

et al. 2024

Cochrane Database of Systematic Reviews

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Converting factor and nonfactor usage into a single metric to facilitate benchmarking the resources consumed for haemophilia care across jurisdictions and over time

Cited by 4 publications

References 31 publications

A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis

A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis

Defining success in haemophilia care – Are we doing it right?

Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B

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