Switching to extended half‐life products in Canada – preliminary data

Keepanasseril, Arun; Stoffman, Jayson; Bouskill, Vanessa; Carção, Manuel; Iorio, Alfonso; Jackson, Stewart M.

doi:10.1111/hae.13245

Cited by 30 publications

(51 citation statements)

References 8 publications

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“…The median reduction of annual IU/kg for prophylaxis was 17% for EHL FVIII and 44% EHL FIX compared to SHL CFCs; however, incremental increase in median CFCs cost per unit of the EHL factor group exceeded use reduction. The decrease in annual factor utilization observed within the ATHNdataset was similar to that reported in a preliminary data analysis of the Canadian Bleeding Disorder Registry (CBDR); however, a national tender system results in a different impact on the annual cost of prophylaxis . Careful assessment of factor consumption and patient outcomes is needed to ensure general cost neutrality of this expensive therapy.…”

Section: Discussionsupporting

confidence: 63%

“…The decrease in annual factor utilization observed within the ATHNdataset was similar to that reported in a preliminary data analysis of the Canadian Bleeding Disorder Registry (CBDR); however, a national tender system results in a different impact on the annual cost of prophylaxis. 22 Careful assessment of factor consumption and patient outcomes is needed to ensure general cost neutrality of this expensive therapy. Unless demonstrably offset by reduction in bleed doses, the net effect could be further increases in annual cost of care for this patient population.…”

Section: Con Clus Ionsmentioning

confidence: 99%

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Regional variation and cost implications of prescribed extended half‐life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia

et al. 2019

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Background Extended half‐life (EHL) factor VIII (FVIII) and IX (FIX) products are intended to decrease the burden of prophylaxis for patients with haemophilia A or B. Whether these newer concentrates have led to meaningful clinical practice change remains vague. Aim To characterize the longitudinal use of standard (SHL) and EHL factor concentrates at haemophilia treatment centres (HTCs), using the ATHNdataset, a US database of 138 ATHN‐affiliated HTCs. Methods Factor concentrate use among moderate and severe haemophilia A and B patients without inhibitors was analysed at three time points over 18 months. Results Use of EHL concentrates rose from 10% of patients to 22% during this study. EHL FVIII prophylaxis is prescribed to the minority of patients, 28%; EHL FIX now predominates for prophylaxis, 52%. Rates of prescribed EHL products varied significantly by age group and HTC region. Median prescribed prophylaxis for SHL compared to EHL products was FVIII 6240 and 5200 and FIX 6968 and FIX 3900 IU/kg/y, respectively. On‐demand EHL use has grown but has minimal contribution to overall usage (2%). Conclusion Haemophilia treatment centre region and patient age impact the rate of adoption of EHL products; however, EHL prescribing continues to rise nationally, particularly for EHL FIX. Careful attention to annual cost of prophylaxis is imperative as the decrease in median EHL prophylaxis consumption is not offset by the higher unit cost of these products. It is unclear how further growth in use of EHLs will be impacted by emerging non‐factor replacement and gene therapies.

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Section: Discussionsupporting

confidence: 63%

Section: Con Clus Ionsmentioning

confidence: 99%

Regional variation and cost implications of prescribed extended half‐life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia

et al. 2019

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“…[20][21][22][23][24][25][26][27][28][29]33,34 Our survey on EHL recombinant FIX showed a significant reduction in infusion frequency at 100% of the centres, with 67% achieving a trough FIX level of at least 5%-10%. [20][21][22][23][24][25][26][27][28][29]33,34 Our survey on EHL recombinant FIX showed a significant reduction in infusion frequency at 100% of the centres, with 67% achieving a trough FIX level of at least 5%-10%.…”

Section: Discussionmentioning

confidence: 99%

“…17,18 EHL products have been licensed at different times in the USA and Europe. [20][21][22][23][24][25][26][27][28][29] In this paper, we report the preliminary data on real-life experiences at European haemophilia treatment centres (HTCs), according to a survey administered by the European Association for 19 At the beginning of 2018, EHL products available in Europe were limited to recombinant FVIII and FIX fusion products.…”

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confidence: 99%

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Real‐life experience in switching to new extended half‐life products at European haemophilia centres

et al. 2019

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The concept of replacement therapy in haemophilia is changing significantly thanks to the switch from standard products to extended half‐life products. These novel drugs are showing beneficial effects overcoming current prophylaxis limitations by reducing the infusion frequency, maintaining a higher trough level to ensure a lower risk of bleeding, and making treatment significantly less distressing to patients by improving the quality of life. Real‐life data on the efficacy of novel drugs and their impact on routine management of haemophilia A and B patients are still limited. This manuscript reports the results of a European survey conducted by the European Association for Haemophilia and Allied Disorders (EAHAD) at the beginning of 2018 on the clinical management of patients using extended half‐life recombinant FVIII and FIX fusion products, since at the time of the survey none of the PEGylated products were available yet. We report data on the efficacy of these novel drugs by 33 European haemophilia centres that have already switched to extended half‐life fusion products, showing a significant reduction in the number of infusions and a satisfactory trough levels in the clinical care of haemophilia patients, with a greater impact for haemophilia B.

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The impact of extended half‐life factor concentrates on prophylaxis for severe hemophilia in the United States

et al. 2020

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With licensure of extended half‐life (EHL) factor products and the changing landscape of available hemophilia products, patients and providers have options for less treatment‐intense prophylaxis. The impact of these products in clinical practice to date remains understudied.We aimed to quantify the use of EHL products in prophylaxis in the US using the ATHN‐dataset, a database of 145 ATHN‐affiliated hemophilia treatment centers (HTCs). Further, we aimed to quantify the impact of EHL on key hemophilia indicators including annualized bleed rates (ABRs), hemophilia joint health scores (HJHS) and quality of life (QOL) metrics. The use of EHL vs standard half‐life (SHL) products in severe hemophilia was compared between June 2018 and March 2019 using the ATHN‐dataset. A cohort of patients was also recruited from seven participating HTCs in order to compare ABR, HJHS and QOL between product classes.By March 2019 the number of individuals with severe Hemophilia A (SHA) receiving EHLs remained relatively stable (28.4%), whereas the number of prescribed non‐factor products increased to 7.1%, with a diminishing majority of patients (64.0%) continuing to receive SHLs. The majority of patients with severe hemophilia B (SHB) received treatment with EHLs including 57.5% by March 2019. There was a trend toward lower ABR with use of EHLs in SHA and SHB, although this did not result in improved HJHS nor QOL.EHL use in the United States in severe hemophilia continues to increase, although at a slower rate in SHA with the availability of non‐factor therapy. The impact of the EHL therapies in clinical practice should continue to be examined prospectively.

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Switching to extended half‐life products in Canada – preliminary data

Cited by 30 publications

References 8 publications

Regional variation and cost implications of prescribed extended half‐life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia

Regional variation and cost implications of prescribed extended half‐life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia

Real‐life experience in switching to new extended half‐life products at European haemophilia centres

The impact of extended half‐life factor concentrates on prophylaxis for severe hemophilia in the United States

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