Modern Treatments of Haemophilia: Review of Cost-Effectiveness Analyses and Future Directions

Cortesi, P.A.; D’Angiolella, Lucia Sara; Lafranconi, Alessandra; Micale, Mariangela; Cesana, Giancarlo; Mantovani, Lorenzo Giovanni

doi:10.1007/s40273-017-0588-z

Cited by 14 publications

(12 citation statements)

References 54 publications

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“…35 The model also assumes that patients on prophylaxis regimen (i.e., emicizumab and BPA prophylaxis) do not experience orthopaedic surgeries because of their (long-term) lower bleed rate, as already assumed in previous haemophilia models. 25,36 The assumption of not requiring orthopaedic surgery is unlikely for patients with BPAs due to the documented bleed rates in the literature. However, no data are available to estimate the true protection of prophylaxis with BPAs and emicizumab started in the early age.…”

Section: Clinical Datamentioning

confidence: 99%

Cost-Effectiveness and Budget Impact of Emicizumab Prophylaxis in Haemophilia A Patients with Inhibitors

et al. 2019

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Recent evidence demonstrated that weekly prophylaxis with subcutaneous bispecific antibody (emicizumab) has shown higher efficacy in adolescent and adults patients affected by haemophilia A (HA) with inhibitor, compared with patients treated on demand or on prophylaxis with bypassing agents (BPAs). However, no economic evaluations assessing the value and sustainability of emicizumab prophylaxis have been performed in Europe. This study assessed the cost-effectiveness of emicizumab prophylaxis compared with BPA prophylaxis and its possible budget impact from the Italian National Health Service (NHS) perspective. A Markov model and a budget impact model were developed to estimate the cost-effectiveness and budget impact of emicizumab prophylaxis in HA patients with inhibitors. The model was populated using treatment efficacy from clinical trials and key clinical, cost and epidemiological data retrieved through an extensive literature review. Compared with BPAs prophylaxis, emicizumab prophylaxis was found to be more effective (0.94 quality adjusted life-years) and cost saving (–€19.4/–€24.4 million per patient lifetime) in a cohort of 4-year-old patients with HA and inhibitors who failed immune tolerance induction. In the probabilistic sensitivity analysis, emicizumab prophylaxis had always 100% probability of being cost-effective at any threshold. Further, the use of emicizumab prophylaxis was associated to an overall budget reduction of €45.4 million in the next 3 years. In conclusion, the clinically effective emicizumab prophylaxis can be considered a cost-saving treatment for HA with inhibitor patients. Furthermore, emicizumab treatment is also associated to a significant reduction of the health care budget, making this new treatment a sustainable and convenient health care option for Italian NHS.

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Section: Clinical Datamentioning

confidence: 99%

Cost-Effectiveness and Budget Impact of Emicizumab Prophylaxis in Haemophilia A Patients with Inhibitors

et al. 2019

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“…In the present review, we also chose to highlight the importance of assessing not only clinical efficacy and injection frequency but also the FVIII IU annual consumption, 19 because data on FVIII IU consumption are important in order to estimate the treatment cost of the different EHL‐rFVIII and thus better define their value. Our combined evaluation of consumption and efficacy data indicated that damoctocog alfa pegol showed at the same time the lowest yearly consumption and injection frequency.…”

Section: Discussionmentioning

confidence: 99%

Comparative analysis of the pivotal studies of extended half‐life recombinant FVIII products for treatment of haemophilia A

et al. 2021

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The need to reduce the burden of injections, and improve adherence and clinical outcomes in haemophilia A led to the development of recombinant FVIII products endowed with an extended plasma half‐life (EHL‐rFVIII) in comparison with standard half‐life products (SHL‐rFVIII). Lack of head‐to‐head studies makes difficult to grasp the relative value of each treatment option. We conducted a combined evaluation of the individual pivotal trials in order to assess between‐product differences regarding the reported efficacy results and FVIII consumption. We evaluated 4 EHL‐rFVIII products available to treat patients with haemophilia A without inhibitors and also a SHL‐rFVIII as a comparator. In the frame of these clinical studies, all the EHL‐rFVIII products showed a decrease in the injection burden coupled with good clinical efficacy, even though there were between‐product differences in terms of reduction in injection frequencies. Further, between‐product differences in terms of weekly/yearly consumption of rFVIII expressed in IU/Kg were identified, suggesting a different economic impact for the different EHL‐rFVIII products in the context of comparable clinical efficacy. The present findings based upon the review of pivotal studies done in the frame of a highly selected clinical scenario should be integrated with real‐life data.

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“…This is particularly relevant in a rare condition as haemophilia where even the biggest regions in a country as Italy reported less than 165 patients. Another important aspect to take into account in assessing FVIII consumption is the distribution of patients according to the different treatment options (eg prophylaxis vs. on‐demand) that significantly increases the variability of consumptions within patients 18‐20 . Furthermore, another issue associated with the consumption variability is related to the measurement of infused factors in the clinical coagulation laboratory, particularly for EHL‐rFVIII.…”

Section: Discussionmentioning

confidence: 99%

Variability of treatment modalities and intensity in patients with severe haemophilia A on prophylaxis: Results from the Italian national registry

Cortesi

Giampaolo²,

Abbonizio³

et al. 2021

European J of Haematology

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Background A shift from a standard to a personalized prophylaxis has been increasingly adopted in patients with severe haemophilia A (SHA). This approach has raised the likelihood of a significant variability in the prophylactic approaches and the relative Factor VIII (FVIII) consumptions. The aim of our study was to assess the treatment variability of SHA patients without inhibitors and on prophylaxis regimen in Italy. Material and methods Data reported in the National Registry of Congenital Coagulopathies (NRCC) were analysed to assess treatment distribution within SHA patients without inhibitors, focussing on FVIII consumption in 2017, associated with prophylaxis regimen. The analysis was stratified based on age groups and Italian regions to describe the variability of FVIII consumption in Italy. Results In 2017, the Registry reported the therapeutic plans of 1068 SHA patients without inhibitors on prophylaxis. The mean (95% CI) individual consumption ranges from 123 127 IU (99 736‐146 518) in the age group 0‐6 years to 345 000 IU (336 000‐354 000) in the age group >20 years. A significant FVIII consumption variability was identified within the adult population. Regions with less than 50 patients reported the higher variability in mean FVIII consumption per patient‐year within the different age groups. Similar difference in FVIII consumption variability was reported also in the age groups comparing “low,” “middle” and “high” patient volume regions. Discussion A reliable estimation of FVIII consumption for patients’ treatment is necessary to manage and plan the appropriate budget and keep treatment's costs affordable. However, without the implementation of a methodology aiming to assess the overall value produced by these FVIII consumptions, the scenario will keep driven by FVIII consumptions, its costs and the budget available. An effort by haemophilic community, haemophilia treatment centres and institutions is required to develop and share this cultural shift in improving haemophilia management and assessment.

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Modern Treatments of Haemophilia: Review of Cost-Effectiveness Analyses and Future Directions

Cited by 14 publications

References 54 publications

Cost-Effectiveness and Budget Impact of Emicizumab Prophylaxis in Haemophilia A Patients with Inhibitors

Cost-Effectiveness and Budget Impact of Emicizumab Prophylaxis in Haemophilia A Patients with Inhibitors

Comparative analysis of the pivotal studies of extended half‐life recombinant FVIII products for treatment of haemophilia A

Variability of treatment modalities and intensity in patients with severe haemophilia A on prophylaxis: Results from the Italian national registry

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