Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A

Ham, Renske M.T. ten; Walker, Sikon M; Soares, Marta; Frederix, Geert; Leebeek, Frank W.G.; Fischer, Kathelijn; Coppens, Michiel; Palmer, Stephen

doi:10.1097/hs9.0000000000000679

Cited by 12 publications

(10 citation statements)

References 65 publications

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“…Results of the five models are presented in Table 4 . Machin et al and Ten Ham et al 81 , 85 conducted a 10-year time horizon starting at approximately 30 years of age. Cook et al 82 presented the latter as a scenario analysis which we use here for comparability purposes.…”

Section: Considerations For Successful Implementation Of Gene Therapy...mentioning

confidence: 99%

“…Differences in costs to control and treat bleeds were driven by major variations in 1) the dosing protocol which varied in the number of units and number of administered doses for prophylaxis and different types of bleeds ( Table 4 ), 2) the assumed weight of the prototypical patient which ranged between 81.4 and 90.2 kg, 3) the FVIII unit cost which ranged between $0.79/IU (discounted) and $1.63/IU (Wholesale Acquisition Cost), and 4) the costing perspective and discounting rate used in the model. Ten Ham et al 85 was the only model that adopted a societal perspective meaning that more cost categories like productivity loss were considered. However, it was also the only model that discounted costs at an annual rate of 4% compared to 3% for the two other models.…”

Section: Considerations For Successful Implementation Of Gene Therapy...mentioning

confidence: 99%

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Gene Therapy and Hemophilia: Where Do We Go from Here?

Bolous¹,

Bhatt²,

Bhakta³

et al. 2022

JBM

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Gene therapy for hemophilia using adeno-associated virus (AAV) derived vectors can reduce or eliminate patients’ disease-related complications and improve their quality of life. Broad implementation globally will lead to societal gains and foster health equity. Several vector products each for factor IX (FIX) or factor VIII (FVIII) deficiency are in advanced clinical development. Safety data are reassuring. Efficacy data for up to 8 and 5 years, respectively, vary considerably among vector types and among individuals, but indicate significant reduction in bleeds and factor use. Products will soon be approved for marketing. This review highlights the relevant considerations for implementation of hemophilia gene therapy, specifically across a broad range of socioeconomic backgrounds globally, based on recent publications and our own experience. We address the current efficacy and safety data and relevant aspects of vector immunology. We then discuss pertinent implementation steps including pre-implementation and readiness assessments, considerations on cost, cost-effectiveness and payment models, approaches to education and informed consent, and the operational needs as well as the need for monitoring of health outcomes and implementation outcomes. To prevent a lag or complete lack of establishing access to this life-changing therapy option for all patients with hemophilia worldwide, adaptable pathways supported by collaborative and international efforts of all stakeholders are needed.

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Section: Considerations For Successful Implementation Of Gene Therapy...mentioning

confidence: 99%

Section: Considerations For Successful Implementation Of Gene Therapy...mentioning

confidence: 99%

Gene Therapy and Hemophilia: Where Do We Go from Here?

Bolous¹,

Bhatt²,

Bhakta³

et al. 2022

JBM

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“…Potentially these will further improve patient outcomes but cost and benefit for patients and society are yet unclear. The effectiveness, (long term) side effects, and therefore positioning and optimal use of these new treatments are still to be established 21 . In addition, we are obligated to identify which patients will benefit from which therapeutic approach.…”

Section: Introductionmentioning

confidence: 99%

SYMPHONY consortium: Orchestrating personalized treatment for patients with bleeding disorders

Cnossen

Moort

Reitsma

et al. 2022

Journal of Thrombosis and Haemostasis

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Background: Treatment choices for individual patients with an inborn bleeding disorder are increasingly challenging due to increasing options and rising costs for society.We have initiated an integrated interdisciplinary national research program. Objectives:The SYMPHONY consortium strives to orchestrate personalized treatment in patients with an inborn bleeding disorder, by unraveling the mechanisms behind interindividual variations of bleeding phenotype.

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“…A recent cost-effective analysis calculated a break-even time of 8 years for valoctocogene roxaparvovec, assuming an annual FVIII decrease of 5.7%. 6 This decrease was based on the earlier BioMarin trial in 15 patients. 4 With the phase 3 data now available, it is clear that this decline seems too optimistic.…”

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confidence: 99%