The reimbursement for expensive medicines: stakeholder perspectives on the SMA medicine nusinersen and the Dutch Coverage Lock policy.

Autor: Scheijmans FEV; Department of Neurology, UMC Utrecht Brain Center, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands. f.e.v.scheijmans@umcutrecht.nl.; University Medical Center Utrecht, Heidelberglaan 100, 3584CX, Utrecht, The Netherlands. f.e.v.scheijmans@umcutrecht.nl.; Department of Public Health, Healthcare Innovation & Evaluation and Medical Humanities, Julius Center for Health Sciences and Primary Care, Utrecht, The Netherlands. f.e.v.scheijmans@umcutrecht.nl., Zomers ML; University Medical Center Utrecht, Heidelberglaan 100, 3584CX, Utrecht, The Netherlands.; Department of Public Health, Healthcare Innovation & Evaluation and Medical Humanities, Julius Center for Health Sciences and Primary Care, Utrecht, The Netherlands., Fadaei S; University Medical Center Utrecht, Heidelberglaan 100, 3584CX, Utrecht, The Netherlands.; Department of Public Health, Healthcare Innovation & Evaluation and Medical Humanities, Julius Center for Health Sciences and Primary Care, Utrecht, The Netherlands., Onrust MR; University Medical Center Utrecht, Heidelberglaan 100, 3584CX, Utrecht, The Netherlands.; Department of Public Health, Healthcare Innovation & Evaluation and Medical Humanities, Julius Center for Health Sciences and Primary Care, Utrecht, The Netherlands., van der Graaf R; University Medical Center Utrecht, Heidelberglaan 100, 3584CX, Utrecht, The Netherlands.; Department of Public Health, Healthcare Innovation & Evaluation and Medical Humanities, Julius Center for Health Sciences and Primary Care, Utrecht, The Netherlands., Delden JJMV; University Medical Center Utrecht, Heidelberglaan 100, 3584CX, Utrecht, The Netherlands.; Department of Public Health, Healthcare Innovation & Evaluation and Medical Humanities, Julius Center for Health Sciences and Primary Care, Utrecht, The Netherlands., van der Pol WL; Department of Neurology, UMC Utrecht Brain Center, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.; University Medical Center Utrecht, Heidelberglaan 100, 3584CX, Utrecht, The Netherlands., van Thiel GJMW; University Medical Center Utrecht, Heidelberglaan 100, 3584CX, Utrecht, The Netherlands.; Department of Public Health, Healthcare Innovation & Evaluation and Medical Humanities, Julius Center for Health Sciences and Primary Care, Utrecht, The Netherlands.
Jazyk: angličtina
Zdroj: BMC health services research [BMC Health Serv Res] 2022 Nov 04; Vol. 22 (1), pp. 1320. Date of Electronic Publication: 2022 Nov 04.
DOI: 10.1186/s12913-022-08690-z
Abstrakt: Background: The reimbursement for expensive medicines poses a growing challenge to healthcare worldwide. In order to increase its control over the costs of medicines, the Dutch government introduced the Coverage Lock (CL) policy in 2015. The CL postpones decisions regarding reimbursement of expensive medicines until detailed advice on i.e., cost-effectiveness has been given. The CL has been in place for six years, has raised many questions and concerns, but currently, no evaluation is known to the authors. A better understanding of the effects of the CL on all stakeholders involved may contribute to reflections on the CL process and help find ways to improve it. An evaluation of Dutch policy will also be relevant for other countries that aim to optimize reimbursement procedures for expensive treatments. To perform this evaluation, we focused on the CL procedure for the medicine nusinersen. Nusinersen is the first treatment for spinal muscular atrophy (SMA). Following EMA approval in May 2017, it was placed in the CL. The analysis of cost-effectiveness and added therapeutic value resulted in an advice for reimbursement limited to children younger than 9.5 years at the start of treatment; this was implemented from August 2018 onwards.
Methods: Qualitative stakeholder perspective analysis of the CL procedure focusing on nusinersen with 15 stakeholders.
Results: Stakeholders raised key issues of the CL based on their experience with nusinersen: emotional impact of the CL, duration of the CL procedure, appropriateness of the CL procedure for different types of medicines, transparency of the CL, a wish for patient-centred decision-making and the lack of uniformity of access to expensive treatments.
Discussion: Stakeholders supported measures to control healthcare expenses and to ensure reasonable pricing. They considered the delay in access to therapies and lack of procedural transparency to be the main challenges to the CL. Stakeholders also agreed that the interests of patients deserve more attention in the practical implementation of the reimbursement decision. Stakeholders suggested a number of adjustments to improve the CL, such as a faster start with conditional reimbursement programs to ensure access and intensify European collaboration to speed up the assessment of the medicine.
(© 2022. The Author(s).)
Databáze: MEDLINE
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