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

Abstract

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.

Ethics of access to newly approved expensive medical treatments: multi-stakeholder dialogues in a publicly funded healthcare system

“…Both the call to use the models strategically to accelerate the CL-procedure and the call for more transparency regarding the CL-procedure stemmed from dissatisfaction with some aspects of the current CL-procedure. Criticism of the lack of transparency and the duration of the CL-procedure has also been found by Scheijmans et al, in their study on the experiences of stakeholders during the CL-procedure of Nusinersen (Scheijmans et al, 2022). The lack of transparency is problematic from the perspective of procedural justice.…”

“…However, it is unclear for how many patients or in how many hospitals access was possible. A previous interview study amongst a diverse group of Dutch stakeholders regarding access to Nusinersen while it was in the lock, showed that stakeholders perceived the time which treatments spent in the lock to be too long ( Scheijmans et al, 2022 ). Another interview study amongst Dutch physicians showed that physicians sometimes encounter problems when they want to prescribe treatments which are in the CL ( Bomhof et al, 2022 ).…”

Efectividad clínica y seguridad del cribado neonatal de la atrofia muscular espinal. Revisión sistemática

“…• Scheijmans et al 2022 (38) : estudio realizado con datos procedentes de un registro de pacientes con AME tratados con nusinersén en Países Bajos. Se estudiaron a 69 pacientes (23, 30 y 16 con AME tipo I, II y III, respectivamente) en los que el inicio precoz del tratamiento se asoció a una mejor respuesta (p<0,001) en la consecución de hitos motores.…”