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

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

Subacute Combined Spinal Cord Degeneration by Recreational Laughing Gas (NO) Use

Background: A few young patients were brought in with subacute combined spinal cord degeneration at the Department of Neurology in our hospital. They all have used laughing gas for recreational purposes. Case: A 30-year-old woman, known with alcohol abuse, was presented to our Department of Neurology for having paresthesia and unstable movements of arms, legs, and trunk for 9 days. She has used 50 laughing gas patterns per day. The diagnosis of laughing gas–induced combined spinal cord degeneration was evident by the low count of vitamin B 12 combined with lesions shown on magnetic resonance imaging (MRI). Abstaining from the laughing gas, weekly intramuscular injections of hydroxocobalamin and revalidation, she was fully recovered in 8 weeks. Conclusions: Recreational use of laughing gas seems to be more used in our society, however, without having any knowledge of the neurological consequences. The right diagnosis and treatment can provide full recovery in these patients. Furthermore, attention for this diagnosis can help increase social awareness