Nusinersen (Spinraza®) for the treatment of 5q Spinal Muscular Atrophy

Registration

Nusinersen is an orphan drug that underwent the accelerated assessment programme of the EMA for the treatment of 5q spinal muscular atrophy (SMA), a rare, inherited, progressive muscular disease. No alternative treatment for SMA currently exists other than symptomatic treatment and compensating the loss of functions. Roughly speaking, four types of SMA can be differentiated, based on the appearance of the first symptoms and motor milestones that have been reached. The younger a person is when the first symptoms occur, the more severe will be the course of the disease.

Assessment

The National Health Care Institute assessed nusinersen for 3 groups of patients:

• SMA with infantile-onset (symptom onset before 6 months of age) whose disease was diagnosed less than 26 weeks before the start of treatment.
• SMA with later childhood-onset (symptom onset 6-20 months of age) in children who were diagnosed no more than 94 months before the start of treatment.
• pre-symptomatic infants genetically diagnosed with 5q spinal muscular atrophy and with 2 or 3 SMN2-copies.

Nusinersen is not cost-effective. The National Health Care Institute's estimate of the cost-effectiveness (ICER) for use in the three groups of patients on whom the product is effective far exceeds the relevant reference value of €80,000 per high-quality life-year gained (QALY). The National Health Care Institute estimated the ICER for type 1 SMA patients at about €600,000 per QALY and for type 2/3a patients at about € 1,700,000 per QALY.

The National Health Care Institute's advice

Based on the assessment, the National Health Care Institute advises the Ministry of VWS not to include nusinersen in the insured package as addition to best supportive care, unless the cost-effectiveness of this treatment can be improved, and its impact on the care budget reduced by means of price negotiation. The price of nusinersen would have to be reduced by at least 85% in order for it to be considered cost-effective.

If price negotiations lead to inclusion in the basic insurance, the National Health Care Institute will conclude an orphan drug arrangement that records agreements on appropriate use (e.g. start and stop criteria and, if possible, a European register for monitoring the effect on Quality of life). Furthermore, the National Health Care Institute will actively monitor its use and costs in the orphan drugs monitor. Initiatives of the SMA centre of expertise for appropriate use offer good leads in this respect. This is to support the policy of the government and health care insurers, and to promote appropriate use.

Apart from the insured package, we advise the Ministry of VWS to make agreements with the manufacturer about research into the effectiveness of nusinersen for all groups of SMA patients, so that - for them too – a statement can eventually be issued on reimbursement. Until then, the costs of research and of the medicine must be borne by the manufacturer. We also took into consideration that the manufacturer is working under the umbrella of European patent protection. This is intended as legal protection against unrestrained competition and serves the public interest, namely: the development of innovative medicines. We feel that, in exchange for this protection, the manufacturer should be prepared to further promote the appropriate use of nusinersen. Our advice is emphatically in line with the ambition expressed by the manufacturer during the meeting of the Insured Package Advisory Committee (ACP): to give patients with SMA a central role.

This report is a summary of recommendations by the National Health Care Institute. The original text of this excerpt is in Dutch.