Advisory report – risdiplam (Evrysdi®) as a possible candidate for conditional inclusion for treating spinal muscular atrophy (SMA)

Risdiplam is intended for certain people with spinal muscular atrophy

Risdiplam is the active substance; the brand name is Evrysdi®. The medicinal product is a liquid you drink.

It can be used in people with the muscular disease SMA. SMA is the abbreviation for spinal muscular atrophy. As the muscle weakness increases, patients become wheelchair-dependent. Ultimately, the muscular weakness also leads to curvature of the spine, loss of arm and hand function and paralysis of the respiratory muscles. 

Advisory report from the National Health Care Institute

The National Health Care Institute concluded in its advisory reports issued in 2022 and 2023 that risdiplam can only be reimbursed for treatment of patients aged up to 25 years at the start of treatment with a clinical diagnosis of 5q SMA types 1, 2 or 3, or with 1 to 4 copies of the SMN2 gene.

For patients aged 26 and older at the start of treatment, there is insufficient evidence to assess whether risdiplam meets the criterion of ‘established medical science and medical practice’ (hereinafter also “SWP”). On 28 May 2025, the parties therefore submitted an application for conditional inclusion as an orphan drug, conditional or exceptional for risdiplam. The requested application covers patients aged 26 and older at the start of treatment with a clinical diagnosis of 5q SMA type 1, 2 or 3, or with 1 to 4 copies of the SMN2 gene.

Based on the data in the dossier and the advisory report of the Scientific Advisory Board (hereinafter also “WAR”), the National Health Care Institute has concluded that treatment with risdiplam for this subset of SMA patients aged 26 and older who cannot or can no longer be treated with nusinersen meets the criteria for conditional inclusion. Those criteria are as follows:

• risdiplam was registered as an orphan drug in 2021
• there is an unmet medical need for the group of patients who cannot or can no longer be treated with nusinersen
• the marketing authorisation holder is the dossier’s lead applicant; the co-applicants are the attending physicians, an independent research centre and the patient association 
• we expect that the data collected by this research will allow the package question to be answered
• we expect that package inclusion question will be answered within 6.5 years.

Based on these conclusions, we recommend that risdiplam should be designated as a potential candidate for conditional inclusion. If the minister adopts our advisory report, phase 2 of the procedure will commence. In this phase, the parties must formulate their plans in greater detail and draw up a covenant setting out the agreements needed to ensure a careful and successful CI process.

Temporary admission to the basic healthcare package

Since 1 January 2012, healthcare that does not meet the criterion of established medical science and medical practice, i.e. healthcare that has not been proved to be effective, can still be temporarily admitted to the basic health care package. We call this “conditional inclusion”. The condition is that the experimental group collects data on the effectiveness and cost-effectiveness of the care during the period of conditional inclusion. At the end of the conditional inclusion period, this data will help to determine whether the care can be a permanent part of the basic healthcare package.

This report is a summary of recommendations by the National Health Care Institute

The original text of this report is in Dutch.