Package advice risdiplam (Evrysdi®) for the treatment of 5q spinal muscular atrophy (SMA)
The National Health Care Institute advises the Minister to include risdiplam in the health insurance package for indications that comply with established medical science and medical practice, subject to the condition that a price reduction is achieved. The National Health Care Institute has concluded that risdiplam complies with established medical science and medical practice for the treatment of 5q SMA in patients from 2 months to 25 years of age, with a clinical diagnosis of SMA type 1, type 2, or type 3, and for presymptomatic patients with one to four copies of the SMN2 gene. The reason for this advice is that risdiplam is being placed in the lock procedure for expensive medicinal products.
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Registered indication
Reimbursement is requested for the treatment of 5q spinal muscular atrophy (SMA) in patients aged 2 months of age and older with a clinical diagnosis of SMA type 1, type 2, or type 3, or with one to four SMN2 copies.
Package advice
Risdiplam complies with established medical science and medical practice for the treatment of 5q SMA in patients from 2 months to 25 years of age, with a clinical diagnosis of SMA type 1, type 2, or type 3, and for presymptomatic patients with one to four copies of the SMN2 gene.
Due to insufficient evidence, risdiplam does not (yet) comply with established medical science and medical practice for the treatment of patients above 25 years of age, with a clinical diagnosis of SMA type 2 or type 3.
The National Health Care Institute advises the Minister to include risdiplam in the health insurance package for indications that comply with established medical science and medical practice, subject to the condition that a price reduction is achieved.
For the treatment of patients above 25 years of age with a clinical diagnosis of SMA type 2 or type 3, the National Health Care Institute, together with all of the stakeholders, will endeavour to make risdiplam available via conditional inclusion. The National Health Care Institute, the patients’ association, and the physicians’ association share the explicit wish that risdiplam be added to the current conditional inclusion process for nusinersen. This would mean that no extra budget needs to be arranged, provided the price of risdiplam is equal to or less than that of nusinersen.