The Health Service Executive (HSE) has statutory responsibility for decisions on pricing and reimbursement of medicinal products under the community drug schemes in accordance with the provisions of the Health (Pricing and Supply of Medical Goods) Act 2013. The decisions on which medicines are reimbursed by the taxpayer are made on objective, scientific and economic grounds by the HSE.
For a medicine to be considered for reimbursement it must first have a marketing authorisation from the European Medicine Agency (EMA), before being assessed under the 2013 Act.
The EMA provides independent recommendations on medicines for human and veterinary use, based on a comprehensive scientific evaluation of data.
The EMA’s evaluations of marketing authorisation applications provide the basis for the authorisation of medicines in Europe. This is separate to the Statutory Assessment process conducted by the HSE.
I understand from the HSE that Biogen have applied to the EMA for market authorisation in the EU for the drug Nusinersen brand name Spinraza. The application to the EMA was submitted in Autumn 2016. The EMA assessment process generally takes around one year.
Should Nusinersen receive European marketing authorisation, the manufacturer would then be open to make an application to the HSE to have the product reimbursed under the community drug schemes.
The operation of patient access schemes is at the discretion of manufacturers. There is no provision in Irish legislation for the approval of compassionate use programmes for specific groups of patients with an unmet medical need. However I understand that following an application from a consultant paediatric neurologist at Temple Street hospital, the company has extended their early access programme to two of his patients with SMA Type 1 and that the patients' families have been informed.
