What steps the Government is taking to help ensure tofersen can be administered to MND patients under the early access programme.
Companies may put in place Early Access Programmes (EAPs) to allow early access to new medicines which have been licensed by the Medicines and Healthcare Products Regulatory Agency, but which do not yet have National Institute for Health and Care Excellence guidance. Participation in such programmes is decided at an individual National Health Service trust level. The manufacturer of tofersen, Biogen, has established an EAP for people diagnosed with SOD1 amyotrophic lateral sclerosis who meet the inclusion and exclusion criteria set out by the company.Under EAPs, the cost of the drug is free both to patients taking part in it and to the NHS, although NHS trusts must still cover the costs of administering the medicine. NHS England does not therefore have any involvement in directing EAP enrolment by trusts and the Department has no plans to issue any national direction to trusts to participate in such programmes.Many NHS hospitals have been able to identify the extra staff and resources needed to take part in the EAP and safely provide tofersen to as many eligible patients as possible. Neither NHS England nor the Department holds any data on the number of NHS trusts or patients accessing tofersen through the company led EAP.