Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, whether his Department has made an assessment of the potential impact of Early Access Programmes on equity of access to (a) tofersen and (b) other innovative treatments.

Early access schemes for unlicenced medicines, where approval may be obtained in other markets, including where a licence exists but a National Institute for Health and Care Excellence decision is not available, are managed by companies to benefit patients.

The Medicines and Healthcare products Regulatory Agency’s (MHRA) Early Access to Medicines Scheme (EAMS) provides a route for the supply of new medicines to United Kingdom patients on an unlicensed basis, prior to receiving a marketing authorisation. The EAMS aims to give patients with life threatening or seriously debilitating conditions early access to medicines that show early signs of having a major advantage over existing therapeutics.

The EAMS is a two-step process, with the first step being a Positive Innovative Medicine designation, and the second step being the publishing of a Scientific Opinion. Medicines have to be successful at both of these stages to have access to patients through this scheme.

We are currently reviewing the recently submitted Torfersen, but cannot comment on its progress. The MHRA recognises the importance of rapid assessment, and is committed to doing so in a timely manner.