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219 First year of the early access to medicines scheme for raxone in duchenne muscular dystrophy
  1. Catherine Lawrence1,
  2. Shabir Hasham2
  1. 1Santhera Pharmaceuticals UK
  2. 2Santhera Pharmaceuticals, Switzerland

Abstract

Raxone (idebenone) received the first positive scientific opinion for a treatment option in DMD for inclusion into the Early Access to Medicines Scheme (EAMS) in June 2017.

EAMS is regulated by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and aims to provide earlier availability of promising new unlicensed medicines to UK patients that have a high unmet clinical need.

The first patient was enrolled 12 weeks after the scheme approval. In the first year of the scheme forty-six patients were able to access Raxone in six centres. Ease of implementation of the scheme across the NHS was variable. In addition to the MHRA positive scientific opinion, and supportive guidance issued by NHS England and Scotland, most clinicians were required to obtain local hospital committee approvals before entering the scheme, which introduced delays in patient enrolment. Local knowledge of EAMS, its objectives and stakeholder responsibilities, was found to be limited and also contributed to delays in implementation and inequality of access.

Under EAMS Raxone is administered under routine clinical practice. In the second year additional data collection has been introduced to obtain information on the effectiveness of the use of Raxone in an environment similar to that which would exist if the medicine receives a future marketing authorisation for DMD.

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