Rare Disease Strategy implementation plans released

The Department of Health and Social Care and NHS England have published their implementation plans for the UK Strategy for Rare Diseases.

The Strategy, which was published in November 2013, contains commitments aimed at improving access to support and treatments for people with rare diseases such as muscular dystrophy and neuromuscular conditions.

Content of the Strategy

The Strategy covers five key themes which are:

• Empowering those with rare diseases
• Identifying and preventing rare diseases
• Diagnosis and early intervention
• Co-ordination of care
• Role of research in rare diseases

Actions to implement the Strategy

The Department of Health and Social Care and NHS England have set out how they plan to implement the Strategy, including:

• From April 2018, Government will introduce an Accelerated Access Pathway (AAP) that aims to streamline regulatory and market access decisions, getting innovative treatments to patients more quickly
• National Institute for Health Research (NIHR), through its INVOLVE programme, actively promoting and leading on activities to encourage greater patient and public involvement, engagement and participation in research
• identifying an individual to co-ordinate care
• emphasis on giving patients alert cards for their condition
• ensuring an active transition from paediatric to adult services

Implementation plans in Scotland, Wales and Northern Ireland have already been produced.

Muscular Dystrophy UK perspective

We welcome the publication of these plans and are keen to work with NHS England and Department of Health and Social Care to ensure their implementation. With an increasing number of potential treatments for muscle-wasting conditions it is critical that specialist centres are well-staffed and well-supported to ensure these new drugs reach those who desperately need them as quickly as possible.