Rare Diseases: Children:Written question - 155186

Asked on: 19 June 2018
Department of Health and Social Care
Rare Diseases: Children
To ask the Secretary of State for Health and Social Care, what steps he is taking to make access available to drugs for children with rare diseases.
Answered by: Steve Brine
Answered on: 27 June 2018

The National Institute for Health and Care Excellence issues guidance for the National Health Service on whether drugs and other treatments represent an effective use of NHS resources through its technology appraisal and highly specialised technologies programmes, including drugs for children with rare diseases.

Through the Early Access to Medicines Scheme, we are already making important drugs available to patients. So far around 1500 patients have benefited from the scheme, which enables drugs to be used in clinical practice in parallel with later stages of the regulatory process.

We have brought together key government, NHS and industry partners to form the Accelerated Access Collaborative (AAC), who will drive the update and adoption of innovation in the NHS. The AAC will also oversee the Accelerated Access Pathway, streamlining regulatory and market access decisions.

The UK Strategy for Rare Diseases includes commitments to improving the lives of all patients affected by rare diseases including in areas of identification/ prevention and diagnosis and early intervention. European Reference Networks (ERNs) are a cornerstone of this Strategy. ERNs are virtual networks between expert healthcare providers across EU member states. Their functions are to pool specialist expertise to improve the diagnosis, treatment and care of rare diseases patients across Europe and act as focal points for highly specialist training and research, for example through the facilitation of clinical trials and development of new drugs and treatments.

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