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<p>The National Institute for Health and Care Excellence (NICE) issues guidance for
the National Health Service on whether drugs and other treatments represent an effective
use of NHS resources through its technology appraisal (TA) and highly specialised
technologies (HST) programmes, including drugs for patients with rare diseases. 86%
of NICE’s TA recommendations, and 100% of its HST recommendations, for orphan drugs
have been positive since 2013.</p><p>Through the Early Access to Medicines Scheme,
we are already making important drugs available to patients. So far around 1,500 patients
have benefited from the scheme, which enables drugs to be used in clinical practice
in parallel with later stages of the regulatory process.</p><p> </p>
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