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<p>The Department regularly meets with the National Institute for Health and Care
Excellence (NICE) to discuss a range of issues including access to and availability
of medicines. NICE’s methods and processes for health technology evaluation have been
proven to be suitable for medicines for rare diseases where companies price their
products responsibly, and NICE introduced a number of changes to its methods and processes
in 2022 that ensure that its processes are appropriate to the evaluation of emerging
new technologies.</p><p>NICE also operates a highly specialised technologies programme
for the evaluation of a small number of medicines for the treatment of very rare diseases.
As of 31 March 2024, NICE has recommended 88% of medicines licensed for the treatment
of rare diseases for some or all of the eligible patient population, which is comparable
to NICE’s approval rate for all medicines.</p><p>The Innovative Medicines Fund, building
on the success of the Cancer Drugs Fund, provides a mechanism for consistent and transparent
managed access process for companies offering promising non-cancer medicines at a
responsible price. The Fund has already provided early access for National Health
Service patients to several innovative new treatments, including for patients with
rare diseases such as graft-versus-host disease and Wolman disease.</p>
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