Eyesight: Rare Diseases
The question
To ask the Secretary of State for Health and Social Care, what steps his Department is taking to ensure that patients living with rare inherited retinal diseases receive (a) timely and (b) equitable access to new treatments.
Answered by Andrew Gwynne
The Government is committed to improving the lives of those living with rare diseases, such as rare inherited retinal diseases. The UK Rare Diseases Framework sets out four priorities collaboratively developed with the rare disease community, including improving access to specialist care, treatments, and drugs. We remain committed to delivering under the framework, and will publish an England action plan in 2025.
The National Institute for Health and Care Excellence (NICE) makes recommendations for the National Health Service on whether all licensed new medicines should be routinely funded by the NHS based on an assessment of their costs and benefits, and NHS England ensures that funding is available for any licensed new medicines recommended by the NICE. The NICE aims to issue guidance on new medicines, including for rare diseases, as close to the time of licensing as possible. The NICE operates a separate Highly Specialised Technologies (HST) programme for very rare diseases, with significantly higher cost-effectiveness thresholds than those evaluated under the NICE’s standard technology appraisal processes. Decisions on whether new medicines should be evaluated through the HST programme are taken by the NICE against published routing criteria.
The NICE is currently appraising one treatment for treating visual impairment caused by a rare inherited retinal disease, and final guidance is expected to be published in April 2025.
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