In order to ensure the maximum number of patients benefit from new innovative treatments coming on stream, choices need to be made about which specialised services and treatments to invest in. Decisions to fund treatments and drugs are made by NHS England based on their effectiveness following clinical trials or other evidence of effectiveness, and taking into account the views of expert clinicians and other stakeholders.
NHS England has advised that the Clinical Priorities Advisory Group (CPAG) was due to discuss Translarna and other treatments for rare conditions at its meeting on 3-4 December 2014. Following the cancellation of that meeting, CPAG is not expected to discuss Translarna until the current consultation on how NHS England will prioritise which specialised services and treatments to invest in has concluded. The consultation closes on 27 April 2015.
Any prioritisation which is urgent on clinical grounds will continue to be dealt with quickly through NHS England’s existing procedures. The route of individual funding requests remains the same.