Jim Shannon – 2016 Parliamentary Question to the Department of Health
The below Parliamentary question was asked by Jim Shannon on 2016-10-17.
To ask the Secretary of State for Health, what steps the Government is taking to reduce the time taken for people with (a) rare and ultra-rare conditions, (b) Morquio A Syndrome and (c) Duchenne Muscular Dystrophy to access treatment.
Nicola Blackwood
The National Institute for Health and Care Excellence (NICE) has advised that, with NHS England, it is currently running a consultation which sets out proposals to enable faster access to therapies for patients with ultra-rare conditions. The proposals outline what conditions need to be met in order to receive a positive recommendation through the NICE highly specialised technologies programme and what mechanisms should be in place to introduce new technologies and manage the affordability challenge for NHS England.
NICE has also advised that a proportion of patients with Morqiuo syndrome who have met the criteria under the managed access agreement are already receiving elosulfase alfa (Vimizim) and are being monitored by their specialist centre. The remainder are being assessed to ensure that they fulfil the eligibility criteria and patients who do will start treatment within the first year of the managed access agreement coming into force. A very small proportion of the population has decided for personal reasons not to participate.
NHS England and PTC Therapeutics, with the brokering of NICE, agreed a managed access agreement that will enable access to translarna (Ataluren) for the treatment of Duchenne muscular dystrophy. Specialist centres are in the process of reviewing potentially eligible patients to assess their suitability to receive the drug under the terms agreed in the managed access agreement.
