NHS Wales Requesting Patient and Carer Views

As you’re probably aware, there is a new medication with the same active ingredient as Cystagon, cysteamine, but in a slow-release formulation – meaning a less frequent dosing schedule and potentially fewer side effects.

That drug is called Procysbi, it’s available to patients in other countries, including the US and parts of Europe. Unfortunately, this is not the case here in the UK. Due to its high-cost NHS decision makers have been reluctant to make Procysbi available to patients, despite it being approved by NICE.

But all is not lost, we have recently been contacted by the ‘All Wales Medicine Strategy Group’, who advise the Welsh Government on whether a new medicine should be used in NHS Wales. They have informed us that they are making a recommendation on Procysbi and they would like feedback from patients and carers to consider as part of their decision-making process.

Please fill in the online survey at the link below, to help them understand what life is like currently for people with cystinosis, you can also tell them about any unmet needs you feel that you or your child has, that could potentially be solved by the new medication.

Even if you don’t live in Wales your feedback is still welcome – if NHS Wales were to provide Procysbi to its patients this would put pressure on NHS decision-makers elsewhere in the UK to follow suit.


For the first field (Name of medicine) please enter ‘mercaptamine bitartrate (Procysbi)’. The deadline for submissions is 7th January 2022. For more information about the appraisal process please visit

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