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In late October 2016 NHS England and NICE launched a 12-week consultation on changes to the arrangements for evaluating and funding drugs and other health technologies appraised through NICE’s Highly Specialised Technologies (HST) programmes.

This is a clear message from NICE and NHS England published on 15th March, that they are unwilling to continue to pay for innovation treatments for rare diseases to the extent that they do now.

In summary, these changes coming into force from April 1 this year, will see the introduction of a £100,000 Quality-adjusted life year (QALY) threshold for medicines evaluated via NICE’s Highly Specialised Technologies (HST) programme, which assesses treatments for ultra- rare diseases. This threshold will effectively stop the flow of new medicines reaching patients with ultra-rare and complex diseases. Many treatments for ultra-rare conditions that are currently funded by NHS England have costs per QALY of more than £500,000 including the three medicines that have been approved by NICE’s HST process to date. It is widely acknowledged that QALY thresholds are not appropriate for evaluating medicines for ultra-rare diseases, due to the small patient populations and often limited data.

The Gauchers Association, Members of other patient groups, and the Pharmaceutical Companies will work tirelessly to put pressure on The UK government who now need to act at lightning speed and reverse this initial NICE & NHS England policy not just in the context of patients with ultra-rare diseases but also that of the life sciences industry who will see no incentive to investing in the UK market if their innovative medicines and technologies have no prospect of reaching the patient.

You can read the full announcement from NICE and NHS England in the NICE board papers released on 15 March 2017 here

We will use our website and Facebook page to keep our patient community informed. If any of our Members or their family Members would like to talk about this further, please call the Gauchers Association on: 01453 549231