UK Government and Members of Parliament
United Kingdom

NICE and NHS England’s decision puts patient access to ultra-rare disease medicine at serious risk. This change to the arrangements for evaluating and funding drugs and other healthcare technologies assessed through NICE’s highly specialised technology appraisal treats children and young adults with ultra-rare diseases as economic pawns, according to Paul Moody, Chair of Trustees for the MPS Society.

If we don’t stop these changes coming into force from 1 April 2017, we 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 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.

Read the NICE press release:

Download the full announcement from NICE and NHS England in the NICE board papers released on 15 March 2017:

Download the MPS Society response to the consultation:

Read the MPS Society press release:

We, the undersigned, call for a PAUSE to the changes to the NICE drug evaluation process to allow time for the decision to be reconsidered by Parliament.

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The Petition for a PAUSE to NICE and NHS England decision to introduce QALYS into the appraisal of highly specialised technologies affecting reimbursement of drugs for ultra-rare diseases in England petition to UK Government and Members of Parliament was written by MPS Society and is in the category Health at GoPetition.