A coalition of almost 200 patient groups representing families affected by rare and genetic conditions have called for a halt to planned changes in the way therapies are appraised for NHS use, as they “threaten patients’ ability to access treatment, and work against the government's goal of accelerating access to innovative medicines”.

NICE recently announced that rare disease treatments deemed to provide significant QALY benefits will be assessed by its Highly Specialised Technologies (HST) committee against a maximum threshold of £300,000 per QALY. However, many treatments for very rare conditions currently funded by NHS England have costs per QALY in excess of £500,000, including three of the four medicines that have been approved by the HST process to date.

Led by Genetic Alliance UK, the coalition argues that proposals that limit recommendations by NICE’s HST Evaluation programme will prevent access to innovative treatments for rare genetic conditions in England, “and send a chilling message to the life sciences sector that runs contrary to messages from other parts of Government”.

In a letter to health secretary Jeremy Hunt, patients are calling for a pause in the implementation of the proposals, planned for 1 April 2017, to ensure that NICE’s and NHS England’s approaches are in line with the needs of UK patients and families affected by rare genetic conditions.

They insist that changes to the HST programme be stopped because: England already has “extremely slow and limited access” to treatments for rare genetic conditions; to allow for an impact assessment as it is “unacceptable” to implement such “drastically damaging proposals” just eighteen days after they are announced; and because the changes are “contradictory to the positive recommendations made in the Accelerated Access Review, and will be restrictive to any attempt through the Industrial Strategy to position the UK as a centre for the development of innovative medicine”.

“These proposals will push the UK even further down the global ladder of countries that respond in a timely and efficient way to scientific breakthroughs,” warned Doug Henderson, SMA (Spinal Muscular Atrophy) Support UK managing director.

Children with the most severe form of SMA rarely survive their second birthday. New drug treatment nusinersen has encouraging evidence of its clinical effectiveness for these children with potential for also treating those with other less severe forms of SMA, he notes.

“If these new rules come into force, there would be no prospect of the drug being licensed in the UK. Imagine how this will be for families who are hoping to access this first and, thus far, only treatment for this condition. We urge the government to halt this devastating decision.”