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Government Urged to Re-think its Proposed Changes to the HST Programme

29 March 2017

SMA Support UK will today join Genetic Alliance UK and Muscular Dystrophy UK at the All Party Parliamentary Group (APPG) for Muscular Dystrophy which NICE will also attend. There is widespread concern that the proposed changes – set to come into force from 1st April – could slow down or prevent patient access to drugs for rare diseases, such as SMA.

You can read Genetic Alliance’s summary of the proposed changes and what their impact would be here and a report in the Pharma Times here.

SMA Support UK today said:

“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.

Spinal Muscular Atrophy (SMA) is a rare genetic condition causing progressive loss of movement and muscle weakness. Children with SMA Type 1, the most severe form, are never able to sit unaided and 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.

The drug was licensed in the USA in December 2016. A European licence decision is pending. Pharmaceutical company Biogen opened an international Expanded Access Programme (EAP) in autumn 2016 so that children who could benefit from treatment wouldn’t have to wait, but due to our commissioning system only 9 of the estimated 100 children who could be eligible have been treated. Compare this to the 131 being treated in Italy and many more in other countries. Some desperate families feel time is against them and that they have no choice but to raise funds to access treatment overseas.

If these new rules come in to force, there would be no prospect of the drug being licensed in the UK and it is likely the EAP would close for new children. Imagine how this will be for families who are hoping to access this first and, thus far, only treatment for this condition. We urge Government to re-think this devastating decision.”