During today’s PM Question Time, Eleanor Smith MP asked the Prime Minister about access to Spinraza. We are grateful to this fantastic MP for Wolverhampton South West for bringing the issue up. Her constituent, little Heidi Prescott battles the disease while the NHS refuses to provide treatment to her and hundreds of other children and adults with spinal muscular atrophy.
We were somehow disappointed with the Prime Minister response as she seemed to express unconditional trust in NICE appraisal procedures. The procedures have for years been a subject of widespread criticism due to their unsuitability for appraising novel drugs for rare diseases, depriving thousands patients of access to breakthrough therapies.