Andrew Miller, MP for Ellesmere Port and Neston, has backed a major inquiry that warns that hundreds of children with life-shortening conditions could be denied cutting-edge therapies.
Andrew joined colleagues, patients, their families, commissioners, regulators and charitable organisations at the launch of a report by the All Party Parliamentary Group for Muscular Dystrophy into access to high-cost rare disease medicines this week.
The hard-hitting report reveals that while potential treatments for the devastating muscle-wasting condition Duchenne muscular dystrophy are finally in clinical trials, parents fear that the ‘race against time’ for their children to be treated may be lost owing to unnecessary delays, funding issues and bureaucracy.
The Muscular Dystrophy Parliamentary Group is calling on: the Government to establish a ring-fenced fund for rare disease drugs; NICE to assess treatments for rare conditions differently from less rare conditions; the Medicines and Healthcare Products Regulatory Agency (MHRA); NICE and NHS England to speed up access to life-changing drugs after the final stages of clinical trials; NHS England to ensure specialist centres are equipped with an appropriate range of health professionals to deliver treatments.
Andrew Miller said: “I am concerned that funds previously earmarked for rare disease drugs, have now been merged into the overall budget for NHS services commissioned across England.”
