Hundreds Of Children With Muscular Dystrophy May Be Denied Therapy

SDLP Foyle MP Mark Durkan has backed a major inquiry that warns hundreds of children with Muscular Dystrophy could be denied cutting-edge therapies, owing to drastic changes to the way drugs are funded and assessed.

Mr Durkan, who has consistently called for better services, planning and support for the 2,000 people in Northern Ireland who suffer from Muscular Dystrophy, joined other MPs, patients, their families, commissioners, regulators and charitable organisations at the launch of a report by the All Party Parliamentary Group for Muscular Dystrophy this week at Westminster into access to high-cost rare disease medicines.

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.

MPs are particularly concerned that funds previously earmarked for rare disease drugs are being merged into the overall budget for NHS services. This leaves expensive therapies for rare conditions competing for funds with medications for prevalent conditions, such as diabetes or heart disease. The MPs also reported serious concerns over the approval process for new drugs used by the National Institute for Clinical Excellence (NICE), which they fear may delay cutting-edge therapies or prevent them reaching children and young people entirely.

Mr Durkan said: "Talking to families at the reception brought home the urgent action required to fulfil the recommendations laid out in this inquiry. Without the right assessment processes and funding in place we risk families enduring agonising delays before their children can benefit from cutting-edge therapies, which could drastically improve their quality of life."

(CD/JP)