Mary backs fight on access to rare disease drugs

Mary has backed a major inquiry that warns hundreds of children with life-shortening conditions could be denied cutting-edge therapies, owing to drastic changes to the way drugs are funded and assessed.

Mary joined MPs, 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 on Tuesday (3 September).

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, have now been merged into the overall budget for NHS services commissioned across England. This leaves expensive therapies for rare conditions competing for funds with medications for prevalent conditions, such as diabetes or heart disease.[1]  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.[2]

Geneticists believe a pioneering genetic therapy, exon skipping[3], may well offer the first potential treatment for Duchenne muscular dystrophy. Results from advanced trials could lead to a licence application for the treatment as early as next year. The pipeline for other such ground breaking potential treatments for rare diseases looks set to grow; an international consortium on rare disease research was set up in 2011 with the goal of developing 200 further potential treatments by 2020 [4].

Karen Robinson has a son Thomas (6) who has Duchenne muscular dystrophy. She gave evidence to the APPG inquiry. Karen said: 
“Thomas has a type of Duchenne muscular dystrophy that exon skipping could potentially work for. So far, the condition only affects Thomas's mobility - he has trouble standing up, running, jumping and climbing stairs - he can do all these things, but not as fast or steadily as his peers. Without a treatment to stop it, his muscles will weaken and waste as he grows, affecting first his legs, then his arms and eventually his heart and lungs. We are racing against the clock. The earlier we are able to treat him, the more likely we are to stop further damage to his muscles. We need to be ready for when a treatment does become available, and plan ahead now so that a day is not lost before it reaches our children."

The All Party Parliamentary Group (APPG) for Muscular Dystrophy is calling on:

 Mary MP, 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.”

Robert Meadowcroft, Chief Executive of the Muscular Dystrophy Campaign, said:

“We greatly appreciate the support of Mary Glindon for this landmark report. This inquiry is a major step in the right direction, highlighting issues that need to be addressed with a sense of utmost urgency. These will not be simple medications, and alongside licensing and funding issues, we must also address the need for access to specialist care for the 3.5 million people living with rare conditions such as muscular dystrophy in the UK. Specialist care is needed in each region, to ensure that these emerging treatments are as effective as possible in extending lives and improving people’s health.”

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