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Muscle-Wasting Conditions

Volume 561: debated on Tuesday 16 April 2013

11. What steps the Government plan to take to ensure that patients with rare and very rare muscle-wasting conditions have access to high cost drugs when such treatments become available. (150774)

Our priority is to ensure that patients in England, including those with rare and very rare muscle-wasting conditions, have access to new and effective treatments on terms that represent value to the NHS and the taxpayer.

The all-party parliamentary group for muscular dystrophy, which has been conducting an inquiry, was told recently that patients who are experiencing muscle-wasting conditions, and their families, are concerned about the possibility that they will be denied access to potential treatment because of regulatory barriers, and/or on cost grounds. Will the Minister meet the all-party group to discuss measures for the removal of any such impediments?

I am certainly willing to meet the all-party group, but I think that significant new opportunities are emerging. For example, from this month the National Institute for Health and Clinical Excellence will be responsible for the evaluation of new drugs for the treatment of rare conditions, and I think that that is a very good thing.

One of my constituents, a seven-year-old boy, has Duchenne muscular dystrophy. His family are pinning their hopes on a new drug called ataluren, which has not yet completed its trials. Can the Minister give me any idea when it might become available?

I understand that the manufacturer of ataluren has applied for conditional approval from the regulatory authorities. We await the outcome of that process, but I am afraid that I cannot give a time scale for it.

Eculizumab is a high-cost drug that makes a profound difference to the lives of people with a rare kidney disorder, yet the Department has rejected the recommendation of the Advisory Group for National Specialised Services to make it available to all patients, instead referring it to the National Institute for Health and Clinical Excellence, which will delay any decision by at least a year. Will the Minister meet me and specialists from my Newcastle constituency to discuss how we can ensure those patients are not adversely affected by this delay?

I am certainly happy to have a discussion with the hon. Lady, and to look into the concerns she raises and get back to her on them.

As the Minister is aware, the active involvement of patients in biomedical research is one of the areas in which Britain is increasingly leading. Yesterday, I chaired a meeting with my hon. Friend the Member for The Cotswolds (Geoffrey Clifton-Brown) and the Empower: Access to Medicine group and Les Halpin, who is suffering from a terminal disease and is launching a campaign for greater patient involvement in access to rare drugs. Will the Minister meet me and my hon. Friend to take that forward?

I seem to be ready to agree to several meetings and I will certainly add that one to my list. I will be delighted to meet my hon. Friend—and I should also pay tribute to him for the work he is doing in this area.