Last week the All Party Parliamentary Group for Muscular Dystrophy published a report on access to high-cost drugs for rare diseases. Baroness Thomas of Winchester, the group’s vice-chair, hopes its message will be heeded, she writes here
Suppose you were the parent of a boy of five who had just been diagnosed with the rare disease of Duchenne Muscular Dystrophy, for which there is no treatment, let alone cure. You might be in despair at the thought of your active five-year-old gradually becoming less active, then wheelchair-bound, and eventually unable to breathe without a ventilator.
And suppose you then heard that some very promising gene therapy, called exon-skipping – exactly relevant to your son’s condition – which might delay the disease’s progression, was in stage-three clinical trials and might be available very soon. The one thing that you would not want to hear is a row about whether the drug could be afforded when it was pitted against drugs for much more common diseases, such as heart disease.
This was the subject of the All Party Parliamentary Group for Muscular Dystrophy’s Report on access to high-cost drugs for rare diseases, which was published last week. As the Vice-Chair of the group, and someone with a much less severe form of the disease, I meet a lot of families who are coping with the day-to-day realities of having a child or young adult with this devastating condition. They know there is not a moment to lose because the disease progresses quickly and treatments have proved very elusive.
The report calls for four key things: for the Government to establish a ring-fenced fund for rare disease drugs, for the National Institute for Health and Care Excellence to assess treatments for rare conditions in a different way from less rare conditions, for the relevant agencies to speed up access to life-changing drugs after the final stages of clinical trials to ensure there are no major delays in treatments reaching children, and for NHS England to ensure that specialist centres are equipped with an appropriate range of healthcare professionals to deliver treatments.
At the launch of the report, the Minister for Care and Support, Norman Lamb, who met several families and boys affected by Duchenne MD, was very encouraging which gave us hope that our report’s message would be heeded.
Published and promoted by Tim Gordon on behalf of the Liberal Democrats, both at LDHQ, 8-10 Great George Street, London, SW1P 3AE.