Drug-funding changes ‘could hinder people with rare diseases’
Your support helps us to tell the story
From reproductive rights to climate change to Big Tech, The Independent is on the ground when the story is developing. Whether it's investigating the financials of Elon Musk's pro-Trump PAC or producing our latest documentary, 'The A Word', which shines a light on the American women fighting for reproductive rights, we know how important it is to parse out the facts from the messaging.
At such a critical moment in US history, we need reporters on the ground. Your donation allows us to keep sending journalists to speak to both sides of the story.
The Independent is trusted by Americans across the entire political spectrum. And unlike many other quality news outlets, we choose not to lock Americans out of our reporting and analysis with paywalls. We believe quality journalism should be available to everyone, paid for by those who can afford it.
Your support makes all the difference.Patients with rare diseases could be denied access to cutting-edge therapies because of changes to the way drugs are funded, MPs have warned.
The All-Party Parliamentary Group for Muscular Dystrophy raised concerns that money previously earmarked for rare-disease medication has been merged into the overall budget for NHS services in England.
They said this could mean expensive therapies for rare conditions will be forced to compete for funds against medications for common conditions such as diabetes or heart disease.
The group called on the Government to establish a ring-fenced fund for rare-disease drugs to ensure people have access to pioneering treatments.
The report also called for health regulator the National Institute for Health and Care Excellence to assess treatments for rare conditions differently from common ones.
They said health officials should also speed up access to drugs following the conclusion of clinical trials.
Labour MP Dave Anderson, who led the inquiry, said: “We have seen that successfully developing an effective treatment is far from the end, with agonising waits for some families through licensing and funding issues.
“We must focus on ensuring that if treatments are proven to be safe and effective, the UK is in a position to license and deliver them swiftly.
“We urge regulators and commissioners to overcome these obstacles and find a feasible, sustainable way of delivering pioneering rare-disease treatments. Otherwise the effective treatments may remain entirely out of patients’ reach.”
A Department of Health spokeswoman said: “Saving more lives by making sure people are diagnosed sooner and get the right treatment is one of our key priorities.
“NHS patients with rare diseases already receive some of the best levels of care in the world, but we are committed to improving this further. That is why we are working closely with other health departments and stakeholders to develop a UK strategy for rare diseases. This will be published by the end of the year.”
PA
Join our commenting forum
Join thought-provoking conversations, follow other Independent readers and see their replies
Comments