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.”