Rare disease patients 'at risk'

Patients with rare diseases could be put at risk because of changes to the way medication is paid for, MPs warn. The All Party Parliamentary Group for Muscular Dystrophy fears they will have to compete against treatments for more common conditions.

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Govt: Rare diseases are a 'key priority'

The Government has denied patients with rare diseases may miss out on vital medication because of the way treatment is funded.

A Department of Health spokeswoman said making sure treatment for patients with rare diseases was a "key priority" and they would be publishing a strategy shortly.

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.

– A Department of Health spokeswoman

Patients with rare diseases face 'agonising wait'

Some patients with rare diseases suffer an "agonising wait" for the medication they need because of the way it is funded, MPs have said.

Labour MP Dave Anderson, who led the inquiry into funding of medication for rare diseases said the UK needed to be in a position to deliver treatment "swiftly".

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 - something which we most certainly are not now.

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.

– Labour MP Dave Anderson


MPs: Rare disease treatment funds should be separate

Patients with rare diseases may suffer if they have to compete for funding with people with more common illnesses, MPs have warned. Credit: PA

Changes to the way cutting-edge therapies are funded on the NHS could prevent patients with rare disease getting the treatment they need, MPs have warned.

Money previously put aside specifically to pay for medication for rare diseases has now been filtered into the overall NHS budget for services in England, warned the All Party Parliamentary Group for Muscular Dystrophy (APPG).

They expressed concerns patients with rare conditions would have to compete for funds alongside medication for more common conditions, like diabetes or heart disease.

The APPG called on the Government to re-establish "ring-fencing" for funds to buy special medication so people suffering from rare diseases still have access to the treatment they need.

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