Drug-funding changes ‘could hinder people with rare diseases’

 

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

Life and Style
ebookNow available in paperback
ebooks
ebookA delicious collection of 50 meaty main courses
Latest stories from i100
Have you tried new the Independent Digital Edition apps?

ES Rentals

    Independent Dating
    and  

    By clicking 'Search' you
    are agreeing to our
    Terms of Use.

    iJobs Job Widget
    iJobs General

    Recruitment Genius: 3rd Line Virtualisation, Windows & Server Engineer

    £40000 - £47000 per annum: Recruitment Genius: A 3rd Line Virtualisation / Sto...

    Recruitment Genius: Refrigeration and Air Conditioning Service Engineer

    £26000 - £35000 per annum: Recruitment Genius: A successful national service f...

    Recruitment Genius: Business Development Executive / Sales - OTE £25,000

    £15000 - £25000 per annum: Recruitment Genius: This is an opportunity to join ...

    Recruitment Genius: Sales Administrator - Fixed Term Contract

    £17500 - £20000 per annum: Recruitment Genius: We currently require an experie...

    Day In a Page

    Syria civil war: Meet the military commander who says his soldiers will not rest until every inch of their war torn country is free of Islamist 'terrorists'

    ‘We won’t stop until Syria is back to normal’

    Near the front lines with Islamist-controlled towns where Assad’s troops were besieged just last month, Robert Fisk meets a commander confidently preparing his soldiers for battle
    Fifa corruption: Strip Qatar of the World Cup? Not likely

    Strip Qatar of the World Cup? Not likely

    But if a real smoking gun is found, that might change things, says Tom Peck
    Twenty two years later Jurassic Park series faces questions over accuracy of the fictional dinosaurs in it

    Tyrannosaurus wrecked?

    Twenty two years on, Jurassic Park faces questions over accuracy
    The inside story of how Bill Clinton built a $2bn global foundation may undermine Hillary's chances

    The inside story of how Bill Clinton built a $2bn global foundation...

    ... and how it may undermine Hillary's chances in 2016
    Genes greatly influence when and how many babies a woman will have, study finds

    Mother’s genes play key role in decision to start a family

    Study's findings suggest that human fertility is still evolving
    12 best olive oils

    Extra-virgin, cold-press, early-harvest, ultra-premium: 12 best olive oils

    Choosing an olive oil is a surprising minefield. Save yourself the hassle with our handy guide
    Rafa Benitez Real Madrid unveiling: New manager full of emotion at Bernabeu homecoming

    Benitez full of emotion at Bernabeu homecoming

    There were tears in the former Liverpool manager’s eyes as he was unveiled as Real Madrid coach. But the Spaniard knows he must make tough decisions if he is to succeed
    England can win the Ashes – and Elvis Presley will present the urn

    England can win the Ashes – and Elvis will present the urn

    In their last five Test, they have lost two and drawn two and defeated an India side last summer who thought that turning up was competing, says Stephen Brenkley
    Sepp Blatter resignation: The beginning of Fifa's long road to reform?

    Does Blatter's departure mean Fifa will automatically clean up its act?

    Don't bet on it, says Tom Peck
    Charles Kennedy: The baby of the House who grew into a Lib Dem giant

    The baby of the House who grew into a Lib Dem giant

    Charles Kennedy was consistently a man of the centre-left, dedicated to social justice, but was also a champion of liberty and an opponent of the nanny-state, says Baroness Williams
    Syria civil war: The harrowing testament of a five-year-old victim of this endless conflict

    The harrowing testament of a five-year-old victim of Syria's endless civil war

    Sahar Qanbar lost her mother and brother as civilians and government soldiers fought side by side after being surrounded by brutal Islamist fighters. Robert Fisk visited her
    The future of songwriting: How streaming is changing everything we know about making music

    The future of songwriting

    How streaming is changing everything we know about making music
    William Shemin and Henry Johnson: Jewish and black soldiers receive World War I Medal of Honor amid claims of discrimination

    Recognition at long last

    Jewish and black soldiers who fought in WWI finally receive medals after claims of discrimination
    Beating obesity: The new pacemaker which helps over-eaters

    Beating obesity

    The new pacemaker which helps over-eaters
    9 best women's festival waterproofs

    Ready for rain: 9 best women's festival waterproofs

    These are the macs to keep your denim dry and your hair frizz-free(ish)