The patients fast-tracked on new drugs BEFORE they've been approved: A little-known scheme offers a dramatic lifeline for those running out of options

As many as 400,000 people a year in England and Wales are told they've reached the end of their treatment options, according to charity Marie Curie. Patients with conditions as varied as advanced cancer, dementia and Parkinson's disease can find themselves in this heartbreaking situation. Patients are not usually allowed to take unlicensed medicines because they are not officially proven to be safe and effective and may have unknown side-effects. The drugs covered by EAMS have gone through medical trials to prove they are safe and effective, but are just awaiting approval. The EAMS scheme allows eligible patients access to drugs early. To date, 30 new treatments have been added to the scheme by watchdog the Medicines and Healthcare Products Regulatory Agency (MHRA), two-thirds of which have been cancer drugs. The 18-year-old has Duchenne muscular dystrophy (DMD), an incurable muscle-wasting condition that affects around 2,500 people in the UK. The condition also affects the muscles around his heart and lungs, causing breathing difficulties. 'A cold can last weeks because the muscles in his chest don't work properly and could cause pneumonia,' says his mother, Alison, 48, a civil servant from Preston, Lancs. Last year, Connor was offered the chance to start taking Raxone. Emily Crossley, from the charity Duchenne UK, says: 'The length of time it takes for new drugs to be approved can be gut-wrenchingly slow when your child has a very serious illness, so we welcome schemes such as the EAMS.' Katelyn, now 15, developed severe eczema — inflammation of the skin that affects 15 million people in the UK — aged two. 'There wasn't a patch of skin that wasn't affected,' recalls her mother Lucy, 41, a beauty therapist, from Essex. Lucy, who is married to Douglas, 44, an account manager, adds: 'We put prescribed creams on seven times a day, but she never felt comfortable.' When they become fathers for the first time, they smoke and drink less, and are less likely to be involved in crime, according to a 2011 U.S. study published in the Journal of Marriage and Family. ‘These decreases [in tobacco and alcohol use and crime] were in addition to the general tendency of boys to engage less in these behaviours as they approach and enter adulthood,’ said David Kerr, an assistant professor of psychology at Oregon State University and lead author of the study. 'This is a brand new way of treating the condition by targeting the beating heart of the inflammation process — two molecules in the skin that are responsible for the inflammation that causes the breakdown of the skin barrier,' says Professor Carsten Flohr, clinical lead for paediatriceczema at Guy's and St Thomas' Hospital, where Katelyn was treated. It means instead of itching and scratching all the time, she can concentrate and this has potentially changed her future.' For Katelyn it means she can finally lead a normal life; she is now studying for her GCSEs. The 55-year-old, from Cornwall, who runs his own website and marketing design company, has haemophilia A, a genetic disorder that prevents the blood clotting properly and affects more than 2,000 adults in the UK. When Paul was four he developed antibodies to his treatment, meaning the drug's effects lasted only for the 15 minutes it took to inject it into his body. The researchers suggest the two are linked as arthritis pain may make people more sedentary. 'Hemlibra is a big step forward,' says Professor Charles Hay, director of the NHS Manchester Haemophilia Comprehensive Care Centre.