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The new deal struck by the NHS will enable provision of a life-saving treatment for babies and young children who suffer with a rare and fatal genetic disease, metachromatic leukodystrophy (MLD). The revolutionary gene therapy treatment, known by its brand name Libmeldy, is used to treat MLD, which causes severe damage to the child's nervous system and organs, leading to a life expectancy of just five to eight years. Having a reported list price of more than £2.8 million, it is the most expensive drug in the world, but can now be offered to young patients on the NHS in England after the health service negotiated a significant confidential discount. the drug works by removing the patient's stem cells and replacing the faulty gene that causes MLD before then re-injecting the treated cells into the patient. The most common form of MLD usually develops in babies younger than 30 months and can cause loss of sight, speech and hearing, as well as difficulty moving, brain impairment, seizures, and eventually death.

The Department of Health and Social Care (DHSC) launched a new Innovative Medicines Fund on Tuesday (June 7) under which £340 million has been made available to purchase potentially life-saving drugs early. This will allow NHS patients in England to have early access to potentially life-saving and cutting-edge treatments Health secretary Sajid Javid said: "I want NHS patients to be the first in the world to access the most promising and revolutionary treatments that could extend or save their lives. "The launch of the Innovative Medicines Fund delivers another manifesto pledge and will fast-track cutting-edge medicines to adults and children to give people renewed hope for a better future." A total of £680 million has been ringfenced for the Innovative Medicines Fund and Cancer Drugs Fund - £340 million each - to fast-track medicines to NHS patients. DHSC said: "The Innovative Medicines Fund will provide quick access to novel treatments, including potentially lifesaving gene therapies for serious conditions with few treatment options. It often takes longer for pharmaceutical companies to collect data on a medicine's clinical and cost effectiveness for rare diseases due to the smaller patient cohort.