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NHS England negotiates price cuts for ultra-rare Morquio A syndrome drugs

People in England who suffer from the ultra-rare Morquio A syndrome should in future be able to access the only drug for their condition after a ground-breaking agreement was reached between NHS England and the manufacturer. NHS England has successfully negotiated a deal on elosulfase alfa (brand name Vimizim) with manufacturers BioMarin Pharma. The five […]

NHS patients with rare bone disease to benefit from potentially life-transforming drug due to “first of a kind” agreement between NHS England and the manufacturer

Children and adults with a rare inherited bone disease will be able to access a potentially life-enhancing drug, thanks to an innovative deal between NHS England, NICE and manufacturer Alexion. Hypophosphatasia (HPP) is a rare inherited bone condition that can appear any time from before birth to adulthood. In babies it is often fatal, and […]

Life-changing treatment on the NHS for children with rare, deadly disease

Children in England with a rare condition which leads to early death are set to receive treatment on the NHS, after the health service struck a deal with manufacturers. Young people with Batten Disease – an incurable illness which affects the nervous system, causing seizures, visual impairment, mobility loss and early death – will now […]

NHS England announces annual investment decisions for certain specialised services

NHS England has today (2 July) set out its planned investment decisions for certain specialised services as part of its annual commissioning round. The decision follows a three-month public consultation on the principles and processes NHS England will follow when making investment decisions. Feedback from the consultation has helped inform the way these decisions have […]

Thousands of patients to benefit from innovative new treatments on the NHS

Stroke-reducing heart implants and a life changing surgical procedure for children with cerebral palsy are among a host of new treatments that will now be routinely available for thousands of patients on the NHS. The NHS announced its plan to provide the latest innovative treatments for patients, just days after it celebrated its 70th anniversary. Hundreds […]

New Cancer Drugs Fund benefiting thousands of patients and releasing £140million for the NHS

Thousands of cancer patients have benefited from speedy access to the new and innovative treatments due to new Cancer Drugs Fund (CDF), which is also expected to release at least £140million into the NHS over the next five years. Since the new CDF opened in July 2016, nearly 15,700 patients have benefited from the 52 drugs […]

Commercial medicines

The commercial medicines directorate undertakes the commercial arrangements that influence the price of medicines to ensure patients have faster access to the most innovative new medicines and ensure we get the best value from the £18.9 billion the NHS spends on medicines. Our mission is: to support patient access to the latest innovative and most […]

F02. Specialised Blood Disorders

Scope This Clinical Reference Group (CRG) covers haemophilia and other bleeding disorder services. Bleeding disorders are medical conditions in which the blood fails to clot properly. These conditions are rare in the general population, affecting around 24,000 people in the UK. Most are genetically inherited. They include Haemophilia A and B (classified as mild, moderate […]