Cystic fibrosis drugs to be available on NHS in England within 30 days

Every patient in England with cystic fibrosis who could benefit from one of the new precision drugs that target certain mutations underlying the condition will have access to them within 30 days, NHS England has said after striking a deal with the US manufacturer Vertex Pharmaceuticals. Last July it rejected what NHS England said was a “fair and final offer” of around £500m over five years for all of the company’s approved drugs and any future drugs.2 At the time the company argued that it couldn’t accept the offer—which amounted to around £10 000 a year per patient—because other countries would demand the same deal, which would not allow it to develop the next generation of drugs to treat cystic fibrosis. In 2016 NICE rejected Orkambi for use in the NHS, saying that while it reduced admissions to hospital and use of antibiotics among people with cystic fibrosis, the short term benefits to lung function were modest and the long term benefits uncertain.3 At the time the Cystic Fibrosis Trust argued that, in the two years since NICE had conducted its appraisal, new data showed that Orkambi almost halves the rate of decline in lung function in people with the disease over two years.