Positive NICE recommendations for cystic fibrosis drugs The BMJ Technology Assessment Group (BMJ-TAG) conducts independent health research, utilising the expertise of its team of clinical evidence analysts and health economists. The group are experts in systematic literature reviews, statistics, and health economic modelling. Their work informs NHS policy, revolutionises patient treatment, and critically evaluates drug safety and effectiveness for NICE (National Institute for Health and Care Excellence) guidance.
Achieving patient access and long term value Cystic fibrosis (CF) is a life limiting genetic condition affecting over 9,000 people in England. Characterised by a progressive decline in lung function and frequent, severe respiratory infections, CF historically has had a significant impact on life expectancy, with an average age of death of just 36 years. Recently, a new generation of treatments has emerged that target the underlying cause of CF. Still, they were only available in clinical trials, and none were routinely available on the NHS. BMJ-TAG examined the costs and benefits of three such treatments (Kaftrio ® , Symkevi ® and Orkambi ® ). Their research concluded that they potentially offer life changing patient benefits, but at an exceptionally high price for the NHS. After discussions between NICE, NHS, and Vertex Pharmaceuticals (manufacturer of the drugs), informed by further research by BMJ-TAG , all three drugs were recommended for use. 30, 31 The primary goal of healthcare is to improve patient outcomes. If a treatment offers significant, transformative benefits, even at a high cost, it can be deemed worthwhile. CF drugs drastically improve life expectancy and quality of life, which substantially benefits patients.
While initially expensive, these drugs may lead to sustained savings by preventing hospital admissions and reducing complications. BMJ-TAG’s analysis would consider these lasting effects. David Ramsden, chief executive of the Cystic Fibrosis Trust, described the decision as a significant achievement for people with CF, ensuring continued access to treatment for thousands in the UK. 32 “This is a fantastic moment for many people with cystic fibrosis and their families – ending uncertainty and helping to ensure that everyone who can benefit can access these vital medicines – now and in the future.” David Ramsden , chief executive of the Cystic Fibrosis Trust The BMJ-TAG team’s research informs negotiations between the NHS, NICE, and pharmaceutical companies, often leading to reduced drug prices and improved patient access. This evidence based work ensures that treatments which extend and improve quality of life are accessible and costeffective for the NHS, ultimately benefitting many patients. • Supporting informed decisions based on evidence • Improving patient lives with effective treatments • Ensuring value for money through careful analysis
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