The BMJ Technology Assessment Group (BMJ-TAG) conducts health research for a range of institutions. Their work has led to some revolutionary changes to the way patients receive treatment through the UK’s National Health Service (NHS). Over the past two years, the group independently assessed the use of new treatments for ovarian cancer and other life threatening conditions.
In April 2022, as a result of TAG’s independent assessment, elosulfase alfa was recommended for use in the NHS by the National Institute for Health and Care Excellence (NICE), the official body that provides national guidance and advice to improve health and social care. It is recommended for treating mucopolysaccharidosis Type IV A (MPS IVA). Treatment is expected to start in children when they are around three years old and will be lifelong. If untreated, MPS IVA can cause skeletal abnormalities, respiratory symptoms, pain, fatigue, and reduced life expectancy. The Society for Mucopolysaccharide Diseases (MPS Society) considers elosulfase alfa to be ‘life changing,’ with one parent reporting that:
"My son Sam has been receiving Vimizim [elosulfase alfa] for almost 10 years. He does not suffer from fatigue. He does not routinely have any pain.
He is 13 [now] but is still very mobile and independent. Vimizim has had an incredible impact on his life, his quality of life and his independence.”
Elosulfase alfa is likely to slow disease progression and delay symptom onset. It also improves children’s quality of life by preserving lung function and improving endurance. However, as it is still highly uncertain if elosulfase alfa actually increases someone’s life compared to being untreated, the company offered a substantial discount on the retail price of the drug, resulting in a large cost saving for the NHS.
Thousands of women with ovarian cancer to access groundbreaking new drug with biggest breakthrough in three decades
BMJ-TAG's work with NICE has also led to the most significant breakthrough in ovarian cancer treatment in decades. Now, more than 3,000 more women each year will have access to niraparib, a medicine that will keep tumours at bay for years.
Our drug assessments have seen ribociclib, a life-extending drug for incurable breast cancer, approved for NHS use – potentially benefiting 3,300 women a year. Onasemnogene is one of the very first gene therapies used to help protect babies from the impact of their inherited muscle-weakening disorder. It was recently approved for NHS treatment by NICE after its appraisal by the BMJ-TAG team. This life-extending and life-enhancing treatment is the most expensive drug in history to be made available by NICE and is likely to become a benchmark for subsequent gene therapies.