Two new treatments, one for tackling osteoporosis after menopause and a gene therapy for the blood disorder beta-thalassaemia, have been approved for us on the NHS.
The green light for both from the National Institute for Health and Care Excellence (NICE) is expected to help with the health and wellbeing – and ability to stay in work – of thousands of people.
First, it is anticipated that more than 14,000 people will benefit after NICE’s final guidance recommendation that the drug abaloparatide can be used as an option for treating osteoporosis after menopause, if there is a very high risk of fracture.
The drug, also called Eladynos, increases bone density by stimulating the cells that make new bone and this reduces the risk of fracture. It can provide an alternative for people who have not responded to, or cannot tolerate, usual treatment options, NICE said.
The institute’s independent committee heard from patients about how debilitating osteoporosis can be and how it affects all aspects of day-to-day life, such as not going out for a walk because they are fearful of falling and having a fracture. This can impair mental and physical health.
Professor Jonathan Benger, NICE chief medical officer and deputy chief executive, said: “During the menopause oestrogen, the hormone that is important for maintaining bone density and strength, decreases and bone density reduces. This can lead to bone thinning, known as osteoporosis, and therefore broken bones become more common. Fractures can be very painful and limit a person’s independence, and they are also associated with increased mortality.
“Our focus is on enabling access to care that improves quality of life while offering value to the taxpayer. Abaloparatide has been found by our independent committee to be clinically and cost effective at reducing the risk of fracture, giving people more independence and therefore a better quality of life,” he added.
The second recommendation will allow patients in England with severe beta-thalassaemia to be among the first in Europe to benefit from a one-time gene therapy called exagamglogene autotemcel.
NICE’s final draft guidance has recommended exagamglogene autotemcel (or ‘exa-cel’ but also called Casgevy) for people aged 12 years and over with severe beta-thalassaemia who need regular blood transfusions to manage their condition and when a blood and bone marrow transplant is suitable but no donor is available.
Exa-cel is the world’s first CRISPR-based gene therapy and the first gene therapy available in Europe for treating severe beta-thalassaemia, NICE said.
Beta-thalassaemia is an inherited blood disorder caused by a genetic mutation that reduces or prevents production of healthy red blood cells and haemoglobin (the protein found in red blood cells that carries oxygen around the body). In the UK the condition mainly affects people of Pakistani, Indian and Bangladeshi ethnic origin.
People with the most severe type of beta thalassaemia need regular blood transfusions. Severe beta-thalassaemia can cause delayed growth, bone problems, problems with endocrine development and affect quality and length of life.
The independent committee, again, heard from patient experts about the significant effect on work, family and friends that intense blood transfusions and their associated side effects and complications can have.
Although there are some uncertainties in the evidence for its long-term benefits, the NICE committee felt exa-cel could represent a potential cure for some people with transfusion-dependent beta-thalassaemia, freeing them from the burden and risks of needing regular blood transfusions, it argued.
Helen Knight, director of medicines evaluation at NICE, said: “Costing £1,651,000 per course of treatment at its list price, exa-cel works by first removing and then modifying (editing) the faulty gene in a patient’s bone marrow stem cells so the body produces functioning haemoglobin – the protein in red blood cells that carries oxygen around the body.
“The edited cells are then put back into the patient. As exa-cel involves people receiving their own edited cells, there is no risk of their body rejecting them,” she added.
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