UK children with growth disorders given access to weekly injection | Children’s health | Daily News Byte


Thousands of children who are not growing normally due to a hormone deficiency have been given access to a weekly injection for the condition following new guidance from the medicines watchdog.

Children with a disorder known as growth disturbance previously required daily injections of the growth hormone somatropin to ensure their healthy growth. But a fast-track assessment by the National Institute for Health and Care Excellence (NICE) means patients can now use a more convenient weekly injection called somatrogan.

A drug recommended under an expedited evaluation process was typically completed in three-quarters of the time it took NICE to compare its price. The drug will now be available for children aged three years and above.

Doctors estimate that around 2,200 children in the UK could benefit from the decision by needing fewer injections to boost their hormone levels. Evidence from clinical trials shows that somatrogen is as effective as somatropin.

“The introduction of a long-acting preparation of growth hormone given once weekly has the potential to improve quality of life in many children on growth hormone treatment,” said Professor Mehul Dattani, head of pediatric endocrinology at UCL. Great Ormond Street Institute of Child Health.

He said the weekly treatment could be particularly convenient for families who struggle to administer daily injections for a variety of reasons. These range from children and adolescents with needle phobia who find it difficult to stick to a daily injection regimen, to children with visual impairments and children with learning difficulties and neurodisabilities. “With fewer injections, many of these children and youth have the potential to improve treatment adherence,” Dattani said.

Growth hormone is produced by the pituitary gland deep within the brain. If there is a problem with the gland, or the part of the brain that communicates with it, hormone production can be disrupted and the baby will not grow. The first sign may be when growth slows at two to three years of age.

Gary Butler, consultant professor of paediatrics and adolescent endocrinology at University College London Hospital, said daily growth hormone injections have been a major success in helping children who cannot make enough of the hormone naturally to grow to a normal height and live their lives without restrictions. He said children who have been treated include children with underactive pituitary glands, those born very small, or with certain genetic disorders that prevent normal growth, and brain tumor survivors.

“Success depends on regular treatment, so children and their families benefit greatly from having just one weekly injection to make things easier and better promote their development,” he said.

Nice’s approval of Somatrogen was completed seven weeks faster than the watchdog’s old cost comparison process, which took 29 weeks. Under the new procedures, NICE aims to make quick, light-touch assessments for simple, low-risk treatments that don’t require more involved assessments.

Helen Knight, Director of Medicines Evaluation at Nice, said: “The recommendation of Somatrogan for the care of children with growth disorders due to growth hormone deficiency is a welcome development. We want to get patients the best care faster, ensuring value for money for the taxpayer, and at the same time creating useful and useful advice for the NHS,” she added.


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