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Rare bone disease drug for more NHS patients under new deal

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Children and adults with a rare bone disease will be able to benefit from a potentially life-enhancing drug due to a new deal between NHS England and the manufacturer.

The drug – asfotase alfa – is used to treat the rare inherited bone condition hypophosphatasia (HPP), which is often fatal in babies and can lead to debilitating bone deformities in older children and adults.

”This innovative deal shows…[we can] offer ground-breaking therapies to NHS patients…while driving maximum value for taxpayers”

Simon Stevens

It was previously only recommended for use in babies by the National Institute for Health and Care Excellence but a new “managed access agreement” plus updated guidance will make it available to a wider range of patients.

Under the agreement with manufacturer Alexion, infants, children and adult patients with paediatric-onset HPP who experience the most disabling symptoms will also be able to benefit from the drug.

The deal is only the second of its kind to be struck by NHS England after it gained new powers in April this year to negotiate directly with pharmaceutical companies offering innovative but expensive drugs.

Last month NHS chief executive Simon Stevens announced the first such agreement with drug company Roche to make high-cost breast cancer drug trastuzumab emtansine – also known as Kadcyla - available on the NHS.

Because of the small numbers of patients with rare diseases such as HPP, it can be difficult to provide clear evidence of health benefits in clinical trials of new treatments.

The deal allows for a five-year period to gather “real-world” data about how well the drug works and the impact on patients’ health and quality of life before longer-term commissioning decisions are made.

“This innovative deal shows that with goodwill and flexibility from responsible life science companies we can square the circle between offering ground-breaking therapies to NHS patients on the one hand, while driving maximum value for taxpayers on the other,” said Mr Stevens.

”Up until now the committee felt the benefits of the drug were too uncertain in the whole population in relation to its very high price”

Carole Longson

Professor Carole Longson, director of the centre for health technology evaluation at NICE, said asfotase alfa was an “important development” in the treatment of HPP that “has been shown to save lives and prevent or delay its progression”.

“However, up until now the committee felt the benefits of the drug were too uncertain in the whole population in relation to its very high price for them to be able to recommend it for any but the most seriously affected,” she said.

“The new deal, which includes a managed access agreement between the company and NHS England, means that people with the greatest clinical need for treatment can be identified and the costs and risks to the NHS have been reduced.

“We are very pleased therefore to be able to recommend it as an option for treating paediatric-onset HPP,” she added.

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