NHS patients with rare bone disease to benefit from potentially life-transforming drug due to “first of a kind” agreement between NHS England and the manufacturer

Children and adults with a rare inherited bone disease will be able to access a potentially life-enhancing drug, thanks to an innovative deal between NHS England, NICE and manufacturer Alexion.

Hypophosphatasia (HPP) is a rare inherited bone condition that can appear any time from before birth to adulthood. In babies it is often fatal, and in older children and adults it can be debilitating, leading to bone deformities that may result in delayed walking, limb weaknesses, skeletal pain and fractures. The disease is more common in older children and adults, affecting 1 per 6,370 of the population. The drug asfotase alfa was previously only recommended for use in babies by NICE in draft guidance, as evidence showed it could be life-saving.

A new ‘managed access agreement’ between NHS England and manufacturer Alexion, alongside new draft NICE guidance issued today, will broaden access of asfotase alfa to infants, children and adult patients with paediatric-onset HPP, who experience the most disabling symptoms and are expected to benefit most from therapy. It is a novel deal because it is a value-based risk sharing agreement to provide wider cost-effective access for patients, informed by their first-hand experience of the ongoing impact that treatment is having on their health and quality of life.

Because of the small numbers of patients with rare diseases it can be challenging to get sufficient numbers treated in research trials to have certainty about the level of health benefits. The agreement helps address this lack of certainty and allows for a five year period to gather real-world data about how well the treatment benefits these patients before longer term commissioning decisions are taken.

From April 2017 NHS England took on new powers to negotiate directly with pharmaceutical companies offering innovative high value medicines to the NHS. Last month chief executive Simon Stevens announced the first such agreement in respect of a modern breast cancer therapy.

Commenting on this latest successful negotiation, NHS England Chief Executive Simon Stevens said: “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. This agreement is another practical demonstration that NHS England is open to collaborative rare disease funding arrangements, tied to whether apparently promising therapies actually deliver for ‘real world’ patients over time.”

Peter Huskinson, Commercial Director for Specialised Commissioning at NHS England said: “NHS England is delighted to have secured an agreement to provide more people with this potentially life-changing drug, working closely with NICE and the manufacturer. The deal is an important departure from usual funding approaches because it is based on the on-going impact that treatment is having on patient’s health and quality of life.”

Professor Carole Longson MBE, Director of the Centre for Health Technology Evaluation at NICE said: “Asfotase alfa is an important development in the treatment of this devastating condition 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.

“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 hypophosphatasia.”